Under the chapter, industry group PhRMA (Pharmaceutical Researchers and Manufacturers of America) notes that physicians can apply for expanded access to an unapproved drug for their patients to treat a serious or life-threatening disease or condition when there are no other comparable or satisfactory alternative treatment options and when patients are ineligible or unable to participate in a clinical trial.
“It is important for the patient’s physician and the medical staff at a biopharmaceutical company to discuss whether it provides the best possible treatment option for the patient before going through the FDA process,” PhRMA says.
The industry group lays out a number of considerations for biopharma companies to follow when providing patients with access to an unapproved investigational drug outside of a clinical trial. The considerations include that the illness must be serious or life-threatening, the investigational drugs should be active in clinical development, and “there should be sufficiently robust preliminary safety and efficacy data, including dosing information, to determine that the preliminary benefit-risk balance is positive for the specific indication for which the request is made.”
In addition, granting access to an investigational drug through an expanded access program should not delay, interfere or compromise the completion of clinical trials that are intended to support the drug’s approval, according to PhRMA.
As far as eligibility for patients seeking expanded access to an investigational drug outside of a clinical trial, PhRMA notes that “geographic limitations alone would generally not be considered a barrier to participation in clinical trials.”
PhRMA also calls on biopharma companies to establish telephone or internet-based information sources to facilitate communication about expanded access programs.
In addition to the information on expanded access programs, the PhRMA chapter also outlines ways for companies to protect research participants, properly conduct clinical trials, ensure objectivity in research, and ways to provide more information about clinical trials.