Canadian experts discuss somatic gene therapy approval and use

By Samantha Black, PhD, ScienceBoard editor in chief

November 6, 2020 -- Gene therapies offer great benefits to patients but could strain healthcare budgets and exacerbate existing treatment inequities in Canada, according to an expert panel that was commissioned to write a report by the Council of Canadian Academies (CCA).

In Canada, two gene therapies for cancer treatment (Novartis' Kymriah and Gilead's Yescarta) and one for treatment of a rare neuromuscular disease (Biogen's Spinraza) have received marketing authorization, with many others being evaluated in clinical trials.

The arrival of somatic gene therapies has prompted an active global discussion about the merits and challenges of their approval and use. The National Research Council of Canada asked the CCA to examine key legal, regulatory, ethical, societal, and policy challenges specific to the approval and use of somatic gene and engineered cell therapies in Canada.

The report is titled "From Research to Reality: The Expert Panel on the Approval and Use of Somatic Gene Therapies in Canada."

"Rapid scientific advances mean potentially life-changing treatments are approaching the clinic at an accelerated pace," said Janet Rossant, PhD, chair of the CCA's expert panel. "These new therapies, however, pose a number of challenges in terms of their introduction into the Canadian healthcare system and ensuring access to those who would most benefit."

Main findings of the report include:

The diversity of gene therapies requires a flexible and tailored approach to addressing access and affordability challenges. Flexibility will be required for regulatory approval, funding decisions, manufacturing, and clinical use. Further, reimbursement approaches will need to be able to accommodate the challenges of cost and accessibility to patients.

Risk-based purchasing agreements and post-marketing surveillance could mitigate the significant clinical and economic uncertainties associated with approved gene therapies. Performance-based agreements between payers and manufacturers could be used to share the risks associated with funding gene therapies. Ongoing monitoring of long-term performance of approved therapies could reduce uncertainty over time.

High prices, complex provision, and the nature of diseases treated by gene therapies exacerbate existing inequities in healthcare access. The value and affordability of gene therapies by public drug plans should be assessed. Rare diseases may also pose additional difficulties for receiving accurate diagnosis and assessing high-cost treatments in public healthcare systems.

Different conceptions of value may lead to disagreement over the merits of publicly funding individual gene therapies. Based on data from Canada and Europe, society supports spending on drugs that offer the greatest improvements at the least cost but favors spending on relatively high-cost drugs in cases where diseases are severe and lack alternative treatments.

Stewardship of public investments in gene therapy research could alleviate challenges associated with commercialization and high drug prices.

In Canada, coordination of provinces, territories, and the federal government could establish common principles and approaches to accessing high-cost therapies.

"The approval and use of gene therapies challenges payers, regulators, industry, patients and families," said Eric Meslin, PhD, president and CEO of the CCA. "This report covers many of these challenges as well as possible paths forward and will help to support evidence-informed decision making in Canada."

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