By Joanne Koskie and Christine Beyaert
Orphan Drugs
It’s not easy being rare An estimated 2.8 million Canadians live with one of the more than 7,000 identified rare diseases. For many there is little relief, but for a select few who suffer from one of these disorders, treatments are available – if they can get access to them.
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he issue of orphan drug reimbursement by public payers has become central to companies that develop these innovative therapies and the patients who need them. Orphan drug research and development costs are amortized across a small patient population, so a drug’s economic viability rests on a relatively higher per-patient price. As a result, most patients cannot afford to pay for these treatments themselves, instead relying on public or private coverage. While our healthcare system is considered among the best in the world, Canadians with rare disorders are at a marked disadvantage when it comes to accessing treatment. Only half of the rare disease drugs available to patients in the U.S. or Europe are approved in Canada, and only half of these are funded by public drug plans. Those recommended for funding are often met with restrictive criteria and conditions, further limiting patient access. It is by no means easy being rare, but orphan drug reimbursement is achievable – even in the most challenging situations – through a strategic, integrated approach to communications.
Price, policy and political will Drugs for rare diseases face unique and seemingly unsurmountable challenges that impact funding decisions, including the cost of treatment, health technology assessment policies and low political will. In any effort
to gain reimbursement, these barriers must be addressed and overcome to ensure patients have timely access to life-transforming treatments. Several orphan drugs approved in the past few years have seemingly hefty price tags, with some in the million-dollar range per patient, annually. Research and development costs, and the associated high risk, are not well understood or widely accepted and are overshadowed by sticker shock. While the price per patient is high, the few approved drugs for rare diseases in Canada consume less than one per cent of the total spent on pharmaceuticals. Further, these drugs can alleviate downstream pressure on the healthcare system, by contributing to a reduction in doctors visits, hospitalizations, surgeries and disability. Representing another significant hurdle, health technology assessment (HTA) processes that evaluate and make funding recommendations for new treatments are virtually the same for rare disease drugs as for other medications. Many orphan drugs are not recommended for reimbursement because the evaluation criteria – safety, comparative clinical-efficacy, and comparative cost-effectiveness – have not been adapted to the unique characteristics of rare diseases. Since these treatments are firsts, there are no benchmarks to gauge cost-effectiveness. While efforts are made to incorporate patient, caregiver, and health care provider
input, drugs for rare disorders remain at a disadvantage because current HTA processes prioritize cost containment over making treatments available to patients who desperately need them. Policy issues are compounded by fiscal constraints with provincial and territorial healthcare budgets under constant scrutiny. Policymakers responsible for those budgets are under pressure to reduce costs while maximizing the public interest. Political will is influenced by the intensity of public sentiment and how closely a particular issue intersects or aligns with the agenda of the day. Thus, governments are responsive to the files that most urgently demand their attention. If only a few people in a province require a treatment, their voices may be difficult to hear. There is little benefit for governments to respond to the demands of small patient groups. In some cases, what dominates is the perceived risk that voters will see the opportunity cost to treat a single patient with a rare disease as simply too high.
Making rare roar by strategically integrating communications Successful reimbursement efforts necessitate a multi-stakeholder, multi-channel approach that strategically integrates advocacy with government and media relations. Moreover, the process of building and sharing the social and emotional value story for treatment February/March 2015 BIOTECHNOLOGY FOCUS 15
