Abstract
Special Collection on Clinical Trial Design: past, present and future
Background
Pulmonary vascular diseases matter. It is a field of high unmet medical need covering orphan diseases and is the special focus of the Pulmonary Vascular Research Institute (PVRI). The Innovative Drug Development Initiative (IDDI) is a unique core activity within the PVRI facilitating the development of novel treatments for patients with pulmonary hypertension (PH) and right ventricular failure and establish them into clinical practice.
The IDDI provides a platform for academia, the pharmaceutical industry (established and emerging biotechnology) as well as drug regulators to openly discuss questions surrounding the future of trials comprising novel drugs based on experience gained so far. It encourages early and continuous dialogue with all stakeholders, including the patient’s perspective, on innovative clinical and regulatory development strategies, with the aim of finding new ways to fight and treat pulmonary vascular disease and pulmonary hypertension as life-threatening conditions.
Content
This special collection presents the collaborative outcome of the work of the four IDDI workstreams on clinical trial design, biomarkers, endpoints and repurposing drugs as presented in this edition of Pulmonary Circulation:
Clinical trial design in phase 2 and 3 trials for pulmonary hypertension Role of biomarkers in evaluation, treatment and clinical studies of PAH Novel composite clinical endpoints and risk scores used in clinical trials in PAH Repurposing of medications for pulmonary arterial hypertension
Outlook
The PVRI, as a global society, is committed to improve the live for patients with PH in both high- and low-income countries. These projects have an increasing range in complexity and format designed to meet the specific needs of this specific populations. Therefore, the IDDI will be closely aligned to another of the core PVRI core activities namely Global Health Initiative, such that some future projects will be aligned with generic directives from the World Health Organisation.
In this collection leading experts in the field reflect on previous experience, review present understanding, and consider how their respective areas of expertise may develop in years ahead. It will be valuable for students learning a special disease area for the first time, for lecturers in their teaching, and for clinicians and drug developers wanting an overview of their field, and for anyone who might be interested in this advancing area of global health.
It is hoped that these manuscripts will provide a useful source of informed comment of direct benefit to patients with PH worldwide, going forward.