BIO Principles on Expanded Access to Investigational, Unapproved Medicines
BIO member companies are committed to making innovative biotechnology medicines available to patients who need them through efficient development of approved, safe and effective products.
BIO believes that the best way to fulfill this commitment is to complete the clinical studies necessary to demonstrate safety and effectiveness. Patient enrollment in such clinical trials is essential for drug approval.
BIO member companies also understand that seriously ill patients with limited or no alternative therapies, and who are ineligible or unable to participate in the clinical trials, may desire access to an investigational medicine being studied in the trials. Such access outside of the clinical trial setting, or “expanded access,” may be sought through a company's expanded access program, or through individual patient expanded access, if available.
In determining whether and how to establish expanded access programs or to provide expanded access to investigational medicines on an individual-patient basis, BIO members generally should consider:
- the clinical situation and need of the patient who seeks access, including whether the patient has a serious or life-threatening illness for which there is no viable alternative therapy; whether the treating physician believes, for that patient, the potential benefits of the experimental product will outweigh any potential risks; and whether regulatory authorities and institutional review boards/ethics committees have approved expanded access to the investigational medicine;
- whether the medicine is under active development (that is, it is not an already approved product nor has the development program been discontinued);
- whether the patient either is ineligible for or is unable to participate in a clinical trial for the investigational product;
- the impact of providing access to an investigational product on the development program for that product, including the ability to complete patient enrollment and other aspects of the clinical trials necessary to secure product approval, and the available supply of the investigational medicine; and
- the extent to which currently available data from early studies, including clinical studies, may be sufficient to make an assessment of the benefit and risk of the proposed use of the investigational product and for the patient to provide informed consent to such use.
To assist patients and their physicians in obtaining necessary information about the availability of access to an investigational medicine, BIO member companies should make publicly available their general policies regarding how they will evaluate requests for expanded access. Such information should include:
- a general description of the factors that will guide the company's decisions regarding expanded access, which may include the considerations above as well as additional factors the company will take into account; and
- a point of contact with whom physicians or patients may discuss their requests for expanded access to an investigational product the company is developing, and contact information that will provide convenient access to such point of contact, including emergency access as appropriate.