A Blueprint For Addressing Underserved Patient Populations

By Martina Flammer, MD, MBA, chief medical officer, Insmed Incorporated

The biopharmaceutical industry has made significant strides in addressing rare and serious diseases, but the path from bench to bedside remains uniquely complex for diseases that affect smaller patient populations. Limited natural history data, geographically dispersed patient populations, and regulatory uncertainty often slow development and deter R&D investment. We must ask ourselves a critical question: Does our current approach to clinical research truly serve the patients who face the greatest needs?

To make real progress, we must reshape the way we design, execute, and operationalize clinical development. This begins by understanding who the patients are, what matters most to them, where the biggest gaps in care lie, and what it will take to reach patients with much-needed information, support, and access.

What Does It Mean To Be Underserved?

Underserved populations can broadly be defined as those with restricted access to or limited benefits from healthcare and can include rural residents, racial and ethnic minorities, low-income individuals, and older adults.¹ While each of these factors presents challenges, I find that when it comes to rare or less prevalent diseases, geography is perhaps the most vital consideration. Patients can live anywhere, from rural areas to major cities, but if they are not near an academic medical center or dedicated center of excellence (COE) — a highly specialized program that takes a comprehensive, interdisciplinary approach to treating a disease — they may struggle to find consistent access to quality care.

In complex conditions, COEs can be a lifeline for fast, accurate diagnosis and ongoing treatment. Yet they are often geographically disparate and not readily accessible to most patients. And many patients are not connected to a consistent care model at all, relying instead on urgent care centers or emergency departments.

One solution that we’ve seen work well in non-cystic fibrosis bronchiectasis (NCFB) and nontuberculous mycobacterial (NTM) lung disease is an interconnected network of COEs, where experts at established sites train providers in other locations. With support from the industry, including Insmed, the Bronchiectasis and NTM Association established the Bronchiectasis and NTM Care Center Network, which trains medical centers in diverse geographic locations on accurate diagnosis and optimal care for patients with these diseases.

Outside of Insmed, I’ve seen similar approaches yield great success in diseases such as cystic fibrosis, pulmonary fibrosis, and scleroderma, where patient advocacy-led initiatives have established networks of care based on patient needs rather than a traditional academic center model. These programs have been effective in delivering care directly to patients, even in isolated pockets of the country, rather than patients having to travel to major metropolitan areas for regular visits.

Engaging Patients As True Partners In Drug Development

When it comes to designing clinical trials, we’ve learned through engagement with patients and patient advocacy groups that patients often tell us what physicians and regulators may overlook: which symptoms affect them most, which aspects of daily living they wish they could regain, and which parts of a trial may be burdensome to navigate. These are critical insights that we aim to incorporate into how we structure and run our trials. In NCFB, for example, while traditional study endpoints focus on cough, sputum production, and respiratory function, many patients tell us that fatigue is the most debilitating symptom they experience. This insight led us to advocate for including fatigue as an endpoint in a Phase 3 trial alongside traditional clinical markers. Although this endpoint is challenging to measure in a clinical trial setting and did not prove favorable when the results were analyzed, we felt it was important to include a measure that mattered most to patients.

Drug formulation is another area where patient feedback is valuable. Patients with respiratory diseases often face challenges, such as difficulty using an inhaler or nebulizer device, intolerability of dry powder inhalers, or the need for frequent dosing. When considering novel treatment approaches for these diseases, conversations with patients help ensure new therapies are not only effective but also practical. At Insmed, we abide by this principle — even if it means going back to square one and investing additional time and resources to ensure a medicine is truly fit for purpose.

Once we start down the path of drug development, the support we provide for patients throughout the clinical trial journey is critical to our shared success. Some examples of best practices we employ include:

• working with patient advocacy groups to raise awareness of clinical trial options with the patients they represent
• selecting clinical trial sites that have a strong infrastructure, deep commitment to quality, and broad geographic reach — not just traditional academic centers
• reducing trial participation burden by offering travel support for participants, flexible clinical trial visit schedules, and, when appropriate, remote clinical trial visits
• supporting trial retention with strategies such as digital engagement platforms and patient-friendly communications
• providing clinical trial results in language participants can understand.

Importance Of Access

Above all, as we pursue patient-centered drug development, we are acutely aware that even the most promising therapy fails to make a difference if patients can't access it.

For our medicines to get into the hands of the patients who need them, we must ensure that our safety and efficacy data translate into compelling value stories for regulators, clinicians, and payers. We think about access and coverage from the very beginning of trial design and incorporate measures into our studies that will demonstrate a real impact. We’ve learned that having these data in hand will give us the support we need later when engaging with payers appropriately and compliantly ahead of bringing a therapy to market.

A sharp focus on access also means thinking about the ways in which we’ll support patients and caregivers once a therapy is approved. We have built a robust patient support program that helps patients understand information about their condition and their treatment, coordinate logistics for prescription dispenses, and keep them informed during the insurance approval process. The program is designed to help patients feel less overwhelmed and alone in what we know can be a challenging moment of being prescribed a new treatment.

A Call To Action For Our Industry

At Insmed, our aim is simple: to pursue first- and best-in-class therapies that stand to make the greatest impact for patients. If we are to advance truly transformative therapies, particularly in small and underserved populations, we must be willing to rethink our approach to clinical development. That means designing trials rooted in science and what matters to patients, bringing care pathways closer to communities, and incorporating access considerations every step of the way.

This approach is central to how we operate today and how we plan for the future. But true progress requires collective movement. I encourage our industry peers, academic partners, regulators, and patient groups to join us in building a clinical development ecosystem that is more informed by the patient community’s expertise and more adaptive to the complexities of real-world care.

Patients have waited long enough, and their needs must serve as our North Star. By aligning our scientific ambition with genuine commitment to patient partnership, we can build a clinical research enterprise that serves the people who need it most.

References:

1. Mehl KR, Morain SR, Largent EA. The Importance of Including Underserved Populations in Research. Pharmaceut Med. 2025 Mar;39(2):59-71. doi: 10.1007/s40290-025-00562-1. Epub 2025 Apr 1. PMID: 40169528; PMCID: PMC11980435. Available at: https://pmc.ncbi.nlm.nih.gov/articles/PMC11980435/

About The Author:

Martina Flammer, MD, MBA, joined Insmed as chief medical officer in December 2019, bringing more than 17 years of industry experience in both medical and commercial roles. She has launched global brands and managed pipeline portfolios across therapeutic areas and geographies, including the U.S., Europe, Japan, and China. Prior to joining Insmed, Martina was head of corporate division customer value, senior vice president, at Boehringer Ingelheim International. Previous roles at Boehringer Ingelheim include vice president clinical development & medical affairs, specialty care business unit, and chief medical officer, vice president of medicine, regulatory affairs & pharmacovigilance, Boehringer Ingelheim Canada. Martina previously held commercial and medical roles at Pfizer. She has a medical degree from the University of Vienna Medical School, Austria, and completed post-doctoral training in internal and emergency medicine. She also holds a an MBA from New York University Stern School of Business.