Most FDA drug approvals in 2024 targeted rare diseases
Precision AQ hires rare-disease expert to address complexities
More than half of all therapies approved by the U.S. Food and Drug Administration (FDA) in 2024 were designed to treat rare diseases, according to an advisory firm that helps life sciences companies navigate the commercialization process.
One such approval was for Kebilidi (eladocagene exuparvovec-tneq), a gene therapy developed by PTC Therapeutics that in November was granted accelerated approval by the FDA to treat aromatic L-amino acid decarboxylase (AADC) deficiency.
Approvals of Kebilidi and other treatments targeting a range of rare disorders hold the potential to substantially improve care for the millions of people who live with rare diseases, in many cases offering treatments where limited or no options are available.
But the increased number of rare disease treatments that are winning approval comes at a cost — literally, as these treatments tend to carry hefty price tags. For example, in Europe and the U.K., where Kebilidi is marketed as Upstaza, the gene therapy carries a list price of around $3.7 million for its single dose.
In light of the high prices associated with treatments for rare diseases, insurance companies and other payers are increasingly scrutinizing the cost-to-benefit profile of these treatments, according to a press release from Precision AQ. This means that treatment developers need to adopt new strategies to ensure that patients are able to access their therapies, the firm said.
A changing landscape for FDA drug approvals
Precision AQ said its access experience team aims to help companies navigate this ever-evolving landscape. Launched in 2009, the team includes more than 25 experts with experience in treatment access. Their goal is to help therapy developers understand payer strategies, barriers to reimbursement, and ongoing changes in policies.
The most recent addition to Precision AQ’s access experience team is Julia Wermerskirchen, whose expertise is focused on working with payers to help people with rare diseases access treatments. Wermerskirchen was vice president of payer and provider relations at Optum Frontier Therapies, where she led national efforts to improve access to rare-disease treatments.
“Julia’s appointment underscores Precision AQ’s commitment to equipping life sciences companies with the expertise needed to drive sustainable access to rare disease therapies,” said Chad Clark, president and chief operating officer at Precision Medicine Group, Precision AQ’s parent company. “Her strategic acumen, deep industry relationships, and proven ability to navigate payer landscapes will be instrumental in ensuring that innovative treatments reach the patients who need them.”
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