Marti Nelson Cancer Foundation Expanded Access Position Statement
Drug sponsors are sometimes permitted by FDA to undertake expanded access programs prior to approval through the Treatment IND mechanism. Under some circumstances, companies may wish to consider establishment of such programs as a complement to their ongoing clinical trials. Expanded access programs may be particularly desirable in connection with drugs that hold great promise in diseases with no satisfactory therapy, as demand for access may be intense in patients who cannot meet the eligibility criteria for the clinical trials. Expanded access programs should be undertaken only in the following circumstances:
- Phase III trials are near completion, and available data indicate efficacy exceeding standard therapy.
- Toxicity is relatively manageable.
- The sponsor has adequate resources (in terms of both drug material and program infrastructure) to enable an expanded access program to function efficiently.
- Access to the drug by the individual patient will not threaten adequacy of supply for purposes of the ongoing clinical trial. In such cases it is incumbent on the industry sponsor to document the existence of supply shortfalls and plans for remediation.
- There is no reasonable prospect that individual access outside clinical trials will contribute to delay of FDA action on the drug.