7 minute read
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.
The 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.
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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
needs to begin long before a rare disease drug is market-ready. The unmet clinical need must be established, and accepted by payers and the public, before the solution is available and access is sought.
Patient-focused messages
Unfortunately, Canadian reimbursement processes for rare disorder drugs currently favour economic analysis over clinical data, making the need for the patient and physician voice even stronger. Regardless of the per-patient cost, treatment outcomes are arguably priceless. These therapies represent the first and often only treatment for a condition that is often progressive, life-shortening or imminently life-threatening.
Reimbursement objections need to be addressed through messages that speak to the clinical significance of the drug from the patient perspective and that of the medical expert, framed in a way that resonates with government and the public. Without a clear understanding of how a treatment can transform lives, the clinical data and impact of the drug on quality of life is at risk of being overshadowed by price.
The patient and physician voice are particularly important as they underscore the real-world impact of a medication that affects so few lives. Clinical experts can put the costs and benefits of a rare disease treatment into perspective, lending credibility to an otherwise emotional appeal. By sharing their personal stories and unique perspectives, patients and caregivers can very effectively make the personal political, and provide insight into the data that the public can identify with, even if they aren’t touched by the disease.
Expanded support base
Communication efforts are critical in rare disease advocacy as many disorders are not widely known and receive little visibility. Initiatives that serve to amplify the patient from multiple credible sources, through a variety of channels. The Internet and social media have made the world smaller and the opportunities to foster connections through communications much easier. They provide advocates with more ways to engage with government and the general public, but they also demand consistent messaging and persistent action.
voice and broaden the base of support will succeed in influencing public opinion and ultimately, the political agenda.
It is essential to engage the core group of stakeholders – people living with the disorder, their friends and families – and help them to unite and mobilize. This natural base of support is especially important because advocacy relies on volunteers who often have limited time, funds, and expertise. Further, the people directly impacted by a lack of access to treatment are often consumed by battling their disease, or taking care of a stricken loved one.
The power of engaging personal networks cannot be understated. While a rare disease drug may only treat a handful of people, its impact extends to families, friends and communities. The ultimate goal is to expand engagement beyond personal networks to the broader public in the hope that they will identify with their plight. Encouraging patients and their families to tap into their networks can build the critical mass necessary to apply sufficient political pressure.
Expanding the base of support requires engagement on many platforms – in person, online through a variety of social media channels, and through traditional print, radio, and television media. It is important to recognize that today’s world is visual and fast-paced, and to tailor efforts to the medium. The message needs to grab someone’s attention within 15 to 30 seconds, which makes compelling images, videos, and personal pleas even more important.
Taking an integrated approach
An integrated approach to advocacy campaigns is a critical component in influencing and driving change. Given today’s rare disease drug reimbursement landscape where political pressure is almost always required, public relations and public affairs are inextricably linked. It is not enough to simply meet one-on-one with government officials or to tell your story to a reporter – the two must go hand-in-hand and be leveraged through social platforms.
To even consider taking action, decisionmakers need to hear messages repeatedly
Hope for change
The challenges surrounding public reimbursement for rare disease drugs are considerable, the stakes are high, and much needed policy changes are very slow to come. Often, rare disease patient groups are neither expecting nor prepared for the fight they are certain to face. Access to orphan drugs can have an undeniable life-changing impact on those patients who are fortunate enough to receive treatment, but come at a high cost in terms of invested time and energy. Employing a strategic approach to market access that integrates advocacy with government and media relations has demonstrated effectiveness in bringing treatments to patients sooner by encouraging positive drug funding decisions. There is hope – at least for small changes – when the power of the patient and physician voice is brought to bear.
Joanne Koskie, Vice President, Cohn & Wolfe, brings two decades of communications experience as a consultant and within the voluntary sector. Joanne supports the work of dozens of patient groups, providing strategic leadership and direction on integrated, nationwide advocacy campaigns for access to care and treatment. Christine Beyaert, Counsellor, Cohn & Wolfe, provides an insider’s view on public relations from within government and the patient community with a focus on social and digital communications.
To see this story online visit www.biotechnologyfocus.ca/ easy-rare