Whenever a gene therapy worth a million dollars is approved, Peter Marks receives an email from FDA chief Robert Califf.

FDA chief Robert Califf sends Peter Marks an email every time a gene therapy valued at a million dollars is approved.

Following the approval of Orchard Therapeutics' gene therapy for a rare genetic condition in March, Dr. Peter Marks received a brief email from Commissioner Robert Califf. Marks, serving as the director of the Center for Biologics Evaluation and Research (CBER), disclosed this at the American Society of Gene and Cell Therapy annual meeting. Califf typically informs Marks about the approval and pricing of gene therapies through concise emails. Marks recounted receiving an email with a "$4.25 million exclamation point," indicating the therapy's price.

In March, Orchard's Lenmeldy gained FDA approval for treating the rare genetic disorder metachromatic leukodystrophy (MLD). Priced at $4.25 million, Lenmeldy swiftly became the most expensive drug, surpassing CSL Behring and uniQure's Hemgenix, priced at $3.5 million. Orchard justified the price, citing the therapy's potential value and long-term healthcare impact. Peter Marks of CBER clarified that pricing isn't factored into the agency's approval decisions but emphasized the need to assess the overall sustainability of gene therapy pricing. He highlighted the FDA's efforts to potentially influence pricing by streamlining regulatory processes and improving manufacturing technologies to reduce costs for biotech companies.

Marks emphasized that unless significant changes are made to the pricing of gene therapies, their global accessibility will remain limited. He asserted that enhancing access to gene therapy is paramount in reducing costs. "I am driven to work every day by the desire to assist more individuals globally," Marks stated. He believes that lowering the cost of these therapies could potentially enable them to replace conventional treatments, such as aiding in significantly reducing cholesterol levels for ordinary individuals. 

Additionally, Marks highlighted the importance of long-term patient monitoring post-approval, especially for patients with conditions like sickle cell disease or beta thalassemia. He emphasized the necessity of continued follow-up to gauge sustained benefits and detect any adverse effects. However, he acknowledged that tracking small populations with rare diseases can be costly. Marks mentioned that the FDA will explore ways to incentivize the collection of long-term follow-up data in the coming years, as this real-world data could be crucial in demonstrating value for payers. 

Despite the high costs associated with gene therapies approved for rare diseases and small populations, Marks noted that they will never match the spending levels seen with the GLP-1 weight loss/obesity trend. He emphasized that even with a significant number of approvals for rare disease gene therapies, their impact on expenditure will pale in comparison to that of GLP-1 agonists.

Source: Fierce Pharma