Approval of new treatments for PH prompts hope, but also concerns

Medical advancements are great, but they need to be accessible to all

Jen Cueva avatar

by Jen Cueva |

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The pulmonary hypertension (PH) community is buzzing with excitement! It’s not every week that the U.S. Food and Drug Administration (FDA) approves two new treatments for a rare disease like PH. This monumental achievement is a testament to the dedication and passion of researchers, scientists, and the rest of the medical community. We’re beyond grateful!

First, in late March, the FDA approved a once-daily, fixed-dose tablet containing a combination of macitentan and tadalafil for adults with pulmonary arterial hypertension (PAH). It will be marketed under the brand name Opsynvi and is the first single-tablet treatment combination to become available to people with PAH in the U.S.

Then, just days later, the FDA approved the under-the-skin injection therapy sotatercept-csrk, which will be marketed under the brand name Winrevair, to treat adults with PAH.

However, amid our joy, there’s also a palpable sense of concern. Over the past few days, I’ve chatted with many members of the PH community, including patients, caregivers, family members, loved ones, and healthcare providers. A common sentiment seems to be that we’re thrilled but also anxious.

Some might wonder, why the worry? Well, unless you’ve navigated the complex world of managing a rare disease and facing insurance and healthcare hurdles when trying to access a medication, it might not be immediately apparent. Let me share some insights from my conversations with PH community members.

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Behind every new treatment for PH are dedicated professionals

Affordability and accessibility concerns

One PH patient contacted me right after she heard about the new FDA-approved medications. Her PH medical team had identified her as an ideal candidate for Opsynvi even before its approval. But she doesn’t have private health insurance, so her biggest concern was affordability.

I also received an email from an old “PHriend” last week who was curious about the latest treatments hitting the market. But instead of thinking about the potential to transform lives in our PH community, he was focused on availability and insurance coverage, particularly for Winrevair. He lives in a rural area, doesn’t drive, and the nearest medical center is a whopping four hours away. So accessibility is a big concern.

From what I’ve observed, unfortunately, many emerging treatments for rare diseases like PH come with hefty price tags, and not all insurance policies offer coverage for them. I wrote about this in a previous column titled “Why I am a ‘Million Dollar Baby.’”

Anecdotally, other patients have told me that their doctors suspected that the cost of the recently approved drugs might be steep. This raises important questions about the extent of coverage with insurance plans, including Medicare and supplemental insurance and private insurance.

I want to celebrate, but my heart also aches due to potential cost and accessibility.

As I scroll through social media, sift through my inbox, and chat with PHriends, I’m experiencing a range of emotions. The truth hits hard: New treatments, developed by tireless scientists and researchers, should be within everyone’s reach. Yet, not everyone can afford or access them.

“Why do you keep PHighting and advocating for the PH and rare disease community?” someone recently asked me. The answer is complex. There are a million reasons, but one that keeps surfacing is my drive to be a voice for others. I want to share the lessons from my 19-year journey with PH and fight for what we deserve. If one person benefits, I’m fulfilling my goal.

But let’s be real: Financial hurdles and other barriers to accessing care are like towering walls. It’s time for all hands on deck to address this issue. Both federal and state lawmakers must hear our stories.

This combination of excitement and concern contributes to the complexity of living with a rare disease like PH. We face these challenges hand in hand, lifted by the hope that new treatments offer. Let’s make some noise, share our stories, and PHight for the future we all deserve.

Have you heard about the new FDA-approved treatments for PH? What are your thoughts? Please share in the comments below.


Note: Pulmonary Hypertension News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Pulmonary Hypertension News or its parent company, BioNews, and are intended to spark discussion about issues pertaining to pulmonary hypertension.

Comments

Randy Reynolds avatar

Randy Reynolds

Jen
Nicely written. If this pill were to replace Remodglin I suspect that the costs would be comparable. On my next visit with my PAH doctor I'll be asking him about Opsynvi and Winrevair.

Randy

Reply
Jen Cueva avatar

Jen Cueva

Hi Randy,
Thanks for taking time to read my column about these new treatments and offer your feedback. Yes, I could see if these oral medications or injectable compared to Remodulin, the cost would not be cheap.But, thankfully the pharma companies often have copay assistance that helps.

Yes, please talk with your PH team and let us know in the forums the thoughts you get from him.You can add that update in the forums for all to read, if you could.

Again, thanks for your time and support, it means a ton. Take care, buddy!

Reply
Kathleen Grant avatar

Kathleen Grant

Jen, thank you so much for putting PAH which is rare. By you bringing PAH to the forefront helps all of us who was diagnosed with PAH. As patients we have the right to know what is new out there such as medications, tests and meds that touch each and every symptom. Sometimes and/or all the time we must be our own advocates for our own health. That is why what you are doing gives us more information.

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Jen Cueva avatar

Jen Cueva

Hi Kathleen,
I'm grateful that you find the information that I write about beneficial. Thanks for taking the time to not only read my column, but also share your kind words.

Yes, we are more likely to make better decisions about our health when we educate ourselves. Then, as you mention, we can self advocate.

Thanks again for your thoughtfulness. Take care, my PHriend.

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Teri Lewis avatar

Teri Lewis

Thank you so much for PHighting and advocating for all of us in the Rare Disease Club of PAH! Our medications are so expensive and hopefully manufacturers will continue to offer assistance funds for paying the outrageous costs with minimal co pays. I have been on a combo of Letaris, Adcirca and Uptravi for the last 9 years and I am always encouraged that new medications are being approved to prolong our life prognosis. Do you know if Opsynvi or Winrevair manufacturers have set up assistance funds or who the manufacturers are of these new medications? Also, are there any know side effects that may be different than those currently on the market for PAH? I will definitely speak with my team of Pulmonologists and Cardiologists at UCHealth Anschutz Medical Health in Denver about these new drugs! Very exciting!
Thanks and Blessings to all!
Teri

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David H. Lindner, D.O. MBA, FCCP, FACOI avatar

David H. Lindner, D.O. MBA, FCCP, FACOI

As I was finally in "formal" discussion with the pharmaceutical companies regarding both of these drugs for my PH patients, cost, availability and how much am I going to have to fight with insurance to obtain these for my patients dominated the conversation. The hours and number of employees involved in obtaining medications for my patients is onerous and while excited at new options, the hoops we will have to jump through are daunting for us, I shudder to think about what my patients will go through. Trying to get our dysfunctional legislative leaders to recognize the ills of our system regarding orphan medications and diseases at times feels fruitless, but only continued pressure on these public servants will hopefully someday lead to a better way.

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Joanne Sperando avatar

Joanne Sperando

I am praying that Medicare will cover some of the astronomical costs of these meds so that I can stave off the need for a heart/lung transplant. The latest proposed budgets want to cut Medicare, Social Security, and Medicaid. It's terrifying. I hope everyone in our community and anyone else fighting illness, votes this year for reps that will protect the most vulnerable among us.

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A Conversation With Rare Disease Advocates