June 7, 2018
Theresa M. Mullin, Ph.D.
Theresa M. Mullin, Ph.D.

It is my pleasure to introduce Theresa M. Mullin, Ph.D., Associate Director for Strategic Initiatives at FDA’s Center for Drug Evaluation and Research (CDER). Dr. Mullin leads the Patient-Focused Drug Development (PFDD) initiative, which includes work related to the FDA Reauthorization Act (FDARA) and implementation of the 21st Century Cures Act. She also oversees CDER’s International Program. 

Stephen I. Katz, M.D., Ph.D.
Director, National Institute of Arthritis and Musculoskeletal and Skin Diseases

Dear Colleagues,

The assessment of human drug benefits and risks is central to the work of FDA scientists in the Center for Drug Evaluation and Research (CDER). Patients who will take these drugs are in a unique position to inform our assessment, which includes an analysis of the disease condition, its severity and the degree of unmet medical need. Although the existing FDA Patient Representative Program provides valuable patient input, it does not provide the broader patient community perspective on living with a disease.

We’ve addressed this need through an initiative called Patient-Focused Drug Development (PFDD). Since 2013, FDA has conducted 25 meetings in different disease areas to hear directly from patients about the impact of disease on daily life, and experiences with current therapies. These patient voices are providing new insights for FDA reviewers and drug development through the PFDD initiative. This includes not only the clinical context for FDA decisions, but more direct evidence regarding drug benefits and risks using methodologically sound clinical outcome assessment (COA) tools. Such evidence can be used as a basis for FDA’s assessment of drug benefits and risks, and could also be incorporated in drug labeling to better inform decisions by doctors and patients.

To enable the collection of this information, we need to continue to address the perceived risks of developing and deploying patient-focused COAs. Inconsistent quality in COAs, a potential byproduct of uncertainty about standards and approaches, may limit their utility for decision makers. To help address these concerns, FDA is currently developing a series of four new guidance documents to provide more information and direction to stakeholders. The goal is to bridge important early-stage meetings that gain patients’ narrative perspectives to subsequent development and deployment of methodologically sound data collection tools in clinical trials.

The four guidance documents that will be developed correspond to FDA commitments for Prescription Drug User Fee Act (PDUFA) VI (PDF - 262 KB) under the Title I of FDA Reauthorization Act of 2017. The projected timeframes for public workshops and guidance publication (PDF - 167 KB) reflect FDA’s published plans aligning these PDUFA commitments with guidance requirements under the 21st Century Cures Act Section 3002.

As outlined in the publication, the guidance will focus on the following set of topics:

  • Guidance 1: Approaches to collecting comprehensive and representative patient and caregiver input on burden of disease and current therapy. 
  • Guidance 2: Processes and methodological approaches to development of holistic sets of impacts that are most important to patients.  
  • Guidance 3: Approaches to identifying and developing measures for an identified set of impacts which may facilitate collection of meaningful patient input in clinical trials. 
  • Guidance 4: Methods and technologies for clinical outcome assessments—revising or supplementing the 2009 Guidance to Industry on Patient-Reported Outcome Measures. 

While these policy documents are critical to help reduce stakeholder uncertainty and support quality, they will not be sufficient to fully achieve integration of the patient’s perspective in drug development and decision making. In parallel with the policy work, we will be updating relevant review processes and available resources to support widespread adoption within our organization. To support long-term sustainability, we will also pursue developing standard core sets of measurement COA concepts and tools by disease area, enlisting the expertise and building on the excellent measures, tools, and resources previously developed by colleagues at NIH, FDA and elsewhere.


Theresa Mullin, Ph.D.
Associate Director for Strategic Initiatives
Center for Drug Evaluation and Research
U.S. Food and Drug Administration

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