A Canadian Approach to Commercializing Cell and Gene Therapies

The field of cell and gene therapies (CGT) is at the forefront of scientific advancement. There is great promise for patients suffering from serious illnesses.

FREMONT, CA: Cell and gene therapies (CGTs) are at the forefront of scientific advancement. They hold great promise for patients with a variety of severe diseases. However, because these therapies are novel, frequently distinct in composition, and often intended for a small patient population, pharmaceutical companies seeking to bring them to market face significant obstacles. Numerous such drugs are developing, but few have made it to commercialization. These trailblazers can succeed if they develop standard commercialization models and leverage the expertise of those who have brought other complex therapies to market.

CANADA IS WELL-POSITIONED FOR INNOVATION

A company must understand the regulatory environment in which it operates before bringing a drug to market. Canada has sought to foster innovation in its pharmaceutical industry and is implementing changes to modernize its regulatory system for therapeutic products.

Canada seeks to support healthcare innovation by establishing a pathway for Advanced Therapeutic Products (ATPs), which are so complex and unique that the current regulatory frameworks and enforcement tools cannot handle them. The government agency in charge of national health policy, Health Canada, designed an ATP pathway using international approaches. This entails individualized requirements, an iterative approach, and the flexible management of risk through the use of terms and conditions and inspection authority. The transfer of an ATP under existing regulations, or if new regulations are necessary, is based on the amount of regulatory experience with an ATP.

The Food and Drug Regulations and Medical Devices Regulations are slated for amendments in the spring of 2022. These enhancements will increase the system's adaptability, thereby strengthening the implementation of the lifecycle approach. The Minister of Health will be able to impose conditions on authorizations, compel risk management plans for drugs, and extend existing flexibilities for COVID-19 drugs to other drugs under certain conditions.

Currently, most of Canada's cell and gene therapies are classified as biologics, although cell therapy may sometimes be regulated as a medical device. The safety and efficacy of a new treatment must be supported by extensive evidence. 

Commercializing biological cell and gene therapy products requires a "New Drug Submission." The NDS must include the results of all preclinical and clinical trials, regardless of whether they were conducted in Canada or elsewhere, including information on the chemistry and manufacturing of the product, therapeutic claims, and proposed risk management measures. Noting that cell and gene therapies may qualify for priority review or conditional authorization is essential. A variety of diseases and conditions will benefit from cell and gene therapies, most of which are life-threatening or severely debilitating. Priority review or NOC/c may be applicable for diseases or conditions for which no drugs are currently available on the Canadian market or for cell and gene therapies that demonstrate a significant improvement over existing therapies or preventatives.

The proposed modifications to the Food and Drug Regulations may also permit rolling submissions and the use of terms and conditions for these medications.

WHAT THERAPY OWNERS MUST UNDERSTAND

To ensure reimbursement success, a company launching a new cell or gene therapy should initiate market access planning earlier than usual. They must comprehend the patient and clinical journey they are supporting, map the access points, and involve stakeholders throughout the procedure.

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, will require submissions from manufacturers (INESSS). These organizations are independent, non-profit entities tasked with providing objective evidence to Canada's healthcare decision-makers to assist them in making informed decisions regarding the optimal use of drugs and medical devices within the Canadian healthcare system. For instance, a manufacturer must demonstrate to patients, providers, and payers the advantages of a high-priced, one-time cure versus a lifetime of ongoing treatments for a chronic condition. Individualized payment structures that premium improve patient outcomes may be necessary to facilitate reimbursement.

Manufacturers may need to invest in strategies that create a standardized approach to patient access and provide scalability since a relatively small number of patients will be eligible for cell and gene therapies. Manufacturers could, for instance, consider joining a single industry-wide platform or system that allows healthcare providers in a country to access multiple cell and gene therapies. A niche strategy for introducing new therapies to Canada is ideal for global implementation. A commercialization partner capable of deploying an integrated model can guide manufacturers through each step of bringing an innovative treatment to market.