Researchers in biopharmaceutical companies strive to discover and develop new medicines and vaccines that will help people to live longer, healthier lives. They go to work every day knowing that patients with life-threatening diseases live with the hope that tomorrow will bring a new medicine that will extend and improve lives.
A critical part of the discovery process is clinical research, the study of an investigational drug or medical device in human volunteers. In sponsoring and conducting clinical research, PhRMA members place great importance on respecting and protecting the safety of everyone who volunteers to participate.
PhRMA and its member companies have long been committed to ensuring that clinical research is carefully conducted and that meaningful results are properly communicated.
Today marks another milestone in this commitment, as we announce the inclusion of Principles on Expanded Access to Investigational Drugs in our Principles on Conduct of Clinical Trials and Communication of Clinical Trial Results. With this addition, PhRMA member companies commit to establish informational resources to help facilitate awareness of expanded access programs.
The new principles will be announced today during a stakeholder workshop featuring participation by patients and patient advocacy groups, academia, health care providers, the Food and Drug Administration (FDA) and biopharmaceutical companies to discuss expanded access and the role of investigational new drugs in patient care.
As the biopharmaceutical industry continues to work to identify, test and bring new medicines to patients, clinical trials are the primary way that patients can participate in the drug development process, receive access to unapproved investigational drugs, and contribute to the collection of safety and efficacy data needed for regulatory approval. Without clinical research studies, new medicines could not be approved by FDA and made available to the patients who need them.
Use of an unapproved investigational drug via an expanded access program may be an option for a patient who is ineligible or unable to participate in a clinical trial [see box]. In these instances, the patient’s physician and the medical staff at a biopharmaceutical company should discuss whether it provides the best possible treatment option for the patient.
As an industry, we are committed to ongoing dialogue with all stakeholders to identify ways to optimize the existing federal expanded access process and modernize the clinical trial, drug development and FDA review processes by harnessing 21st Century science to accelerate the availability of new medicines for the patients who need them.
The FDA process for a patient to gain access to an investigational drug through expanded access was established in 2009 in close consultation with patients, physicians and the biopharmaceutical industry. The key considerations include:
- Whether the patient has a serious or life-threatening illness and what treatment options are available;
- Whether the patient is eligible to participate in the clinical trial;
- Whether there is sufficient preliminary evidence of safety and efficacy to justify the use of the investigational medicine for treatment of the patient and what the potential benefit - risk balance would be;
- Whether there is sufficient supply of the investigational medicine to support the ongoing clinical trial as well as expanded access;
- Whether the expanded access program could compromise the conduct of clinical trials needed to obtain regulatory approval.