SUPPORT FOR FDA EXPANDED ACCESS (COMPASSIONATE USE) PROGRAM

To advocate that the Food and Drug Administration (FDA) Expanded Access (Compassionate Use) Program be the primary mechanism for patient access to drugs for which an investigational new drug application (IND) has been filed, in order to preserve the integrity of the drug approval process and assure patient safety; further,

To advocate for broader patient access to such drugs under the FDA Expanded Access Program; further,

To advocate that IND applicants expedite review and release of drugs for patients who qualify for the program; further,

To advocate that the drug therapy be recommended by a physician and reviewed and monitored by a pharmacist to assure safe patient care; further,

To advocate for the patient's right to be informed of the potential benefits and risks via an informed consent process, and the responsibility of an institutional review board to review and approve the informed consent and the drug therapy protocol; further,

To support the use of the Right-to-Try pathway in instances in which all other options have been exhausted, provided there is (1) a robust informed consent process, and (2) institutional and clinical oversight by a physician and a pharmacist.

Rationale

Patient access to drugs for which an investigational new drug application (IND) has been filed is made available on a limited basis to individual patients under a compassionate-use program regulated by the FDA. With information about clinical trials and drugs under development readily available to patients, there is an increased demand for access to these therapies. In addition, three states have passed laws to permit patients who have exhausted approved drugs and treatment to have access to these potentially lifesaving drugs. Other states may follow suit in the future, and the FDA has begun to respond to this growing patient demand by streamlining its application process for individual patient expanded access. In order to respond to state legislative proposals, ASHP advocates preserving the integrity of drug development through strengthening the evidence-based clinical trial process and expanded patient access.

In 2018, Congress passed Right-to-Try legislation, which, per FDA, “is one pathway for patients diagnosed with life-threatening diseases or conditions who have exhausted all approved treatment options and are unable to participate in a clinical trial to access certain drugs that have not been approved by the Food and Drug Administration (FDA).” The program functions outside of FDA control, with patients and their physicians coordinating directly with manufacturers for access to investigational new drugs. ASHP advocates that the FDA’s Compassionate Use Program remain the primary access point for investigational new drugs, but supports the use of Right-to-Try for patients who have exhausted all other options. Furthermore, ASHP advocates for additional patient safety requirements related to informed consent and clinician monitoring for patients accessing investigational new drugs through the Right-to-Try pathway.