Release time:2018/04/08author:admin

Statement from FDA Commissioner Scott Gottlieb, M.D., on FDA’s efforts to enhance the patient perspective and experience in drug development and review

Benefit-risk assessment is at the heart of what we do to ensure that Americans have access to medical products that are safe, effective and meet their needs.

But we’re also deeply aware that serious chronic illnesses aren’t monolithic. Patient perception of the benefits and risks of different treatment options can vary based on the stage of the disease, the age of onset, alternative therapies available to treat the disease (if any) and whether a novel therapy improves a patient’s ability to function normally, slows the rate of disease progression or impacts other aspects of a patient’s quality of life.

A 45-year-old father of two who is diagnosed with aggressive prostate cancer may have very different goals than an 80-year-old man diagnosed with the same disease. 

To address these realities, we’ll continue working in close partnership with patients to incorporate their experience into our benefit-risk assessments. We know that first-hand knowledge of living with a serious illness – communicated in science-based terms that patients value and understand – is integral to facilitating the successful development of safe and effective products that can deliver meaningful benefits in each disease, or disease state. 

Today we have many more tools to measure these patient benefits – including wearable devices, medical apps and even machine-learning programs. These tools can bring us a better understanding of how patients experience their illness, including how it affects their day-to-day feeling or functioning and how a given treatment may impact the course of that illness.

Tools for capturing the patient experience may be quantitative or qualitative, but they are transforming nearly every aspect of medical product development. Patients are teaching us about the benefits that matter most to them and the risks that they are most concerned about. Patients are, rightly so, becoming the driving force of the medical research enterprise.