New FDA will ‘enhance patient perspective in drug development’

Aug 15, 2018
New FDA will ‘enhance patient perspective in drug development’

The Federal Drug Agency (FDA) is developing tools to help incorporate the patient voice into its benefit-risk assessments.  

In a statement, FDA Commissioner Dr. Scott Gottlieb, said an update to the body’s implementation plan, Benefit-Risk Assessment in Drug Regulatory Decision Making, provides a roadmap for enhancing its Benefit-Risk Framework by June 2020.  

 “We are 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 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,” said Dr. Gottlieb. 

 “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.” 

 The aim of the FDA’s Benefit-Risk Framework is to improve the clarity and consistency in communicating the reason behind regulatory decisions. 

 In carrying out the process, reviewers consider evidence related to the benefit and risk and effects reported in clinical studies, but also, importantly, the “clinical context” of the disease, added Dr. Gottlieb. 

 “This context encompasses two major considerations: an analysis of the disease condition, including the severity of the condition, and the degree of unmet medical need.” 

 “As part of this work, the FDA recognizes a need to learn about the clinical context more comprehensively and directly from the perspective of the patients who live with the disease and their caregivers.” 

 After conducting patient-focused drug development meetings in more than 20 disease areas, the FDA is developing tools to collect data on patient experience during trials. 

 “Patient input can inform the clinical context and provide insights to frame the assessment of benefits and risk, and provide a direct source of evidence regarding the benefits and risks, if methodologically-sound data collection tools could be developed and used within clinical studies of an investigational therapy,” said Dr. Gottlieb, adding that the FDA was working on a number of projects designed to incorporate the patient perspective into its work. 

 These include patient-focused public meetings to develop ways of systematically collecting data and encouraging patient stakeholders to conduct their own development meetings. 

 Advisory groups, to help patients, advocates and caregivers contribute to decision making more systematically, have been set up. The body is also working with the medical community to design trials that places less burden on patients. 

 The plan, said Dr. Gottlieb, was part of the FDA’s ongoing commitment to advancing its mission to protect and promote public health.  

 “It marks another important step forward in increasing transparency of FDA decisions as well as streamlining the process by which we obtain input from the patient and stakeholder communities.  

 “We know that, in the battle against disease, engaged and informed patients are our best allies and our greatest resource,” he added. 


 Statement from FDA Commissioner Scott Gottlieb, M.D., on FDA’s efforts to enhance the patient perspective and experience in drug development and review. (2018, March 30). Available from (accessed June 2018). 


New FDA will ‘enhance patient perspective in drug development’

Astellas Patient Advocacy is a function within Corporate Affairs at Astellas that focuses on creating, building and maintaining third-party relationships. We serve as a conduit between Astellas and external stakeholders to help improve patient outcomes, improve access issues and address patients’ unmet needs head on.

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