The Oncology Center of Excellence (OCE) Patient-Focused Drug Development (PFDD) program fosters collaboration between FDA Centers and external stakeholders involved in patient outcomes research in cancer populations.
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 identifies rigorous methods to assess the patient experience which complements existing survival and tumor information to provide additional evidence about the effects of cancer therapies on patients.
The program focuses on three key areas:
In 2021, the OCE along with CDER and CBER published a guidance for industry providing recommendations for the collection of a core set of patient-reported clinical outcomes in cancer clinical trials and related considerations for instrument selection and trial design: “Core Patient-Reported Outcomes in Cancer Clinical Trials.”
Vishal Bhatnagar, M.D.
Oncology Center of Excellence
Vishal Bhatnagar, MD, is a medical oncologist/hematologist and the Associate Director for Patient Outcomes in the OCE. His interests include patient reported outcomes, patient preference and incorporation of patient experience in oncology trials. His work focuses on the operational management of the OCE’s Patient-Focused Drug Development program. Additionally, Dr. Bhatnagar has a strong clinical interest in multiple myeloma and has previously served as an Office of Hematology and Oncology multiple myeloma scientific liaison. Dr. Bhatnagar received his BA in Political Science and his medical degree at the George Washington University. He completed his internal medicine residency and hematology/oncology fellowship at the University of Maryland.
Erica Horodniceanu, MPH
Oncology Center of Excellence
Erica Horodniceanu, MPH, is a health scientist in the OCE’s Patient-Focused Drug Development program at the FDA. Over the past 20 years, Erica has provided healthcare research, health education, health communications, and project management services to pharmaceutical, biotech, and medical device companies. She has previously held positions in industry within consulting firms focused on outcomes research and has been working in the field of clinical outcome assessments (COAs) and patient-reported outcomes (PROs) for the past 10 years. Erica holds a Bachelor of Science degree in Health Science Education, with a concentration in Health Promotion from the University of Florida and a Master’s in Public Health degree in Public Health Practice and Policy from the University of Maryland.
Meena Murugappan, PharmD, MPH
Oncology Center of Excellence
Meena Murugappan, PharmD, MPH, is a pharmacist and pharmacoepidemiologist serving as a visiting postdoctoral scientist for Patient-Focused Drug Development at the Oncology Center of Excellence (OCE) within the US Food and Drug Administration (FDA). She is also a PhD candidate in health economics and outcomes research at the University of Minnesota College of Pharmacy. Meena has led numerous projects during her time at FDA including a landscape analysis of Patient Reported Outcomes in pediatric oncology trials, examination of floor/ceiling effects associated with the EORTC QLQ-C30’s Physical Functioning subscale, and evaluation of frailty in patients with Multiple Myeloma. Prior to joining FDA, she served as a research associate at the University of Minnesota, conducting large commercial claims data analyses using datasets such as Truven MarketScan and Medicare. She is interested in the role of Patient Reported Outcomes and Real-World Evidence in regulatory and healthcare decision making.
Paul G. Kluetz, M.D.
Oncology Center of Excellence
Paul Kluetz is a medical oncologist and the Deputy Director of the Oncology Center of Excellence at the U.S. FDA. In addition to his broader role in OCE’s strategic oversight and management, Dr. Kluetz founded the OCE’s PFDD program and continues to support its strategic mission. He has engaged the global cancer drug development and health outcomes community in leading a sustained effort to advance patient reported outcomes (PRO) data, wearable technologies, and other methods to obtain rigorous patient experience data in both the clinical trial and “real-world” settings.
- Collaborator: Kaiser Permanente, Division of Research – PANcreatic cancer Patient Reported Outcomes using the Electronic medical record (PanPROE)
- Collaborator: Northwestern University and University of Tasmania – Evaluation of a global item for side effect bother
- Collaborator: Duke University - Evaluating physical functioning using patient-reported outcome measures: How does the question form and recall period influence patients’ interpretation?
- Collaborator: Mayo Clinic and Yale University - Quantifying physical function in cancer patients undergoing chemotherapy using clinician- and patient-reports along with wearable device data
- Collaborator: Mayo Clinic and Yale University - Longitudinal analysis & visualization of patient-reported physical function & symptom data
- Collaborator: Mayo Clinic and Yale University - Qualitative techniques to define meaningful within-patient change in symptoms of advanced cancer patients
- Collaborator: National Cancer Institute– Advancing the use of digital health technologies and digital symptom reporting for patient-reported outcome measures
- National Cancer Institute (NCI) Cancer Moonshot Tolerability Consortium Steering Committee
- Patient-Reported Outcomes Tools: Engaging Users & Stakeholders (PROTEUS) consortium
- 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)
Clinical Outcomes Assessment in Clinical Cancer Trials (COA-CCT) Workshops
June 29, 2022: 7th COA-CCT Workshop
July 21 and 23, 2021: 6th COA-CCT Workshop
April 25, 2017: 2nd COA-CCT Workshop with the Critical Path Institute
April 26, 2016: COA-CCT Workshop with the Critical Path Institute
October 8, 2019: 3rd Partners in Progress
November 27, 2018: 2nd Partners in Progress
November 13, 2017: 1st Partners in Progress
The OCE PFDD Program continuously publishes on topics to advance patient-focused drug development. Recent articles are listed below:
Piccinin C, Basch E, Bhatnagar V, Calvert M, Campbell A, Cella D, et al. Recommendations on the use of item libraries for patient-reported outcome measurement in oncology trials: findings from an international, multidisciplinary working group. The Lancet Oncology. 2023 Feb 1;24(2):e86-95.
Bhatnagar V, Dutcus C, Ghiorghiu S, Kluetz P, Jones L, McJunkins KR, et al. Supporting a Patient-Centric Approach to Dose Optimization in Oncology: The Essential Role of Patient-Reported Outcomes (PROs). Friends of Cancer Research White Paper. 2022.
Bhatnagar V, Kluetz PG. Encouraging Rigorous Patient-Generated Data All Along the Drug Development Continuum. Journal Of The National Cancer Institute. 2022 Oct;114(10):1313-4.
King-Kallimanis BL, Bhatnagar V, Horodniceanu EG, Chen TY, Kluetz PG. Timing is everything: The importance of patient-reported outcome assessment frequency when characterizing symptomatic adverse events. Clinical Trials. 2022 Jun;19(3):267-73.
Fiero MH, Roydhouse JK, Bhatnagar V, Chen TY, King-Kallimanis BL, Tang S, et al. Time to deterioration of symptoms or function using patient-reported outcomes in cancer trials. The Lancet Oncology. 2022 May 1;23(5):e229-34.
Chen TY, King-Kallimanis BL, Merzoug L, Horodniceanu EG, Fiero MH, Gao JJ, et al. US Food and Drug Administration Analysis of Patient-Reported Diarrhea and Its Impact on Function and Quality of Life in Patients Receiving Treatment for Breast Cancer. Value in Health. 2022 Apr 1;25(4):566-70.
Murugappan MN, King‐Kallimanis BL, Mangir C, Howie L, Bhatnagar V, Beaver JA, et al. Floor and ceiling effects in the EORTC QLQ‐C30 Physical Functioning subscale among patients with advanced or metastatic breast cancer. Cancer. 2022 Feb 15;128(4):808-18.
Murugappan MN, King-Kallimanis BL, Reaman GH, Bhatnagar V, Horodniceanu EG, Bouchkouj N, et al. Patient-reported outcomes in pediatric cancer registration trials: A US Food and Drug Administration perspective. JNCI: Journal of the National Cancer Institute. 2022 Jan;114(1):12-9.
King-Kallimanis BL, Lederer NM, Kim J, Nair A, Horodniceanu E, Bhatnagar V, et al. Patient-reported outcomes after treatment discontinuation: commercial clinical trial data from four cancer types. Value in Health. 2021 Sep 1;24(9):1302-7.
Fernandes LL, Zhou J, Kanapuru B, Horodniceanu E, Gwise T, Kluetz PG, et al. Review of patient-reported outcomes in multiple myeloma registrational trials: highlighting areas for improvement. Blood Cancer Journal. 2021 Aug 31;11(8):148.
Calvert M, King M, Mercieca-Bebber R, Aiyegbusi O, Kyte D, Slade A, et al. SPIRIT-PRO Extension explanation and elaboration: guidelines for inclusion of patient-reported outcomes in protocols of clinical trials. BMJ open. 2021 Jun 1;11(6):e045105.
Roydhouse JK, Mishra-Kalyani PS, Bhatnagar V, Gutman R, King-Kallimanis BL, Sridhara R, et al. Does knowledge of treatment assignment affect patient report of symptoms, function, and health status? An evaluation using multiple myeloma trials. Value in Health. 2021 Jun 1;24(6):822-9.
Kluetz PG, King-Kallimanis BL, Suzman D, Chang E, Brave MM, Weinstock C, et al. Advancing assessment, analysis, and reporting of safety and tolerability in cancer trials. JNCI: Journal of the National Cancer Institute. 2021 May;113(5):507-8.
Kluetz PG, Bhatnagar V. The FDA's patient-focused drug development initiative. Clinical Advances in Hematology & Oncology: H&O. 2021 Feb 1;19(2):70-2.
Bhatnagar V, Hudgens S, Piault‐Louis E, Jones L, Beaver JA, Lyerly HK, et al. Patient‐reported outcomes in oncology clinical trials: Stakeholder perspectives from the accelerating anticancer agent development and validation workshop 2019. The Oncologist. 2020 Oct;25(10):819-21.
Roydhouse JK, King-Kallimanis BL, Roy P, Weinstock C, Krol D, Daniels SR, et al. Exploration of baseline patient-reported side effect bother from cancer therapy. Clinical Trials. 2020 Jun;17(3):332-7.
Roydhouse JK, Gutman R, Bhatnagar V, Kluetz PG, Sridhara R, Mishra‐Kalyani PS. Analyzing patient‐reported outcome data when completion differs between arms in open‐label trials: an application of principal stratification. Pharmacoepidemiology and Drug Safety. 2019 Oct;28(10):1386-94.
King-Kallimanis BL, Howie LJ, Roydhouse JK, Singh H, Theoret MR, Blumenthal GM, et al. Patient reported outcomes in anti-PD-1/PD-L1 inhibitor immunotherapy registration trials: FDA analysis of data submitted and future directions. Clinical Trials. 2019 Jun;16(3):322-6.
Roydhouse JK, King-Kallimanis BL, Howie LJ, Singh H, Kluetz PG. Blinding and patient-reported outcome completion rates in US food and drug administration cancer trial submissions, 2007–2017. JNCI: Journal of the National Cancer Institute. 2019 May 1;111(5):459-64.
Roydhouse JK, Fiero MH, Kluetz PG. Investigating potential bias in patient-reported outcomes in open-label cancer trials. JAMA oncology. 2019 Apr 1;5(4):457-8.
Kluetz PG, Kanapuru B, Lemery S, Johnson LL, Fiero MH, Arscott K, et al. Informing the tolerability of cancer treatments using patient-reported outcome measures: summary of an FDA and critical path institute workshop. Value in Health. 2018 Jun 1;21(6):742-7.
Kluetz PG, O'Connor DJ, Soltys K. Incorporating the patient experience into regulatory decision making in the USA, Europe, and Canada. The Lancet Oncology. 2018 May 1;19(5):e267-74.
Kim J, Singh H, Ayalew K, Borror K, Campbell M, Johnson LL, et al. Use of PRO Measures to Inform Tolerability in Oncology Trials: Implications for Clinical Review, IND Safety Reporting, and Clinical Site Inspections. Clinical Cancer Research. 2018 Apr 15;24(8):1780-4.
Kluetz PG, Chingos DT, Basch EM, Mitchell SA. 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). American Society of Clinical Oncology Educational Book. 2016 May;36:67-73.
Kluetz PG, Slagle A, Papadopoulos EJ, Johnson LL, Donoghue M, Kwitkowski VE, et al. Focusing on core patient-reported outcomes in cancer clinical trials: symptomatic adverse events, physical function, and disease-related symptoms. Clinical Cancer Research. 2016 Apr 1;22(7):1553-8.