Science for the people: enabling impactful innovation by improving drug accessibility
Erika Siren, PhD
Harvard Stem Cell Institute
Throughout the many areas of scientific research, the one constant that unites the majority of scientists is our desire to make an impact on the world around us. We fastidiously work at our benches and in the field to develop innovative solutions to global challenges. We assume that if the science is sound, the rest will be taken care of. In the context of drug discovery (my own field of expertise), the path forward is arduous but clear: develop paradigm-shifting biotechnology, push it through commercialization, obtain regulatory approval and market access, treat patients, improve healthcare.
This process is grossly oversimplified but has more-or-less rung true throughout the history of drug approval. However, the recent emergence of advanced therapies and the attempts to push them through the existing regulatory process has been akin to trying to fit a square peg into a round hole. Although therapeutically powerful, advanced therapies such as cell, gene, and immunotherapies are expensive and require complex patient care regimens. As advanced therapies gain federal approval across the globe, there is an increasing fear that these complex treatment modalities will provoke a disparity in quality-of-care, favouring those who reside in urban areas. Furthermore, the steep price tag of these therapies (which cost hundreds of thousands of dollars) would have prospective patients scrambling to find coverage in already exasperated public and private reimbursement channels. For those with fewer reimbursement options, access to these therapies may be limited even if they’re located in large, urban areas. These issues are even more substantial in Canada, as healthcare standards and reimbursements are decided at the provincial level. This means that even if rural patients were able to travel to larger hospitals, the province they live in may be the deciding factor between being able to afford an advanced therapy or not.
Scientists and physicians have worked tirelessly to push through scientific barriers and generate paradigm-shifting treatments like cell and gene therapies. With science and technology showing no sign of slowing down, it is imperative that politicians and regulators push through their own barriers to modernize Canada’s healthcare system. This is not the first-time advanced therapies have attempted to integrate into Canadian healthcare. Promising therapies like Prochymal, Provenge and Orkambi have failed because of reimbursement issues and a lack of supporting infrastructure. Though the failure of these therapies is not due to infrastructure and reimbursement alone, they reveal an unresolved gap between scientific innovation and implementation.
For those actively involved in the development of advanced therapies, the feeling of helplessness in getting these therapies to patients can be disheartening. Grant applications and graduate student presentations are littered with promises that science can help people, with many of us unaware of how little power we have in making this a reality. To implement scientific progress into society, scientists are dependent on policymakers and politicians to connect innovation to the people.
This month, Bill Morneau announced that the 2019 federal budget will allocate funds toward creating a national strategy to improve access to high-cost drugs for rare diseases, investing up to C$1 billion over two years starting in the 2022-23 fiscal year. They also announced $35 million towards the development of a national drug agency to help cut the cost of prescription drugs and eventually harmonize drug coverage across Canada. As many emerging cell and gene therapies target rare diseases, this is a clear effort to catch up with advances in medicine and biotechnology. Moreover, a well designed national drug agency will enable the government to purchase drugs in bulk, a strategy other countries have used to negotiate lower prices for cancer-targeting cell therapies. Together, these could serve as important steps to ensure that Canadians from Vancouver Island to Bonavista have equal access to the treatments that scientists worked so hard to develop.
In a world where partisanship is increasingly stalling societal progress, implementations like these show that that change may still be possible. This facet of the 2019 federal budget is perhaps not as directly related to research as academic funding; however, the impact on Canadian scientists and innovation is substantive. Impactful science does not end at the bench and there are many hurdles that must be overcome for innovation to be felt in society. There are still many unknowns however, and the execution of these initiatives will ultimately determine whether the aspirations of the Canadian drug agency and national strategy for rare diseases are achieved. From my lab bench, I will watch with cautious optimism.