The Ethics and Politics of Accelerated Access to Medicines

Introduction: Australia’s systems for registering and funding medicines are well-established and internationally respected for their success in facilitating access while also protecting patients from harm and ensuring system sustainability. However, in recent years, there have been increasing concerns about whether these systems hinder access to medicines that patients want or need. In response, a number of “accelerated access” initiatives have been implemented to facilitate faster access. However, the risk-benefit balance of such schemes is currently unclear—while they may provide patients with a wider range of treatment options and earlier access to therapies, there is also increased uncertainty surrounding the safety, efficacy and cost-effectiveness of medicines made available via these pathways. Their impacts for stakeholders—including patients, physicians, members of the pharmaceutical industry and the general public—are likely to be significant and stakeholder engagement is therefore essential to determine the best way to proceed. Methods: This ethically-informed stakeholder analysis consisted of two phases. The first involved semi-structured interviews with physicians about their views on and experiences with accelerated access and standards of efficacy, safety and cost-effectiveness that they want to be applied by regulatory and funding bodies. The second was a mixed methods phase exploring consumer perspectives on this phenomenon. This consisted of an in-depth investigation of media debates surrounding three different forms of accelerated access (coverage with evidence development, cancer drugs funds and initiatives to promote access to medicines used to treat rare diseases) and semi-structured interviews with patient advocates and focus group interviews with patients. Main findings: Through this study, I have generated an-depth description of the diverse views of patients and physicians regarding accelerated access. In phase 1, we identified three “archetypes” of physicians: “confident accelerators”, “cautious accelerators” and “decelerators”. Although all acknowledged the potential risks and benefits of accelerated access, they disagreed on their magnitude and extent and how they should be balanced in both policy formation and clinical practice. In phase 2, analysis of media debates illustrated an emotive discourse, with stakeholders emphasising the importance of timely access to new therapies and barriers that current regulatory and reimbursement systems pose and little discussion of issues such as uncertainty surrounding safety and efficacy and cost-effectiveness of new therapies and the opportunity costs of funding these. However, interview participants were more cautious. While they noted a number of reasons for alternative access mechanisms, they were also cognisant of potential risks and viewed ongoing data collection and disinvestment and market withdrawal as ways to address these. Participants in both phases also identified a number of factors—such as transparent decisions made by people with relevant expertise, based on a thorough consideration of both scientific evidence and stakeholder perspectives—required to ensure appropriate decision-making processes. Overall, stakeholder views are varied, nuanced, and largely reasonable, with consideration of both benefits and risks and respect for and understanding of perspectives that differ from their own. Discussion and Conclusions: Stakeholder analysis suggests that there are a number of reasonable views on how we should respond to increasing calls for accelerated access to medicines. I draw on theories of protopianism, pragmatism and incrementalism to argue that the best way to proceed here is to focus on incremental changes to our current systems. While not perfect, these have evolved into something stable, workable and, on the whole, just. While small changes might at times be warranted, calls to drastically change our standards of evidence—as advocated by some—are unjustified and could have serious consequences. However, policy makers cannot simply ignore these calls and must respond to them. I argue that they can do this by focusing on legitimate policy and decision-making (looking at both the structure of the system and specific decisions made about particular therapies). I use theories of legitimate decision-making—such as Daniels and Sabin’s Accountability for Reasonableness, Gutmann and Thompson’s Standards for Assessing the Process of Health Policy Decision-Making and Clark and Weale’s Process Values for Healthcare Priority Setting—to identify both forces that may undermine legitimacy in this area and actions that policy makers can take to address this. I conclude with a number of practical suggestions to ensure both that the benefits of our current systems are maintained and harms to individual patients and the broader healthcare system are minimised