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Patient-Focused Drug Development

Patient-Focused Drug Development

The Oncology Center of Excellence PFDD program fosters collaboration between FDA Centers and external stakeholders involved in patient outcomes research in cancer populations. The program focuses on three key areas:

  • Actively engaging with patients and advocacy groups.
  • Fostering research into measurement of the patient experience.
  • Generating science-based recommendations for regulatory policy.

Cancer patients experience disease symptoms and symptomatic treatment side effects that can impact their ability to function and other aspects of their health-related quality of life. The PFDD Program is working to identify rigorous methods to assess the patient experience that will complement existing survival and tumor information to provide additional evidence about the effects of cancer therapies on patients.

photo of Paul Kluetz, M.D.

Paul G. Kluetz, M.D.
Oncology Center of Excellence

Paul Kluetz is a medical oncologist and the Acting Associate Director of Patient Outcomes in the Oncology Center of Excellence at the U.S. FDA. His interests include defining clinical benefit in oncology trials, the use of expedited programs such as accelerated approval, and opportunities and challenges associated with patient reported outcomes (PRO) data, wearable technologies, and other methods to obtain data on the patient experience both in the clinical trial and “real-world” settings. He is currently leading a team to develop regulatory science and policy initiatives to advance patient-focused drug development in cancer trials. Dr. Kluetz remains clinically active, practicing inpatient medicine as a teaching attending at the Georgetown University Hospital.


photo of Bellinda King-Kallimanis, Ph.D.

Bellinda King-Kallimanis, Ph.D.
Senior Staff Fellow
Oncology Center of Excellence

Bellinda King-Kallimanis is a Senior Staff Fellow in the Oncology Center of Excellence at the FDA. She has a PhD in psychometrics and works on patient-focused drug development initiatives that focus on using clinical outcome assessments (COAs) in cancer clinical trials. Her current projects involve looking at open label response bias, understanding responsiveness of physical function measures and investigating current COA strategies being submitted to the agency to learn how these can be improved. Bellinda has been working in the field of COAs for the past 11 years across both academia and industry. She has investigated how patients’ responses to questionnaires can shift over time using structural equation modeling and worked on qualitative projects developing new patient-reported outcome instruments.  


photo of Janice Kim

Janice Kim, Pharm.D., M.S.
Regulatory Health Project Manager
Office of Hematology and Oncology Products

Janice Kim is a Regulatory Health Project Manager in the Office of Hematology and Oncology Products – Immediate Office and is a practicing pharmacist. She graduated from the University of Virginia with a Bachelor’s degree in biomedical engineering. In addition, she received her Master’s degree in biochemistry at Georgetown University before completing her pharmacy degree at the Medical College of Virginia. She completed her ambulatory care residency at a free clinic. She contributes to the policy development for the patient-focused drug development program in the Oncology Center of Excellence at the FDA. She enjoys working with patients and continues to work in the free clinic setting.


 

Projects

  • Collaborator: Division of Research of The Permanente Medical Group PanPROE
    Pancreatic Cancer Patient Reported Outcomes Using the Electronic Medical Record
    Investigating the use of patient-reported outcome (PRO) measurement of physical function in pancreatic cancer patients who undergo treatment and follow up for their illness. 
  • Collaborator: University of North Carolina and Alliance Foundation Trials 
    Exploring Self-Reported Physical Functioning in Cancer Patients
    To further our understanding of the responsiveness of physical functioning outside of the clinical trial setting so that we can understand how sensitive a measure is across the drug life cycle (NCT03249090)

Working Groups

  • Lungevity: Patient Preference, Industry Roundtable
  • National Cancer Institute (NCI) Cancer Moonshot Tolerability Consortium Steering Committee
  • Patient-Reported Outcome (PRO) Consortium Electronic PRO Working Group
  • Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data (SISAQOL) 
  • Standard Protocol Items: Recommendations for Interventional Trials Patient-Reported Outcomes (SPIRIT-PRO)

The OCE PFDD group is working to advance several initiatives in the upcoming year. 

Image displays a large circle in the center representing the OCE Patient-Focused Drug Development Program connected with lines to three smaller circles representing Regulatory Policy, Regulatory Science, and Education and Engagement

Regulatory/Policy: 

  • Create standard analyses for physical function and symptomatic adverse events
  • Foster efficient regulatory review of clinical outcome assessment submissions to the FDA
  • Develop guidance for industry

Science: 

  • Engage and participate with international scientific research in PRO measurement with international organizations
  • Identify analyses and visualizations to interpret clinical outcome data from cancer trials
  • Actively engage in scientific collaboration with clinicians, social scientists and statisticians across FDA

Education/Engagement: 

  • Create educational opportunities for the patient community
  • Continue efforts to make oncology clinical trial terms more patient friendly language
  • Engage with patient advocacy groups in patient-centered qualitative research
  • Foster educational opportunities for FDA oncology reviewers related to measurement of symptoms and function

Upcoming Events:

July 12, 2019: 4th Clinical Outcomes Assessment in Clinical Cancer Trials (COA-CCT) Workshop with American Society of Clinical Oncology

October 8, 2019: 3rd Partners in Progress Workshop to provide basic training on the role of the FDA and cancer patient advocates in oncology product development.

Past Events:

April 26, 2016: COA-CCT Workshop with the Critical Path Institute (Slides only)

November 13, 2017: 1st Partners in Progress

April 25, 2017: 2nd COA-CCT Workshop with the Critical Path Institute- Safety and Tolerability (Slides and Audio)

November 27, 2018: 2nd Partners in Progress

June 22, 2018: 3rd COA-CCT Workshop with American Society for Clinical Oncology (Slides and Video)

 

The OCE PFDD program has published multiple manuscripts in peer-reviewed journals to advance patient-focused drug development. Some selected articles are listed below:

Patient-Reported Outcomes in Anti-PD-1/PD-L1 Inhibitor Immunotherapy Registration Trials: FDA Analysis of Data Submitted and Future Directions. 
King-Kallimanis BL, Howie LJ, Roydhouse JK, Singh H, Theoret MR, Blumenthal GM, Kluetz PG. 
Clinical Trials. 2019. doi.org/10.1177/1740774519836991

FDA Approval Summary: Pertuzumab for adjuvant treatment of HER2-positive early breast cancer.
Howie LJ, Scher NS, Amiri-Kordestani L, Zhang L, King-Kallimanis BL, Choudhry Y, Schroeder J, Goldberg KB, Kluetz PG, Ibrahim A, Sridhara R, Blumenthal GM, Pazdur R, Beaver JA.
Clin Cancer Res. 2018 Dec 14. pii: clincanres.3003.2018. doi: 10.1158/1078-0432.CCR-18-3003. [Epub ahead of print]

Blinding and Patient-Reported Outcome Completion Rates in US Food and Drug Administration Cancer Trial Submissions, 2007-2017.
Roydhouse JK, King-Kallimanis BL, Howie LJ, Singh H, Kluetz PG.
J Natl Cancer Inst. 2018 Dec 17. doi: 10.1093/jnci/djy181. [Epub ahead of print]

Learning from Patients: Reflections on Use of Patient-Reported Outcomes in Lung Cancer Trials.
Basu Roy U, King-Kallimanis BL, Kluetz PG, Selig W, Ferris A.
J Thorac Oncol. 2018 Dec;13(12):1815-1817. doi: 10.1016/j.jtho.2018.09.003. Epub 2018 Oct 15. No abstract available. 

Moving forward toward standardizing analysis of quality of life data in randomized cancer clinical trials.
Bottomley A, Pe M, Sloan J, Basch E, Bonnetain F, Calvert M, Campbell A, Cleeland C, Cocks K, Collette L, Dueck AC, Devlin N, Flechtner HH, Gotay C, Greimel E, Griebsch I, Groenvold M, Hamel JF, King M, Kluetz PG, Koller M, Malone DC, Martinelli F, Mitchell SA, Moinpour CM, Musoro JZ, O'Connor D, Oliver K, Piault-Louis E, Piccart M, Pimentel FL, Quinten C, Reijneveld JC, Schürmann C, Smith AW, Soltys KM, Sridhara R, Taphoorn MJB, Velikova G, Coens C.
Clin Trials. 2018 Dec;15(6):624-630. doi: 10.1177/1740774518795637. Epub 2018 Aug 24.

Overview of Oncology and Hematology Drug Approvals at US Food and Drug Administration Between 2008 and 2016.
Zhou J, Vallejo J, Kluetz P, Pazdur R, Kim T, Keegan P, Farrell A, Beaver JA, Sridhara R.
J Natl Cancer Inst. 2018 Aug 4. doi: 10.1093/jnci/djy130. [Epub ahead of print]

Metastasis-free Survival - A New End Point in Prostate Cancer Trials.
Beaver JA, Kluetz PG, Pazdur R.
N Engl J Med. 2018 Jun 28;378(26):2458-2460. doi: 10.1056/NEJMp1805966. No abstract available. 

Informing the Tolerability of Cancer Treatments Using Patient-Reported Outcome Measures: Summary of an FDA and Critical Path Institute Workshop.
Kluetz PG, Kanapuru B, Lemery S, Johnson LL, Fiero MH, Arscott K, Barbachano Y, Basch E, Campbell M, Cappelleri JC, Cella D, Cleeland C, Coens C, Daniels S, Denlinger CS, Fairclough DL, Hillard JR, Minasian L, Mitchell SA, O'Connor D, Patel S, Rubin EH, Ryden A, Soltys K, Sridhara R, Thanarajasingam G, Velikova G, Coons SJ.
Value Health. 2018 Jun;21(6):742-747. doi: 10.1016/j.jval.2017.09.009. Epub 2017 Nov 7.

Incorporating the patient experience into regulatory decision making in the USA, Europe, and Canada.
Kluetz PG, O'Connor DJ, Soltys K.
Lancet Oncol. 2018 May;19(5):e267-e274. doi: 10.1016/S1470-2045(18)30097-4. Review.

Guidelines for Inclusion of Patient-Reported Outcomes in Clinical Trial Protocols: The SPIRIT-PRO Extension.
Calvert M, Kyte D, Mercieca-Bebber R, Slade A, Chan AW, King MT; the SPIRIT-PRO Group, Hunn A, Bottomley A, Regnault A, Chan AW, Ells C, O'Connor D, Revicki D, Patrick D, Altman D, Basch E, Velikova G, Price G, Draper H, Blazeby J, Scott J, Coast J, Norquist J, Brown J, Haywood K, Johnson LL, Campbell L, Frank L, von Hildebrand M, Brundage M, Palmer M, Kluetz P, Stephens R, Golub RM, Mitchell S, Groves T.
JAMA. 2018 Feb 6;319(5):483-494. doi: 10.1001/jama.2017.21903.

Use of PRO Measures to Inform Tolerability in Oncology Trials: Implications for Clinical Review, IND Safety Reporting, and Clinical Site Inspections.
Kim J, Singh H, Ayalew K, Borror K, Campbell M, Johnson LL, Karesh A, Khin NA, Less JR, Menikoff J, Minasian L, Mitchell SA, Papadopoulos EJ, Piekarz RL, Prohaska KA, Thompson S, Sridhara R, Pazdur R, Kluetz PG.
Clin Cancer Res. 2018 Apr 15;24(8):1780-1784. doi: 10.1158/1078-0432.CCR-17-2555. Epub 2017 Dec 13.

Commentary on Heath et al.
Kwitkowski V, Daniels S, Reaman G, Farrell A, Kluetz P.
Clin Trials. 2017 Dec;14(6):572-574. doi: 10.1177/1740774517723308. Epub 2017 Aug 2. No abstract available. 

Patient-Reported Outcomes in Cancer Clinical Trials: Measuring Symptomatic Adverse Events With the National Cancer Institute's Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE).
Kluetz PG, Chingos DT, Basch EM, Mitchell SA.
Am Soc Clin Oncol Educ Book. 2016;35:67-73. doi: 10.14694/EDBK_159514. Review.

Focusing on Core Patient-Reported Outcomes in Cancer Clinical Trials-Response.
Kluetz PG, Papadopoulos EJ, Johnson LL, Donoghue M, Kwitkowski VE, Chen WH, Sridhara R, Farrell AT, Keegan P, Kim G, Pazdur R.
Clin Cancer Res. 2016 Nov 15;22(22):5618. doi: 10.1158/1078-0432.CCR-16-2140. No abstract available. 

Analysing data from patient-reported outcome and quality of life endpoints for cancer clinical trials: a start in setting international standards.
Bottomley A, Pe M, Sloan J, Basch E, Bonnetain F, Calvert M, Campbell A, Cleeland C, Cocks K, Collette L, Dueck AC, Devlin N, Flechtner HH, Gotay C, Greimel E, Griebsch I, Groenvold M, Hamel JF, King M, Kluetz PG, Koller M, Malone DC, Martinelli F, Mitchell SA, Moinpour CM, Musoro J, O'Connor D, Oliver K, Piault-Louis E, Piccart M, Pimentel FL, Quinten C, Reijneveld JC, Schürmann C, Smith AW, Soltys KM, Taphoorn MJ, Velikova G, Coens C; Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data (SISAQOL) consortium.
Lancet Oncol. 2016 Nov;17(11):e510-e514. doi: 10.1016/S1470-2045(16)30510-1. Epub 2016 Oct 18. Review.

Looking to the future in an unprecedented time for cancer drug development.
Kluetz PG, Pazdur R.
Semin Oncol. 2016 Feb;43(1):2-3. doi: 10.1053/j.seminoncol.2016.01.001. Epub 2016 Jan 15.

Focusing on Core Patient-Reported Outcomes in Cancer Clinical Trials: Symptomatic Adverse Events, Physical Function, and Disease-Related Symptoms.
Kluetz PG, Slagle A, Papadopoulos EJ, Johnson LL, Donoghue M, Kwitkowski VE, Chen WH, Sridhara R, Farrell AT, Keegan P, Kim G, Pazdur R.
Clin Cancer Res. 2016 Apr 1;22(7):1553-8. doi: 10.1158/1078-0432.CCR-15-2035. Epub 2016 Jan 12.

Patient-Reported Outcomes in Cancer Drug Development and US Regulatory Review: Perspectives From Industry, the Food and Drug Administration, and the Patient.
Basch E, Geoghegan C, Coons SJ, Gnanasakthy A, Slagle AF, Papadopoulos EJ, Kluetz PG.
JAMA Oncol. 2015 Jun;1(3):375-9. doi: 10.1001/jamaoncol.2015.0530. Review.

Pain palliation measurement in cancer clinical trials: the US Food and Drug Administration perspective.
Basch E, Trentacosti AM, Burke LB, Kwitkowski V, Kane RC, Autio KA, Papadopoulos E, Stansbury JP, Kluetz PG, Smith H, Justice R, Pazdur R.
Cancer. 2014 Mar 1;120(5):761-7. doi: 10.1002/cncr.28470. Epub 2013 Dec 5.

Several examples of the OCE engaging with the community in the form of blogs and podcasts can be found below.

The FDA's Oncology Center of Excellence — Quantifying the Patient Experience. By Paul G. Kluetz, MD. 

Utilizing Patient-reported Outcomes in Cancer Clinical trials. By Karen Olsen, PhD.

PROs in regulatory decision making. By Paul G. Kluetz, MD.