- The FDA is closing in on plans to ease the burden on physicians seeking to access investigational drugs through compassionate use, Regulatory Focus reports. The agency aims to reduce the time needed to complete an application to 45 minutes.
- Compassionate use allows a patient to use an investigational drug outside of a clinical trial and before the drug is approved. However, the process can require hours of paperwork by physicians.
- At the same time as the agency works to simplify applications, the Reagan-Udall Foundation (the nonprofit arm of the FDA) is designing an online navigator to assist physicians and patients through the process.
The FDA approves nearly all of the compassionate use requests it receives. But both the physician treating the patient and the company producing the drug must agree to proceed with a compassionate use application, creating a hurdle to early access.
Patient advocates and lawmakers have pressured the FDA to make it easier for terminally ill patients to access potentially helpful new drugs.
The Reagan-Udall Foundation is hoping its online navigator will ease the process for physicians to request compassionate use through the current process. The navigator will give doctors a directory on FDA policies and points of contact, along with more information and context on the process.
On Monday, the RUF held a half-day workshop, to discuss the best way to handle information and design the navigator. However, the tool will only be a source of information, not a determinant of eligibility or access.
Detractors suggest that the money could be better spent helping the FDA process drug applications more quickly, notes Regulatory Focus. Additionally, some companies are leery compassionate use cases could require alterations to or otherwise compromise an ongoing clinical trial.
But advocates can point to the example of Syntex Corporation's drug ganciclovir, used to treat viral eye infections in patients with AIDS, which was approved based on efficacy demonstrated in a compassionate use case.