Proposed FDA rules provide reasonable balance
FDA’s mandate as far as human drugs are concerned is to ensure that drugs sold in the U.S. are safe and effective for their intended usage. So when faced with criticisms that their rules prevent some very ill patients from receiving life-saving medicines, FDA was faced with a challenging problem–how can regulators ensure that only safe and effective medications are sold while also ensuring that patients don’t die or suffer unnecessarily during the investigational period?
It’s a tough problem. Too much weight given to the needs of ill patients can jeopardize completion of the investigational program. This is a particular concern for therapies aimed at rare diseases, where clinical-trial subjects are few and far between. Delay or non-completion of an investigational program leaves sponsors without a timely marketed product and jeopardizes therapy access to a broad population. Too much weight given to the evidenciary requirements of regulators, and the patients who might benefit most from investigational drugs could be left inadequate therapeutic options.
Rules already exist for access to investigational drugs, but these rules are somewhat vague in their criteria for implementation and provide for treatment use of investigational drugs in circumstances that make it difficult for individual physicians to intervene on a patient’s behalf, namely the treatment IND (aka “treatment protocol”) and emergency use. I won’t go into detail about existing rules. You can read descriptions of them in the proposed rule (expandedaccess).
In the proposed rule, FDA keeps the older terminology (but clarifies its meaning) and adds two new expanded access protocols aimed at addressing the needs of individual patients and intermediate-sized (~10 to ~100) patient populations, respectively. Evidentiary requirements for treating seriously ill individual patients with investigational drugs are lowest (with animal data often sufficing for those with life-threatening diseases), with increasing evidence of safety and efficacy required for intermediate-sized pools of patients and larger groups (under the treatment IND). Individual physicians will still have to work with sponsors and collect drug disposition and adverse event data, but cooperative sponsors can include treatment protocols for individual patients in an amendment to their existing IND. Physicians won’t have to convince sponsors to open a treatment IND or provide drug under emergency use.
FDA has also wisely created checks that should ensure that investigational drugs’ paths to the market are not unnecessarily delayed, for example by limiting treatment to patients with serious diseases who have no other approved-treatment options. FDA will also maintain oversight of treatment and will have 30 days to review each IND amendment.
Overall, I think both sponsors and patient advocates should be pleased with these new rules. FDA’s mandate to protect patients by ensuring the safety and effectiveness of marketed therapies is fulfilled without jeopardizing the health of patients for whom marketed therapies are not an option. Well done.
