FDA on the Path of Patient Focused Medicine

The FDA is entering a new era of patient-centered medicine.

After holding patient-focused workshops for 3 years, the FDA has taken a step further into its patient advocacy program by creating a new advisory committee: the Patient Engagement Advisory Committee.

All of these workshops are being covered under both the CDER/CBER office regulation, while this new committee has been created under the CDRH office.

From 2013, the FDA has followed a new approach for patient advocacy beginning with the Patient-Focused Drug Development (PFDD) initiative[1] and a proposed list of workshops. The PPFD initiative was created to fulfill the performance commitments of the agency under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V). The first PFDD meeting was held in April 2013 on the topic of chronic fatigue syndrome and myalgic encephalomyelitis[2].

The various PPFD disease areas and topics were announced in a Federal Register[3] notice in April 2013 for the first 3 years of the 5-year PDUFA V timeframe (2013-2015).

Topics for FY2015 included:

  • Female sexual dysfunction: October 27-28, 2014
  • Breast cancer: April 2, 2015
  • Chagas disease: April 28, 2015
  • Functional GI disorders: May 11, 2015 
  • Parkinson’s disease and Huntington’s disease: September 22, 2015
  • Alpha-1 antitrypsin deficiency:  September 29, 2015

In July 2015, the Federal Register[4] published the list of disease areas that would be addressed at PFDD meetings in FY2016-2017. Disease areas include:

  • Non-tuberculous mycobacterial infections: October 15, 2015
  • Psoriasis: March 17, 2016
  • Neuropathic pain associated with peripheral neuropathy: June 10, 2016
  • Patients who have received an organ transplant: September 27, 2016

Meeting dates are to be announced for the following:

  • Alopecia areata
  • Autism
  • Hereditary angioedema
  • Neuropathic pain associated with peripheral neuropathy
  • Sarcopenia

The previous meetings topics were:

For 2014:

  • Sickle cell disease: February 7, 2014
  • Fibromyalgia: March 26, 2014
  • Pulmonary arterial hypertension: May 13, 2014
  • Neurological manifestations of inborn errors of metabolism: June 10, 2014  
  • Hemophilia A, hemophilia B, von Willebrand disease, and other heritable bleeding disorders:
    September 22, 2014
  • Idiopathic pulmonary fibrosis: September 26, 2014

For 2013:

  • Chronic fatigue syndrome and myalgic encephalomyelitis: April 25-26, 2013
  • Human immunodeficiency virus (HIV): June 14, 2013
  • Lung cancer: June 28, 2013
  • Narcolepsy: September 24, 2013

Following each meeting, the FDA releases a public report in the “Voice of the Patient[5] to summarize the public comments and input provided by patients and patient representatives during the meeting.These reports offer a more systematic approach to collecting information on patients’ knowledge on their condition and treatment options, and provide to the FDA “the therapeutic context” for weighing benefits and risks of investigational products for the given disease. In particular, the reports reflect the content of the meetings and the docket comment submissions as they relate to patient perspectives on the various disease symptoms, impacts, and current treatments.

Alongside CDER and CBER, CDRH started to consider the patient perspective in September 2013, when it held a workshop entitled “The Patient Preference Initiative: Incorporating Patient Preference Information into the Medical Device Regulatory Processes[6].

Following this first step, CDRH announced in the Federal Register[7] its focus on engaging patients throughout the regulatory process in medical device development by considering patient perspectives in the decision making.

The FDA’s first ever Patient Engagement Advisory Committee will provide advice to the FDA on issues relating to medical devices, their regulation, and their use by patients. As announced by the FDA: “The Committee will provide advice to the Commissioner or designee, on complex issues relating to medical devices, the regulation of devices, and their use by patients. The Committee may consider topics such as: Agency guidance and policies, clinical trial or registry design, patient preference study design, benefit-risk determinations, device labeling, unmet clinical needs, available alternatives, patient reported outcomes and device-related quality of life or health status issues, and other patient-related topics. The Committee will provide relevant skills and perspectives, in order to improve communication of benefits, risks, clinical outcomes, and increase integration of patient perspectives into the regulatory process for medical devices. It will perform its duties by discussing and providing advice and recommendation in ways such as: Identifying new approaches, promoting innovation, recognizing unforeseen risks or barriers, and identifying unintended consequences that could result from FDA policy.”

The science of patient input is a high priority area for both the FDA and the pharmaceutical industry. But the real challenge is to create a standard way to collect and use patients’ opinions.

This new interest for patient input into the drug and device development process has yet to find its role, and the FDA still needs to provide guidance on how to use that information in the development process. Nonetheless, some initiatives have appeared; for example, in a white paper[8] released in June 2015, the Biotechnology Industry Organization (BIO) recommended that the perspectives of patients be a standard component of decisions that are made during discussions between sponsors and the FDA.

Other positive aspects of this type of standardized data collection approach is for pharmaceutical companies that believe it can ease the task of recruiting patients into clinical trials and help relations between sponsors and the FDA, the PFDD initiative has the potential to change product development.

A first stone in this patient input into FDA regulation has been laid with the new “Draft Guidance: Patient Preference Information – Submission, Review in PMAs, HDE Applications, and De Novo Requests, and Inclusion in Device Labeling,”[9] published in May 2015.

The objective of the FDA to develop the ability to take the direct experience of patients with particular diseases and conditions, and the effect of their current therapies, and then use that data to modify and improve potential treatments is a new tool in the drug or device development process and a great step toward personalized medicine.


[1] http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm347317.htm

[2] http://www.fda.gov/Drugs/NewsEvents/ucm369563.htm

[3] Federal resister, April 11, 2013 - Volume 78, Number 70/Pages 21613 – 21614.

[4] Federal Register, July 02, 2015- Volume 80, Number 127/Pages 38216 - 38217

[5] http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm368342.htm

[6] http://www.fda.gov/MedicalDevices/NewsEvents/WorkshopsConferences/ucm361864.htm

[7] Federal Register September 21, 2015 - Volume 80, Number 182/Pages 57007 – 57008.

[8] https://www.bio.org/FDAwhitepaper

[9] http://www.fda.gov/downloads/medicaldevices/deviceregulationandguidance/guidancedocuments/ucm446680.pdf