Breakthroughs in pulmonary hypertension didn’t happen by chance; they started with clinical trials. While progress over the past few decades has transformed care, there is still no cure, and some forms of PH remain limited in treatment options. Anna Hemnes, chair of the Department of Medicine at Vanderbilt Health, shares how patients play a vital role in advancing research and shaping the future of PH care.

Hemnes said patient participation in clinical trials helps health care professionals understand how drugs may affect pulmonary vascular disease.

Without clinical trials and the patients who agreed to participate, Hemnes said there would be no FDA-approved therapies for pulmonary arterial hypertension, chronic thromboembolic pulmonary hypertension or interstitial lung disease-associated pulmonary hypertension.

Compared to 30 years ago, survival today has improved substantially in each of these conditions because of what we learned from clinical trials

“Compared to 30 years ago, survival today has improved substantially in each of these conditions because of what we learned from clinical trials,” Hemnes said. “I am thankful every day for the patients who took risks and participated.”

Common misconceptions

Hemnes said many patients hope that participating in drug trials may help manage their own disease, but that is not always the case. Even if the trial is not positive and the drug is not approved for the treatment of pulmonary hypertension, the field still learns how to improve future trials and whether specific patients, including those with other forms of pulmonary hypertension, may benefit from the findings.

“Everyone who participates in a clinical trial helps us understand more about the disease and how to treat it better,” Hemnes said.

During a clinical trial, patients are involved at all stages. Patients are given details about risks, benefits and procedures before agreeing to participate so they know what will happen every step of the way. Patients may also withdraw at any point during the trial, as participation is voluntary.

For complex conditions such as PH, your doctor and care team remain involved or coordinate with the research team to ensure continuity of care.

Who should participate?

Any patient who wants to help providers better understand and treat pulmonary hypertension should consider participating in a clinical trial.

Hemnes recommends patients ask their physician or health care provider about current and upcoming opportunities to participate in research, as they are always changing. Patients should also inform their care team of any trials they are considering or decide to participate in to ensure optimal care continues.

When considering participation, Hemnes encourages patients to focus on advancing understanding of the disease and best practices instead of the personal benefit. Although improvement of symptoms can happen with new therapies, it is not guaranteed, so Hemnes recommends managing expectations to avoid false hope.

“Participation is a highly personal choice,” said Hemnes. “But every patient has the opportunity to help us and the world understand pulmonary vascular disease better.”

Clinical trials today

New drugs and interventions are still needed to improve pulmonary hypertension care for those living with PH and those yet to be diagnosed. Any patient who participates in clinical trials helps researchers better understand PH. Trials are the foundation for medical progress.

“We owe tremendous gratitude to the patients who agreed to participate in prior clinical trials as they have all advanced our knowledge and moved the field forward,” Hemnes said. “Patients who participated in these trials, understanding their risks and benefits, have helped shape how we treat patients today, and we hope for more innovation in the future.”