Laurie Burke, RPh, MPH
Founder of LORA Group, LLC and former Associate Director for Study Endpoints and Labeling, Office of New Drugs, CDER, FDA
I started my career at the FDA in 1976, two years after Bernard Jambon founded Mapi! Now, in 2014, when I contemplate all these years and all the things accomplished to take the patient’s voice into consideration in drug development, I say to myself “We’ve come a long way.” Therefore, when Catherine asked me to participate in the Mapi 40th anniversary celebration, I thought that sharing with you my perspective on the history of patient-reported outcome measurement at the FDA would be relevant.
A recent Medline review with health-related quality of life (HRQL) and patient-reported outcome (PRO) as key words (see Figure 1) showed that the first publication with “PRO” in the title was out in 1976,1 and the first with “HRQL” in 1982.2 Since then, the growth has been parallel and almost exponential, showing the importance of the field in the biomedical literature.
Figure 1. PRO and HRQL publications 1976-2013
Historically, there were many events that provided the foundation for the patient’s voice to be heard in clinical trial outcomes. I provide a table that lists just a few of these. As you can see, the first academic research on measurement of attitudes was published in 1932. Since then, academic research has continued while, in parallel, regulators established the rules of evidence for treatment benefit claims in labeling and promotion,3-4 launched the first steps for collaboration between agencies (FDA, EMA), published the EMA Reflection Paper on HRQL5 and the draft and final guidance on PRO measures,6-7 and launched the qualification programs for clinical outcome assessments (FDA and EMA).8-10
It was because of two men in particular, Dan Shames and Bernard Jambon, that I acquired the courage and inspiration to establish the Study Endpoints and Labeling Development Staff and pursue the development of regulatory standards for PRO measurement. Dan, because of his role as the director of the FDA division that reviewed drug products based entirely on PRO endpoints, saw the need for consistent policy and oversight, and Bernard who reinforced for me that it really is all about the patients. Subsequently, it was Donald Patrick, who provided the hands-on scientific support as a special government employee, and Dee Kennedy, who collated all the public comments on the draft and enabled the finalization process. The Guidance stands as a line in the sand for all outcomes measurement in clinical trials by providing a standard for all “well-defined and reliable” assessments of treatment benefit.
Many others deserve mentioning who have picked up the mantle and continue to move the field of PRO measurement forward. Mapi plays a critical role in that work, and I look forward to their continued success!
Happy anniversary, Mapi, and happy anniversary to Bernard!
1. Levine DM, Morlock LL, Mushlin AI, Shapiro S, Malitz FE. The role of new health practitioners in a prepaid group practice: provider differences in process and outcomes of medical care. Med Care. 1976;14(4):326-47.
2. Bush JW, Anderson JP, Kaplan RM, Blischke WR. “Counterintuitive” preferences in health-related quality-of-life measurement. Med Care. 1982 May;20(5):516-25.
3. TITLE 21—Food and Drugs. Chapter I—FDA, DHHS. Subchapter D—Drugs for Human Use. Part 314—Applications for FDA Approval to Market a New Drug. Subpart D—FDA action on Applications and Abbreviated Applications. §314.126 Adequate and well-controlled studies. http://www.gpo.gov/fdsys/pkg/CFR-2010-title21-vol5/pdf/CFR-2010-title21-vol5-sec314-126.pdf
4. DHHS FDA CDER. Draft Guidance for Industry Promoting Medical Products in a Changing Healthcare Environment; I. Medical Product Promotion by Healthcare Organizations or Pharmacy Benefits Management Companies (PBMs). 1997. http://www.fda.gov/downloads/BiologicsBloodVaccines/DevelopmentApprovalProcess/AdvertisingLabelingPromotionalMaterials/UCM161546.pdf
5. EMA. Reflection Paper on the Regulatory Guidance for the Use of Health-Related Quality of Life (HRQL) Measures in the Evaluation of Medicinal Products. 2005. http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003637.pdf
6. DHHS FDA CDER. Draft Guidance for Industry – Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. 2006.
7. DHHS FDA CDER. Guidance for Industry – Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. 2009. http://www.fda.gov/downloads/Drugs/Guidances/UCM193282.pdf.
8. EMA. Qualification of novel methodologies for drug development: guidance to applicants. 2008. http://www.ema.europa.eu/docs/en_GB/document_library/Regulatory_and_procedural_guideline/2009/10/WC500004201.pdf
9. DHHS FDA CDER. Clinical Outcome Assessment Qualification Program. http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugDevelopmentToolsQualificationProgram/ucm284077.htm
10. DHHS FDA CDER. Guidance for Industry and FDA Staff – Qualification Process for Drug Development Tools. 2014. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM230597.pdf
11. DHHS FDA CDER. Draft Guidance for Industry Analgesic Indications: Developing Drug and Biological Products. 2014. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM384691.pdf
12. DHHS FDA CDER. Draft Guidance for Industry Chronic Fatigue Syndrome/ Myalgic Encephalomyelitis: Developing Drug Products for Treatment. 2014. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM388568.pdf
13. DHHS FDA CDER. Draft Guidance for Industry Labeling for Human Prescription Drug and Biological Products Approved Under the Accelerated Approval Regulatory Pathway. 2014. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM390058.pdf