New Approaches Needed to Speed up New Drug Development

Scientific advances are bringing the time closer to when personalized approaches will become more common in treating illnesses. Medicines would take into account the patient’s history, disease risk and pathology and be tailor-made to be most effective for that specific patient. But, slowing the developments of these therapies is today’s clinical trial process, which has fallen behind scientific advances. Currently, the U.S. Food and Drug Administration (FDA) approves new drugs based on lengthy Phase III trials that use a diverse population to study a drug’s safety and effectiveness. In these cases, large groups of subjects are needed for statistical purposes. Such trials contribute to the rapidly increasing cost of bringing drugs to market and are not necessarily the best way to evaluate more targeted medicines. There has been a call in the scientific community to institute new, more efficient clinical trial designs to speed up the approval and marketing of personalized medicines. In an article in the Brooking Institution’s Up Front newsletter, the authors discuss using “adaptive trials”, drug trials which use data gathered during the trial to design the drug. Unlike traditional trials that have fixed parameters, adaptive trials would allow parameters such as treatment regimen, study population and sample size to be modified based on interim results. Such trials could speed the development of personalized treatments by allowing researchers to identify the most responsive subpopulations and the best drugs for them. Of course, the pharmaceutical industry and the FDA are hesitant about using the unfamiliar adaptive approach for Phase III pivotal trials. But, there are several factors that could help make them successful. These include: Experience: Traditional trials were at one time also new. Getting started with adaptive trials will lead to a better understanding of the practical and statistical aspects involved and to more confidence in the results. Transparency: Regular communication between drug developers and the FDA regarding what is learned during these trials will make both sides more comfortable with them. Clarity: Clear standards for approval are needed if pharmaceutical companies are going to be confident enough to try adaptive designs. The FDA is currently working on draft guidance for adaptive trials. An efficient approach to speeding up the development and approval of new, targeted medicines is important to the health of the pharmaceutical industry as well as to patients, and adaptive trials can be a big part of the effort to do this.