Drug Development for Rare Diseases

Bo Yang · Yang Song · Yijie Zhou

feb. 2023 · CRC Press

E-bok

232

Sider

Kvalifisert

Vurderinger og anmeldelser blir ikke kontrollert Finn ut mer

Om denne e-boken

A disease is defined as rare if the prevalence is fewer than 200,000 in the United States. It is estimated that there are more than 7,000 rare diseases, which collectively affect 30 million Americans or 10% of the US population. This diverse and complex disease area poses challenges for patients, caregivers, regulators, drug developers, and other stakeholders. This book is proposed

to give an overview of the common issues facing rare disease drug developers, summarize challenges specific to clinical development in small populations, discuss drug development strategies in the evolving regulatory environment, explain generation and utilization of different data and evidence

inside and beyond clinical trials, and use recent examples to demonstrate these challenges and the development strategies that respond to the challenges.

Key Features:

• Rare disease.

• Drug development.

• Innovative clinical trial design.

• Regulatory approval.

• Real-world evidence.

Om forfatteren

Bo Yang is the Vice President, Biometrics and Real World Evidence at Vertex Pharmaceuticals, USA.

Yang Song is the Executive Director, Biostatistics Group Head of Pipeline Development at Vertex Pharmaceuticals, USA.

Yijie Zhou is the Executive Director, Real World Statistics and Analytics at Vertex Pharmaceuticals, USA.

Vurder denne e-boken

Fortell oss hva du mener.

Hvordan lese innhold

Smarttelefoner og nettbrett

Installer Google Play Bøker-appen for Android og iPad/iPhone. Den synkroniseres automatisk med kontoen din og lar deg lese både med og uten nett – uansett hvor du er.

Datamaskiner

Du kan lytte til lydbøker du har kjøpt på Google Play, i nettleseren på datamaskinen din.

Lesebrett og andre enheter

For å lese på lesebrett som Kobo eReader må du laste ned en fil og overføre den til enheten din. Følg den detaljerte veiledningen i brukerstøtten for å overføre filene til støttede lesebrett.

Rapporter ulovlig innhold