The US Food and Drug Administration (FDA) has released a statement outlining its plans to promote patient voices in the development and regulation of medical products. You can find more information in the source statement that this article is based on at the FDA’s website here.
The Importance of Patient Voices
The FDA says in their statement, “we learn through scientific advances, but also by listening to patients.” Continual feedback and engagement with patient communities can help the FDA to understand what patients and their families value most in medical drugs and products. Patients can help drug manufacturers and regulators to understand the context of conditions, and how patients view risks, benefits, and impact on quality of life of different medical options.
Oncology Drugs
Patient input is particularly important, the FDA says, for targeted therapies. These increasingly common drugs focus on treating specific elements of a condition, or specific sub-groups of patients.
The FDA cites recent changes in approach to oncology (tumour) drug development and regulation as an example of the importance of patient perspectives. In the past, clinical trials in this area often investigated combination cytotoxic chemotherapies, which typically have significant risks, but might not have provided a substantial improvement in effectiveness over other drugs. The endpoints (aims) of clinical trials reflected this. However, recent advances in scientific research have led to more targeted therapies being trialled. These may give certain groups of patients more benefits, and in some cases with fewer risks. The FDA says that for this type of drug patients often say they prefer surrogate endpoints. According to the NIH, surrogate endpoints use less strong indicators (such as tumour shrinkage) rather than other indicators such as longer survival. This may lead to earlier approval of drugs, since the surrogate indicators may be measured sooner. Consulting with patients, therefore, is an important factor when modernising clinical trial designs. For more detailed information about this, you can view the original statement from the FDA here.
How the FDA Plans to Advance Patient Voices
To support patient input, the FDA is planning to encourage companies to listen to patient perspectives when developing medical products, and to provide tools that can help organisations to gather information on patient views. The FDA plans to release four guidances focused on how to collect and use information about patient experiences to inform the development and regulation of medicines and other medical products. The first of these, which has already been released, covers topics such as sampling methods of data collection, how to standardise the collection, and how to analyse patient data.
The FDA is also holding Patient Focused Drug Development meetings to better understand patient experiences around specific conditions. So far, the FDA has held meetings about over twenty disease areas, including HIV and Parkinson’s disease. The FDA says, “we have gained from this experience.”
