Abstract
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.
Highlights
The development of new medicines is characterized by very high attrition rates, with up to 10,000 compounds failing to show clinical efficacy per each new medicine reaching the market
Past failures have been attributed to the heterogeneous pathogenesis of the disease, the inappropriateness of study designs and endpoints used in clinical trials [5,6,7], and the lack of merit of the products studied, which in many cases were repurposed and not developed for idiopathic pulmonary fibrosis (IPF)
To clarify the usefulness and limitations of the bleomycin model as proof of concept in IPF and other interstitial lung diseases (ILDs), standardization is needed of methodological aspects, such as the timing of administration of candidate products—which are currently administered in most preclinical studies before or immediately after bleomycin challenge [20] as opposed to the clinical situation where lung fibrosis is already present at the start of treatment
Summary
The development of new medicines is characterized by very high attrition rates, with up to 10,000 compounds failing to show clinical efficacy per each new medicine reaching the market. IPF drug discovery between present and future To date, 12 medicinal products, most in preclinical phase of development, have obtained orphan designation from the European Medicines Agency (EMA) [10] for the treatment of IPF.
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