Innovation in pulmonary hypertension (PH) does not happen in isolation. It requires coordinated global effort, cross-sector collaboration, and a willingness to confront long-standing clinical challenges with fresh thinking. That is precisely the ambition behind the Innovative Drug Discovery Initiative (IDDI) Workstreams, a flagship initiative of the Pulmonary Vascular Research Institute (PVRI).

A collaborative and targeted leadership approach

In 2025, IDDI entered a new phase with a refreshed leadership team. Sandeep Sahay, Associate Professor of Medicine at Weill Cornell Medical College and Director of the Pulmonary Hypertension Program at Houston Methodist Hospital, and Luke Howard, Consultant Pulmonologist and Lead Clinician for the Pulmonary Hypertension Service, based at Hammersmith Hospital, Imperial College Healthcare NHS Trust, were joined by industry leaders Gil Golden, Senior Vice President and Chief Medical Officer of United Therapeutics Corporation, and Sidra Hoffman, Senior Director Clinical Development Pulmonary Vascular Disease at Gossamer Bio.

This multiskilled leadership team reflects IDDI’s defining characteristic: alignment across academia, clinical practice, and industry.

Photo: Luke Howard, Gil Golden, Karen Osborn (PVRI CEO), Sidra Hoffman and Sandeep Sahay in Amsterdam, 2025

“The IDDI was only created a short time ago – several years with a pandemic in between – and if you look at where we are today, we’re hitting attendance records and filling conference rooms. The conversations are robust, on point, timely and leading to future collaborations that can drive the field forward in an efficient way that really benefits all the parties.”

“There is a tendency in big groups working together to become distracted, so as a leadership team, we are trying to be targeted in our focus so that we can achieve our aims in the best way possible.” – Gil Golden, MD, PhD.

With the publication of its 2025-26 IDDI Workstream & Task Forces Report, PVRI provides a blueprint for how the PH community can accelerate discovery, refine clinical research, and expand access to care worldwide. At its core, IDDI represents a shift from siloed research toward structured, purpose-built collaboration. Its 7 global workstreams tackle the most pressing barriers in PH drug development and clinical translation – from trial design and endpoint selection to paediatric research and real-world data integration. Rather than pursuing innovation in the abstract, each workstream focuses on clearly defined gaps in PH research and care.

Redefining access to care

Global inequities in PH diagnosis and treatment remain stark. The Access to Care Workstream seeks to map these disparities and identify practical strategies to improve the availability of therapies and specialist expertise – particularly in under-resourced regions. By combining local insight with international collaboration, the group is working toward a more equitable global standard of care.

“We published a paper in January 2026 – Global Resource Disparities Between Pulmonary Hypertension Centers: Results From the International Survey by the PVRI IDDI Access to Care Workstream – in PVRI’s Pulmonary Circulation journal highlighting significant global disparities in PH resources, with varied availability of diagnostic tools and therapies across regions and income levels – because the first step to making change is to know what the magnitude of the problem and what the challenges are.”

“By clearly defining where inequities exist across geography and income levels, we have an evidence base to inform targeted solutions rather than broad assumptions.” – Sandeep Sahay, MD, MSc, FCCP. ATSF.

Re-engineering clinical trials

Clinical trials in PH have evolved, but persistent challenges remain in endpoint selection, recruitment, and trial conduct. Improved endpoints are not merely academic refinements; they determine how quickly promising therapies reach patients and how convincingly the benefit is demonstrated to regulators and payers.

“We want to be ambitious. We met with industry leaders in Amsterdam in 2025, and the message was clear: clinical trials in PH need to become more efficient. We have a growing number of new targets and drugs entering development, but the patient population available for trials hasn’t grown. In fact, many patients are now so well treated that they no longer meet traditional study criteria, while others don’t qualify for different reasons. That creates a real challenge. We need smarter ways to study smaller groups of patients and reach faster ‘go or no-go’ decisions before moving into large, expensive phase II and III trials.” 

“Over the next 5 years, we hope to reshape trial design — by supporting approaches such as digital monitoring and advanced imaging endpoints — so decisions can be made more quickly and with greater confidence. It’s ambitious, but it’s exactly the kind of challenge we believe we should take on.” – Luke Howard, MA, DPhil, FRCP.

The IDDI Leadership Team are currently looking at the best way to address the challenges of clinical trial design: through a dedicated workstream, or as a thread running through all the workstreams.

Advancing Paediatric Research

Children with PH face distinct biological and clinical challenges, yet paediatric trial design has historically lagged adult research. The dedicated Paediatric Clinical Trial Design & Endpoints Workstream is addressing this gap, advocating for development frameworks that recognize the unique needs of younger patients while maintaining scientific rigour.

Megan Griffiths, Paediatric Cardiologist and PH Specialist, did a phenomenal talk, If you had it all: designing the ideal paediatric PH clinical trial, at the PVRI 2026 IDDI meeting in Dublin, that really got to the heart of those in the room. PVRI’s Head of Communications and Marketing, Deborah Driscoll, interviewed Megan Griffiths and Sidra Hoffman at PVRI 2026 in Dublin on the challenges facing paediatric PH patients and how the IDDI can be part of the solution.

Watch the full interview here.

“The question is, why do we still not have access to medications that we have decades of research on, but are not yet approved for treatment in children – why?! I want to credit my paediatric colleagues for being steadfast in their request to gather assistance. At PVRI 2026, we, as a leadership team, discussed novel methods for addressing this. The goal is to file an application with the medications that we believe meet the criteria. It’s time!” – Sidra Hoffman, PhD, MSc, CMD.

Elevating the patient voice

Patient engagement has matured from consultation to co-creation. The former Patient Engagement & Empowerment Workstream helped catalyse the global PH Patient Survey, which has since become a standalone PHGPS Task Force – underscoring the growing importance of structured patient input in research prioritization and care design.

In today’s research environment, meaningful patient partnership is not optional; it is foundational.

Embracing new modalities and technologies

Therapeutic innovation in PH increasingly extends beyond traditional pharmacology. The New Modalities & Technologies Workstream explores advances ranging from novel delivery systems and inhaled platforms to device-based interventions and emerging diagnostic tools. The emphasis is on future-proofing the field – ensuring that regulatory frameworks, clinical pathways, and scientific understanding evolve alongside technological progress.

Tackling Group III PH

PH associated with chronic lung disease (Group III PH) remains one of the most complex and therapeutically challenging areas. IDDI’s focused PH Group III Workstream reflects recognition that disease heterogeneity demands targeted investigation rather than extrapolation from other PH groups.

Harnessing real-world evidence

Randomized controlled trials remain the gold standard – but they are not the whole story. The Real-World Evidence & Data Workstream aims to complement trial data with insights drawn from clinical registries and routine care. By refining methodologies for analysing real-world datasets, the group seeks to answer questions that traditional trials cannot fully address, including long-term effectiveness and diverse patient representation.

Extending global reach with PVRI task forces

Beyond its IDDI Workstreams, PVRI has a network of Task Forces, including Regional Task Forces of Sri Lanka, Central Asia, China, India, Latin America, and Saudi Arabia Task Forces, alongside Speciality Task Forces dedicated to imaging, exercise physiology, infection in pulmonary vascular disease, women’s health, and genetic research, such as PAH-ICON.

This decentralized yet coordinated model ensures that innovation does not remain concentrated in a handful of academic centres but is informed by diverse healthcare systems and patient populations.

A collaborative model for the future

By structuring collaboration through defined workstreams, the IDDI demonstrates how a global network can move beyond discussion toward measurable impact. It has strengthened industry engagement and its patient-centred philosophy position it as a model for therapeutic development in rare and complex diseases.

“Our purpose is collaboration. When you bring the best and brightest into the room with all their passion, and you have academics working hand in hand with industry folks, I believe we can move mountains. We do it piece by piece, but as a whole, it is truly impressive. There is no other organisation in the therapeutic area of pulmonary vascular disease, I think that can move mountains the way PVRI can, and IDDI is a key component of that.” – Gil Golden, MD, PhD.

The message to the PH community is clear: progress will not be driven by isolated breakthroughs alone, but by sustained, coordinated effort. As IDDI enters its next chapter, its workstreams represent both a map of current priorities and a catalyst for the breakthroughs still to come.

To keep up to date with upcoming PVRI events, visit https://pvrinstitute.org/upcoming-events.

Visit PVRI’s comprehensive library of abstracts, scientific talks, scientific papers, and research on pulmonary vascular disease.