PMID- 30790065
OWN - NLM
STAT- MEDLINE
DCOM- 20190314
LR  - 20200225
IS  - 1573-904X (Electronic)
IS  - 0724-8741 (Linking)
VI  - 36
IP  - 4
DP  - 2019 Feb 21
TI  - Ophthalmic Drug Discovery and Development: Regulatory Aspects of Patient Focused 
      Drug Development in Ophthalmology.
PG  - 54
LID - 10.1007/s11095-019-2577-8 [doi]
AB  - In 2009, members of the ophthalmic research community held a joint meeting with 
      members of the Food and Drug Administration (FDA) and the National Eye Institute 
      (NEI) to define and describe the types of patient-focused drug development (PFDD) 
      tools used in ophthalmology. Since then numerous reports have been published 
      which indicate that many of the questionnaires used for patient-reported outcomes 
      (PROs) in ophthalmic clinical development lack rigor and reliability according to 
      modern methods. In 2017, the FDA began development of a series of four 
      methodological guidances for sponsors of clinical trials on the significance of 
      PFDD. The new guidances delineate the FDA's thinking and commitments under the 
      Prescription Drug User Fee Act to implement a more structured approach to the 
      assessment of risks and benefits in clinical trials. In these guidances, the FDA 
      provides steps that drug and device manufacturers should follow, not only to 
      obtain, but also to develop reliable and validated tools that measure patients' 
      experience in clinical trials. Subsequent efforts have resulted in the 
      development and validation of PROs specifically for ophthalmology. The purpose of 
      this paper is to assesses the PROs currently used in ophthalmology and to provide 
      practical strategies for incorporating them into clinical trials.
FAU - Rowe-Rendleman, Cheryl L
AU  - Rowe-Rendleman CL
AUID- ORCID: 0000-0002-3024-2356
AD  - Ophthalmic Medical and Research Consulting, Omar Consulting Group, LLC, Princeton 
      Junction, NJ, 08550, USA. crrendleman@omarconsultants.com.
LA  - eng
PT  - Journal Article
PT  - Review
DEP - 20190221
PL  - United States
TA  - Pharm Res
JT  - Pharmaceutical research
JID - 8406521
RN  - 0 (Ophthalmic Solutions)
SB  - IM
MH  - Administration, Ophthalmic
MH  - Clinical Trials as Topic/*legislation & jurisprudence/methods
MH  - Drug Approval/*legislation & jurisprudence/methods
MH  - Drug Compounding
MH  - Drug Development/*legislation & jurisprudence/methods
MH  - Drug Discovery/*legislation & jurisprudence/methods
MH  - Endpoint Determination
MH  - Eye/*drug effects/metabolism/pathology/physiopathology
MH  - Eye Diseases/*drug therapy/metabolism/pathology/physiopathology
MH  - *Government Regulation
MH  - Humans
MH  - Ocular Absorption
MH  - Ophthalmic Solutions
MH  - *Patient Reported Outcome Measures
MH  - Patient Safety
MH  - Psychometrics
MH  - Risk Assessment
MH  - Treatment Outcome
MH  - United States
MH  - United States Food and Drug Administration/*legislation & jurisprudence
OTO - NOTNLM
OT  - FDA
OT  - PROMs
OT  - PROs
OT  - clinical trials
OT  - patient-reported outcomes
EDAT- 2019/02/23 06:00
MHDA- 2019/03/15 06:00
CRDT- 2019/02/22 06:00
PHST- 2018/11/06 00:00 [received]
PHST- 2019/01/17 00:00 [accepted]
PHST- 2019/02/22 06:00 [entrez]
PHST- 2019/02/23 06:00 [pubmed]
PHST- 2019/03/15 06:00 [medline]
AID - 10.1007/s11095-019-2577-8 [pii]
AID - 10.1007/s11095-019-2577-8 [doi]
PST - epublish
SO  - Pharm Res. 2019 Feb 21;36(4):54. doi: 10.1007/s11095-019-2577-8.