Clinical Research and Trials

Clinical trials are carefully controlled tests of new medicines in people to evaluate their safety and effectiveness and compile the evidence needed by the FDA and other regulators around the world to approve new treatments for commercialization.

Clinical testing of new medicines for approval is conducted in three phases in which the number of clinical trial participants increases as investigators learn more about the medicine being tested. At the beginning of a clinical trial and throughout the testing itself, companies and clinical investigators inform the FDA and other regulators about plans, outcomes and any issue regarding the trial. Even after a new therapy reaches the market, companies are required to submit periodic reports to the FDA, including all cases of adverse reactions and new information learned about the medicine. For some medicines, clinical studies may be required after it is approved for marketing in order to evaluate safety and efficacy in a broader patient population and over a longer period of time than what is required in the pre-approval studies.

Clinical trials involve the participation of thousands of volunteer patients and the generation of thousands of pages of scientific, medical and technical data. They account for seven of the 10 to 15 years and 45 to 75 percent of the $1.2 billion average cost of developing one new cutting-edge medicine.

Participation in clinical trials may provide opportunities for patients to gain access to a new treatment (sometimes the only option for their condition) before it becomes widely available, obtain high-quality care in leading medical facilities during their participation in the trial, and play a more active role in their healthcare while helping others by contributing to medical research.

Protecting patient safety and confidentiality is of the utmost importance in clinical development. Different entities and individuals contribute to the safe and proper conduct of clinical trials including sponsoring companies, regulatory agencies, healthcare professionals, and other institutions involved in the clinical study. Clinical trials follow carefully controlled protocols that must be reviewed and approved by Institutional Review Boards, which are independent committees of scientists, physicians, and community advocates, to make sure the trial is conducted ethically. Clinical trial participants learn about potential benefits and risks associated with the clinical trial through the informed consent process.

PhRMA member companies take great care to respect and protect the safety and individual privacy of research participants and have had a longstanding commitment to sponsoring clinical studies that fully comply with all legal and regulatory requirements.

By committing to PhRMA’s Principles on Conduct of Clinical Trials and Communication of Clinical Trial Results, America’s biopharmaceutical research companies help to ensure that their clinical research is scientifically and ethically rigorous, and that clinical trial results are properly communicated to healthcare professionals, patients, and the public.

Research conducted by biopharmaceutical research companies is already among the most transparent of any industry. As stated in PhRMA’s Principles on Conduct of Clinical Trials and Communication of Clinical Trial Results, biopharmaceutical research companies are committed to transparency through the timely submission and registration of all clinical trials involving patients on the NIH's clinical trial database, www.clinicaltrials.gov. As clinical trials advance, results are frequently reported at scientific meetings and in medical journals.

Biopharmaceutical companies have also committed to releasing the results of all clinical trials involving products that are approved for marketing, or investigational products whose development programs are discontinued – regardless of outcome.

PhRMA recognizes and supports the important public health benefits associated with making appropriate clinical trial information available to healthcare practitioners, patients and the public, and believes that such data are best disseminated in a fashion that is scientifically robust, while maintaining protection for individual privacy and incentives for future innovation.