The US Food and Drug Administration (FDA) has finalised guidance for the clinical development of drugs and biologics to treat amyotrophic lateral sclerosis (ALS), a neurological disease.
In the guidance, the regulator provides information on general product development, clinical trial designs, effectiveness measurement, safety considerations and specific effectiveness trial considerations.
The guidance recommends researchers and companies to interact with the FDA in the early stages of product development for advice on development programmes and the trial design required to generate data for regulatory approval.
The FDA also highlighted the need to minimise placebo exposure and speed-up the trials via master protocols, adaptive designs and enrichment approaches, among others.
A statement from the regulator read: “Specific to clinical trial design, this final guidance provides clarity around some of the questions raised by stakeholders. It emphasises that all patients in ALS trials should receive the best standard of care, and no patient should be denied effective therapies in order to be randomised to a placebo-only arm.”
FDA added that the expedited development and approval programmes are available to provide patients with faster access to new treatments.
FDA acting commissioner Norman Sharpless said: “We also know that many patients with ALS are seeking access to investigational products for this devastating disease.
“The FDA remains committed to helping patients and health care professionals evaluate options for accessing investigational products, such as participating in a clinical trial, obtaining access to an unapproved, investigational product outside of a clinical trial through expanded access (compassionate use) or providing information for patients about the Right to Try Act.”
Furthermore, the FDA urged drugmakers to provide information on their research status and availability of products, as well as sponsors to provide access to investigational drugs even after the trials are complete.