Gene therapies offer a "world of wonders" for patients, but with some 10,000 of the pricey therapies in development, pharma companies and payers need to get outcomes-based payments nailed down, a top Pfizer exec told Bloomberg.
What's more, Pfizer biopharma president Angela Hwang told Bloomberg's Cynthia Koons, gene therapies present some unique scientific and manufacturing challenges.
Gene therapies currently target monogenic diseases, or diseases that are characterized by a single genetic mutation, Hwang said. There are about 3,000 such disorders, and they're all considered rare diseases. Pfizer itself has three gene therapies in development for hemophilia A, hemophilia B and Duchenne muscular dystrophy.
Overall, there are 10,000 gene therapy programs in development, Hwang said. If 10% of them end up working, “you start to see that potentially gene therapy can become a mainstay therapy in how we manage diseases," she said in the Bloomberg interview.
That necessitates the key question about payment. Currently, payment systems are centered on paying for individual drug doses. Hwang said the industry needs to get outcomes-based payments worked out, adding that the process could become easier as science advances and data collection improves.
Looking ahead, new gene therapy launches will “necessarily drive us to come up with different solutions other than the ones we have today,” Hwang said in the interview, referencing subscription-based payments as one option. Her company is already “brainstorming” with payers, she added.
There are just two launched U.S. gene therapies—Novartis' Zolgensma and Spark Therapeutics' Luxturna. Roche is in the process of acquiring Spark, but the buyout has been held up by antitrust regulators.
With already approved gene therapies, the market is adapting. Novartis has offered to allow payers to fund its spinal muscular atrophy gene therapy Zolgensma—which costs $2.125 million—over a period of five years. The drugmaker has also proposed pay-for-performance deals.
Bluebird bio recently won European approval for its beta-thalassaemia gene therapy Zynteglo and is allowing payers to pay for the drug over several years as long as the med continues to work for patients.
For its part, Pfizer is building a gene therapy manufacturing plant in North Carolina to manufacture clinical trial supplies and potential commercial products following approvals.