The gene therapy race

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Crystal Yednak Senior Manager, Health Research Institute, PwC US February 05, 2020

Driven by scientific advances, life sciences companies are rushing to develop and invest in gene therapies that can treat and even cure cancers and diseases. 

The FDA expects to receive more than 200 investigational new drug applications per year for gene and cell therapies starting in 2020. By 2025, the federal regulator expects to approve between 10 and 20 cell and gene therapy products per year.

Four considerations

Biotechnology companies are focused on obtaining regulatory approval for these therapies, but the post-approval period contains significant tests, too. That includes getting therapies to patients and getting paid for them. Speed—for manufacturing, distribution and reimbursement—is of the essence. For patients waiting for cancer therapy, delays in treatment may mean the difference between life and death. Delays may also undermine outcomes-based contracts with insurers and government payers.

Rapid production is difficult. In addition to the challenges of making the gene therapy products, biotechnology companies producing autologous gene therapy products will need to manage a complex supply chain to get a patient’s cells from a hospital to a processing facility and back, vein-to-vein. A disruption at any point could ruin the product or harm a patient. Companies also should recognize that the lengthy process can induce patient anxiety, and work on ways to keep them actively informed. Without addressing these barriers to market with new manufacturing, operational and commercial models, it will take longer for companies to realize the full sales potential of their therapies.

The FDA expects to receive more than 200 investigational new drug applications per year for gene and cell therapies starting in 2020. By 2025, the federal regulator expects to approve between 10 and 20 cell and gene therapy products per year.

While gene therapies likely will create opportunities for the life sciences sector, it may also produce new strains on existing business models and perhaps even society. Low reimbursement levels may discourage investment given the high costs of production and small patient populations. High prices may put essential treatments out of reach for the patients who need them most. As the life sciences industry enters this new, gene-focused world of development, it will need to think about their place in it, and how it plans to compete on behalf of patients and shareholders. 

Staffing will be another key challenge for producers of gene therapies. Companies should prepare to train new employees or upskill existing ones. Competition for existing talent will be fierce, making upskilling of existing staff a potentially more feasible and cost-effective pathway to success.

For citations, implications and insights, please read our full report, Gene therapies require advanced capabilities to succeed after approval.

For more of HRI’s insights and content, visit our Regulatory Center and report library.

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Trine K. Tsouderos

HRI Regulatory Center Leader, PwC US

Tel: +1 (312) 241 3824

Crystal Yednak

Senior Manager, Health Research Institute, PwC US

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