FDA proposes new pathway for custom treatments
Federal health officials have proposed a new framework aimed at facilitating the development of customized treatments for patients with hard-to-treat diseases, including rare genetic conditions, that the pharmaceutical industry has often deemed unprofitable, reports BritPanorama.
The preliminary Food and Drug Administration guidelines, if adopted, would create an alternative pathway for bespoke therapies that have typically been tested on only a limited number of patients, owing to the difficulties associated with larger trials. The FDA announcement highlights gene editing as a focus, while officials noted that this new approach could extend to various other drugs and treatments.
This represents a significant shift sought by patients, advocates, and researchers who deal with rare diseases, which frequently do not conform to the traditional business models of the pharmaceutical industry or the FDA’s standard drug-approval processes.
“It is our priority to remove barriers and exercise regulatory flexibility to encourage scientific advances and deliver more cures and meaningful treatments for patients suffering from rare diseases,” stated FDA Commissioner Marty Makary in a release.
The announcement follows Makary’s previous statement indicating that the FDA would discontinue its longstanding requirement for two clinical trials for standard drug evaluations. This revision is part of a series of changes to FDA norms and standards that have often bypassed the conventional federal procedures typically required to amend agency regulations.
Senior FDA officials clarified that the recent changes, including the newly proposed pathway, do not amount to new FDA standards. The agency plans to accept public comments on its draft guidance for a 60-day period prior to finalizing it.
In recent years, academic institutions have demonstrated the potential of using emerging technologies to rectify specific defects in a patient’s genetic makeup. For instance, a team at the Children’s Hospital of Philadelphia and the University of Pennsylvania designed a therapy using CRISPR, a groundbreaking gene-editing tool, to treat a baby diagnosed with a rare illness that leads to excessive ammonia accumulation in the blood.
Traditionally, the FDA mandates that drug manufacturers establish the safety and efficacy of experimental treatments via clinical studies that compare participants receiving the therapy with those undergoing a placebo treatment or an alternate intervention. A greater enrolment typically correlates with stronger evidence.
However, for conditions affecting only a small percentage of the global population, pharmaceutical companies often lack the motivation to invest substantial amounts to complete trials and navigate the rigorous FDA approval steps, which can extend for a decade or more.
The proposed pathway aims to establish a standardized system for authorizing experimental treatments and significantly offers firms the opportunity to commercialize these therapies.
The FDA currently allows the application of experimental drugs under the “compassionate use” provision for individuals without other medical alternatives, but this process can be complex and strictly bars companies or researchers from profiting from treatments not cleared by the FDA.
The newly introduced pathway, labeled “plausible mechanism,” refers to the criteria that FDA regulators will need to see met before approving any experimental therapies.
According to FDA officials, this approach will primarily apply to well-understood conditions where there exists a credible basis to believe that the therapy will impact the underlying genetic or cellular biology of the disease. Researchers must also confirm the therapy’s successful targeting of the specific genetic or biological anomaly affecting the patient.
