TY - JOUR
T1 - Can Innovative Trial Designs in Orphan Diseases Drive Advancement of Treatments for Common Neurological Diseases?
AU - Stephenson, Diane
AU - Ollivier, Cecile
AU - Brinton, Roberta
AU - Barrett, Jeffrey
N1 - Funding Information:
The Critical Path Institute recognizes the strong leadership and support of US FDA Center of Drug Evaluation Research staff, including Teresa Mullin, Michelle Campbell, Issam Zineh, and Billy Dunn. The Critical Path Institute is supported by the FDA of the US Department of Health and Human Services (HHS) and is 56.5% funded by FDA/HHS, totaling $16,749,891, and 43.5% funded by non‐government source(s), totaling $12,895,366. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the US Government. For more information, please visit FDA.gov. We acknowledge the C‐Path leadership team with special recognition of Klaus Romero, Chief Science Officer and Quantitative Medicine leader, and Terina Martinez, Executive Director of The Duchenne Regulatory Science Consortium. Special recognition goes to Jesse Cedarbaum for his strong support of C‐Path and leadership in PD and ALS. The authors acknowledge Kimberly Ward Barowicz for development of Figure 2 and Jane Larkindale for review of the manuscript. We recognize the support of the EMA for their role in supporting C‐Path. C‐Path acknowledges FDA’s Dr. Michelle Campbell for her strong support of Critical Path for Parkinson’s, Duchenne Regulatory Science, and Rare Disease Cures Accelerator Programs at C‐Path.
Funding Information:
No funding was received for this work. The Critical Path Institute recognizes the strong leadership and support of US FDA Center of Drug Evaluation Research staff, including Teresa Mullin, Michelle Campbell, Issam Zineh, and Billy Dunn. The Critical Path Institute is supported by the FDA of the US Department of Health and Human Services (HHS) and is 56.5% funded by FDA/HHS, totaling $16,749,891, and 43.5% funded by non-government source(s), totaling $12,895,366. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the US Government. For more information, please visit FDA.gov. We acknowledge the C-Path leadership team with special recognition of Klaus Romero, Chief Science Officer and Quantitative Medicine leader, and Terina Martinez, Executive Director of The Duchenne Regulatory Science Consortium. Special recognition goes to Jesse Cedarbaum for his strong support of C-Path and leadership in PD and ALS. The authors acknowledge Kimberly Ward Barowicz for development of Figure 2 and Jane Larkindale for review of the manuscript. We recognize the support of the EMA for their role in supporting C-Path. C-Path acknowledges FDA’s Dr. Michelle Campbell for her strong support of Critical Path for Parkinson’s, Duchenne Regulatory Science, and Rare Disease Cures Accelerator Programs at C-Path.
Publisher Copyright:
© 2022 The Authors. Clinical Pharmacology & Therapeutics published by Wiley Periodicals LLC on behalf of American Society for Clinical Pharmacology and Therapeutics.
PY - 2022/4
Y1 - 2022/4
N2 - Global regulatory agencies have transformed their approach to approvals in their processes for formal review of the safety and efficacy of new drugs. Opportunities for innovation have expanded because of the coronavirus disease 2019 (COVID-19) pandemic. Several regulatory-led initiatives have progressed rapidly during the past year, including patient-focused drug development, model-informed drug development, real-world evidence, and complex innovative trial designs. Collectively, these initiatives have accelerated the rate of approvals. Despite demands to focus on urgent needs imposed by the COVID-19 pandemic, the number of new drug approvals over the past year, particularly for rare diseases, has outpaced expectations. Advancing therapeutics for nervous system disorders requires adaptive strategies that align with rapid developments in the field. Three relentlessly progressive diseases, amyotrophic lateral sclerosis, Duchenne muscular dystrophy, and Parkinson’s disease are in urgent need of new treatments. Herein, we propose new regulatory initiatives, including innovative trial designs and patient-focused drug development that accelerate clinical trial conduct while meeting critical regulatory requirements for therapeutic approval.
AB - Global regulatory agencies have transformed their approach to approvals in their processes for formal review of the safety and efficacy of new drugs. Opportunities for innovation have expanded because of the coronavirus disease 2019 (COVID-19) pandemic. Several regulatory-led initiatives have progressed rapidly during the past year, including patient-focused drug development, model-informed drug development, real-world evidence, and complex innovative trial designs. Collectively, these initiatives have accelerated the rate of approvals. Despite demands to focus on urgent needs imposed by the COVID-19 pandemic, the number of new drug approvals over the past year, particularly for rare diseases, has outpaced expectations. Advancing therapeutics for nervous system disorders requires adaptive strategies that align with rapid developments in the field. Three relentlessly progressive diseases, amyotrophic lateral sclerosis, Duchenne muscular dystrophy, and Parkinson’s disease are in urgent need of new treatments. Herein, we propose new regulatory initiatives, including innovative trial designs and patient-focused drug development that accelerate clinical trial conduct while meeting critical regulatory requirements for therapeutic approval.
UR - http://www.scopus.com/inward/record.url?scp=85124460178&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85124460178&partnerID=8YFLogxK
U2 - 10.1002/cpt.2528
DO - 10.1002/cpt.2528
M3 - Review article
C2 - 35034352
AN - SCOPUS:85124460178
VL - 111
SP - 799
EP - 806
JO - Clinical Pharmacology and Therapeutics
JF - Clinical Pharmacology and Therapeutics
SN - 0009-9236
IS - 4
ER -