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Sandra Anderson outlines what manufacturers need to know about the regulatory environment and commercialization solutions for the Canadian market.
Cell and gene therapies (CGT) are currently at the forefront of scientific innovation. They hold great promise for patients suffering from a range of serious ailments. But because these therapies are new, often distinct in their makeup, and often targeted to a small patient population, they pose considerable challenges for the pharmaceutical companies seeking to bring them to market. While there are many such drugs in development, few have made it through the commercialization process to date. Still, by seeking to create standard models for commercialization and leveraging the expertise of those with experience bringing other complex therapies to market, these trailblazers can find success.
In order to bring a drug to market, a company must understand the specific regulatory environment in which it is operating. Canada has sought to foster innovation in its pharmaceutical industry and is making changes to continue modernization of the therapeutic product regulatory system.
Canada is seeking to support healthcare innovation by establishing a pathway for Advanced Therapeutic Products (ATPs),1 products that are so complex and distinct that the existing regulatory frameworks and enforcement tools are not equipped to handle them. Health Canada, the division of the government responsible for national health policy, has leveraged international approaches in their design of an ATP pathway. This involves tailored requirements, an iterative approach, and flexible management of risk with use of terms and conditions and inspection powers. Once there is enough regulatory experience with an ATP, it is transitioned under existing regulations or new regulations will be created as needed.
Amendments to the Food and Drug Regulations and Medical Devices Regulations are proposed for spring of 2022. These updates will allow for greater agility of the system, strengthening implementation of the lifecycle approach. The Minister of Health will have the ability to impose terms and conditions on authorizations, compel risk management plans for drugs and extend flexibilities in place for COVID-19 drugs to other drugs under specific circumstances.
Currently, most cell and gene therapies are classified as biologics in Canada, although in certain cases a cell therapy may be regulated as a medical device. Extensive evidence is required to support the safety and efficacy of a new therapy. In order to bring biologic cell and gene therapy products to market, a “New Drug Submission" must be filed. The NDS must contain the results of preclinical and clinical trials, whether completed in Canada or elsewhere, as well as details on chemistry and manufacturing, therapeutic claims and proposed risk management measures. It's important to note that cell and gene therapies may qualify for priority review or conditional authorization. Many of the patients that cell and gene therapies are designed to support are living with serious, life-threatening, or severely debilitating diseases or conditions. Where these are diseases or conditions for which there are no drugs currently available in the Canadian market, or where the cell and gene therapies show significant improvement over existing therapies or preventatives priority review or NOC/c may apply.
Proposed amendments to the Food and Drug Regulations may also allow for rolling submissions and use of terms and conditions for these types of drugs.
Regardless of where they're launching a new product, companies launching cell and gene therapies should start planning earlier than usual for their market access strategy to enable successful reimbursement. They must understand the patient and clinical journey they are supporting and map the access points, engaging stakeholders throughout the process.
Manufacturers will need to submit to the Health Technology agencies in Canada, such as the Canadian Agency for Drugs and Technologies in Health (CADTH) and the Institut national d'excellence en santé et en services sociaux (INESSS). These bodies are independent, not-for-profit organizations responsible for providing Canada's healthcare decision-makers with objective evidence to help make informed decisions about the optimal use of drugs and medical devices in the Canadian healthcare system. For instance, a manufacturer will need to demonstrate for patients, providers, and payers the benefits of a high-cost, one-time cure against a lifetime of ongoing treatments for a chronic illness. To facilitate reimbursement, it may also be necessary to develop custom payment structures that place value on better patient outcomes.
Given that a relatively small number of patients will be a candidate for cell and gene therapies, it may be necessary for manufacturers to invest in strategies that create a standardized approach to patient access while providing the opportunity to scale up. For instance, manufacturers could consider joining a single industry-wide platform or system that healthcare providers in a country can use to access multiple cell and gene therapies. Canada is an ideal place to launch these types of niche strategies for rolling out new therapies that could then be expanded globally. A commercialization partner that can deploy an integrated model can help manufacturers navigate every part of the process of bringing an innovative therapy to market.
Manufacturers should reevaluate the comprehensive services and capabilities that they traditionally provide and look at a customized commercial approach that emphasizes and delivers on the therapy's true value. Some considerations for a seamless patient journey include streamlined enrollment and follow-up/engagement, integrated logistics models, rapid access to therapy, and flexible financial assistance programs.
In addition, the manufacturer's market access strategy should consider the importance of engaging with the clinical community and academic centres across Canada, along with the role of patient advocacy groups, specifically within the rare disease and oncology areas.
Moreover, the right partner can help manufacturers address questions around:
Because these questions are complex, planning must be done more upstream than typical in a drug launch plan.
Finding the right commercialization partner is important for any new product, but perhaps even more so when working with cell and gene therapies, especially given the challenges CGTs face and the demand for new models for access and reimbursement that can be standardized across the healthcare industry. Manufacturers must choose a partner with extensive knowledge of local regulatory requirements within the country as well as the international footprint necessary to realize economies of scale. They must seek out a partner that can draw on expertise and data gained from years of bringing other complex therapies to market, while also having the flexibility to create a new, customized approach where appropriate.
Sandra Anderson is Senior Vice President of Commercialization & Strategy at Innomar Strategies.