Advancing clinical trials for inherited retinal diseases: Recommendations from the second monaciano symposium

The Monaciano Consortium

doi:10.1167/tvst.9.7.2

Advancing clinical trials for inherited retinal diseases: Recommendations from the second monaciano symposium

The Monaciano Consortium

Ophthalmology

Research output: Contribution to journal › Review article › peer-review

57 Scopus citations

Abstract

Major advances in the study of inherited retinal diseases (IRDs) have placed efforts to develop treatments for these blinding conditions at the forefront of the emerging field of precision medicine. As a result, the growth of clinical trials for IRDs has increased rapidly over the past decade and is expected to further accelerate as more therapeutic possibilities emerge and qualified participants are identified. Although guided by established principles, these specialized trials, requiring analysis of novel outcome measures and endpoints in small patient populations, present multiple challenges relative to study design and ethical considerations. This position paper reviews recent accomplishments and existing challenges in clinical trials for IRDs and presents a set of recommendations aimed at rapidly advancing future progress. The goal is to stimulate discussions among researchers, funding agencies, industry, and policy makers that will further the design, conduct, and analysis of clinical trials needed to accelerate the approval of effective treatments for IRDs, while promoting advocacy and ensuring patient safety.

Original language	English (US)
Article number	2
Journal	Translational Vision Science and Technology
Volume	9
Issue number	7
DOIs	https://doi.org/10.1167/tvst.9.7.2
State	Published - Jun 2020

Keywords

Clinical trials
Counseling patients
Inherited retinal diseases
Natural history studies
Outcome measures

ASJC Scopus subject areas

Biomedical Engineering
Ophthalmology

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10.1167/tvst.9.7.2

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@article{b2aa74e7f30d4f4faa39fdf754a5402e,

title = "Advancing clinical trials for inherited retinal diseases: Recommendations from the second monaciano symposium",

abstract = "Major advances in the study of inherited retinal diseases (IRDs) have placed efforts to develop treatments for these blinding conditions at the forefront of the emerging field of precision medicine. As a result, the growth of clinical trials for IRDs has increased rapidly over the past decade and is expected to further accelerate as more therapeutic possibilities emerge and qualified participants are identified. Although guided by established principles, these specialized trials, requiring analysis of novel outcome measures and endpoints in small patient populations, present multiple challenges relative to study design and ethical considerations. This position paper reviews recent accomplishments and existing challenges in clinical trials for IRDs and presents a set of recommendations aimed at rapidly advancing future progress. The goal is to stimulate discussions among researchers, funding agencies, industry, and policy makers that will further the design, conduct, and analysis of clinical trials needed to accelerate the approval of effective treatments for IRDs, while promoting advocacy and ensuring patient safety.",

keywords = "Clinical trials, Counseling patients, Inherited retinal diseases, Natural history studies, Outcome measures",

author = "{The Monaciano Consortium} and Thompson, {Debra A.} and Alessandro Iannaccone and Ali, {Robin R.} and Arshavsky, {Vadim Y.} and Isabelle Audo and Bainbridge, {James W.B.} and Besirli, {Cagri G.} and Birch, {David G.} and Branham, {Kari E.} and Cideciyan, {Artur V.} and Daiger, {Steven P.} and Deniz Dalkara and Duncan, {Jacque L.} and Fahim, {Abigail T.} and Flannery, {John G.} and Roberto Gattegna and Heckenlively, {John R.} and Elise Heon and Jayasundera, {K. Thiran} and Khan, {Naheed W.} and Henry Klassen and Leroy, {Bart P.} and Molday, {Robert S.} and Musch, {David C.} and Pennesi, {Mark E.} and Petersen-Jones, {Simon M.} and Pierce, {Eric A.} and Rao, {Rajesh C.} and Reh, {Thomas A.} and Sahel, {Jose A.} and Dror Sharon and Sieving, {Paul A.} and Enrica Strettoi and Paul Yang and Zacks, {David N.}",

note = "Publisher Copyright: {\textcopyright} 2020 The Authors.",

year = "2020",

month = jun,

doi = "10.1167/tvst.9.7.2",

language = "English (US)",

volume = "9",

journal = "Translational Vision Science and Technology",

issn = "2164-2591",

publisher = "Association for Research in Vision and Ophthalmology Inc.",

number = "7",

}

TY - JOUR

T1 - Advancing clinical trials for inherited retinal diseases

T2 - Recommendations from the second monaciano symposium

AU - The Monaciano Consortium

AU - Thompson, Debra A.

AU - Iannaccone, Alessandro

AU - Ali, Robin R.

AU - Arshavsky, Vadim Y.

AU - Audo, Isabelle

AU - Bainbridge, James W.B.

AU - Besirli, Cagri G.

AU - Birch, David G.

AU - Branham, Kari E.

AU - Cideciyan, Artur V.

AU - Daiger, Steven P.

AU - Dalkara, Deniz

AU - Duncan, Jacque L.

AU - Fahim, Abigail T.

AU - Flannery, John G.

AU - Gattegna, Roberto

AU - Heckenlively, John R.

AU - Heon, Elise

AU - Jayasundera, K. Thiran

AU - Khan, Naheed W.

AU - Klassen, Henry

AU - Leroy, Bart P.

AU - Molday, Robert S.

AU - Musch, David C.

AU - Pennesi, Mark E.

AU - Petersen-Jones, Simon M.

AU - Pierce, Eric A.

AU - Rao, Rajesh C.

AU - Reh, Thomas A.

AU - Sahel, Jose A.

AU - Sharon, Dror

AU - Sieving, Paul A.

AU - Strettoi, Enrica

AU - Yang, Paul

AU - Zacks, David N.

PY - 2020/6

Y1 - 2020/6

N2 - Major advances in the study of inherited retinal diseases (IRDs) have placed efforts to develop treatments for these blinding conditions at the forefront of the emerging field of precision medicine. As a result, the growth of clinical trials for IRDs has increased rapidly over the past decade and is expected to further accelerate as more therapeutic possibilities emerge and qualified participants are identified. Although guided by established principles, these specialized trials, requiring analysis of novel outcome measures and endpoints in small patient populations, present multiple challenges relative to study design and ethical considerations. This position paper reviews recent accomplishments and existing challenges in clinical trials for IRDs and presents a set of recommendations aimed at rapidly advancing future progress. The goal is to stimulate discussions among researchers, funding agencies, industry, and policy makers that will further the design, conduct, and analysis of clinical trials needed to accelerate the approval of effective treatments for IRDs, while promoting advocacy and ensuring patient safety.

AB - Major advances in the study of inherited retinal diseases (IRDs) have placed efforts to develop treatments for these blinding conditions at the forefront of the emerging field of precision medicine. As a result, the growth of clinical trials for IRDs has increased rapidly over the past decade and is expected to further accelerate as more therapeutic possibilities emerge and qualified participants are identified. Although guided by established principles, these specialized trials, requiring analysis of novel outcome measures and endpoints in small patient populations, present multiple challenges relative to study design and ethical considerations. This position paper reviews recent accomplishments and existing challenges in clinical trials for IRDs and presents a set of recommendations aimed at rapidly advancing future progress. The goal is to stimulate discussions among researchers, funding agencies, industry, and policy makers that will further the design, conduct, and analysis of clinical trials needed to accelerate the approval of effective treatments for IRDs, while promoting advocacy and ensuring patient safety.

KW - Clinical trials

KW - Counseling patients

KW - Inherited retinal diseases

KW - Natural history studies

KW - Outcome measures

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UR - http://www.scopus.com/inward/citedby.url?scp=85086021830&partnerID=8YFLogxK

U2 - 10.1167/tvst.9.7.2

DO - 10.1167/tvst.9.7.2

M3 - Review article

AN - SCOPUS:85086021830

SN - 2164-2591

VL - 9

JO - Translational Vision Science and Technology

JF - Translational Vision Science and Technology

IS - 7

M1 - 2

ER -

Advancing clinical trials for inherited retinal diseases: Recommendations from the second monaciano symposium

Abstract

Keywords

ASJC Scopus subject areas

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