백서
희귀 질환 신약 개발의 과제와 기회
새로운 희귀 질환 치료법의 개발에는 기회와 복잡한 문제가 공존합니다. 해당 적응증에 대한 환자 풀이 적어 임상시험의 설계 및 수행과 그 결과로 얻은 데이터의 해석, 그리고 최종적인 등록 과정에 어려움이 있습니다.
The top 3 critical downfalls in rare diseases development include
Poor understanding of the disease process and natural history
Incomplete understanding of clinically meaningful endpoints
Inability to assess clinical benefit and achieve full approval
This whitepaper by drug development experts, Drs. Julie Bullock and Rajesh Krishna, reflect on some of these challenges and opportunities in rare diseases drug development.
Key learning points
- An overview of the biology and genetics of rare diseases
- How to leverage regulatory frameworks to reduce regulatory uncertainty
- The challenges of the accelerated approval mechanism
- How to streamline rare disease drug development using model-informed drug development
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