The US FDA has released its first patient focused drug development (PFDD) guidance. This guidance is the first methodological guidance( referred as guidance 1) and that will be followed by 3 more guidance’s on this topic. The FDA addresses how patients, researchers and medical product developers can collect and submit patient experience data. The FDA hopes that these series of guidance will fulfill their commitment to the Cures Act and Prescription Drug User Fee Act that outlines the goal to “enhancing the Incorporation of the Patient’s Voice in Drug Development and Decision-Making.”

Guidance 1 discusses sampling methods that could be used when planning a study to collect patient input. It also provides a general overview of the relationship between potential research question(s) and method(s) when deciding from whom to get input (including defining the target population and development of the sampling strategy).

What is “Patient Experience Data”?

The Cures Act defines the term “patient experience data” to include data that:  

(1) are collected by any persons (including patients, family members and caregivers of patients, patient advocacy organizations, disease research foundations, researchers and drug manufacturers);


 (2) are intended to provide information about patients’ experiences with a disease or condition,

  • the ‘impact (including physical and psychosocial impacts) of such disease or condition or a related therapy or clinical investigation;
  • patient preferences with respect to treatment of the disease or condition.

Types of Patient Experience data to collect and measure:

These may include:

Impact of the disease and its treatment on the patient:

  • Signs/symptoms of disease or condition
  • Chief complaints
  • Burden of living with or managing a disease or condition (including effect of the disease or condition on activities of daily living and functioning)
  • Burden of treatment
  • Burden of participating in clinical studies

Patients’ perspectives about potential and current treatments

  • Expectations of benefits
  • Tolerance for harms or risks
  • Acceptable trade-offs of benefits and risks
  • Attitudes towards uncertainty

Views on unmet medical needs and available treatment options


Enhanced understanding of the natural history of the disease or condition, including progression, severity, and chronicity

FDA’s Recommendation: General Steps for Conduction Studies about Patient Experience:

DATA Management Plan:

The FDA recommends that Data management considerations be addressed in the early stages of a research study. Thereby , a data management plan (DMP) should be formulated. A DMP is a written document that describes the data expected to be acquired or generated during the research study; how to manage, describe, analyze, and store said data; and what mechanisms will use at the end of the study to preserve and share your data (Stanford University Libraries n.d.).

About Global Regulatory Partners

Global Regulatory Partners Inc, (GRP) is an American company that provides regulatory affairs, clinical, quality and safety services to medical devices and pharmaceutical companies globally. GRP headquarters is located in Massachusetts USA and its main affiliates are located in China, Japan, Brazil, Mexico and Argentina. GRP helps many life science companies register their products in different countries in compliance with local regulations.  To learn more, please contact us at  info@globalregulatorypartners.com

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