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Master Protocols: A Promising Approach to Accelerate Drug Development in Rare Kidney Diseases
Julie Lin1, Jai Radhakrishnan2
1Sanofi, Cambridge, MA and Brigham and Women's Hospital, MRB-4, 75 Francis Street, Boston, MA, USA. julierenal@gmail.com.
Abstract:
Kidney diseases have been a highly challenging area for new drug development because of traditional requirements for reaching doubling of serum creatinine, dialysis, or transplantation endpoints for regulatory approval, which translates into clinical trials needing several years of follow up and large numbers of study participants to achieve adequate power. In recent years, however, progress in surrogate endpoints (specifically proteinuria reduction and slowing of estimated glomerular filtration rate decline in rare glomerular diseases) has resulted in greatly increased interest by biotechnology and pharmaceutical sponsors in investing in these indications.
Insights
Drug development for kidney diseases is advancing with new surrogate endpoints. Proteinuria reduction and slowing estimated glomerular filtration rate decline are accelerating clinical trials and investment.
Area of Science:
- Nephrology and drug development.
- Advancements in clinical trial design for kidney diseases.
Background:
- Traditional kidney disease drug development faced hurdles due to lengthy clinical trial endpoints like dialysis or transplantation.
- These lengthy trials required significant time and participant numbers, hindering therapeutic innovation.
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