Pulmonary hypertension advocates recently participated in an Institute for Clinical and Economic Review meeting about a new PH treatment expected to receive FDA approval in March. The advocates shared scientific information and personal insight about the impact of PH, the importance of robust insurance coverage for treatment and benefits of the new drug, sotatercept.

Julia Feitner, who was diagnosed with pulmonary arterial hypertension 15 years ago, said her experience in the sotatercept clinical trial over the past two years “has been course-altering, life-rejuvenating and hope-restoring.”

The testimony by Feitner and other PHA advocates was part of PHA’s ongoing work to educate ICER about PH during the organization’s 2023 review of sotatercept. ICER is a private, non-governmental organization that publishes cost-analysis reports on emerging “health technologies.”

Katie Kroner, PHA’s vice president of advocacy and patient education, reminded the panel that sotatercept addresses a new disease pathway that can benefit people for whom current therapy options are less effective.

Each year, ICER reviews select new therapies, such as prescription drugs, and issues recommendations about their value compared to existing treatments and how they should be priced. Prescription drug manufacturers can use ICER’s recommendations to guide pricing. Health insurance companies can use the recommendations to determine how much to reimburse patients for treatments and how they distribute costs between the company and the patient. ICER’s final report on sotatercept is expected this month.

“I hope that your final report underscores just how much quality of life is currently impacted by PH, and how the potential for physical improvement leads to quality of life improvement,” said Collen Brunetti, immediate past chair of PHA. “I hope that you can settle on a presentation of data and recommendations that take the whole patient in mind and centers the patient and providers as drivers of shared decision making.”

Brunetti reminded the panel that “behind every number is a patient like me — impacted every day by PH, and desperate for a better tomorrow. Anything we can do to move the needle forward has inherent value, and that is why I chose to be here.”

PHA board member and support group leader Colleen Connor said, “We need new treatment options for PH. Death and transplant are far too common in my support group … We have lost far too many patients.

‘My dream is to see my children mature, attend their weddings – no, dance at their weddings — meet my grandchildren … And maybe, just maybe, do the things with my grandchildren that I could not do with my own kids.”

Katie Tobias described her rollercoaster experience with IV therapy to a three-drug treatment combination and back to IV therapy.

“The terrifying truth is I may collapse in death’s front yard with very little warning,” she said “I have no other treatment options [that will work for me]. I started the evaluation process for a double-lung transplant as a precaution, but that’s just trading one battle for another.

“As we fight for our own lives, we must honor those who have gone before us … I’ve loved and lost some of the best people because of this disease. We all have. I can’t help wondering … what if they had access to this new treatment? What would the world look like if our angels were standing with us today, still living, still breathing?”

Although ICER has acknowledged sotatercept’s clinical benefits, the group has said the drug’s expected price was unlikely to meet common measures of cost-effectiveness.

PHA’s Kroner noted that expert clinical judgment and the patient-clinician relationship must drive care, including all available treatments.

“Any delay in placing patients on the therapy options that are best for them, including delays created by step therapy requirements, can lead to irreversible decline and an overall increase in health care costs,” Kroner said.

ICER has questioned how the design of the clinical trial might affect health insurance coverage of sotatercept. Because the original clinical trial excluded people with functional class IV PAH, some health insurance companies might hesitate to cover the drug for those patients. However, Merck, which manufactures sotatercept, is recruiting participants for new trials that would include children and people with functional class III and IV PAH.