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The views of stakeholders on controlled access schemes for high-cost antirheumatic biological medicines in Australia

The views of stakeholders on controlled access schemes for high-cost antirheumatic biological... Background: In Australia, government-subsidised access to high-cost medicines is "targeted" to particular sub-sets of patients under the Pharmaceutical Benefits Scheme to achieve cost-effective use. In order to determine how this access system could be improved, the opinions of key stakeholders on access to biological agents for rheumatoid arthritis were explored. Methods: Thirty-six semi-structured interviews were conducted with persons from relevant stakeholder groups. These were transcribed verbatim, and analysed thematically. Results: Controlled access to expensive medicines was considered to be equitable and practical; however, there was disagreement as to the method of defining the target patient populations. Other concerns included timeliness of access, excessive bureaucracy, and the need for additional resources to facilitate the scheme. Collaboration between stakeholders was deemed important because it allows more equitable distribution of limited resources. The majority considered that stakeholder consultation should have been broader. Most wanted increased transparency of the decision-making process, ongoing and timely review of access criteria, and an increased provision of information for patients. More structured communication between stakeholders was proposed. Conclusion: The Pharmaceutical Benefit Scheme is adapting to meet the changing needs of patients. Provision of subsidised access to high-cost medicines in a manner that is affordable for individuals and society, and that is equitable and efficiently managed is challenging. The views of stakeholders on targeted access to anti-rheumatic biological medicines in Australia acknowledged this challenge and provided a number of suggestions for modifications. These could serve as a basis to inform the debate on how to change the processes and policies so as to improve the scheme. prescribers. In Australia, government-subsidised access to Introduction Drug reimbursement systems grapple with the challenges prescription medicines is provided through the Pharma- of funding innovative but costly medicines in the face of ceutical Benefits Scheme (PBS). Decisions on drug sub- increasing cost constraints, in part due to rapidly increas- sidy are based on assessment by the Pharmaceutical ing demands for access to these drugs by consumers and Benefits Advisory Committee (PBAC), which evaluates Page 1 of 9 (page number not for citation purposes) Australia and New Zealand Health Policy 2007, 4:26 http://www.anzhealthpolicy.com/content/4/1/26 incremental effectiveness and cost-effectiveness of the sidised access to the anti-rheumatic biologicals is only medicine [1]. Criteria for access to PBS listed medicines provided for patients who meet criteria for both starting are based on clinical trial evidence and economic evalua- and continuing therapy. These criteria include both clini- tions. cal and laboratory based measures (Table 1) [2]. Other biologicals subsequently subsidised by the PBS for treat- Increasingly, there is collaboration between the PBAC, the ing RA are: infliximab, adalimumab, anakinra, and rituxi- sponsor, and medical organisations (e.g. Australian Rheu- mab. matology Association) to develop access criteria. A repre- sentative example of such a collaboration is the 'Stakeholders', for the purpose of this study, were defined consultation process that contributed to the decision to as individuals or groups of people having the potential to subsidise and the criteria to access etanercept, a high-cost influence the decisions on the access arrangements, as biological medicine for treating rheumatoid arthritis well as those affected by the PBS restrictions. In reviewing (RA). This process set a new paradigm for analogous PBS the published literature, no studies apart from our own decisions [2,3]. A broad definition of "high-cost medi- have examined the views of stakeholders regarding this cines" in Australia is medicines whose acquisition cost is novel approach to control subsidised access to high-cost greater than A$10,000 per patient per treatment course medicines. This study examined the perceptions and expe- [4]. Although not directly involved in the consultation riences of stakeholders with respect to access to high-cost process, there was considerable support and lobbying medicines under Australia's PBS using, as an example, the activities by consumer representatives (namely the Arthri- anti-rheumatic biologicals. tis Foundation of Australia) for 'targeted' access to high- cost biologicals. Access to high-cost medicines under the Methods PBS is tightly regulated, requiring approval by Medicare In-depth semi-structured interviews were conducted with Australia (a government body that administers the PBS) participants drawn from the stakeholder groups that were before it can be prescribed with subsidy by a doctor. Sub- involved in access to biologicals through the PBS. Based Table 1: Access arrangements for biological agents for RA under the PBS Authority requirements Criteria for initiating • Severe active disease: treatment a) elevated levels of inflammatory markers (ESR > 25 mm/hour or CRP > 15 mg/L) b) swollen and tender joints – a total of > 20 joints, or > 4 major joints (elbow, wrist, knee, ankle, shoulder, hip) • A record of rheumatoid factor positive status (this requirement was removed as of June 2005) • Failure to achieve adequate response to a step-up sequence of treatment with conventional DMARDs: a) monotherapy with methotrexate (20 mg per week) b) a combination of methotrexate (> 7.5 mg per week) and 2 other DMARDs for at least 3 months c) leflunomide, leflunomide with methotrexate, or cyclosporin for at least 3 months • Evidence of intolerance or contraindication to DMARDs • Patients required to sign a 'patient acknowledgement form' • Treatment is approved for 16 weeks only (treatment of 22 weeks is approved for infliximab) A patient agreement process • A Patient Acknowledgement Form to be signed by patients to acknowledge that PBS subsidised treatment will only continue if the predetermined response criteria are achieved at 12 weeks Criteria for continuing treatment • Clinical outcomes are evaluated according to predetermined quantifiable criteria at 12 weeks: a) Reduction in levels of inflammatory markers, ESR < 25 mg/hour, or CRP < 15 mg/L, or 20% from baseline levels b) Reduction in the total number of joint count by 50% 'Interchangeability' (introduced • Patients approved to commence PBS subsidised biological treatment are allowed to switch to an December 2004) alternate biological agent at any time Restricted prescribing rights • Prescription only by specialist rheumatologists initially. Prescribing rights were extended to clinical immunologists with expertise in the management of RA as of February 2004 'Risk-sharing' arrangement • Annual PBS expenditure for the tumour necrosis factor inhibitors group was predicted to be up to A$140 million • Expenditure above this figure to be covered by the sponsoring pharmaceutical companies (details not clear from public documents) ESR = erythrocyte sedimentation rate CRP = C-reactive protein DMARDs = disease-modifying anti-rheumatic drugs Page 2 of 9 (page number not for citation purposes) Australia and New Zealand Health Policy 2007, 4:26 http://www.anzhealthpolicy.com/content/4/1/26 on our definition of "stakeholders" in the case of anti- The study was approved by the Human Research Ethics rheumatic biologicals, interviewees included were rheu- Committees of St Vincent's Hospital Sydney, and the Uni- matologists, patients with RA, government advisors, con- versity of New South Wales, Australia. sumer advocates, public servants, and pharmaceutical company spokespersons. Interviewees were asked to Results declare any potential conflicts of interest, that is, any pre- Thirty-six interviews were conducted between 2004–2005 vious or current advisory role with a pharmaceutical com- (Table 3). None of the government advisors, public serv- pany, or in any other committees or organisations that ants, consumer representatives, or patients had held advi- have a vested interest in the PBS listings. sory positions or roles on behalf of a pharmaceutical company. Four of the eight rheumatologists had been or Purposeful sampling of 'information-rich' individuals were on at least one advisory committee of a pharmaceu- [5,6], especially those involved in formulating the criteria, tical company. Marked differences were not found in the was undertaken. This led to inclusion of additional inter- views of rheumatologists who had held an advisory posi- viewees i.e. the so-called 'snowball sampling technique' tion with a pharmaceutical company versus those who [5]. Interviews were conducted using a guide based on had not. Most rheumatologists (7 out of 8) had been in findings from the first phase of the study [7] by the same practice for more than 10 years; the number of patients investigator (CL), a research pharmacist. The main ques- each rheumatologist treated with biologicals under the tions put to interviewees are listed in Table 2. Each inter- PBS ranged from 2 to 16. Most patients (5 out of 6) were view, lasting 45–80 minutes, was recorded and prescribed etanercept. One nurse was included in the transcribed verbatim. study opportunistically. NVivo 2.0 software was used to manage the data and to Five major themes emerged: resource rationing, excessive assist the coding for major concepts arising inductively bureaucracy, partnerships and inclusive decision-making, edu- from the data. Three investigator independently catego- cation, and review. All were related to the perceived fairness rised these concepts thematically before meeting to reach of the process and were essential determinants of support consensus on theme choice [8,9]. for the access criteria by stakeholders. A variety of techniques was used to confirm the methodo- 1. Resource rationing logical rigour. These included verifying the meaning Providing access to expensive medicines through a pub- ascribed to interviewees by offering them the opportunity licly-funded system (the PBS) was seen by all participants to review edited transcripts [9], using researcher triangula- as equitable and a feasible approach. Targeting access was tion to arrive at consensus on interpretation of the data supported as a necessary form of "resource rationing". The [9], using triangulation of multiple data sources (namely, vast majority acknowledged that there were finite individuals from different stakeholder groups) to counter- resources available for the possible range of diseases. Tar- balance weaknesses of one source by strengths in another geting access to sub-groups of patients who most need [9], searching for negative or discrepant cases to enhance treatment and would gain most benefit was in keeping interrogation of the data, and continuing to add inter- with spending "for value". However, interviewees disa- views until overall data saturation was achieved [10]. greed about how the appropriate target patient popula- Table 2: Main interview questions 1. What are the sources of information you used before prescribing the drug, or during the course of the treatment? 2. What do you see as the primary objective of the PBS arrangements for access? 3. What do you see as the strengths and weaknesses of the access scheme? 4. Have you been involved in the consultation process in regards to developing restrictions or arrangements to access the TNF inhibitors via the PBS? If yes, i. Can you briefly describe your role in the consultation process? ii. Who else took part in the consultation process? 5. Who do you think should take part in the consultation process for formulating the arrangements or restrictions for access? 6. What is the extent of your contact with other rheumatologists, local general practitioners, administrators, consumer organization, the PBAC? And what were the purposes of these contacts? 7. What do you see as the role and responsibility of the prescribers? The PBAC? The industry? Arthritis Foundation? 8. Who, in your view, has responsibility in informing/'educating' the prescribers (and the public) regarding PBS restriction changes, or new complex PBS restrictions? 9. How important an advance do you think the consultation approach and access arrangements represent? 10. If a new and expensive drug comes along that is a significant advance for the treatment of a chronic disease, what differences would you like to see in the process of getting it listed and using it? Page 3 of 9 (page number not for citation purposes) Australia and New Zealand Health Policy 2007, 4:26 http://www.anzhealthpolicy.com/content/4/1/26 Table 3: Characteristics of interview participants (n = 36) Number of participants Age group 18–29 years 1 30–39 years 5 40–49 years 8 50–59 years 14 60 years and over 8 Sex Male 13 Female 23 Stakeholder group Rheumatologist 8 Patient 6 Government advisor 5 Public servant 8 Consumer representative 5 Pharmaceutical industry representative 3 Clinical nurse 1 tion should be defined. A government advisor recognised immunologists) was deemed safe and appropriate, given the tensions between system-wide and individual needs: the expense of the medicines, the severity of RA in the affected patient population, and long-term safety con- "It [controlled access] is entirely defensible in terms of cerns. A patient voiced the predominant view: a population utility concept. It's very difficult at an individual patient level." "I think it's good that people have to have tried all the other cheaper medicines, that's fair enough. Why Some participants suggested broader, initial access. The should the government pay for a really expensive med- risk of excessive uptake of the biologicals by patients out- icine when methotrexate works and also, we don't side the PBS criteria was considered, by these participants, know yet what the long-term side effects will be." to be limited given the other controls that were applied, namely, risk-sharing between sponsors and government While monitoring patient outcomes was supported, via price-volume agreements, and the continuation rule that patients were apprehensive that an effective treatment limited ongoing access to those who responded to treat- might be withdrawn if one missed the threshold response ment. All agreed that the criteria should be based on to qualify for ongoing treatment. Further, prescribers sound clinical evidence. However, some criteria were seen might feel pressured to 'modulate' measurements of dis- as "arbitrary" and "potentially unfair" particularly when ease activity to benefit individual patients while, in effect, they were "unsupported by published literature". A rheu- denying treatment for others. Some interviewees sug- matologist gave his opinion: gested that the continuation rule be removed, and others proposed increased flexibility with respect to the timing of "I think ... the restriction to seropositive patients is the assessment, including less frequent assessment. A rep- unfortunate. Secondly, the reliance on laboratory indi- resentative quote from one rheumatologist illustrates this ces is a concern ... " view: The requirement for 'positive rheumatoid factor' (serop- "I think a system that had more flexibility for the ositive) was removed shortly after the completion of this renewal, possibly by making the reapplication less fre- study and such opinions may have been rescinded subse- quent, or having a larger window within which the quently. appropriate blood test could be acquired, or allowing so-called unacceptably high ESR [erythrocyte-sedi- The requirement to first trial existing, less costly therapies mentation-rate] or CRP [C-reactive-protein] or joint was considered to be reasonable. Limiting prescribing count for a period of time, until things settle down rights to sub-specialist physicians (rheumatologists, Page 4 of 9 (page number not for citation purposes) Australia and New Zealand Health Policy 2007, 4:26 http://www.anzhealthpolicy.com/content/4/1/26 again... a little less anxiety-provoking for the patients order to obtain ongoing supply. Physicians experienced would be good." difficulties in locating documents including records of laboratory tests and details of treatment history, some of However, government advisors and public servants which depended upon other clinicians who had treated believed that the interpretation of joint tenderness and patients previously. However, improved documentation swelling by clinicians and patients, being subjective, was seen as a good outcome overall, and an application allowed some flexibility. Further, they believed that for access was easier for more recently diagnosed patients. assessment of eligibility was flexible because applications for subsidised treatment were assessed by pharmacists and Prescribers were uneasy that there was no recompense for medical advisors from Medicare Australia, the responsible the substantial additional time to undertake the tasks to agency. Both prescribers and patients believed that clini- apply for access for their patients. Assistance (e.g. from cians who see individual patients should have such discre- nurses) was in general unavailable as the majority of rheu- tion because, as a patient put it: "people in government matologists in Australia work in private practice. Where aren't educated about the disease [RA]". A government nurses were available, considerable workload was advisor voiced the opposing view about allowing flexibil- imposed on them. A nurse was concerned about the ity with borderline cases: impact on hospital resources: "the problem with the high-cost drugs is it's very diffi- "In a lot of places I think that nurses are doing a lot of cult to be flexible because small changes in flexibility the work. And there's nowhere that this has been taken start to blow budgets out to massive amounts of into consideration with staff and issues and so on. It's money and that means that the whole principle of added at least eight hours a week to my workload. ...." equity and equitable access to these things are not obeyed. ... I don't think the uncertainty can be borne There was also an increase in the use of other resources. A only by Australian taxpayers." public servant noted that greater resources were needed to administer this complex scheme: The Patient Acknowledgement Form was seen as a contract between the patient and the government, and reduced the "We've got pharmacists on call and they are paid more direct pressure on the individual rheumatologists. Some than admin staff would be paid. We have to go rheumatologists and patients saw this agreement as through a more intensive process in order to approve "pointless" because treatment would be discontinued the application because we lose more money if we even if the patients disagreed, and it was unlikely that approve it wrongly." patients would wish to continue if the treatment was inef- fective. A common view of patients was: "when you're des- The requirement that there be an adequate trial of conven- perate you'll sign anything." tional anti-rheumatic drugs promoted re-evaluation of previous treatments and a more comprehensive and 2. Excessive Bureaucracy aggressive use of anti-rheumatic drugs. Patients were more The access scheme controlled the usage and expenditure easily convinced to go through these steps with the 'incen- of biologicals effectively in the first years of operation. tive' that they might access the 'new' biologicals. A rheu- This was seen as a good outcome by the majority. How- matologist voiced the predominant view: ever, there was concern by some that the use of biologics was inappropriately low. "It's forced our rheumatologists to re-look at the treat- ment that patients have already received ... in reassess- Most had the view that medical care had become "more ing the patient and aiming to meet the PBS criteria, bureaucratic" as a result of the PBS processes. A rheuma- they actually get their rheumatoid arthritis under con- tologist commented: trol before requiring a biological..." "It seems to be a very bureaucratic way of giving med- The majority considered that this effect was beneficial and ical care, rather than a normal doctor-patient relation- potentially systematised the treatment of RA nationwide. ship... it's eliminating the ability of the doctor to show some level of discretion..." 3. Partnerships and inclusive decision-making There was uniform support for the stakeholder collabora- The majority thought the application process was admin- tion and increased communication that contributed to istratively burdensome. Patients were anxious about coor- the decision to subsidise biologicals. However, while gov- dination of laboratory tests, joint assessment, application ernment advisors and public servants commented that the forms, and ordering the medicine from pharmacies in representative rheumatologists were cooperative, the Page 5 of 9 (page number not for citation purposes) Australia and New Zealand Health Policy 2007, 4:26 http://www.anzhealthpolicy.com/content/4/1/26 companies felt that the representative rheumatologists them-and-us system, this is our system." (government were less willing to collaborate. The consumers felt that advisor) communication with the Rheumatology Association and the government was insufficient. Further, some interview- A comment by a rheumatologist summarised the two key ees suggested that consultation among rheumatologists elements for future processes: should be wider. An industry spokesperson commented on the negotiation skills of the representing rheumatolo- "I would like to see a more collaborative and more gists: open approach, because I think a lot of the negotia- tions that eventually resulted in this listing were, "I don't think they [rheumatologists] had the skills, whether deliberately or accidentally, shrouded in which is built up over experience, to be able to handle some secrecy and I know from the PBAC perspective negotiations with government. People like 'gastros' there's been some talk of deliberations being made have had good experience over quite a long time with more transparent. If we were doing this process again new therapies, and oncologists and cardiologists as and if I had the power to change something, those are well. Rheumatologists are babes in the wood when it the things I would like to see: direct consumer repre- comes to dealing with government." sentation and an open and transparent process that we could watch evolving." Some believed that doctors would adequately represent 4. Education the views of patients while the majority view was that patients should have a more direct role. A common view A common view of stakeholders was that the clinicians at was that patient representatives would need to be care- Medicare Australia readily assist with queries regarding fully selected and well-informed. Interviewees had mixed applications. However, the majority felt that better provi- opinions about when patients should be involved in the sion of information on the criteria and application proce- process, but that they should at least be involved in devel- dures, particularly in the early stages of implementation, oping the Patient Acknowledgement Form. Some inter- was important. In addition to avoiding confusion and viewees believed that negotiation between the three increasing efficiency, it would enhance support for the primary groups (PBAC, medical specialists, companies) access criteria. was sufficient and would avoid difficulties and lengthy negotiations potentially associated with wider consulta- Concerns were expressed about insufficient information tion. for patients and inadequate clinical knowledge of the bio- logicals among health professionals in general. Clinicians An urgent need recognised by the majority was for open emphasised the importance of providing information on discussion to engage the broader community to work out adverse reactions, notably risks of infection. This was the overarching principles of allocation and access to potentially a difficult task because some patients feared medicines. A government advisor voiced the predominant that treatment might be stopped if they reported infec- view: tions. Most patients felt that quality-of-life outweighed other considerations including the potential risks of treat- "The population as a whole needs to have some ment. debate about where they want to spend their money, they are the ones that are funding it ultimately..." The industry interviewees felt that education was a major responsibility of industry particularly when medicines are Greater transparency around deliberations between stake- subject to complex criteria for access. It was agreed that holders and the rationale behind the selected criteria, cur- company representatives had been helpful in providing rently constrained by confidentiality requirements, was information to clinicians. However, most interviewees considered essential to enhance understanding of the sys- expressed a lack of faith in the companies to supply tem and to enable the PBAC to better defend its decisions appropriate materials for patients or the public. Concerns and garner broader support. were also raised about fragmented and misleading mes- sages delivered by the media. "We need greater transparency. It's [important] people understanding the system, taking some ownership of Enhanced provision of information on the rationale for the system, involving them through transparency, restricting access, how criteria were decided, and the dis- through education, being prepared to speak to them. tinction between "effectiveness" and "cost-effectiveness" The PBAC should defend the decisions. It's all to do of medicines was advocated. The Patient Acknowledgement with dialogue, with the partnerships. This is not a Form represented an opportunity to increase patient understanding that there were responsibilities and risks to Page 6 of 9 (page number not for citation purposes) Australia and New Zealand Health Policy 2007, 4:26 http://www.anzhealthpolicy.com/content/4/1/26 be shared by all parties. Most thought that information Discussion provided by the Australian Rheumatology Association By including individuals who represented the range of (clinical information on the medicines) and the National stakeholder groups with their different and potentially Prescribing Service (information about the PBS and its competing interests, this study sought to present a well- decisions) would be most trustworthy. Consumer organi- rounded picture of the access scheme. In-depth insights sations were identified as having a role in disseminating into stakeholders' views provide a basis to inform the information and providing consumer support. Interview- broader debate on how subsidy systems for high-cost ees suggested that general practitioners and other health medicines can be improved. The major consensus finding professionals, such as pharmacists, could be more was that while the the current access scheme for anti-rheu- involved in educating patients. matic biologicals can be viewed as successful, stakeholder communication and involvement needs to be increased. A 5. Review limitation of the study was that insights into some mana- Regular review of access criteria was recommended. Drug gerial perspectives were not obtained because administra- utilisation patterns and feedback from stakeholders tors who assessed patient applications for access declined should be analysed to refine the access arrangements. The to participate due to concerns about privacy. risk-sharing agreement should also be reviewed. The proc- esses of PBAC decision-making and stakeholder consulta- The innovations introduced to establish the access scheme tion would also benefit from regular evaluation. A public were considered concordant with the expectation that servant also proposed examining the administrative government is responsible for providing access to effective expenses associated with such a scheme: medicines while balancing the need to use public resources wisely. Limiting access to sub-sets of patients, "The administration costs [of Medicare Australia] are where need has been established and where use was cost- not a consideration for the PBAC. We need to do some effective, was viewed as practical and equitable. The stake- cost-benefit analysis around the cost of building the holders were supportive of the proposition that it is possi- appropriate systems [of access] and the benefit in ble to make high-cost medicines available and affordable reduction of use [of medicines] on PBS." for the community and individual patients. That this had been achieved with these drugs was accepted as an impor- Some were disappointed that a prospective, formal evalu- tant accomplishment. However, timeliness of access to ation of the access scheme had not been established. This innovative medicines via the PBS in Australia, in compar- was partly because stakeholders could not reach consen- ison to comparable countries, was a concern. This was sus on who should be responsible or what were appropri- partly due to the registration process which often occurred ate funding sources. Australian rheumatologists had later in Australia than in the USA or Europe. A definition implemented independently a patient registry to track of "timeliness of access to medicines" by interviewees of patient outcomes. Some interviewees suggested that gov- the six stakeholder groups was not actively explored in ernment and the pharmaceutical industry should fund this study; an important issue that warrants further inves- such a registry collaboratively. The fact that a comprehen- tigation. sive information system enabling evaluation had not been developed was a concern for most interviewees. Criteria for access were seen to be potentially 'unfair' when the evidence was not publicly available. For exam- Some rheumatologists and patients considered it a weak- ple, the requirement for rheumatoid-factor-positive-status to ness that a review panel for contentious cases had not gain access was contentious at the time of the interviews. been established, while others felt that, to a degree, special Most published pivotal studies did not exclude rheuma- consideration had occurred informally via interactions toid-factor-negative patients or analyse them as a sub- between Medicare Australia and rheumatologists. A group. There was insufficient evidence in the public patient voiced frustration about the delayed review of the domain to support the view that rheumatoid-factor-posi- PBS criteria: tive-status was a factor predicting a better response to treatment with etanercept [11]. However, efficacy in rheu- "I would like a true appeal system. I would like who- matoid-factor-negative patients had not been clearly ever's in power to try and rectify the situation quickly established because of the small number of such patients and that when they put in future criteria for any med- in these trials [12]. This requirement for rheumatoid fac- icine, that they are very, very careful about the crite- tor positivity has subsequently been removed. Sustaina- ria." bility of a subsidy system such as the PBS is dependent upon a rigorous and consistent process of drug review and increased transparency around the rationale underpin- ning decisions [13]. How the PBAC arrives at its recom- Page 7 of 9 (page number not for citation purposes) Australia and New Zealand Health Policy 2007, 4:26 http://www.anzhealthpolicy.com/content/4/1/26 mendations for PBS listing was significantly limited by the to administer systems of access; and improving communi- "commercial-in-confidence" restrictions at the time of cation and information based on increased transparency. etanercept listing. An important milestone in this respect is that summaries of PBAC decisions have become availa- Competing interests ble publicly recently [3,14]. Increasing transparency also CL and JR have nothing to declare. KW has been a mem- increases accountability of all parties for decisions and ber of the Advisory Board to the sponsor for adalimumab. performance of the system. These moves towards trans- RD has been a member of the Advisory Board to sponsors parency were supported by the participants. for adalimumab, infliximab, and anakinra in Australia. KW and RD have also been contracted to undertake clini- Open dialogue and declaration of potential conflicts of cal trials of etanercept, infliximab, adalimumab, and ana- interest are important to building trust [15]. Increased kinra. Recompense for these activities is placed in audited communication and collaboration between stakeholders hospital trust funds for use in the research activities of the have been crucial steps that were initiated during the Clinical Pharmacology Department, St Vincent's Hospital, effort to subsidise the anti-rheumatic biologicals. How- Sydney. ever, the consumer participants identified a need for increasing the voice of patients and the public in order to Authors' contributions enhance the quality of decisions, and acceptance of access CL, KW, and RD have made substantial contributions to criteria, a position supported by the majority of partici- the conception and design of the study, analysis and inter- pants. pretation of the data, and drafting and revising the manu- script. JR has been involved in advising on the analysis, Increasing bureaucratic requirements are a threat to the drafting the manuscript and revising it critically for intel- acceptance and efficient running of such access systems. lectual content. All authors read and approved the final The administrative burden imposed on prescribers can be manuscript. a barrier to enrolling deserving patients and a source of increased costs [16]. Most stakeholders, with the excep- Acknowledgements The authors thank the interviewees for their time and participation in this tion of the government advisors and public servants, pro- research. posed that more reliance on physicians' integrity to comply with PBS criteria should be considered an appro- Funding/support: CL was supported by an Australian National Health & priate approach and would benefit patients. Medical Research Council postgraduate scholarship (Grant No. 351040). A significant gap in knowledge was identified by health References professionals (namely, general practitioners, pharmacists 1. Commonwealth Department of Health and Aged Care: The Aus- tralian Health Care System, An Outline. Canberra, ACT, Pub- and nurses) and the community about the PBS and the lications Unit, Commonwealth Department of Health and Aged Care; increasing need to control access to medicines, in particu- lar, high-cost medicines. General practitioners and phar- 2. 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Patton MQ: Qualitative research and evaluation methods. 3rd processes [17]. edition. Thousand Oaks, CA, Sage Publications; 2002. 6. Pope C, van Royen P, Baker R: Qualitative methods in research on healthcare quality. Quality and Safety in Health Care 2002, Conclusion 11:148-152. 7. Lu CY, Ritchie J, Williams KM, Day RO: Recent developments in The limited resources must be carefully used to provide targeting access to high cost medicines in Australia. Aus NZ needed, effective and safe medicines that are affordable Health Policy 2005, 2:28. for the individual and society in order to achieve optimal 8. Pope C, Ziebland S, Mays N: Qualitative research in health care: Analysing qualitative data. BMJ 2000, 320:114-116. outcomes. Policy makers dealing with subsidised access to 9. Patton MQ: Enhancing the quality and credibility of qualitative high-cost medicines might focus upon: increasing stake- analysis. Health Serv Res 1999, 34:1189-1208. 10. 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Morgan SG, McMahon M, Mitton C, Roughead E, Kanavos P, Menon D: Centralized drug review processes in Australia, Canada, New Zealand, and the United Kingdom. Health Aff (Millwood) 2006, 25:337-347. 14. Australian Government Department of Health and Ageing: Pharma- ceutical Benefits Scheme - Public Summary Documents. [http://www.health.gov.au/internet/wcms/publishing.nsf/Content/ public-summary-documents]. 15. Smith R: Transparency: a modern essential. BMJ 2004, 328:. 16. Burton SL, Randel L, Titlow K, Emanuel EJ: The ethics of pharma- ceutical benefit management. Health Aff (Millwood) 2001, 20:150-163. 17. Gibson JL, Martin DK, Singer PA: Priority setting in hospitals: fairness, inclusiveness, and the problem of institutional power differences. Soc Sci Med 2005, 61:2355-2362. Publish with Bio Med Central and every scientist can read your work free of charge "BioMed Central will be the most significant development for disseminating the results of biomedical researc h in our lifetime." Sir Paul Nurse, Cancer Research UK Your research papers will be: available free of charge to the entire biomedical community peer reviewed and published immediately upon acceptance cited in PubMed and archived on PubMed Central yours — you keep the copyright BioMedcentral Submit your manuscript here: http://www.biomedcentral.com/info/publishing_adv.asp Page 9 of 9 (page number not for citation purposes) http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png Australia and New Zealand Health Policy Springer Journals

The views of stakeholders on controlled access schemes for high-cost antirheumatic biological medicines in Australia

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Copyright © 2007 by Lu et al; licensee BioMed Central Ltd.
Subject
Medicine & Public Health; Public Health; Social Policy
eISSN
1743-8462
DOI
10.1186/1743-8462-4-26
pmid
18096055
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Abstract

Background: In Australia, government-subsidised access to high-cost medicines is "targeted" to particular sub-sets of patients under the Pharmaceutical Benefits Scheme to achieve cost-effective use. In order to determine how this access system could be improved, the opinions of key stakeholders on access to biological agents for rheumatoid arthritis were explored. Methods: Thirty-six semi-structured interviews were conducted with persons from relevant stakeholder groups. These were transcribed verbatim, and analysed thematically. Results: Controlled access to expensive medicines was considered to be equitable and practical; however, there was disagreement as to the method of defining the target patient populations. Other concerns included timeliness of access, excessive bureaucracy, and the need for additional resources to facilitate the scheme. Collaboration between stakeholders was deemed important because it allows more equitable distribution of limited resources. The majority considered that stakeholder consultation should have been broader. Most wanted increased transparency of the decision-making process, ongoing and timely review of access criteria, and an increased provision of information for patients. More structured communication between stakeholders was proposed. Conclusion: The Pharmaceutical Benefit Scheme is adapting to meet the changing needs of patients. Provision of subsidised access to high-cost medicines in a manner that is affordable for individuals and society, and that is equitable and efficiently managed is challenging. The views of stakeholders on targeted access to anti-rheumatic biological medicines in Australia acknowledged this challenge and provided a number of suggestions for modifications. These could serve as a basis to inform the debate on how to change the processes and policies so as to improve the scheme. prescribers. In Australia, government-subsidised access to Introduction Drug reimbursement systems grapple with the challenges prescription medicines is provided through the Pharma- of funding innovative but costly medicines in the face of ceutical Benefits Scheme (PBS). Decisions on drug sub- increasing cost constraints, in part due to rapidly increas- sidy are based on assessment by the Pharmaceutical ing demands for access to these drugs by consumers and Benefits Advisory Committee (PBAC), which evaluates Page 1 of 9 (page number not for citation purposes) Australia and New Zealand Health Policy 2007, 4:26 http://www.anzhealthpolicy.com/content/4/1/26 incremental effectiveness and cost-effectiveness of the sidised access to the anti-rheumatic biologicals is only medicine [1]. Criteria for access to PBS listed medicines provided for patients who meet criteria for both starting are based on clinical trial evidence and economic evalua- and continuing therapy. These criteria include both clini- tions. cal and laboratory based measures (Table 1) [2]. Other biologicals subsequently subsidised by the PBS for treat- Increasingly, there is collaboration between the PBAC, the ing RA are: infliximab, adalimumab, anakinra, and rituxi- sponsor, and medical organisations (e.g. Australian Rheu- mab. matology Association) to develop access criteria. A repre- sentative example of such a collaboration is the 'Stakeholders', for the purpose of this study, were defined consultation process that contributed to the decision to as individuals or groups of people having the potential to subsidise and the criteria to access etanercept, a high-cost influence the decisions on the access arrangements, as biological medicine for treating rheumatoid arthritis well as those affected by the PBS restrictions. In reviewing (RA). This process set a new paradigm for analogous PBS the published literature, no studies apart from our own decisions [2,3]. A broad definition of "high-cost medi- have examined the views of stakeholders regarding this cines" in Australia is medicines whose acquisition cost is novel approach to control subsidised access to high-cost greater than A$10,000 per patient per treatment course medicines. This study examined the perceptions and expe- [4]. Although not directly involved in the consultation riences of stakeholders with respect to access to high-cost process, there was considerable support and lobbying medicines under Australia's PBS using, as an example, the activities by consumer representatives (namely the Arthri- anti-rheumatic biologicals. tis Foundation of Australia) for 'targeted' access to high- cost biologicals. Access to high-cost medicines under the Methods PBS is tightly regulated, requiring approval by Medicare In-depth semi-structured interviews were conducted with Australia (a government body that administers the PBS) participants drawn from the stakeholder groups that were before it can be prescribed with subsidy by a doctor. Sub- involved in access to biologicals through the PBS. Based Table 1: Access arrangements for biological agents for RA under the PBS Authority requirements Criteria for initiating • Severe active disease: treatment a) elevated levels of inflammatory markers (ESR > 25 mm/hour or CRP > 15 mg/L) b) swollen and tender joints – a total of > 20 joints, or > 4 major joints (elbow, wrist, knee, ankle, shoulder, hip) • A record of rheumatoid factor positive status (this requirement was removed as of June 2005) • Failure to achieve adequate response to a step-up sequence of treatment with conventional DMARDs: a) monotherapy with methotrexate (20 mg per week) b) a combination of methotrexate (> 7.5 mg per week) and 2 other DMARDs for at least 3 months c) leflunomide, leflunomide with methotrexate, or cyclosporin for at least 3 months • Evidence of intolerance or contraindication to DMARDs • Patients required to sign a 'patient acknowledgement form' • Treatment is approved for 16 weeks only (treatment of 22 weeks is approved for infliximab) A patient agreement process • A Patient Acknowledgement Form to be signed by patients to acknowledge that PBS subsidised treatment will only continue if the predetermined response criteria are achieved at 12 weeks Criteria for continuing treatment • Clinical outcomes are evaluated according to predetermined quantifiable criteria at 12 weeks: a) Reduction in levels of inflammatory markers, ESR < 25 mg/hour, or CRP < 15 mg/L, or 20% from baseline levels b) Reduction in the total number of joint count by 50% 'Interchangeability' (introduced • Patients approved to commence PBS subsidised biological treatment are allowed to switch to an December 2004) alternate biological agent at any time Restricted prescribing rights • Prescription only by specialist rheumatologists initially. Prescribing rights were extended to clinical immunologists with expertise in the management of RA as of February 2004 'Risk-sharing' arrangement • Annual PBS expenditure for the tumour necrosis factor inhibitors group was predicted to be up to A$140 million • Expenditure above this figure to be covered by the sponsoring pharmaceutical companies (details not clear from public documents) ESR = erythrocyte sedimentation rate CRP = C-reactive protein DMARDs = disease-modifying anti-rheumatic drugs Page 2 of 9 (page number not for citation purposes) Australia and New Zealand Health Policy 2007, 4:26 http://www.anzhealthpolicy.com/content/4/1/26 on our definition of "stakeholders" in the case of anti- The study was approved by the Human Research Ethics rheumatic biologicals, interviewees included were rheu- Committees of St Vincent's Hospital Sydney, and the Uni- matologists, patients with RA, government advisors, con- versity of New South Wales, Australia. sumer advocates, public servants, and pharmaceutical company spokespersons. Interviewees were asked to Results declare any potential conflicts of interest, that is, any pre- Thirty-six interviews were conducted between 2004–2005 vious or current advisory role with a pharmaceutical com- (Table 3). None of the government advisors, public serv- pany, or in any other committees or organisations that ants, consumer representatives, or patients had held advi- have a vested interest in the PBS listings. sory positions or roles on behalf of a pharmaceutical company. Four of the eight rheumatologists had been or Purposeful sampling of 'information-rich' individuals were on at least one advisory committee of a pharmaceu- [5,6], especially those involved in formulating the criteria, tical company. Marked differences were not found in the was undertaken. This led to inclusion of additional inter- views of rheumatologists who had held an advisory posi- viewees i.e. the so-called 'snowball sampling technique' tion with a pharmaceutical company versus those who [5]. Interviews were conducted using a guide based on had not. Most rheumatologists (7 out of 8) had been in findings from the first phase of the study [7] by the same practice for more than 10 years; the number of patients investigator (CL), a research pharmacist. The main ques- each rheumatologist treated with biologicals under the tions put to interviewees are listed in Table 2. Each inter- PBS ranged from 2 to 16. Most patients (5 out of 6) were view, lasting 45–80 minutes, was recorded and prescribed etanercept. One nurse was included in the transcribed verbatim. study opportunistically. NVivo 2.0 software was used to manage the data and to Five major themes emerged: resource rationing, excessive assist the coding for major concepts arising inductively bureaucracy, partnerships and inclusive decision-making, edu- from the data. Three investigator independently catego- cation, and review. All were related to the perceived fairness rised these concepts thematically before meeting to reach of the process and were essential determinants of support consensus on theme choice [8,9]. for the access criteria by stakeholders. A variety of techniques was used to confirm the methodo- 1. Resource rationing logical rigour. These included verifying the meaning Providing access to expensive medicines through a pub- ascribed to interviewees by offering them the opportunity licly-funded system (the PBS) was seen by all participants to review edited transcripts [9], using researcher triangula- as equitable and a feasible approach. Targeting access was tion to arrive at consensus on interpretation of the data supported as a necessary form of "resource rationing". The [9], using triangulation of multiple data sources (namely, vast majority acknowledged that there were finite individuals from different stakeholder groups) to counter- resources available for the possible range of diseases. Tar- balance weaknesses of one source by strengths in another geting access to sub-groups of patients who most need [9], searching for negative or discrepant cases to enhance treatment and would gain most benefit was in keeping interrogation of the data, and continuing to add inter- with spending "for value". However, interviewees disa- views until overall data saturation was achieved [10]. greed about how the appropriate target patient popula- Table 2: Main interview questions 1. What are the sources of information you used before prescribing the drug, or during the course of the treatment? 2. What do you see as the primary objective of the PBS arrangements for access? 3. What do you see as the strengths and weaknesses of the access scheme? 4. Have you been involved in the consultation process in regards to developing restrictions or arrangements to access the TNF inhibitors via the PBS? If yes, i. Can you briefly describe your role in the consultation process? ii. Who else took part in the consultation process? 5. Who do you think should take part in the consultation process for formulating the arrangements or restrictions for access? 6. What is the extent of your contact with other rheumatologists, local general practitioners, administrators, consumer organization, the PBAC? And what were the purposes of these contacts? 7. What do you see as the role and responsibility of the prescribers? The PBAC? The industry? Arthritis Foundation? 8. Who, in your view, has responsibility in informing/'educating' the prescribers (and the public) regarding PBS restriction changes, or new complex PBS restrictions? 9. How important an advance do you think the consultation approach and access arrangements represent? 10. If a new and expensive drug comes along that is a significant advance for the treatment of a chronic disease, what differences would you like to see in the process of getting it listed and using it? Page 3 of 9 (page number not for citation purposes) Australia and New Zealand Health Policy 2007, 4:26 http://www.anzhealthpolicy.com/content/4/1/26 Table 3: Characteristics of interview participants (n = 36) Number of participants Age group 18–29 years 1 30–39 years 5 40–49 years 8 50–59 years 14 60 years and over 8 Sex Male 13 Female 23 Stakeholder group Rheumatologist 8 Patient 6 Government advisor 5 Public servant 8 Consumer representative 5 Pharmaceutical industry representative 3 Clinical nurse 1 tion should be defined. A government advisor recognised immunologists) was deemed safe and appropriate, given the tensions between system-wide and individual needs: the expense of the medicines, the severity of RA in the affected patient population, and long-term safety con- "It [controlled access] is entirely defensible in terms of cerns. A patient voiced the predominant view: a population utility concept. It's very difficult at an individual patient level." "I think it's good that people have to have tried all the other cheaper medicines, that's fair enough. Why Some participants suggested broader, initial access. The should the government pay for a really expensive med- risk of excessive uptake of the biologicals by patients out- icine when methotrexate works and also, we don't side the PBS criteria was considered, by these participants, know yet what the long-term side effects will be." to be limited given the other controls that were applied, namely, risk-sharing between sponsors and government While monitoring patient outcomes was supported, via price-volume agreements, and the continuation rule that patients were apprehensive that an effective treatment limited ongoing access to those who responded to treat- might be withdrawn if one missed the threshold response ment. All agreed that the criteria should be based on to qualify for ongoing treatment. Further, prescribers sound clinical evidence. However, some criteria were seen might feel pressured to 'modulate' measurements of dis- as "arbitrary" and "potentially unfair" particularly when ease activity to benefit individual patients while, in effect, they were "unsupported by published literature". A rheu- denying treatment for others. Some interviewees sug- matologist gave his opinion: gested that the continuation rule be removed, and others proposed increased flexibility with respect to the timing of "I think ... the restriction to seropositive patients is the assessment, including less frequent assessment. A rep- unfortunate. Secondly, the reliance on laboratory indi- resentative quote from one rheumatologist illustrates this ces is a concern ... " view: The requirement for 'positive rheumatoid factor' (serop- "I think a system that had more flexibility for the ositive) was removed shortly after the completion of this renewal, possibly by making the reapplication less fre- study and such opinions may have been rescinded subse- quent, or having a larger window within which the quently. appropriate blood test could be acquired, or allowing so-called unacceptably high ESR [erythrocyte-sedi- The requirement to first trial existing, less costly therapies mentation-rate] or CRP [C-reactive-protein] or joint was considered to be reasonable. Limiting prescribing count for a period of time, until things settle down rights to sub-specialist physicians (rheumatologists, Page 4 of 9 (page number not for citation purposes) Australia and New Zealand Health Policy 2007, 4:26 http://www.anzhealthpolicy.com/content/4/1/26 again... a little less anxiety-provoking for the patients order to obtain ongoing supply. Physicians experienced would be good." difficulties in locating documents including records of laboratory tests and details of treatment history, some of However, government advisors and public servants which depended upon other clinicians who had treated believed that the interpretation of joint tenderness and patients previously. However, improved documentation swelling by clinicians and patients, being subjective, was seen as a good outcome overall, and an application allowed some flexibility. Further, they believed that for access was easier for more recently diagnosed patients. assessment of eligibility was flexible because applications for subsidised treatment were assessed by pharmacists and Prescribers were uneasy that there was no recompense for medical advisors from Medicare Australia, the responsible the substantial additional time to undertake the tasks to agency. Both prescribers and patients believed that clini- apply for access for their patients. Assistance (e.g. from cians who see individual patients should have such discre- nurses) was in general unavailable as the majority of rheu- tion because, as a patient put it: "people in government matologists in Australia work in private practice. Where aren't educated about the disease [RA]". A government nurses were available, considerable workload was advisor voiced the opposing view about allowing flexibil- imposed on them. A nurse was concerned about the ity with borderline cases: impact on hospital resources: "the problem with the high-cost drugs is it's very diffi- "In a lot of places I think that nurses are doing a lot of cult to be flexible because small changes in flexibility the work. And there's nowhere that this has been taken start to blow budgets out to massive amounts of into consideration with staff and issues and so on. It's money and that means that the whole principle of added at least eight hours a week to my workload. ...." equity and equitable access to these things are not obeyed. ... I don't think the uncertainty can be borne There was also an increase in the use of other resources. A only by Australian taxpayers." public servant noted that greater resources were needed to administer this complex scheme: The Patient Acknowledgement Form was seen as a contract between the patient and the government, and reduced the "We've got pharmacists on call and they are paid more direct pressure on the individual rheumatologists. Some than admin staff would be paid. We have to go rheumatologists and patients saw this agreement as through a more intensive process in order to approve "pointless" because treatment would be discontinued the application because we lose more money if we even if the patients disagreed, and it was unlikely that approve it wrongly." patients would wish to continue if the treatment was inef- fective. A common view of patients was: "when you're des- The requirement that there be an adequate trial of conven- perate you'll sign anything." tional anti-rheumatic drugs promoted re-evaluation of previous treatments and a more comprehensive and 2. Excessive Bureaucracy aggressive use of anti-rheumatic drugs. Patients were more The access scheme controlled the usage and expenditure easily convinced to go through these steps with the 'incen- of biologicals effectively in the first years of operation. tive' that they might access the 'new' biologicals. A rheu- This was seen as a good outcome by the majority. How- matologist voiced the predominant view: ever, there was concern by some that the use of biologics was inappropriately low. "It's forced our rheumatologists to re-look at the treat- ment that patients have already received ... in reassess- Most had the view that medical care had become "more ing the patient and aiming to meet the PBS criteria, bureaucratic" as a result of the PBS processes. A rheuma- they actually get their rheumatoid arthritis under con- tologist commented: trol before requiring a biological..." "It seems to be a very bureaucratic way of giving med- The majority considered that this effect was beneficial and ical care, rather than a normal doctor-patient relation- potentially systematised the treatment of RA nationwide. ship... it's eliminating the ability of the doctor to show some level of discretion..." 3. Partnerships and inclusive decision-making There was uniform support for the stakeholder collabora- The majority thought the application process was admin- tion and increased communication that contributed to istratively burdensome. Patients were anxious about coor- the decision to subsidise biologicals. However, while gov- dination of laboratory tests, joint assessment, application ernment advisors and public servants commented that the forms, and ordering the medicine from pharmacies in representative rheumatologists were cooperative, the Page 5 of 9 (page number not for citation purposes) Australia and New Zealand Health Policy 2007, 4:26 http://www.anzhealthpolicy.com/content/4/1/26 companies felt that the representative rheumatologists them-and-us system, this is our system." (government were less willing to collaborate. The consumers felt that advisor) communication with the Rheumatology Association and the government was insufficient. Further, some interview- A comment by a rheumatologist summarised the two key ees suggested that consultation among rheumatologists elements for future processes: should be wider. An industry spokesperson commented on the negotiation skills of the representing rheumatolo- "I would like to see a more collaborative and more gists: open approach, because I think a lot of the negotia- tions that eventually resulted in this listing were, "I don't think they [rheumatologists] had the skills, whether deliberately or accidentally, shrouded in which is built up over experience, to be able to handle some secrecy and I know from the PBAC perspective negotiations with government. People like 'gastros' there's been some talk of deliberations being made have had good experience over quite a long time with more transparent. If we were doing this process again new therapies, and oncologists and cardiologists as and if I had the power to change something, those are well. Rheumatologists are babes in the wood when it the things I would like to see: direct consumer repre- comes to dealing with government." sentation and an open and transparent process that we could watch evolving." Some believed that doctors would adequately represent 4. Education the views of patients while the majority view was that patients should have a more direct role. A common view A common view of stakeholders was that the clinicians at was that patient representatives would need to be care- Medicare Australia readily assist with queries regarding fully selected and well-informed. Interviewees had mixed applications. However, the majority felt that better provi- opinions about when patients should be involved in the sion of information on the criteria and application proce- process, but that they should at least be involved in devel- dures, particularly in the early stages of implementation, oping the Patient Acknowledgement Form. Some inter- was important. In addition to avoiding confusion and viewees believed that negotiation between the three increasing efficiency, it would enhance support for the primary groups (PBAC, medical specialists, companies) access criteria. was sufficient and would avoid difficulties and lengthy negotiations potentially associated with wider consulta- Concerns were expressed about insufficient information tion. for patients and inadequate clinical knowledge of the bio- logicals among health professionals in general. Clinicians An urgent need recognised by the majority was for open emphasised the importance of providing information on discussion to engage the broader community to work out adverse reactions, notably risks of infection. This was the overarching principles of allocation and access to potentially a difficult task because some patients feared medicines. A government advisor voiced the predominant that treatment might be stopped if they reported infec- view: tions. Most patients felt that quality-of-life outweighed other considerations including the potential risks of treat- "The population as a whole needs to have some ment. debate about where they want to spend their money, they are the ones that are funding it ultimately..." The industry interviewees felt that education was a major responsibility of industry particularly when medicines are Greater transparency around deliberations between stake- subject to complex criteria for access. It was agreed that holders and the rationale behind the selected criteria, cur- company representatives had been helpful in providing rently constrained by confidentiality requirements, was information to clinicians. However, most interviewees considered essential to enhance understanding of the sys- expressed a lack of faith in the companies to supply tem and to enable the PBAC to better defend its decisions appropriate materials for patients or the public. Concerns and garner broader support. were also raised about fragmented and misleading mes- sages delivered by the media. "We need greater transparency. It's [important] people understanding the system, taking some ownership of Enhanced provision of information on the rationale for the system, involving them through transparency, restricting access, how criteria were decided, and the dis- through education, being prepared to speak to them. tinction between "effectiveness" and "cost-effectiveness" The PBAC should defend the decisions. It's all to do of medicines was advocated. The Patient Acknowledgement with dialogue, with the partnerships. This is not a Form represented an opportunity to increase patient understanding that there were responsibilities and risks to Page 6 of 9 (page number not for citation purposes) Australia and New Zealand Health Policy 2007, 4:26 http://www.anzhealthpolicy.com/content/4/1/26 be shared by all parties. Most thought that information Discussion provided by the Australian Rheumatology Association By including individuals who represented the range of (clinical information on the medicines) and the National stakeholder groups with their different and potentially Prescribing Service (information about the PBS and its competing interests, this study sought to present a well- decisions) would be most trustworthy. Consumer organi- rounded picture of the access scheme. In-depth insights sations were identified as having a role in disseminating into stakeholders' views provide a basis to inform the information and providing consumer support. Interview- broader debate on how subsidy systems for high-cost ees suggested that general practitioners and other health medicines can be improved. The major consensus finding professionals, such as pharmacists, could be more was that while the the current access scheme for anti-rheu- involved in educating patients. matic biologicals can be viewed as successful, stakeholder communication and involvement needs to be increased. A 5. Review limitation of the study was that insights into some mana- Regular review of access criteria was recommended. Drug gerial perspectives were not obtained because administra- utilisation patterns and feedback from stakeholders tors who assessed patient applications for access declined should be analysed to refine the access arrangements. The to participate due to concerns about privacy. risk-sharing agreement should also be reviewed. The proc- esses of PBAC decision-making and stakeholder consulta- The innovations introduced to establish the access scheme tion would also benefit from regular evaluation. A public were considered concordant with the expectation that servant also proposed examining the administrative government is responsible for providing access to effective expenses associated with such a scheme: medicines while balancing the need to use public resources wisely. Limiting access to sub-sets of patients, "The administration costs [of Medicare Australia] are where need has been established and where use was cost- not a consideration for the PBAC. We need to do some effective, was viewed as practical and equitable. The stake- cost-benefit analysis around the cost of building the holders were supportive of the proposition that it is possi- appropriate systems [of access] and the benefit in ble to make high-cost medicines available and affordable reduction of use [of medicines] on PBS." for the community and individual patients. That this had been achieved with these drugs was accepted as an impor- Some were disappointed that a prospective, formal evalu- tant accomplishment. However, timeliness of access to ation of the access scheme had not been established. This innovative medicines via the PBS in Australia, in compar- was partly because stakeholders could not reach consen- ison to comparable countries, was a concern. This was sus on who should be responsible or what were appropri- partly due to the registration process which often occurred ate funding sources. Australian rheumatologists had later in Australia than in the USA or Europe. A definition implemented independently a patient registry to track of "timeliness of access to medicines" by interviewees of patient outcomes. Some interviewees suggested that gov- the six stakeholder groups was not actively explored in ernment and the pharmaceutical industry should fund this study; an important issue that warrants further inves- such a registry collaboratively. The fact that a comprehen- tigation. sive information system enabling evaluation had not been developed was a concern for most interviewees. Criteria for access were seen to be potentially 'unfair' when the evidence was not publicly available. For exam- Some rheumatologists and patients considered it a weak- ple, the requirement for rheumatoid-factor-positive-status to ness that a review panel for contentious cases had not gain access was contentious at the time of the interviews. been established, while others felt that, to a degree, special Most published pivotal studies did not exclude rheuma- consideration had occurred informally via interactions toid-factor-negative patients or analyse them as a sub- between Medicare Australia and rheumatologists. A group. There was insufficient evidence in the public patient voiced frustration about the delayed review of the domain to support the view that rheumatoid-factor-posi- PBS criteria: tive-status was a factor predicting a better response to treatment with etanercept [11]. However, efficacy in rheu- "I would like a true appeal system. I would like who- matoid-factor-negative patients had not been clearly ever's in power to try and rectify the situation quickly established because of the small number of such patients and that when they put in future criteria for any med- in these trials [12]. This requirement for rheumatoid fac- icine, that they are very, very careful about the crite- tor positivity has subsequently been removed. Sustaina- ria." bility of a subsidy system such as the PBS is dependent upon a rigorous and consistent process of drug review and increased transparency around the rationale underpin- ning decisions [13]. How the PBAC arrives at its recom- Page 7 of 9 (page number not for citation purposes) Australia and New Zealand Health Policy 2007, 4:26 http://www.anzhealthpolicy.com/content/4/1/26 mendations for PBS listing was significantly limited by the to administer systems of access; and improving communi- "commercial-in-confidence" restrictions at the time of cation and information based on increased transparency. etanercept listing. An important milestone in this respect is that summaries of PBAC decisions have become availa- Competing interests ble publicly recently [3,14]. Increasing transparency also CL and JR have nothing to declare. KW has been a mem- increases accountability of all parties for decisions and ber of the Advisory Board to the sponsor for adalimumab. performance of the system. These moves towards trans- RD has been a member of the Advisory Board to sponsors parency were supported by the participants. for adalimumab, infliximab, and anakinra in Australia. KW and RD have also been contracted to undertake clini- Open dialogue and declaration of potential conflicts of cal trials of etanercept, infliximab, adalimumab, and ana- interest are important to building trust [15]. Increased kinra. Recompense for these activities is placed in audited communication and collaboration between stakeholders hospital trust funds for use in the research activities of the have been crucial steps that were initiated during the Clinical Pharmacology Department, St Vincent's Hospital, effort to subsidise the anti-rheumatic biologicals. How- Sydney. ever, the consumer participants identified a need for increasing the voice of patients and the public in order to Authors' contributions enhance the quality of decisions, and acceptance of access CL, KW, and RD have made substantial contributions to criteria, a position supported by the majority of partici- the conception and design of the study, analysis and inter- pants. pretation of the data, and drafting and revising the manu- script. JR has been involved in advising on the analysis, Increasing bureaucratic requirements are a threat to the drafting the manuscript and revising it critically for intel- acceptance and efficient running of such access systems. lectual content. All authors read and approved the final The administrative burden imposed on prescribers can be manuscript. a barrier to enrolling deserving patients and a source of increased costs [16]. Most stakeholders, with the excep- Acknowledgements The authors thank the interviewees for their time and participation in this tion of the government advisors and public servants, pro- research. posed that more reliance on physicians' integrity to comply with PBS criteria should be considered an appro- Funding/support: CL was supported by an Australian National Health & priate approach and would benefit patients. Medical Research Council postgraduate scholarship (Grant No. 351040). A significant gap in knowledge was identified by health References professionals (namely, general practitioners, pharmacists 1. Commonwealth Department of Health and Aged Care: The Aus- tralian Health Care System, An Outline. Canberra, ACT, Pub- and nurses) and the community about the PBS and the lications Unit, Commonwealth Department of Health and Aged Care; increasing need to control access to medicines, in particu- lar, high-cost medicines. General practitioners and phar- 2. Lu CY, Williams KM, Day RO, March LM, Sansom L, Bertouch J: Access to high cost drugs in Australia: Risk sharing scheme macists were identified as potentially important may set a new paradigm. BMJ 2004, 329:415-416. contributors to the success of targeted access schemes. 3. Sansom L: The subsidy of pharmaceuticals in Australia: proc- esses and challenges. Aust Health Rev 2004, 28:194-205. They have an important role to play in educating patients 4. High Cost Drugs Working Party: Drug and Therapeutics Com- about the use of medicines in chronic diseases such as RA mittee Template for Formulary Submissions - Decision algo- and the PBS system in general. Adequate provision of rithm; NSW Therapeutic Advisory Group (cited 12 Oct 2006). [http://www.ciap.health.nsw.gov.au/nswtag/high_cost_drugs- information is critical to managing patient expectations wp.html]. and empowering them to participate in decision-making 5. Patton MQ: Qualitative research and evaluation methods. 3rd processes [17]. edition. Thousand Oaks, CA, Sage Publications; 2002. 6. Pope C, van Royen P, Baker R: Qualitative methods in research on healthcare quality. Quality and Safety in Health Care 2002, Conclusion 11:148-152. 7. Lu CY, Ritchie J, Williams KM, Day RO: Recent developments in The limited resources must be carefully used to provide targeting access to high cost medicines in Australia. Aus NZ needed, effective and safe medicines that are affordable Health Policy 2005, 2:28. for the individual and society in order to achieve optimal 8. Pope C, Ziebland S, Mays N: Qualitative research in health care: Analysing qualitative data. BMJ 2000, 320:114-116. outcomes. Policy makers dealing with subsidised access to 9. Patton MQ: Enhancing the quality and credibility of qualitative high-cost medicines might focus upon: increasing stake- analysis. Health Serv Res 1999, 34:1189-1208. 10. Strauss A, Corbin J: Basics of qualitative research. 2nd Edition holder involvement in decisions; better methods of defin- edition. California, SAGE; 1998:312. ing target populations; improving the timeliness of access; 11. Lu CY, Williams KM, March LM, Bertouch J, Day RO: Subsidised increasing the flexibility for clinicians to make decisions access to TNFa inhibitors: is the rationale for exclusion of rheumatoid factor negative patients defensible? Med J Aust while reducing bureaucratic red-tape; enhancing resources 2004, 181:457. Page 8 of 9 (page number not for citation purposes) Australia and New Zealand Health Policy 2007, 4:26 http://www.anzhealthpolicy.com/content/4/1/26 12. Sansom L: Subsidised access to TNFa inhibitors: is the ration- ale for exclusion of rheumatoid factor negative patients defensible? [comment]. Med J Aust 2004, 181:457-458. 13. Morgan SG, McMahon M, Mitton C, Roughead E, Kanavos P, Menon D: Centralized drug review processes in Australia, Canada, New Zealand, and the United Kingdom. Health Aff (Millwood) 2006, 25:337-347. 14. Australian Government Department of Health and Ageing: Pharma- ceutical Benefits Scheme - Public Summary Documents. [http://www.health.gov.au/internet/wcms/publishing.nsf/Content/ public-summary-documents]. 15. Smith R: Transparency: a modern essential. BMJ 2004, 328:. 16. Burton SL, Randel L, Titlow K, Emanuel EJ: The ethics of pharma- ceutical benefit management. Health Aff (Millwood) 2001, 20:150-163. 17. Gibson JL, Martin DK, Singer PA: Priority setting in hospitals: fairness, inclusiveness, and the problem of institutional power differences. Soc Sci Med 2005, 61:2355-2362. Publish with Bio Med Central and every scientist can read your work free of charge "BioMed Central will be the most significant development for disseminating the results of biomedical researc h in our lifetime." Sir Paul Nurse, Cancer Research UK Your research papers will be: available free of charge to the entire biomedical community peer reviewed and published immediately upon acceptance cited in PubMed and archived on PubMed Central yours — you keep the copyright BioMedcentral Submit your manuscript here: http://www.biomedcentral.com/info/publishing_adv.asp Page 9 of 9 (page number not for citation purposes)

Journal

Australia and New Zealand Health PolicySpringer Journals

Published: Dec 20, 2007

References