This paper discusses the data for and against the use of molecular biomarkers as an enrichment strategy in future clinical trials in PAH. This is a call for the collection of prospective data to create a biomarker panel that would then be employed in future trials for PAH.
This review is from the PVRI Innovative drug Development Initiative which includes academic, regulatory and pharmaceutical leaders in the field of PAH. They address changes in the traditional endpoints in the trials for PAH and offer newer approaches including the use of risk scores to enrich the patient pool appropriate for the type of treatment used.
This paper discusses the various possible clinical trial designs that are well suited for a disease like PAH. The authors point out that orphan diseases make the typical parallel design of randomized blinded trials difficult and often risky. Novel trial designs can result in smaller trials which are less costly, while at the same enriched to allow for larger treatment effects.
This review focuses on the pitfalls of traditional randomized clinical trials, and suggest more modern approaches with new endpoints. Co-authored by members of the FDA, they also discuss newer treatments under development which may be better suited for these strategies.
NIH Workshop Summary from leading experts. A review of precision medicine approaches to treatments for PAH including inadequacies of therapy of PAH using the traditional approach, considerations in the conduct of clinical trials for a precision medicine approach, considerations in the design of clinical trials, and pharmaceutical industry challenges with precision medicine clinical trials.