TY - JOUR
T1 - Integration of Patient Reported Outcomes in Drug Development in Genitourinary Cancers
AU - Wong, Risa L.
AU - Morgans, Alicia K.
PY - 2020/3/1
Y1 - 2020/3/1
N2 - Purpose of Review: Patient reported outcomes (PROs) are increasingly utilized in cancer drug development, and are of particular importance in genitourinary cancers due to symptom burden, multiple treatment options with similar efficacy, and often prolonged duration of disease. Here we review current data and perspectives related to use of PROs in drug development for genitourinary cancers, including insights on the regulatory process for drug approval. Recent Findings: The FDA is committed to incorporating PRO data into the regulatory process for development and approval of new cancer drugs, but challenges exist due to lack of standardization of PRO instrument choice and analytic approach, missing data, and difficulty isolating treatment effect from disease-related effects. We review guidance for standardization of PRO methodology that is nonetheless tailored to disease state and anticipated effects of treatment. PRO and efficacy data should be simultaneously analyzed and reported for best clinical practice. Multiple disease-specific PRO instruments exist for genitourinary cancers. Summary: While clinicians, researchers, and regulatory bodies alike recognize the importance of PROs in cancer drug development, challenges remain regarding implementation of best practices.
AB - Purpose of Review: Patient reported outcomes (PROs) are increasingly utilized in cancer drug development, and are of particular importance in genitourinary cancers due to symptom burden, multiple treatment options with similar efficacy, and often prolonged duration of disease. Here we review current data and perspectives related to use of PROs in drug development for genitourinary cancers, including insights on the regulatory process for drug approval. Recent Findings: The FDA is committed to incorporating PRO data into the regulatory process for development and approval of new cancer drugs, but challenges exist due to lack of standardization of PRO instrument choice and analytic approach, missing data, and difficulty isolating treatment effect from disease-related effects. We review guidance for standardization of PRO methodology that is nonetheless tailored to disease state and anticipated effects of treatment. PRO and efficacy data should be simultaneously analyzed and reported for best clinical practice. Multiple disease-specific PRO instruments exist for genitourinary cancers. Summary: While clinicians, researchers, and regulatory bodies alike recognize the importance of PROs in cancer drug development, challenges remain regarding implementation of best practices.
KW - Drug development
KW - Genitourinary cancer
KW - Patient reported outcomes
KW - Prostate cancer
KW - Quality of life
KW - Regulatory decision making
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UR - http://www.scopus.com/inward/citedby.url?scp=85079109851&partnerID=8YFLogxK
U2 - 10.1007/s11912-020-0890-3
DO - 10.1007/s11912-020-0890-3
M3 - Review article
C2 - 32036478
AN - SCOPUS:85079109851
VL - 22
JO - Current Oncology Reports
JF - Current Oncology Reports
SN - 1523-3790
IS - 3
M1 - 21
ER -