Recent changes at the US Food and Drug Administration (FDA) have changed the circumstances for rare disease drug development, but this remains an area of opportunity for biotechs.
At the recent 3rd Annual Clinical Trials in Rare Diseases (CTRD) meeting, Rik Mehta, ex- FDA consumer safety officer, discussed the effect of the Trump Administration on policies surrounding rare disease drug development. Mehta is also the co-founder and CEO of Lactiga, a biotech developing innovative mucosal antibody therapies.
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The debate over confirmatory studies has long shaped regulatory science. However, the appointment of Dr. Vinay Prasad, a noted critic of trials that use surrogate endpoints, as the head of FDA’s Center for Biologics Evaluation and Research, has meant the topic has come to the forefront at the agency. Different appointments highlight differing perspectives, with some pushing to accelerate products for unmet medical needs, while others emphasise rigorous evidence and questioning the relevance of surrogate endpoints, Mehta said.
It remains to be seen how Prasad’s appointment will change the agency’s view. The administration is pushing for faster market access and more involvement of the patient voice, but on the other hand, the FDA’s leadership could also set more rigid expectations for endpoints.
Nonetheless, the FDA review divisions have become more open to surrogate endpoints and biomarkers, especially in rare diseases, allowing confirmatory studies post-approval, said Mehta. He advised companies to engage with the FDA early, propose endpoints, and negotiate with agency, especially in rare disease indications as the FDA’s mission remains centered on public health and transformative therapies.
Incentives in rare disease research
Initiatives like the Rare Disease Evidence Principles programme aim to accelerate approvals, sometimes within months, using approaches such as natural history data and single-arm studies and reducing the need for confirmatory studies, which Mehta cites as an indication that rare diseases remain a focus for this administration. However, he acknowledged that there is still room for better alignment with patients, advocates, and stakeholders.
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By GlobalDataThe pediatric priority review voucher program, was considered at risk of expiry since it wasn’t renewed until recently. However, the Give Kids a Chance Act of 2025 (H.R. 1262) was recently reported in the House of Representatives, to support its reauthorization. According to Mehta, voucher programmes have the potential to “inject federal priorities into drug development by offering valuable incentives that can transform company trajectories, especially in a volatile economic market”.
Although media and politics influence the process, ultimately, decisions largely remain with FDA review teams and there is no big shift involving higher levels in decision-making under this administration, unless controversies over efficacy escalate, he says.
With the commissioner introducing a separate national health priority voucher programs, questions remain about the future of the rare pediatric voucher program. The outcome of the midterm elections, depending on the control of the House and Senate, will affect policy priorities, particularly regarding the rare disease voucher programmes.
Regulatory announcements on drug repurposing and pricing
Last month, the administration also announced an initiative involving collaboration between the National Institutes of Health (NIH) and the Food and Drug Administration (FDA) to facilitate research and streamline the approval process for new applications of existing drugs under the Make America Healthy Again (MAHA) initiative. Such steps will be important for financing, because repurposing offers a more de-risked pathway since safety profiles of drugs are better established, making them more attractive to both the FDA and investors, says Mehta. Therefore, repurposed drugs may become more significant in future innovation strategies, he added.
Policies such as the Most Favored Nation (MFN) model will inevitably shape the affordability landscape. Also, political and economic pressures could influence regulatory decisions, and companies not aligned with MFN-type approaches might risk having their voucher withheld or revoked, said Mehta.
The fireside chat ended on the topic of judgement calls on clinical value. Following a Complete Response Letter (CRL), the FDA will make a judgment of clinical value and there are questions surrounding how strict or flexible it will choose to be. The question of “a softer or more stringent FDA approach” is particularly evident in rare diseases, where patient recruitment is difficult, Mehta said.
The CTRD meeting took place on 17-18 September in Princeton, New Jersey
