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Neuropathy Action Foundation Releases White Paper On Adjusting Policies for Gene Therapies


The paper discusses multiple steps the industry can take to ensure that patients have access to these treatments.



Gene therapy is changing healthcare, but healthcare may have to change for gene therapy.

The Neuropathy Action Foundation (NAF) announced that it is releasing a new white paper that details the ways that the healthcare system may have to adapt in order to ensure that gene therapy is generally an option for patients.1 In a press release, the foundation outlined several specific adaptions the industry should consider.

In a press release, NAF board president Dominick Spatafora said, “The promise of gene therapy is immense, particularly for patients with neuropathy and other conditions with limited or no treatment options. Neuropathy patients, researchers, and clinicians are all hopeful that gene therapy products can create new options for treatment for certain kinds of neuropathy, including inherited peripheral neuropathy (IPN), which is caused by generic mutations in more than 100 genes. For patients to access these innovative therapies, however, innovative policies will also be required."

Over the past several years, there has been a renewed energy behind gene therapy. Researchers have developed a significant number of new treatments using the method. However, gene therapy treatments are often quite complex and expensive. This creates a situation where these therapies are highly effective, but out of reach for many patients.

According to NAF, the first gene-therapy treatment was only approved for use in the U.S. in 2017, with more than 30 having been approved by December, 2023.

The foundation details three specific steps the industry should consider. First, it says that policy and coverage decisions must be centered on patient and family input. This simply means that patients and their families should have input on which treatments are used based on their unique circumstances.

Second, the ways that medical interventions are valued needs to be reassessed. A “broader range of outcomes” must be considered. Lastly, in order to make these treatments truly available to patients, new payment models must be considered. Gene therapies aren’t just expensive, they also have a wider range of uncertain outcomes associated with them. Traditional pricing and payment models aren’t well suited for these treatments, so the industry needs to consider new models to meet this challenge.

In the past, experts have discussed using value-based care models to determine payment coverage for gene therapies and other cutting-edge treatments.

The NAF is a neuropathy-focused organization, and is approaching the issue from that perspective. Gene therapies can treat a wide variety of conditions and diseases, and the suggestions made by NAF in its white paper could be applied across the industry.


  1. Neuropathy Action Foundation Launches White Paper on Future of Gene Therapy & Urges Innovative Policy Approaches to Ensure Patient Access. Neuropathy Action Foundation. March 4, 2024. https://www.prnewswire.com/news-releases/neuropathy-action-foundation-launches-white-paper-on-future-of-gene-therapy--urges-innovative-policy-approaches-to-ensure-patient-access-302076790.html
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