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The Promises and Challenges of Gene Therapy


Gene therapy offers great hope for patients, but comes with a bevy of challenges for patients and payers alike.

DNA strands

Gene therapies-infamous for their high prices, as well as their great potential for patients-are slowly becoming a larger part of the healthcare landscape.

As more of these therapies come to market, payers have been searching for a way to deal with the high up-front costs. Compared with traditional medicines, gene therapies largely frontload their costs-rather than a series of payments over the course of months or years, many gene therapies require fewer doses overall, or are a one-time treatment.

At the Association of Managed Care Pharmacy Annual Meeting 2019, Kellie Rademacher PharmD, vice president, access experience team for Precision for Value, outlined some of the pros and cons of gene therapy in her talk, Managing Access for Gene Therapy: Learnings from an International Perspective.

Overall, Rademacher said, gene therapies have been accessible to patients, but payers are still adapting and attempting to find the best models.

“There is a significant need in the United States to develop payment models that support potentially curable therapies that come with substantial price tags,” she said. “For gene therapies to be sustainable, the healthcare system needs to adapt the reimbursement and valuation of these products.”

As more of these high-cost therapies enter the market, Rademacher explained, it will be up to managed care organizations to evaluate them, decide on their place in a formulary, consider access problems, and also to provide reimbursement.

“With only a couple of agents on the market, established techniques in managed care have been sufficient: however, as multiple gene therapies enter the market and competition within a category increases, managed care needs a more dynamic way to provide access, manage affordability, and support the sustainability of new therapies,” said Rademacher.

But how can organizations meet those challenges? The answer, according to Rademacher, is not a one-size-fits-all solution. Rather, it is “more likely that managed care organizations will need to be nimble enough to have multiple modalities for payment based on the archetype of the population (e.g., orphan diseases without other options, orphan diseases with a current standard of care, populations with larger incidence and prevalence).” 

Overall, Rademacher said, scientific innovation is moving faster than the healthcare system, and it’s time for healthcare to “adapt and catch up” with what is an undeniably “innovative scientific advancement with large-scale applications.”

“Both the government and private sector will need to collaborate across stakeholders to face these challenges,” concluded Radmacher. “As the market shifts, adaptation and reimbursement innovation needs to be rapid and fluid.”

Nicholas Hamm is an editor with Managed Healthcare Executive

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