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Recent developments in targeting access to high cost medicines in Australia

Recent developments in targeting access to high cost medicines in Australia Background: In Australia, the Pharmaceutical Benefits Scheme (PBS) has developed a set of arrangements to control access to high-cost medicines to ensure their use is cost-effective. These medicines include the tumour necrosis factor-alpha inhibitors (TNFIs) for the treatment of rheumatoid arthritis. The aim of this first phase of a qualitative study was to explore basic views on the restricted access to TNFIs in order to confirm where further investigation should take place in the next phase. Methods: Semi-structured interviews were conducted in 2004 with a member of the four relevant stakeholder groups. Participants were asked their opinions about features of the establishment, process and effects of the system of restricted access to TNFIs. Views on the collaboration between stakeholder groups in the decision-making process were also collected. Results: The principle of 'controlled access' to TNFIs was supported in general. There were concerns regarding some of the specific eligibility criteria. Wider and more transparent stakeholder consultation was judged desirable. Some flexibility around prescribing of TNFIs by physicians, and regular review of the arrangements were proposed. These themes will inform the next phase of the study. Conclusion: This first phase highlighted a range of issues associated with the PBS arrangements restricting access to TNFIs. Timely review and report of issues and concerns associated with such policy developments that arose in practice are essential. There is a need for a more comprehensive exploration across a wide range of stakeholders with different perspectives that will in turn be helpful in guiding policy and practice around national arrangements to manage access to high-cost medicines. Background cally in all countries both in the developed and the devel- Expenditure on pharmaceuticals has increased dramati- oping world in recent years [1]. Provision of an increasing Page 1 of 8 (page number not for citation purposes) Australia and New Zealand Health Policy 2005, 2:28 http://www.anzhealthpolicy.com/content/2/1/28 number of effective but expensive pharmaceuticals by formulated to secure the most cost-effective use of these drug reimbursement systems is challenging because the expensive agents, were agreed upon after extensive negoti- costs per patient are high. Payers in the public and private ations [3,10]. The respective consumer organisation, the health systems face similar challenges due to increasing Arthritis Foundation of Australia, also coordinated a consumer expectations to access these medicines in the strong lobbying effort by consumer representatives to the context of substantive cost constraints. The Pharmaceuti- government. This collaborative model has set a new para- cal Benefits Scheme (PBS) is Australia's national program digm for future PBS decisions. Subsidised access was lim- which provides subsidised access to prescriptions medi- ited to a subset of patients whose disease "has not been cines for the community [2]. Decisions on drug reim- adequately controlled using conventional anti-rheumatic bursement ('listing') under the PBS are based on drugs and these patients must meet strict criteria for both assessment by the Pharmaceutical Benefits Advisory Com- starting and continuing biological therapy" [11]. Patients mittee (PBAC), which evaluates efficacy, safety and incre- are required to sign a Patient Acknowledgement Form mental cost-effectiveness of new pharmaceutical products which specifies that continuation of therapy beyond four compared to other existing treatments [3]. Australia was months "will only be approved if objective, substantial the first country to introduce an explicit requirement for response is achieved". Prescribing rights are limited to economic analysis to select pharmaceuticals for a publicly specialists with expertise in the management of rheuma- funded formulary [4]. This system has attracted consider- toid arthritis. Also agreed upon was a risk-mitigation able attention worldwide as Australian pharmaceutical arrangement between the government and the sponsors prices are markedly lower than those in other comparable that established a ceiling level for government outlays countries [5]. The national goal, as expressed in Australia's annually [10]. Similarly complex arrangements for access National Medicines Policy and, in particular, the Quality are being applied to other high-cost medicines such as Use of Medicines component, states that limited resources imatinib (Glivec ) for the treatment of patients with should be utilised in such a way that there is provision of chronic myeloid leukaemia [11]. needed, effective and safe medicines that are affordable for the individual in order to achieve optimal health out- Decision makers' and stakeholders' perspectives on prior- comes [6]. itising decisions about drug reimbursement and decision- making processes in comparable countries such as Fin- Biological agents licensed in Australia for treating rheu- land, Canada, and the United Kingdom have been matoid arthritis include three tumour necrosis factor- described [12-14]. However, to the best of our knowledge, alpha inhibitors (TNFIs), etanercept (Enbrel ), infliximab there are no published data on perceptions regarding ® ® (Remicade ), and adalimumab (Humira ), and an inter- approaches used in Australia, such as arrangements for leukin-1 receptor antagonist, anakinra (Kineret ). These access to high-cost medicines operated through the PBS, new medicines markedly reduce disease activity in a or studies examining stakeholder consultation processes majority of patients, but there are concerns about their to reach consensus on arrangements for subsidised access high cost (approximately AUD$20,000 per patient per to high-cost medicines. We considered that careful exam- year), in particular given the annual growth in govern- ination of the recent developments in targeting access to ment expenditure on pharmaceuticals averaging 10.5% high-cost medicines, using TNFIs as an example, would be between 1992–93 and 2002–03 (representing an increase instructive in informing the debate concerning the princi- from AUD$1.88 billion to AUD$5.12 billion) [7]. Fur- ples and processes that might underpin appropriate and ther, there are uncertainties regarding their long-term ethical access to expensive pharmaceuticals under the PBS safety, including serious and opportunistic infections and or similar access systems. Stakeholders are defined here as the risk of lymphoma [8,9]. groups of people that have the potential to influence the decisions on the arrangements for access, or those who are The PBS has evolved a set of arrangements to control affected by the PBS-restrictions. The views, attitudes, con- access to high-cost medicines in an attempt to maintain cerns, and level of support for these arrangements by the the viability of the PBS. Representative, but most innova- stakeholders are critical determinants for a successful tive of this set of arrangements are those established for implementation of the arrangements. Qualitative tech- the TNFIs, implemented since August 2003. The decision niques are useful to explore perceptions and experiences to subsidise TNFIs was based on a collaborative decision- across a range of relevant stakeholders with respect to making model to enable the listing of the TNFIs on the restricted access to TNFIs, and to understand the effects of PBS. This involved the PBAC which consulted the relevant the access criteria when implemented in practice. From a key stakeholders: the respective pharmaceutical compa- policy perspective, issues and concerns that arise in prac- nies, and rheumatologists via the Australian Rheumatol- tice need to be reviewed in a timely manner thereby ena- ogy Association Therapeutics Subcommittee. The bling appropriate management of any implications. arrangements for access under the PBS (PBS-restrictions), Research guiding policy development can often best be Page 2 of 8 (page number not for citation purposes) Australia and New Zealand Health Policy 2005, 2:28 http://www.anzhealthpolicy.com/content/2/1/28 Table 1: Major themes around restricted access to tumour aims to elicit a basic viewpoint from a member of each necrosis factor inhibitors stakeholder group. The rheumatologist received an invita- tion to participate through an opinion-leader rheumatol- Predetermined Interview topics ogist, purposively selected to have had experiences Perceptions of PBS-restrictions on access Experience with application process prescribing TNFIs through the PBS at the time of the Pre-PBS collaboration between the different stakeholder groups study. The health advisor who participated belonged to a Post-PBS collaboration between the different stakeholder groups like-group of the PBAC, and the pharmaceutical employee Sources of information was invited to give views of the industry but was not from a company that marketed TNFIs. The patient was nomi- Major themes emerging from data nated by a rheumatologist. Participant information sheets Access to medicines and consent forms were provided to all participants prior Targeting access Review of the PBS-restrictions to the interviews. These four interviews were conducted by Implications of access arrangements in practice one trained researcher (CL); interviews were of 45 to 60 Effects on the practice of rheumatology minutes duration. Ethical issues Roles and responsibilities Interviews were recorded and transcribed verbatim. The PBS Decision-making process transcripts were coded for major concepts. Categories Stakeholder consultation process related to the discussion topics were created. Coded seg- Transparency Information provision ments were then analysed and categorised thematically, thereby developing a thematic framework. NVivo soft- ware 2.0 was used to help manage the qualitative data. undertaken as an iterative approach with each phase The transcripts were also coded independently by another building on the one before, in particular where the impact investigator (RD or KW). The researchers then met to of implementing new access criteria on practice is examine the analyses in order to reach agreement on cat- unknown. The scope and content of such a study are not egories and identified themes. Some differences with specified in advance but are developed iteratively. This regard to labelling the themes were found, but agreement paper reports on the initial phase of a qualitative study was reached on the central meanings. Interviewees were using an iterative approach. This first phase sought to offered opportunities to review an edited interview tran- explore the views of one member of each of the four stake- script to check that their views had been accurately repre- holder groups with a vested interest in any new policy sented. Only one interviewee reviewed the transcript. It about provision of medicines, in order to confirm where was accepted without change. The study was approved by further exploration should take place in the next iteration. the Human Research Ethics Committees of St Vincent's Hospital Sydney, and the University of New South Wales, Methods Australia. Qualitative techniques were used for data collection and analysis. In depth, semi-structured interviews were con- Results and Discussion ducted in Sydney in 2004 with four individuals to explore This initial phase identified a range of concerns, as held by four different perspectives about access to TNFIs under the individuals from different stakeholder groups, that arose PBS. Interviewees were asked their opinions about fea- in practice associated with the recent developments to tures of the establishment, process and effects of the control access to a group of high-cost medicines in Aus- arrangements for targeting access to TNFIs. Views on the tralia. These are imperative issues that decision makers in decision-making process and collaboration between Australia and other countries need to consider and man- stakeholder groups were also collected. age appropriately and in a timely manner. The pre-deter- mined interview topics and major themes that emerged Due to a finite number of key stakeholder representatives from the data are listed in Table 1. The four major themes who participated in the PBAC decision-making process are described below with selected quotes from partici- that formulated the arrangements for access to TNFIs, they pants. were purposively not invited in this initial phase of the study. Purposeful sampling was used to select participants 1. Access to medicines on the basis of their primary membership of different 1.1 Targeting access stakeholder groups with respect to the controlled access to Interviewees expressed an understanding for the finite TNFIs: a rheumatologist, a health advisor to the govern- resources that can be allocated to treat the possible range ment, an employee of a pharmaceutical company, and a of diseases. The principle of 'controlled access' to TNFIs patient (who had used a TNFI). Representation of partici- was supported in general. All perceived that the purpose pants was not sought in this first phase of the study which of restricting access to TNFIs was to control costs while tar- Page 3 of 8 (page number not for citation purposes) Australia and New Zealand Health Policy 2005, 2:28 http://www.anzhealthpolicy.com/content/2/1/28 geting those patients identified by the access criteria as of their disease without reaching the 'disease activity' spec- most likely to derive benefit. ified in the eligibility criteria. "The government is trying to target the therapy to those "It's forced our rheumatologists to re-look at the treat- who need it ... and control their budget." (advisor) ment that patients have already received and to re-evalu- ate ... in reassessing the patient and aiming to meet the The PBS-restrictions on access, including the limiting of PBS criteria, they actually get their rheumatoid arthritis prescribing rights to qualified specialists, were considered under control before requiring a biological..." (rheuma- to be safe and cost-effective, generally evidence-based and tologist) appropriate, given the expense and some lingering uncer- 2.2 Ethical issues tainty about long-term safety. One of the ethical issues raised by the interviewees was the "TNF inhibitors are fairly new ... there are no long-term possible benefit that might be achieved in some patients data as yet on their use ... they are not without side effects, if the PBS-restrictions would allow a switch to another and because of their expense, trying existing therapies first TNFI if they failed to respond to the first agent. is a reasonable approach" (rheumatologist) "If patients didn't meet the criteria for continuing treat- Participants disagreed with some of the specific eligibility ment and at the time they stopped, were not bad enough criteria which they interpreted as inflexible and not obvi- to meet the initial criteria for another agent, they can't ously evidence-based. For example, a number of patients switch to another agent, because they are required to meet with severe rheumatoid arthritis suffered because of the the initial criteria again." (rheumatologist). requirement that rheumatoid factor be positive. The original PBS-restrictions did not address this issue, "The requirement for a rheumatoid factor is a concern but these have been altered shortly after the completion of because I don't believe that there's any scientific evidence this study's initial phase. The PBS introduced an 'inter- ... and they [application assessors] seem to be inflexible changeability' rule from December 2004, allowing about that criterion ... There are unfortunate people who patients who meet the eligibility criteria for commencing would qualify on every criteria except rheumatoid factor therapy to trial any of the listed TNFIs as well as anakinra, alone." (rheumatologist) another anti-rheumatic biological agent [11]. This change demonstrates that some review of the PBS-restrictions is 1.2 Review of the PBS-restrictions occurring. Availability of TNFIs through the PBS, although tightly restricted, was welcomed. However, ongoing review of the Despite the 'objective' clinical measures upon which con- PBS-restrictions in accordance with emerging new evi- tinued access to TNFIs is determined, there are many fac- dence and analysis of utilisation data was seen as both tors influencing the medical condition of an individual. important and necessary. Interviewees believed the key Concerns were raised about withdrawing patients from an stakeholder representatives who had participated in the effective treatment when they may have just missed the initial stakeholder consultation should again participate threshold for continued access as well as a lack of flexibil- in these activities. The risk-mitigation agreement should ity for physicians to make decisions in cases that are also be reviewed for its effectiveness and value. equivocal based on their professional judgement. "There should be some process of review of criteria, after "There's a disparity between the patients' clinical response some agreed period, particularly if they're sharing a and joint count and their laboratory parameters ... the [financial] risk, to see whether they still continue as inflammatory markers may be staying up when they actu- appropriate based on new evidence and data of utilisa- ally clinically are a lot better." (rheumatologist) tion, etcetera." (advisor) "There's got to be a level of discretion there to the experts 2. Implications of access arrangements in practice because they're the guys who know. It's not some bureau- 2.1 Effects on the practice of rheumatology crat." (patient) The arrangements for access to TNFIs requiring evidence of exposure to and failure of a number of anti-rheumatic The application process was seen to be complex, time- drugs appear to have promoted re-evaluation of previous consuming and an administrative burden for everyone treatments and responses in patients with insufficiently involved: controlled rheumatoid arthritis. A proportion of patients may have benefited from this process with better control Page 4 of 8 (page number not for citation purposes) Australia and New Zealand Health Policy 2005, 2:28 http://www.anzhealthpolicy.com/content/2/1/28 "The administrative burden for the doctors, for the Health patients who don't strictly have rheumatoid arthritis." Insurance Commission...on the pharmacists, and also the (rheumatologist) patients...and the whole being caught up in that bureau- cratic tangle." (pharmaceutical employee) A view was put that if physicians disagree with any of the restrictions, they should lobby through their professional Another ethical concern was that a Patient Information organisation for revision because they are ultimately Sheet had not been developed to accompany the Patient responsible for their patients' well-being. More reliance Acknowledgement Form at the time these interviews took on physicians was considered appropriate and desirable. place, i.e. 12 months after the first TNFI was made availa- ble under the PBS. "Prescribers are obliged to adhere to the PBS arrange- ments... If we don't like the criteria and have concerns "They're [the patients] signing a contract and they should with it then the appropriate thing is to lobby through the be able to understand the benefits and also the implica- ARA [Australian Rheumatology Association]..." (rheuma- tions...they need to do it in a manner where they have all tologist) the information they need to decide." (advisor) Concerns were voiced about the need to achieve substan- 2.3 Roles and responsibilities tial clinical improvement for continuing access at the fol- Broadly, government is expected to provide affordable low-up assessment. This requirement may have access to effective medicines for patients with medical introduced potential perverse incentives to 'fudge' meas- needs while using taxpayers' money wisely and appropri- urements of disease activity in order to ensure continued ately. A related and necessary responsibility of govern- access to TNFIs. This raises the issue of the responsibility ment is to monitor the PBAC process to ensure and accountability of prescribers with respect to their legal appropriate assessment and selection of medicines for obligation to comply with the PBS requirements for PBS- reimbursement by the PBS. subsidised treatments. However, this does place powerful ethical concerns on doctors in cases where they become "The government has an obligation to ensure that the the agents whereby patients are denied or withdrawn from processes that the PBAC uses are rigorous, are regularly effective medicines, particularly if the patient's response is evaluated, and are well supported." (advisor) near the threshold level of eligibility. The Australia Rheu- matology Association was seen to be responsible for The pharmaceutical industry interviewee felt that infor- reviewing the PBS-restrictions and providing information mation provision by industry was a major responsibility to their members, i.e. rheumatologists, as well as the and needed particularly when high-cost medicines subject patients. to complex eligibility criteria become available through the PBS. This interviewee considered that updates should "The ARA [Australia Rheumatology Association] has an be provided to prescribers about the clinical uses of med- obligation to provide information to their own members, icines, including the application procedures for PBS-sub- it's essential that they support with adequate provision of sidised treatment; and to promote marketing and education and advice and support about this new treat- publicity with the content being concordant with the PBS- ment ... and they also have a role to provide education to restrictions (A consequence of risk-mitigation agreements the public." (rheumatologist) is a strong negative incentive for inappropriate marketing and publicity leading to use outside approved PBS-restric- The role and responsibilities of patient organisations were tions). The risk-mitigation agreement that is part of the not explored in detail in this initial phase. Consumer arrangements for access to TNFIs is an indication of col- organisations could represent patients in the consultation laboration between the government and the pharmaceuti- processes, but qualifications of the staff working in such cal company. organisations were thought to be important if they are to contribute to the consultation processes or to produce Physicians have a responsibility to comply with the PBS- educational information. Consumer organisations were restrictions by law [15]. The PBS-restrictions were worded perceived to primarily interact with patients and to dis- in such stringent and exhaustive detail that they were seen seminate information. to demonstrate a lack of faith in the prescribers by the PBS. "It's difficult to allocate the Arthritis Foundation a more active role in making recommendations, depending on "There seems ... mistrust from the PBS that rheumatolo- who is actually staffing the Arthritis Foundation and gists could have a loophole for the prescribing ... that where those opinions are coming from...the Arthritis they're worried that we will be prescribing the drug for Page 5 of 8 (page number not for citation purposes) Australia and New Zealand Health Policy 2005, 2:28 http://www.anzhealthpolicy.com/content/2/1/28 Foundation is primarily interfacing with the patients." the process, which could provide a humane view; and (rheumatologist) patients should at least be involved in the development of the Patient Acknowledgement Form. The staff at the Health Insurance Commission, a statutory organisation administering the PBS and other health pro- "No one really understands what you go through except grams [16], was considered in general to be helpful with you ... not even my rheumatologist or other specialist." respect to queries regarding the application procedure for (patient) subsidised TNFI treatments. "Patient groups should have a seat at the table...they 3. PBS Decision-making process should be represented formally, rather than by the doc- 3.1 Stakeholder consultation process tors... We need to have a process where consumers, pre- The interviewees were in substantive accord with the initi- scribers, people with interest, come together on an equal ative instituted with TNFIs, that negotiations between the footing to discuss what the issues are and what the criteria stakeholders should take place early in the decision-mak- are and can follow the progress." (advisor) ing process to enhance the likelihood that the final arrangements for access to such medicines are reasonable There is increasing need for the voices of patients, their and can be supported. carers and the public to be heard because it is appropriate and crucial for acceptance, change and improvement in "Negotiations should start early so that there is clear com- the process [17-19]. The representativeness of some con- munication between the expectation and the calibre of sumer groups was raised along with concern that some are evidence needed." (advisor) funded in part by pharmaceutical companies. This con- cern may be reduced if conflicts of interests are declared "...so that the final criteria are the correct criteria, the right openly, which links to the theme of "transparency". patient group is being identified, and also the initiation and continuation rules, etcetera, are workable and man- 3.2 Transparency ageable" (pharmaceutical employee) Transparency of the PBAC process and their decisions was a recurrent theme in the interviews. Interviewees thought However, the interviewees had different views about the that wider involvement in the consultation process and stakeholder consultation process that had taken place. greater empowerment of stakeholders to contribute to dis- cussion of the issues would be desirable. Disclosure of the "The major strength is the negotiations and the partner- rationale behind the PBAC's selection of access criteria, ing." (advisor) currently not available in the public domain, was consid- ered to be desirable and a helpful improvement to the sys- "It seems that each group has their own agenda and it's tem. Greater empowerment (and transparency) by not really like a collaboration but more each trying to put provision of appropriate information was proposed. forward their opinion." (rheumatologist) "A public document which summarised why different While the representativeness of the Australian Rheumatol- decisions were made, and the evidence base on which ogy Association Therapeutics Sub-committee in the deci- they were made would be a most fruitful thing to help sion-making process was believed to be appropriate, a patients to understand why they have potentially limited wider consultation among ordinary members of the asso- access to some of these medicines ... that's about empow- ciation was wanted: erment in information." (advisor) "The therapeutics committee should have sought infor- In addition, the issue of the requirement for a positive mation from the members of the ARA [Australian Rheu- rheumatoid factor has been the subject of debate since the matology Association], or have consulted more widely PBS-restrictions for TNFIs were implemented [20,21]. before they made their decisions because it seems that Greater transparency would be helpful to resolve these they made their decisions on our behalf but without con- concerns and gain increased community support. The sulting us." (rheumatologist) recent recommendation by PBAC to remove this criterion [22] is a welcome and rational outcome of the lengthy On the subject of representing the views of patients in debate on this issue. such processes, the rheumatologist was of the opinion that doctors would appropriately represent the views of The interviewees who participated in this study were 'out- their patients. On the contrary, the three other partici- siders' to the consultation process that led to establish- pants felt that patients should have a more direct role in ment of the arrangements for access. Minimal Page 6 of 8 (page number not for citation purposes) Australia and New Zealand Health Policy 2005, 2:28 http://www.anzhealthpolicy.com/content/2/1/28 involvement in the decision-making process and the cur- Interviewees had different views on whose responsibility rent level of transparency around PBAC's decisions lim- it was to provide information. The rheumatologist consid- ited their support for some of the eligibility criteria. ered that information about the efficacy and safety of Further, their minimal awareness of the negotiations that TNFIs is most important and that the Australian Rheuma- took place between key stakeholders suggests that there is tology Association would be appropriate to provide ongo- a need for better communication between those key stake- ing updates. Other proposed providers of information: holder representatives with their constituencies. Increased pharmacists, the Medicare Australia (previously the communication within and between the stakeholder Health Insurance Commission), the PBS, the National groups is also critical to obtain stronger support for the Prescribing Service (an independent educational organi- final PBS-restrictions in practice. sation funded by the Federal Government) [23], and the consumer organisations. 4. Information provision The PBS-restrictions to control access to high-cost medi- Further investigation cines, exemplified by TNFIs, are particularly complex, and The initial phase of the study has explored concerns of thus adequate provision of information is all the more individuals from the four relevant stakeholder groups. important. Information from pharmaceutical companies Our study was not designed to support statistical general- to the prescribers and general practitioners were noted to isability. Findings have established the basis for the next be abundant, however, interviewees considered that the phase of the study to explore perceptions of a wide range content needed to be more balanced. In particular, the of stakeholders that will be helpful in gaining a compre- rheumatologist and the government advisor expressed a hensive and broad societal view of these significant devel- lack of faith in the pharmaceutical companies' ability to opments of controlling access to high-cost medicines. A supply appropriate materials for patients or the public. written interview guide has been developed on the basis of the findings of this phase for use in subsequent inter- "It is not the sponsor's responsibility to talk to patients views. Diversity in sample characteristics and inclusion of because they will always get it wrong... direct-to-consumer individuals who were engaged in the decision-making advertising is a crazy way to go..." (advisor) process will expand understanding of the issues affecting different members of the stakeholder groups and provide Some concern was also expressed about industry-funded some insight into the negotiations during the decision- 'educational' functions: making process. Interviewees will include government advisors, participants in the stakeholder consultation "The way it was presented was one-sided, that the govern- process that formulated the arrangements for access to ment has put all these barriers forward, the barrier nature TNFIs (who are in a position to comment on what actu- was highlighted..." (advisor) ally occurred and what could be improved), rheumatolo- gists and patients who have gone through the application Interviewees' had concerns about the understanding of process, pharmaceutical company personnel, consumer the PBS system and the PBAC process among health pro- organisation representatives, and government administra- fessionals and in the community. Better provision of tion staff. We anticipate that this broad range of interview- information on the structure of the PBS and the PBAC ees will provide deep and rich insights to decision makers process was proposed. In particular, interviewees noted regarding the future decision-making process and devel- that doctors are the most appropriate health professionals opment of national arrangements to manage access to to inform patients with regards to the PBS-restrictions, medicines from the perspectives of enhancing appropriate and that they therefore need to be better informed about access to effective and expensive medicines. the cost-effective analyses that underpin the PBAC deci- sions. For example, patients should be made aware that Conclusion self-funding is an option, although in the case of TNFIs The PBS has developed a set of arrangements to target this is impractical for most people. Interviewees proposed access to expensive medicines such as the TNFIs in an that communication about the need to targeting use of attempt to sustain this national drug reimbursement sys- highly specialised medicines should extend to (potential) tem. Findings from this initial phase of the study high- patients and the public generally, and also to general prac- lighted a number of issues associated with the restricted titioners who could have an important role and contribu- access to TNFIs. The principle of 'controlled access' to tion in this area. Increased community awareness and TNFIs was supported in general by all interviewees each understanding of the principle of quality-use-of-medi- from a different stakeholder group. However, it is clear cines should be part of the overall educational strategy. that some views, such as those concerning patient direct participation or the varying responsibilities of each stake- holder group, were different. The pre-determined ques- Page 7 of 8 (page number not for citation purposes) Australia and New Zealand Health Policy 2005, 2:28 http://www.anzhealthpolicy.com/content/2/1/28 5. Productivity Commission: International pharmaceutical price differences: tions appeared to adequately explore the key issues. A research report Canberra: AusInfo; 2001. thematic framework has now been developed on the basis 6. Sansom L: The Australian National Medicinal Drug Policy. of the major themes that emerged from the interviews. We Journal of Quality Clinical Practice 1999, 19:31-35. 7. Australian Institute of Health and Welfare (AIHW): Health expenditure believe there is a need for a more comprehensive explora- in Australia 2002–03. AIHW Cat No HWE 27 (Health and Welfare tion, through a second phase of the study, to elicit differ- Expenditure Series No 20) Canberra: AIHW; 2004. 8. Symmons DP, Silman AJ: Anti-tumor necrosis factor alpha ther- ent perspectives across a variety of stakeholders, that will apy and the risk of lymphoma in rheumatoid arthritis: no in turn be helpful in guiding policy and practice around clear answer. Arthritis & Rheumatism 2004, 50:1703-1706. national arrangements to manage access to high-cost 9. Fleischmann RM, Iqbal I, Stern RL: Considerations with the use of biological therapy in the treatment of rheumatoid arthritis. medicines. Expert Opinion on Drug Safety 2004, 3:391-403. 10. Lu CY, Williams K, Day R, March L, Sansom L, Bertouch J: Access to Australia's Pharmaceutical Benefits Scheme, as part of the high cost drugs in Australia: Risk sharing scheme may set a new paradigm. BMJ 2004, 329:415-416. National Medicines Policy, is committed to providing 11. Commonwealth Department of Health and Ageing: Sched- access to cost-effective medicines to the community. ule of Pharmaceutical Benefits for Approved Pharmacists and Medical Practitioners (1 April 2005) [http:// When resources are constrained, restricting access is an www1.health.gov.au/pbs/] inevitable outcome. Greater and more trust-based com- 12. Vuorenkoski L, Toiviainen H, Hemminki E: Drug reimbursement munication and discussion among a wide range of stake- in Finland – a case of explicit prioritising in special catego- ries. Health Policy 2003, 66:169-177. holder groups will enhance community awareness and 13. Singer PA, Martin DK, Giacomini M, Purdy L: Priority setting for support to limit access to effective medicines. Acceptable new technologies in medicine: qualitative case study. BMJ 2000, 321:1316-1319. but different approaches to control access and manage the 14. Wirtz V, Cribb A, Barber N: Reimbursement decisions in health associated implications should be explored with the goal policy – extending our understanding of the elements of of enhancing health outcomes for all. decision-making. Health Policy 2005, 73:330-338. 15. Australian Government, The National Health (Pharmaceu- tical Benefits) Regulations 1960 (made under The National Declaration of competing interests Health Act 1953) [http://www.comlaw.gov.au/] Christine Lu and A/Prof Jan Ritchie have nothing to 16. Medicare Australia [http://www.medicareaustralia.gov.au/] 17. Wiseman V, Mooney G, Berry G, Tang KC: Involving the general declare. A/Prof Ken Williams is a member of the Advisory public in priority setting: experiences from Australia. Social Board to the sponsor for adalimumab. Prof Ric Day is a Science & Medicine 2003, 56:1001-1012. 18. Cayton H: Patient and public involvement. Journal of Health Serv- member of the Advisory Board to sponsors for adalimu- ices Research & Policy 2004, 9:193-194. mab, infliximab, and anakinra in Australia. A/Prof Wil- 19. Florin D, Dixon J: Public involvement in health care. BMJ 2004, liams and Prof Day have also been contracted to 328:159-161. 20. Lu CY, Williams KM, March L, Bertouch JV, Day RO: Subsidised undertake clinical trials with etanercept, infliximab, adal- access to TNF-alpha inhibitors: is the rationale for exclusion imumab, and anakinra. Recompense for these activities of rheumatoid factor negative patients defensible? Medical received was placed in audited hospital trust funds for use Journal of Australia 2004, 181:457-458. 21. Mann EL: Subsidised access to TNF-alpha inhibitors: is the in the research activities of the Clinical Pharmacology rationale for exclusion of rheumatoid factor negative Department, St Vincent's Hospital, Sydney. patients defensible? Medical Journal of Australia 2005, 182:47-48. 22. Commonwealth Department of Health and Ageing: Recom- mendations made by the Pharmaceutical Benefits Advisory Authors' contributions Committee in March 2005 [http://www.health.gov.au/internet/ CL, KW, and RD have made substantial contributions to wcms/publishing.nsf/Content/pbacrec-pbacrecmar05] 23. Weekes LM, Mackson JM, Fitzgerald M, Phillips SR: National pre- the conception and design of the study, analysis and inter- scribing service: creating an implementation arm for pretation of the data; and drafting and revising the manu- national medicines policy. British Journal of Clinical Pharmacolology script. JR has been involved in advising on the analysis, 2005, 59:113-116. drafting the manuscript and revising it critically for intel- lectual content. All authors read and approved the final manuscript. Publish with Bio Med Central and every Acknowledgements scientist can read your work free of charge The authors thank the interviewees for their participation in this research. "BioMed Central will be the most significant development for disseminating the results of biomedical researc h in our lifetime." References Sir Paul Nurse, Cancer Research UK 1. Organisation for Economic Co-operation and Development (OECD): OECD Health Data: Statistics and indicator for 30 countries 2005. Your research papers will be: 2. Commonwealth Department of Health and Aged Care: The Australian available free of charge to the entire biomedical community health care system: an outline. Canberra 2000. 3. Sansom L: The subsidy of pharmaceuticals in Australia: proc- peer reviewed and published immediately upon acceptance esses and challenges. Australian Health Review 2004, 28:194-205. cited in PubMed and archived on PubMed Central 4. Henry D: Economic analysis as an aid to subsidisation deci- sions: the development of Australian guidelines for pharma- yours — you keep the copyright ceuticals. Pharmacoeconomics 1992, 1:54-67. BioMedcentral Submit your manuscript here: http://www.biomedcentral.com/info/publishing_adv.asp Page 8 of 8 (page number not for citation purposes) http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png Australia and New Zealand Health Policy Springer Journals

Recent developments in targeting access to high cost medicines in Australia

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Springer Journals
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Copyright © 2005 by Lu et al; licensee BioMed Central Ltd.
Subject
Medicine & Public Health; Public Health; Social Policy
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1743-8462
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1743-8462
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10.1186/1743-8462-2-28
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16305742
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Abstract

Background: In Australia, the Pharmaceutical Benefits Scheme (PBS) has developed a set of arrangements to control access to high-cost medicines to ensure their use is cost-effective. These medicines include the tumour necrosis factor-alpha inhibitors (TNFIs) for the treatment of rheumatoid arthritis. The aim of this first phase of a qualitative study was to explore basic views on the restricted access to TNFIs in order to confirm where further investigation should take place in the next phase. Methods: Semi-structured interviews were conducted in 2004 with a member of the four relevant stakeholder groups. Participants were asked their opinions about features of the establishment, process and effects of the system of restricted access to TNFIs. Views on the collaboration between stakeholder groups in the decision-making process were also collected. Results: The principle of 'controlled access' to TNFIs was supported in general. There were concerns regarding some of the specific eligibility criteria. Wider and more transparent stakeholder consultation was judged desirable. Some flexibility around prescribing of TNFIs by physicians, and regular review of the arrangements were proposed. These themes will inform the next phase of the study. Conclusion: This first phase highlighted a range of issues associated with the PBS arrangements restricting access to TNFIs. Timely review and report of issues and concerns associated with such policy developments that arose in practice are essential. There is a need for a more comprehensive exploration across a wide range of stakeholders with different perspectives that will in turn be helpful in guiding policy and practice around national arrangements to manage access to high-cost medicines. Background cally in all countries both in the developed and the devel- Expenditure on pharmaceuticals has increased dramati- oping world in recent years [1]. Provision of an increasing Page 1 of 8 (page number not for citation purposes) Australia and New Zealand Health Policy 2005, 2:28 http://www.anzhealthpolicy.com/content/2/1/28 number of effective but expensive pharmaceuticals by formulated to secure the most cost-effective use of these drug reimbursement systems is challenging because the expensive agents, were agreed upon after extensive negoti- costs per patient are high. Payers in the public and private ations [3,10]. The respective consumer organisation, the health systems face similar challenges due to increasing Arthritis Foundation of Australia, also coordinated a consumer expectations to access these medicines in the strong lobbying effort by consumer representatives to the context of substantive cost constraints. The Pharmaceuti- government. This collaborative model has set a new para- cal Benefits Scheme (PBS) is Australia's national program digm for future PBS decisions. Subsidised access was lim- which provides subsidised access to prescriptions medi- ited to a subset of patients whose disease "has not been cines for the community [2]. Decisions on drug reim- adequately controlled using conventional anti-rheumatic bursement ('listing') under the PBS are based on drugs and these patients must meet strict criteria for both assessment by the Pharmaceutical Benefits Advisory Com- starting and continuing biological therapy" [11]. Patients mittee (PBAC), which evaluates efficacy, safety and incre- are required to sign a Patient Acknowledgement Form mental cost-effectiveness of new pharmaceutical products which specifies that continuation of therapy beyond four compared to other existing treatments [3]. Australia was months "will only be approved if objective, substantial the first country to introduce an explicit requirement for response is achieved". Prescribing rights are limited to economic analysis to select pharmaceuticals for a publicly specialists with expertise in the management of rheuma- funded formulary [4]. This system has attracted consider- toid arthritis. Also agreed upon was a risk-mitigation able attention worldwide as Australian pharmaceutical arrangement between the government and the sponsors prices are markedly lower than those in other comparable that established a ceiling level for government outlays countries [5]. The national goal, as expressed in Australia's annually [10]. Similarly complex arrangements for access National Medicines Policy and, in particular, the Quality are being applied to other high-cost medicines such as Use of Medicines component, states that limited resources imatinib (Glivec ) for the treatment of patients with should be utilised in such a way that there is provision of chronic myeloid leukaemia [11]. needed, effective and safe medicines that are affordable for the individual in order to achieve optimal health out- Decision makers' and stakeholders' perspectives on prior- comes [6]. itising decisions about drug reimbursement and decision- making processes in comparable countries such as Fin- Biological agents licensed in Australia for treating rheu- land, Canada, and the United Kingdom have been matoid arthritis include three tumour necrosis factor- described [12-14]. However, to the best of our knowledge, alpha inhibitors (TNFIs), etanercept (Enbrel ), infliximab there are no published data on perceptions regarding ® ® (Remicade ), and adalimumab (Humira ), and an inter- approaches used in Australia, such as arrangements for leukin-1 receptor antagonist, anakinra (Kineret ). These access to high-cost medicines operated through the PBS, new medicines markedly reduce disease activity in a or studies examining stakeholder consultation processes majority of patients, but there are concerns about their to reach consensus on arrangements for subsidised access high cost (approximately AUD$20,000 per patient per to high-cost medicines. We considered that careful exam- year), in particular given the annual growth in govern- ination of the recent developments in targeting access to ment expenditure on pharmaceuticals averaging 10.5% high-cost medicines, using TNFIs as an example, would be between 1992–93 and 2002–03 (representing an increase instructive in informing the debate concerning the princi- from AUD$1.88 billion to AUD$5.12 billion) [7]. Fur- ples and processes that might underpin appropriate and ther, there are uncertainties regarding their long-term ethical access to expensive pharmaceuticals under the PBS safety, including serious and opportunistic infections and or similar access systems. Stakeholders are defined here as the risk of lymphoma [8,9]. groups of people that have the potential to influence the decisions on the arrangements for access, or those who are The PBS has evolved a set of arrangements to control affected by the PBS-restrictions. The views, attitudes, con- access to high-cost medicines in an attempt to maintain cerns, and level of support for these arrangements by the the viability of the PBS. Representative, but most innova- stakeholders are critical determinants for a successful tive of this set of arrangements are those established for implementation of the arrangements. Qualitative tech- the TNFIs, implemented since August 2003. The decision niques are useful to explore perceptions and experiences to subsidise TNFIs was based on a collaborative decision- across a range of relevant stakeholders with respect to making model to enable the listing of the TNFIs on the restricted access to TNFIs, and to understand the effects of PBS. This involved the PBAC which consulted the relevant the access criteria when implemented in practice. From a key stakeholders: the respective pharmaceutical compa- policy perspective, issues and concerns that arise in prac- nies, and rheumatologists via the Australian Rheumatol- tice need to be reviewed in a timely manner thereby ena- ogy Association Therapeutics Subcommittee. The bling appropriate management of any implications. arrangements for access under the PBS (PBS-restrictions), Research guiding policy development can often best be Page 2 of 8 (page number not for citation purposes) Australia and New Zealand Health Policy 2005, 2:28 http://www.anzhealthpolicy.com/content/2/1/28 Table 1: Major themes around restricted access to tumour aims to elicit a basic viewpoint from a member of each necrosis factor inhibitors stakeholder group. The rheumatologist received an invita- tion to participate through an opinion-leader rheumatol- Predetermined Interview topics ogist, purposively selected to have had experiences Perceptions of PBS-restrictions on access Experience with application process prescribing TNFIs through the PBS at the time of the Pre-PBS collaboration between the different stakeholder groups study. The health advisor who participated belonged to a Post-PBS collaboration between the different stakeholder groups like-group of the PBAC, and the pharmaceutical employee Sources of information was invited to give views of the industry but was not from a company that marketed TNFIs. The patient was nomi- Major themes emerging from data nated by a rheumatologist. Participant information sheets Access to medicines and consent forms were provided to all participants prior Targeting access Review of the PBS-restrictions to the interviews. These four interviews were conducted by Implications of access arrangements in practice one trained researcher (CL); interviews were of 45 to 60 Effects on the practice of rheumatology minutes duration. Ethical issues Roles and responsibilities Interviews were recorded and transcribed verbatim. The PBS Decision-making process transcripts were coded for major concepts. Categories Stakeholder consultation process related to the discussion topics were created. Coded seg- Transparency Information provision ments were then analysed and categorised thematically, thereby developing a thematic framework. NVivo soft- ware 2.0 was used to help manage the qualitative data. undertaken as an iterative approach with each phase The transcripts were also coded independently by another building on the one before, in particular where the impact investigator (RD or KW). The researchers then met to of implementing new access criteria on practice is examine the analyses in order to reach agreement on cat- unknown. The scope and content of such a study are not egories and identified themes. Some differences with specified in advance but are developed iteratively. This regard to labelling the themes were found, but agreement paper reports on the initial phase of a qualitative study was reached on the central meanings. Interviewees were using an iterative approach. This first phase sought to offered opportunities to review an edited interview tran- explore the views of one member of each of the four stake- script to check that their views had been accurately repre- holder groups with a vested interest in any new policy sented. Only one interviewee reviewed the transcript. It about provision of medicines, in order to confirm where was accepted without change. The study was approved by further exploration should take place in the next iteration. the Human Research Ethics Committees of St Vincent's Hospital Sydney, and the University of New South Wales, Methods Australia. Qualitative techniques were used for data collection and analysis. In depth, semi-structured interviews were con- Results and Discussion ducted in Sydney in 2004 with four individuals to explore This initial phase identified a range of concerns, as held by four different perspectives about access to TNFIs under the individuals from different stakeholder groups, that arose PBS. Interviewees were asked their opinions about fea- in practice associated with the recent developments to tures of the establishment, process and effects of the control access to a group of high-cost medicines in Aus- arrangements for targeting access to TNFIs. Views on the tralia. These are imperative issues that decision makers in decision-making process and collaboration between Australia and other countries need to consider and man- stakeholder groups were also collected. age appropriately and in a timely manner. The pre-deter- mined interview topics and major themes that emerged Due to a finite number of key stakeholder representatives from the data are listed in Table 1. The four major themes who participated in the PBAC decision-making process are described below with selected quotes from partici- that formulated the arrangements for access to TNFIs, they pants. were purposively not invited in this initial phase of the study. Purposeful sampling was used to select participants 1. Access to medicines on the basis of their primary membership of different 1.1 Targeting access stakeholder groups with respect to the controlled access to Interviewees expressed an understanding for the finite TNFIs: a rheumatologist, a health advisor to the govern- resources that can be allocated to treat the possible range ment, an employee of a pharmaceutical company, and a of diseases. The principle of 'controlled access' to TNFIs patient (who had used a TNFI). Representation of partici- was supported in general. All perceived that the purpose pants was not sought in this first phase of the study which of restricting access to TNFIs was to control costs while tar- Page 3 of 8 (page number not for citation purposes) Australia and New Zealand Health Policy 2005, 2:28 http://www.anzhealthpolicy.com/content/2/1/28 geting those patients identified by the access criteria as of their disease without reaching the 'disease activity' spec- most likely to derive benefit. ified in the eligibility criteria. "The government is trying to target the therapy to those "It's forced our rheumatologists to re-look at the treat- who need it ... and control their budget." (advisor) ment that patients have already received and to re-evalu- ate ... in reassessing the patient and aiming to meet the The PBS-restrictions on access, including the limiting of PBS criteria, they actually get their rheumatoid arthritis prescribing rights to qualified specialists, were considered under control before requiring a biological..." (rheuma- to be safe and cost-effective, generally evidence-based and tologist) appropriate, given the expense and some lingering uncer- 2.2 Ethical issues tainty about long-term safety. One of the ethical issues raised by the interviewees was the "TNF inhibitors are fairly new ... there are no long-term possible benefit that might be achieved in some patients data as yet on their use ... they are not without side effects, if the PBS-restrictions would allow a switch to another and because of their expense, trying existing therapies first TNFI if they failed to respond to the first agent. is a reasonable approach" (rheumatologist) "If patients didn't meet the criteria for continuing treat- Participants disagreed with some of the specific eligibility ment and at the time they stopped, were not bad enough criteria which they interpreted as inflexible and not obvi- to meet the initial criteria for another agent, they can't ously evidence-based. For example, a number of patients switch to another agent, because they are required to meet with severe rheumatoid arthritis suffered because of the the initial criteria again." (rheumatologist). requirement that rheumatoid factor be positive. The original PBS-restrictions did not address this issue, "The requirement for a rheumatoid factor is a concern but these have been altered shortly after the completion of because I don't believe that there's any scientific evidence this study's initial phase. The PBS introduced an 'inter- ... and they [application assessors] seem to be inflexible changeability' rule from December 2004, allowing about that criterion ... There are unfortunate people who patients who meet the eligibility criteria for commencing would qualify on every criteria except rheumatoid factor therapy to trial any of the listed TNFIs as well as anakinra, alone." (rheumatologist) another anti-rheumatic biological agent [11]. This change demonstrates that some review of the PBS-restrictions is 1.2 Review of the PBS-restrictions occurring. Availability of TNFIs through the PBS, although tightly restricted, was welcomed. However, ongoing review of the Despite the 'objective' clinical measures upon which con- PBS-restrictions in accordance with emerging new evi- tinued access to TNFIs is determined, there are many fac- dence and analysis of utilisation data was seen as both tors influencing the medical condition of an individual. important and necessary. Interviewees believed the key Concerns were raised about withdrawing patients from an stakeholder representatives who had participated in the effective treatment when they may have just missed the initial stakeholder consultation should again participate threshold for continued access as well as a lack of flexibil- in these activities. The risk-mitigation agreement should ity for physicians to make decisions in cases that are also be reviewed for its effectiveness and value. equivocal based on their professional judgement. "There should be some process of review of criteria, after "There's a disparity between the patients' clinical response some agreed period, particularly if they're sharing a and joint count and their laboratory parameters ... the [financial] risk, to see whether they still continue as inflammatory markers may be staying up when they actu- appropriate based on new evidence and data of utilisa- ally clinically are a lot better." (rheumatologist) tion, etcetera." (advisor) "There's got to be a level of discretion there to the experts 2. Implications of access arrangements in practice because they're the guys who know. It's not some bureau- 2.1 Effects on the practice of rheumatology crat." (patient) The arrangements for access to TNFIs requiring evidence of exposure to and failure of a number of anti-rheumatic The application process was seen to be complex, time- drugs appear to have promoted re-evaluation of previous consuming and an administrative burden for everyone treatments and responses in patients with insufficiently involved: controlled rheumatoid arthritis. A proportion of patients may have benefited from this process with better control Page 4 of 8 (page number not for citation purposes) Australia and New Zealand Health Policy 2005, 2:28 http://www.anzhealthpolicy.com/content/2/1/28 "The administrative burden for the doctors, for the Health patients who don't strictly have rheumatoid arthritis." Insurance Commission...on the pharmacists, and also the (rheumatologist) patients...and the whole being caught up in that bureau- cratic tangle." (pharmaceutical employee) A view was put that if physicians disagree with any of the restrictions, they should lobby through their professional Another ethical concern was that a Patient Information organisation for revision because they are ultimately Sheet had not been developed to accompany the Patient responsible for their patients' well-being. More reliance Acknowledgement Form at the time these interviews took on physicians was considered appropriate and desirable. place, i.e. 12 months after the first TNFI was made availa- ble under the PBS. "Prescribers are obliged to adhere to the PBS arrange- ments... If we don't like the criteria and have concerns "They're [the patients] signing a contract and they should with it then the appropriate thing is to lobby through the be able to understand the benefits and also the implica- ARA [Australian Rheumatology Association]..." (rheuma- tions...they need to do it in a manner where they have all tologist) the information they need to decide." (advisor) Concerns were voiced about the need to achieve substan- 2.3 Roles and responsibilities tial clinical improvement for continuing access at the fol- Broadly, government is expected to provide affordable low-up assessment. This requirement may have access to effective medicines for patients with medical introduced potential perverse incentives to 'fudge' meas- needs while using taxpayers' money wisely and appropri- urements of disease activity in order to ensure continued ately. A related and necessary responsibility of govern- access to TNFIs. This raises the issue of the responsibility ment is to monitor the PBAC process to ensure and accountability of prescribers with respect to their legal appropriate assessment and selection of medicines for obligation to comply with the PBS requirements for PBS- reimbursement by the PBS. subsidised treatments. However, this does place powerful ethical concerns on doctors in cases where they become "The government has an obligation to ensure that the the agents whereby patients are denied or withdrawn from processes that the PBAC uses are rigorous, are regularly effective medicines, particularly if the patient's response is evaluated, and are well supported." (advisor) near the threshold level of eligibility. The Australia Rheu- matology Association was seen to be responsible for The pharmaceutical industry interviewee felt that infor- reviewing the PBS-restrictions and providing information mation provision by industry was a major responsibility to their members, i.e. rheumatologists, as well as the and needed particularly when high-cost medicines subject patients. to complex eligibility criteria become available through the PBS. This interviewee considered that updates should "The ARA [Australia Rheumatology Association] has an be provided to prescribers about the clinical uses of med- obligation to provide information to their own members, icines, including the application procedures for PBS-sub- it's essential that they support with adequate provision of sidised treatment; and to promote marketing and education and advice and support about this new treat- publicity with the content being concordant with the PBS- ment ... and they also have a role to provide education to restrictions (A consequence of risk-mitigation agreements the public." (rheumatologist) is a strong negative incentive for inappropriate marketing and publicity leading to use outside approved PBS-restric- The role and responsibilities of patient organisations were tions). The risk-mitigation agreement that is part of the not explored in detail in this initial phase. Consumer arrangements for access to TNFIs is an indication of col- organisations could represent patients in the consultation laboration between the government and the pharmaceuti- processes, but qualifications of the staff working in such cal company. organisations were thought to be important if they are to contribute to the consultation processes or to produce Physicians have a responsibility to comply with the PBS- educational information. Consumer organisations were restrictions by law [15]. The PBS-restrictions were worded perceived to primarily interact with patients and to dis- in such stringent and exhaustive detail that they were seen seminate information. to demonstrate a lack of faith in the prescribers by the PBS. "It's difficult to allocate the Arthritis Foundation a more active role in making recommendations, depending on "There seems ... mistrust from the PBS that rheumatolo- who is actually staffing the Arthritis Foundation and gists could have a loophole for the prescribing ... that where those opinions are coming from...the Arthritis they're worried that we will be prescribing the drug for Page 5 of 8 (page number not for citation purposes) Australia and New Zealand Health Policy 2005, 2:28 http://www.anzhealthpolicy.com/content/2/1/28 Foundation is primarily interfacing with the patients." the process, which could provide a humane view; and (rheumatologist) patients should at least be involved in the development of the Patient Acknowledgement Form. The staff at the Health Insurance Commission, a statutory organisation administering the PBS and other health pro- "No one really understands what you go through except grams [16], was considered in general to be helpful with you ... not even my rheumatologist or other specialist." respect to queries regarding the application procedure for (patient) subsidised TNFI treatments. "Patient groups should have a seat at the table...they 3. PBS Decision-making process should be represented formally, rather than by the doc- 3.1 Stakeholder consultation process tors... We need to have a process where consumers, pre- The interviewees were in substantive accord with the initi- scribers, people with interest, come together on an equal ative instituted with TNFIs, that negotiations between the footing to discuss what the issues are and what the criteria stakeholders should take place early in the decision-mak- are and can follow the progress." (advisor) ing process to enhance the likelihood that the final arrangements for access to such medicines are reasonable There is increasing need for the voices of patients, their and can be supported. carers and the public to be heard because it is appropriate and crucial for acceptance, change and improvement in "Negotiations should start early so that there is clear com- the process [17-19]. The representativeness of some con- munication between the expectation and the calibre of sumer groups was raised along with concern that some are evidence needed." (advisor) funded in part by pharmaceutical companies. This con- cern may be reduced if conflicts of interests are declared "...so that the final criteria are the correct criteria, the right openly, which links to the theme of "transparency". patient group is being identified, and also the initiation and continuation rules, etcetera, are workable and man- 3.2 Transparency ageable" (pharmaceutical employee) Transparency of the PBAC process and their decisions was a recurrent theme in the interviews. Interviewees thought However, the interviewees had different views about the that wider involvement in the consultation process and stakeholder consultation process that had taken place. greater empowerment of stakeholders to contribute to dis- cussion of the issues would be desirable. Disclosure of the "The major strength is the negotiations and the partner- rationale behind the PBAC's selection of access criteria, ing." (advisor) currently not available in the public domain, was consid- ered to be desirable and a helpful improvement to the sys- "It seems that each group has their own agenda and it's tem. Greater empowerment (and transparency) by not really like a collaboration but more each trying to put provision of appropriate information was proposed. forward their opinion." (rheumatologist) "A public document which summarised why different While the representativeness of the Australian Rheumatol- decisions were made, and the evidence base on which ogy Association Therapeutics Sub-committee in the deci- they were made would be a most fruitful thing to help sion-making process was believed to be appropriate, a patients to understand why they have potentially limited wider consultation among ordinary members of the asso- access to some of these medicines ... that's about empow- ciation was wanted: erment in information." (advisor) "The therapeutics committee should have sought infor- In addition, the issue of the requirement for a positive mation from the members of the ARA [Australian Rheu- rheumatoid factor has been the subject of debate since the matology Association], or have consulted more widely PBS-restrictions for TNFIs were implemented [20,21]. before they made their decisions because it seems that Greater transparency would be helpful to resolve these they made their decisions on our behalf but without con- concerns and gain increased community support. The sulting us." (rheumatologist) recent recommendation by PBAC to remove this criterion [22] is a welcome and rational outcome of the lengthy On the subject of representing the views of patients in debate on this issue. such processes, the rheumatologist was of the opinion that doctors would appropriately represent the views of The interviewees who participated in this study were 'out- their patients. On the contrary, the three other partici- siders' to the consultation process that led to establish- pants felt that patients should have a more direct role in ment of the arrangements for access. Minimal Page 6 of 8 (page number not for citation purposes) Australia and New Zealand Health Policy 2005, 2:28 http://www.anzhealthpolicy.com/content/2/1/28 involvement in the decision-making process and the cur- Interviewees had different views on whose responsibility rent level of transparency around PBAC's decisions lim- it was to provide information. The rheumatologist consid- ited their support for some of the eligibility criteria. ered that information about the efficacy and safety of Further, their minimal awareness of the negotiations that TNFIs is most important and that the Australian Rheuma- took place between key stakeholders suggests that there is tology Association would be appropriate to provide ongo- a need for better communication between those key stake- ing updates. Other proposed providers of information: holder representatives with their constituencies. Increased pharmacists, the Medicare Australia (previously the communication within and between the stakeholder Health Insurance Commission), the PBS, the National groups is also critical to obtain stronger support for the Prescribing Service (an independent educational organi- final PBS-restrictions in practice. sation funded by the Federal Government) [23], and the consumer organisations. 4. Information provision The PBS-restrictions to control access to high-cost medi- Further investigation cines, exemplified by TNFIs, are particularly complex, and The initial phase of the study has explored concerns of thus adequate provision of information is all the more individuals from the four relevant stakeholder groups. important. Information from pharmaceutical companies Our study was not designed to support statistical general- to the prescribers and general practitioners were noted to isability. Findings have established the basis for the next be abundant, however, interviewees considered that the phase of the study to explore perceptions of a wide range content needed to be more balanced. In particular, the of stakeholders that will be helpful in gaining a compre- rheumatologist and the government advisor expressed a hensive and broad societal view of these significant devel- lack of faith in the pharmaceutical companies' ability to opments of controlling access to high-cost medicines. A supply appropriate materials for patients or the public. written interview guide has been developed on the basis of the findings of this phase for use in subsequent inter- "It is not the sponsor's responsibility to talk to patients views. Diversity in sample characteristics and inclusion of because they will always get it wrong... direct-to-consumer individuals who were engaged in the decision-making advertising is a crazy way to go..." (advisor) process will expand understanding of the issues affecting different members of the stakeholder groups and provide Some concern was also expressed about industry-funded some insight into the negotiations during the decision- 'educational' functions: making process. Interviewees will include government advisors, participants in the stakeholder consultation "The way it was presented was one-sided, that the govern- process that formulated the arrangements for access to ment has put all these barriers forward, the barrier nature TNFIs (who are in a position to comment on what actu- was highlighted..." (advisor) ally occurred and what could be improved), rheumatolo- gists and patients who have gone through the application Interviewees' had concerns about the understanding of process, pharmaceutical company personnel, consumer the PBS system and the PBAC process among health pro- organisation representatives, and government administra- fessionals and in the community. Better provision of tion staff. We anticipate that this broad range of interview- information on the structure of the PBS and the PBAC ees will provide deep and rich insights to decision makers process was proposed. In particular, interviewees noted regarding the future decision-making process and devel- that doctors are the most appropriate health professionals opment of national arrangements to manage access to to inform patients with regards to the PBS-restrictions, medicines from the perspectives of enhancing appropriate and that they therefore need to be better informed about access to effective and expensive medicines. the cost-effective analyses that underpin the PBAC deci- sions. For example, patients should be made aware that Conclusion self-funding is an option, although in the case of TNFIs The PBS has developed a set of arrangements to target this is impractical for most people. Interviewees proposed access to expensive medicines such as the TNFIs in an that communication about the need to targeting use of attempt to sustain this national drug reimbursement sys- highly specialised medicines should extend to (potential) tem. Findings from this initial phase of the study high- patients and the public generally, and also to general prac- lighted a number of issues associated with the restricted titioners who could have an important role and contribu- access to TNFIs. The principle of 'controlled access' to tion in this area. Increased community awareness and TNFIs was supported in general by all interviewees each understanding of the principle of quality-use-of-medi- from a different stakeholder group. However, it is clear cines should be part of the overall educational strategy. that some views, such as those concerning patient direct participation or the varying responsibilities of each stake- holder group, were different. The pre-determined ques- Page 7 of 8 (page number not for citation purposes) Australia and New Zealand Health Policy 2005, 2:28 http://www.anzhealthpolicy.com/content/2/1/28 5. Productivity Commission: International pharmaceutical price differences: tions appeared to adequately explore the key issues. A research report Canberra: AusInfo; 2001. thematic framework has now been developed on the basis 6. Sansom L: The Australian National Medicinal Drug Policy. of the major themes that emerged from the interviews. We Journal of Quality Clinical Practice 1999, 19:31-35. 7. Australian Institute of Health and Welfare (AIHW): Health expenditure believe there is a need for a more comprehensive explora- in Australia 2002–03. AIHW Cat No HWE 27 (Health and Welfare tion, through a second phase of the study, to elicit differ- Expenditure Series No 20) Canberra: AIHW; 2004. 8. Symmons DP, Silman AJ: Anti-tumor necrosis factor alpha ther- ent perspectives across a variety of stakeholders, that will apy and the risk of lymphoma in rheumatoid arthritis: no in turn be helpful in guiding policy and practice around clear answer. Arthritis & Rheumatism 2004, 50:1703-1706. national arrangements to manage access to high-cost 9. Fleischmann RM, Iqbal I, Stern RL: Considerations with the use of biological therapy in the treatment of rheumatoid arthritis. medicines. Expert Opinion on Drug Safety 2004, 3:391-403. 10. Lu CY, Williams K, Day R, March L, Sansom L, Bertouch J: Access to Australia's Pharmaceutical Benefits Scheme, as part of the high cost drugs in Australia: Risk sharing scheme may set a new paradigm. BMJ 2004, 329:415-416. National Medicines Policy, is committed to providing 11. Commonwealth Department of Health and Ageing: Sched- access to cost-effective medicines to the community. ule of Pharmaceutical Benefits for Approved Pharmacists and Medical Practitioners (1 April 2005) [http:// When resources are constrained, restricting access is an www1.health.gov.au/pbs/] inevitable outcome. Greater and more trust-based com- 12. Vuorenkoski L, Toiviainen H, Hemminki E: Drug reimbursement munication and discussion among a wide range of stake- in Finland – a case of explicit prioritising in special catego- ries. Health Policy 2003, 66:169-177. holder groups will enhance community awareness and 13. Singer PA, Martin DK, Giacomini M, Purdy L: Priority setting for support to limit access to effective medicines. Acceptable new technologies in medicine: qualitative case study. BMJ 2000, 321:1316-1319. but different approaches to control access and manage the 14. Wirtz V, Cribb A, Barber N: Reimbursement decisions in health associated implications should be explored with the goal policy – extending our understanding of the elements of of enhancing health outcomes for all. decision-making. Health Policy 2005, 73:330-338. 15. Australian Government, The National Health (Pharmaceu- tical Benefits) Regulations 1960 (made under The National Declaration of competing interests Health Act 1953) [http://www.comlaw.gov.au/] Christine Lu and A/Prof Jan Ritchie have nothing to 16. Medicare Australia [http://www.medicareaustralia.gov.au/] 17. Wiseman V, Mooney G, Berry G, Tang KC: Involving the general declare. A/Prof Ken Williams is a member of the Advisory public in priority setting: experiences from Australia. Social Board to the sponsor for adalimumab. Prof Ric Day is a Science & Medicine 2003, 56:1001-1012. 18. Cayton H: Patient and public involvement. Journal of Health Serv- member of the Advisory Board to sponsors for adalimu- ices Research & Policy 2004, 9:193-194. mab, infliximab, and anakinra in Australia. A/Prof Wil- 19. Florin D, Dixon J: Public involvement in health care. BMJ 2004, liams and Prof Day have also been contracted to 328:159-161. 20. Lu CY, Williams KM, March L, Bertouch JV, Day RO: Subsidised undertake clinical trials with etanercept, infliximab, adal- access to TNF-alpha inhibitors: is the rationale for exclusion imumab, and anakinra. Recompense for these activities of rheumatoid factor negative patients defensible? Medical received was placed in audited hospital trust funds for use Journal of Australia 2004, 181:457-458. 21. Mann EL: Subsidised access to TNF-alpha inhibitors: is the in the research activities of the Clinical Pharmacology rationale for exclusion of rheumatoid factor negative Department, St Vincent's Hospital, Sydney. patients defensible? Medical Journal of Australia 2005, 182:47-48. 22. Commonwealth Department of Health and Ageing: Recom- mendations made by the Pharmaceutical Benefits Advisory Authors' contributions Committee in March 2005 [http://www.health.gov.au/internet/ CL, KW, and RD have made substantial contributions to wcms/publishing.nsf/Content/pbacrec-pbacrecmar05] 23. Weekes LM, Mackson JM, Fitzgerald M, Phillips SR: National pre- the conception and design of the study, analysis and inter- scribing service: creating an implementation arm for pretation of the data; and drafting and revising the manu- national medicines policy. British Journal of Clinical Pharmacolology script. JR has been involved in advising on the analysis, 2005, 59:113-116. drafting the manuscript and revising it critically for intel- lectual content. All authors read and approved the final manuscript. Publish with Bio Med Central and every Acknowledgements scientist can read your work free of charge The authors thank the interviewees for their participation in this research. "BioMed Central will be the most significant development for disseminating the results of biomedical researc h in our lifetime." References Sir Paul Nurse, Cancer Research UK 1. Organisation for Economic Co-operation and Development (OECD): OECD Health Data: Statistics and indicator for 30 countries 2005. Your research papers will be: 2. Commonwealth Department of Health and Aged Care: The Australian available free of charge to the entire biomedical community health care system: an outline. Canberra 2000. 3. Sansom L: The subsidy of pharmaceuticals in Australia: proc- peer reviewed and published immediately upon acceptance esses and challenges. Australian Health Review 2004, 28:194-205. cited in PubMed and archived on PubMed Central 4. Henry D: Economic analysis as an aid to subsidisation deci- sions: the development of Australian guidelines for pharma- yours — you keep the copyright ceuticals. Pharmacoeconomics 1992, 1:54-67. 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Australia and New Zealand Health PolicySpringer Journals

Published: Nov 23, 2005

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