Here are the best takeaways:
Access remains a problem
Gene therapies can cost millions per dose, and insurers and government health programs have been slow to cover them, said Erica Cischke, vice president of government affairs at the Alliance for Regenerative Medicine, an industry group for their makers.
« We have to make sure that people can actually access therapies once they’re approved, » Cischke said, noting that patients often have to cross state lines to receive treatment.
It can also take many years for patients to be diagnosed with rare diseases that gene therapies can cure, delaying treatment, said Annie Kennedy, chief of policy, advocacy and engagement at the EveryLife Foundation for Rare Diseases, a group of patient advocacy.
The FDA is working to make gene therapies marketable
The FDA is working to make treatments more commercially viable, said Peter Marks, director of the agency’s Center for Biologics Evaluation and Research.
« Part of that is brute force production, » Marks said. « We have to reorganize and probably develop more automation in production. »
Another part is taking advantage of redundancy. Often, viral vector gene therapies have the same backbones that are reused, Marks said.
Marks also touted a provision passed by Congress the 2022 government funding package aimed at making drug development more efficient by reducing regulatory burdens.
Regular communication with the FDA can also help smaller companies speed up the regulatory process, he said, pointing to Operation Warp Speed, which brought Covid vaccines to market in record time.
Eyes on health equity
Officials and advocates have stressed that health equity concerns must be at the forefront.
The best way to reach underprivileged populations is to have a « bustling commercial enterprise » and allow Medicare and Medicaid to cover treatments, Marks said.
Carla Rodriguez-Watson, director of research at the Reagan-Udall Foundation, which Congress created to advise the FDA, said real-world data from clinic visits can help identify which demographic groups need the drugs.
Joni Rutter, director of the National Center for Advancing Translational Sciences at the National Institutes of Health, said her agency engages with the community to see what research gaps need to be filled and accounts for inequities from the outset.
Buzz around artificial intelligence
Speakers were optimistic about AI’s potential to support gene therapies, with some caveats.
Marks called AI a potential « game changer » in helping produce complex treatments and said the FDA has used it to detect potential safety issues.
AI can help ensure production is efficient and can help analyze data, he said, but it’s not a silver bullet.
“Some of that won’t be through artificial intelligence. Some will be through well-chosen endpoints that can be used for expedited approval,” Marks said.
Rodriguez-Watson said tech developers need to make sure the data underpinning AI is representative of the population, or they risk making disparities worse.
Pay for performance
The government should continue to promote new ways of paying providers, emphasizing patient outcomes, not the number of services provided, the rapporteur said.
Gene therapy advocates say they should ensure that insurers and government programs pay for them.
“They help states mitigate some of the uncertainty around the coverage of these new therapies,” Cischke said.
To this end, he pushed for the passage of the MVP law by the chairman of the House subcommittee on health, energy and commerce Brett Guthrie (R-Ky.), which would promote value-based compensation agreements in Medicaid.