The future of the development of medicines in idiopathic pulmonary fibrosis

BMC Medicine

Irmgard Eichler¹  Laura Fregonese² 

[1] Paediatric Medicines Office, European Medicines Agency, London, UK;Orphan Medicines Office, European Medicines Agency, 30 Churchill Place, Canary Wharf, London, E14 5EU, UK

关键词: Regulatory approval;    Outcome measures;    Idiopathic pulmonary fibrosis;    Early access;    Drug discovery;    Clinical development;   

Others  :  1227196
DOI  :  10.1186/s12916-015-0480-7

 received in 2015-09-03, accepted in 2015-09-03,  发布年份 2015

【 摘 要 】

The development of treatments for idiopathic pulmonary fibrosis (IPF) has been often disappointing. Building on authorized treatments that can benchmark the validity of treatment effect measures, the time has come to standardize endpoints and achieve consensus on their use for different clinical questions and specific IPF phenotypes. In order to facilitate the development of new medicines for IPF it is crucial that the knowledge of the disease and lessons learnt from past trials are taken forward to create international trial networks with involvement of patients, including biobanks and clinical data collection through a multinational registry. Interaction with regulators may be useful to align the initiatives of academia and pharmaceutical companies with the bodies ultimately responsible for licensing new products. Interaction can occur through the use of qualification programs for biomarkers and endpoints, and participation in innovative regulatory pathways and initiatives. Finally, the experience of IPF should be used to benefit even rarer interstitial lung diseases for which no treatment is available, including pediatric interstitial lung diseases. This commentary provides a perspective on the hurdles slowing the development and regulatory approval of medicines for IPF, and encourages close cooperation between investigators and drug regulators.

【 授权许可】

   
2015 Fregonese and Eichler.

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