Gene Therapy: International Regulatory and Health Technology Assessment (HTA) Activities and Reimbursement Status
March 20, 2019

Gene Therapy: International Regulatory and Health Technology Assessment (HTA) Activities and Reimbursement Status

Gene therapy is a growing area of interest for the treatment of several diseases. These therapies are intended to offer a one-time treatment to modify a person’s genes through a variety of mechanisms in order to treat or cure a disease.

Over the last decade, several important scientific advances have been made in this field and these therapies are now being submitted for regulatory and reimbursement evaluation. For instance, in August 2017 the FDA approved the first gene therapy for use in the US: Kymriah® (tisagenlecleucel) for acute lymphoblastic leukemia (ALL) in children and young adults. Soon after, in October 2017, a second gene therapy, Yescarta® (axicabtagene ciloleucel), was approved to treat patients with certain types of large B-cell lymphoma in adults. In December 2017, the FDA also approved the novel gene therapy Luxturna® (voretigene neparvovec-rzyl) for the treatment of a rare inherited form of vision loss.

However, transitioning these new therapies into clinical practice poses challenges given the lack of long-term evidence related to efficacy and safety as well as the costs associated with these therapies; therefore, the regulatory and reimbursement processes for these therapies may require novel pathways and interpretations of value.


Helping Canadian jurisdictions understand and prepare for gene therapy technology

 To inform the approach and process for evaluating gene therapy and to help Canadian jurisdictions understand and prepare for this new health technology, Medlior worked with the Canadian Agency for Drugs and Technologies in Health’s (CADTH) to prepare an Environmental Scan entitled Gene Therapy: International Regulatory and Health Technology Assessment (HTA) Activities and Reimbursement Status. This Environmental Scan enhances the current understanding of international regulatory, HTA, and reimbursement activities related to gene therapies.

The environmental scan found that evidence generation was commonly cited as a key challenge for gene therapies. For instance, randomized controlled trials are often not appropriate for the intended populations, identifying appropriate comparators for gene therapy is difficult, and long term safety outcomes are often not available. In addition to challenges with evaluating the efficacy and safety of these new products, evaluating the cost-effectiveness of gene therapies may be difficult within the existing approaches to HTA and new approaches may need to be developed.

Click here to read the full Environmental Scan.

Gene Therapy in Canada

 Since the publication of this Environmental Scan, the first gene therapy was approved for use in Canada. Similar to the United States, in September 2018 Kymriah® received regulatory approval for some patients with relapsing/refractory hematological malignancies. Most recently, a second gene therapy, Yescarta® for the treatment of adults with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, received regulatory approval in February of 2019. Both of these therapies are chimeric antigen receptor T-cell (CAR-T) therapies which involve collecting and modifying a patient’s own T-cells.

Click here to read Medlior’s latest white paper on the implementation of CAR-T Therapies.

About Medlior

At Medlior, our diverse group of carefully selected experts offer substantial health system, academic and industry experience which ensures every Medlior project provides meaningful insights.

Our seasoned biostatisticians and epidemiologists use de-identified patient data from provincial health systems to address a variety of research questions. Our specialties include real-world data solutions, statistical analysis, health technology assessment, economic evaluation, systematic literature reviews, environmental scans and medical writing.

Each member of the Medlior team holds an advanced degree, which is paired with relevant work experience from academia, the health system, consulting and/or the pharmaceutical industry for a clear advantage in conducting health outcomes research.

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