Patient Focused Drug Development – Listening to The Patient’s Voice

Gone are the days when patients were merely participants in clinical trials. Today, integrating the patient’s perspective about their diagnosis and treatment experience with reference to the disease plays an important role in developing patient-focused treatment.

Developing patient-focused treatment involves prioritizing current unmet needs in a particular disease condition, designing patient-centric trials, and utilizing patient-appropriate outcomes. To develop efficient treatments within budgets, it is imperative that the systems and the stakeholders are aligned with patient needs and preferences at the earliest stage of development.

In 2012, the FDA launched the Patient-Focused Drug Development (PFDD) — an initiative to integrate patient perspectives at an early stage of drug development.

What is Patient-Focused Drug Development

The FDA introduced the PFDD initiative “to provide a new, more systematic, and expansive approach to obtaining patient perspectives on disease severity or unmet medical need in a therapeutic area to benefit the drug review process.” PFDD is thus “a systematic method to ensure that experiences, perspectives, needs, and priorities of patients are captured and meaningfully incorporated into drug development and evaluation.”

The main goals of the initiative include:

  • Encourage the use of systematic approaches to collect meaningful data from patients and their caregivers, which can be utilized during the drug development, evaluation process, and regulatory stages.
  • Identify and incorporate the best approaches to enhance patient enrolment and engagement in clinical trials.
  • Augment the use of appropriate methods to collect information on patient choices and explain the potential trade-off between the risks and benefits of treatment.
  • Facilitate informed decision-making using meaningful data related to patient’s perception of benefits, risks, and burden of treatment.

Patient-Focused Drug Development – Series of Four Guidance Documents

The FDA developed a series of four guidance documents to support drug developers and regulatory professionals in making informed decisions when collecting information related to patient preferences.  The documents guide decision-makers to follow a systematic, step-wise approach to collect meaningful information from patients and caregivers.

Guidance 1 covers the various sampling approaches that can be utilized when collecting patient data. It also outlines the relationship between a possible research question and the methodology to be followed when determining the target population and the sampling strategy. 

Guidance 2 explores methods that can be used to obtain information from the target population selected in Guidance 1. It includes methods to collect information about how the disease and its symptoms impact the patients. It covers information on how to conduct effective qualitative research, including ways to develop surveys, interview guides, etc. It also includes details on how to prevent misleading outcomes that may not truly represent specifics that are of significance to the patient.

In Guidance 3, methods to select, develop, modify, and validate various clinical trial outcomes of significance to the patients are discussed.

Guidance 4 explores techniques, methods, and standards to collect, store, and analyse clinical outcome assessment data. It also outlines approaches that can be used to amalgamate clinical outcome assessments and endpoints that are important for regulatory purposes. In its move to support patient-centred research, the FDA recommends the use of traditional randomized controlled trials in conjunction with computerized adaptive testing whenever possible. To reduce patient burden in clinical trials, the FDA supports the involvement of patient representatives and communities at an early stage of trial planning.

Patients as the primary stakeholder in healthcare decision-making

Recent years have witnessed increased efforts to involve patients in the drug development process. However, more structured efforts are required to maximize patient involvement in the process. Previous methods of data collection were fragmented with resultant increased efforts and patient burden. The FDA’s PFDD initiative aims to provide a more structured approach for the collection of patient preferences and associated data that may help streamline the understanding of patient experiences across multiple stakeholder groups.

As patients live with their condition, they have unique understanding of what makes the most meaningful impact on their disease condition and overall life. They are the primary stakeholders in healthcare decision-making; thus, considering their preferences, background, and experiences during drug development can help deliver better health outcomes.

The IWANA team routinely expands its knowledge of the changing face of the healthcare industry and never fails to apply the recommendations made by governing bodies to ensure documents are created in compliance. Moreover, we thoroughly understand the significance of increased patient involvement in the drug development process as discussed in the blog and are able to offer our expertise in the understanding and use of the guidance documents included in the PFDD initiative.


  1. FDA-led Patient-Focused Drug Development (PFDD) Public Meetings. U.S. FDA. Available at: https://www.fda.gov/industry/prescription-drug-user-fee-amendments/fda-led-patient-focused-drug-development-pfdd-public-meetings.
  2. CDER Patient-Focused Drug Development. U.S. FDA. Available at: https://www.fda.gov/drugs/development-approval-process-drugs/cder-patient-focused-drug-development
  3. CDER Patient-Focused Drug Development. Available at: https://www.fda.gov/drugs/development-approval-process-drugs/cder-patient-focused-drug-development.
  4. FDA Patient-Focused Drug Development Guidance Series for Enhancing the Incorporation of the Patient’s Voice in Medical Product Development and Regulatory Decision Making. Available at: https://www.fda.gov/drugs/development-approval-process-drugs/fda-patient-focused-drug-development-guidance-series-enhancing-incorporation-patients-voice-medical.
  5. Schroeder K, Bertelsen N, Scott J, et al. Building from Patient Experiences to Deliver Patient-Focused Healthcare Systems in Collaboration with Patients: A Call to Action. Ther Innov Regul Sci. 2022;56(5):848-858.
  6. Lowe MM, Blaser DA, Cone L, et al. Increasing Patient Involvement in Drug Development. Value Health. 2016;19(6):869-878.
  7. Perfetto EM, Burke L, Oehrlein EM, Epstein RS. Patient-Focused Drug Development: A New Direction for Collaboration. Med Care. 2015;53(1):9-17.

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