Placeholder Banner

BIO Principles on Expanded Access to Investigational, Unapproved Medicines

April 23, 2015
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 investigational, unapproved medicines.  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.

We believe that the best way to make innovative biotechnology medicines available to patients is through the efficient development of approved, safe and effective products.

As such, we recently released general principles for determining whether and how to establish expanded access programs or to provide expanded access to investigational medicines on an individual-patient basis.

These principles include considering the clinical situation and need of the patient who seeks access; determining whether the medicine is under active development; determining whether the patient either is ineligible for or is unable to participate in the clinical trial; assessing the impact of providing access based on the development program for the product; and evaluating the benefit and risk of the proposed use of the investigational product based on currently available data.

We also recommend that BIO member companies make publicly available their general policies regarding how they will evaluate requests for expanded access to assist patients and their physicians in obtaining information about the availability of access. Such information should include a general description of the factors that will guide the company's decisions regarding expanded access, as well as designating a point of contact with whom physicians or patients may discuss their requests for expanded access.

The principles can be accessed at this link.