NHC Comments on FDA-2020-D-2316 for Benefit-Risk Assessment for New Drug and Biological Products; Draft Guidance for Industry


Food and Drug Administration
Dockets Management Staff (HFA-305)
5630 Fishers Lane, Rm. 1061
Rockville, MD 20852

RE: FDA-2020-D-2316 for “Benefit-Risk Assessment for New Drug and Biological Products; Draft Guidance for Industry.”

Dear Acting Commissioner Woodcock:

The National Health Council (NHC) appreciates the opportunity to provide a response to the Food and Drug Administrations (FDA’s) request for comments on the draft guidance “Benefit-Risk Assessment for New Drug and Biological Products.”

Created by and for patient organizations over 100 years ago, the NHC brings diverse organizations together to forge consensus and drive patient-centered health policy. We promote increased access to affordable, high-value, sustainable health care. Made up of more than 140 national health-related organizations and businesses, the NHC’s core membership includes the nation’s leading patient organizations. Other members include health-related associations and nonprofit organizations including the provider, research, and family caregiver communities; and businesses representing biopharmaceutical, device, diagnostic, generic, and payer organizations.

The NHC appreciates the FDA’s attention to integrating the perspectives of individual patients and family caregivers in benefit-risk assessments and its overall acknowledgement of involving patient experience data throughout the regulatory review process. Such changes will enable FDA to better accommodate the dynamic nature of benefit-risk assessments, integrate new findings from clinical investigations, and entail patient preferences and risk tolerances for developing a patient-focused treatment landscape.

The NHC applauds the FDA’s commitment to patient-centered benefit-risk assessment by recognizing that “patient experience data can inform nearly every aspect of the FDA’s benefit-risk assessment throughout the drug lifecycle as patients are experts in the experience of their disease or condition, and they are the ultimate stakeholders in the outcomes of medical treatment” in this guidance. We value the FDA’s commitment to patient-focused drug development and welcome this opportunity to encourage the agency to further clarify whether and how the Agency considers patient experience data (PED) in its benefit-risk assessment.

While we note this guidance refers to other existing and forthcoming guidances, we believe additional clarity can be added to this guidance in several key areas. To ensure the benefit-risk assessment fully considers PED and best serves the needs of patients, we have identified these potential modifications and improvements:

  • The FDA should elaborate on how they intend to use various types of PED in their benefit-risk assessments and the role the Agency will play in advancing the use of PED in benefit-risk assessment;
  • The FDA should ensure opportunities are available for early interaction between FDA and sponsors to obtain feedback on PED collection strategy; and
  • The FDA should enhance the transparency and communication of the collection and use of PED to create consistent benefit-risk assessments across therapeutics areas.


Read our full comments: Benefit Risk Assessment Letter