FDA Expanded Access: What the Right to Try Act Actually Removed

Two statutes govern a patient's access to a drug that is not yet approved: FDA's expanded access program under 21 CFR Part 312, Subpart I, in place since 2009, and the Right to Try Act, enacted May 30, 2018. Sponsors routinely treat the second as a replacement for the first — a way around FDA entirely. It is not. Right to Try removes one specific step: FDA's authorization of the use. It leaves the decision that actually controls whether a patient gets the drug exactly where expanded access already put it — with the company.

Three tiers, one set of criteria

Subpart I is not a single program; it is three mechanisms sized to the population requesting access, all gated by the same general criteria in 312.305: the patient has a serious or immediately life-threatening disease with no comparable or satisfactory alternative therapy, the potential benefit justifies the risk in the context of that disease, and authorizing the use will not interfere with the clinical investigations needed to support marketing approval. A sponsor's IND application strategy should account for all three tiers before the first request lands, not after.

• Individual patients, including emergency use (§312.310). A single patient's physician requests access; emergency authorization can be obtained by phone, with written follow-up required.
• Intermediate-size patient populations (§312.315). A defined cohort larger than one patient but smaller than the population a treatment IND targets, often used while a pivotal trial is still enrolling.
• Treatment IND or treatment protocol (§312.320). Sustained, widespread access during late-stage development, typically once a drug shows promise for a serious condition and trials are ongoing.
• Cost recovery (§312.8). A sponsor may charge for an investigational drug under expanded access, generally limited to direct costs and generally requiring separate FDA authorization.

What the Right to Try Act actually changed

The Right to Try Act, codified at 21 U.S.C. §360bbb-0a, defines its own eligibility on both sides. An eligible patient must be diagnosed with a life-threatening disease or condition as defined at 21 CFR 312.81, must have exhausted approved treatment options and be unable to participate in a clinical trial for the drug — both physician-certified — and must provide written informed consent. An eligible investigational drug must have completed a Phase 1 trial, must not be approved or licensed under FD&C Act Section 505 or PHS Act Section 351, and must either remain under active investigation intended to support a future marketing claim or be the subject of a pending application. Meet both sets of conditions and the patient can obtain the drug without the FDA authorization that expanded access otherwise requires.

Right to Try removes a federal approval step. It does not create a right to the drug — the company still decides whether to supply it, exactly as it would under expanded access. The distinction sponsors miss

The decision that doesn't move

This is the point most access-request discussions skip: the Right to Try Act creates no obligation on a manufacturer or sponsor to make an investigational drug available, to set a price, or to agree to any specific patient's request. It removes a federal-approval step for a narrowly defined set of patients and drugs; it does not displace the company's own gatekeeping. A sponsor evaluating a rare disease program with limited drug supply, an actively enrolling pivotal trial, or a molecule whose safety signal is still being characterized faces the same internal decision whether the request cites Subpart I or the Right to Try Act. Building that decision process once — who evaluates a request, what criteria apply, how it is documented — is the actual work, and it sits upstream of either statute.

None of this requires a novel legal position. It requires a written policy that exists before a physician calls about a specific patient, maps that call to the correct Subpart I tier or confirms Right to Try eligibility, and keeps the manufacturer's own discretion — unchanged by either pathway — explicit rather than improvised. Programs that build this alongside their broader expedited pathway strategy, rather than as a reactive one-off, are the ones that answer the first request correctly instead of the fifth.