Not Worth Less

Jul 23, 2019

Guest Blog by Jennifer McNary, Patient Advocate

ICER. What is it? Who is it? Who put them in charge of my children’s health care? I ask this question – urgently – because Thursday, this group called ICER is meeting in Massachusetts to decide whether my sons, Austin and Max, should continue to have access to new drugs that are treating their rare, fatal disease.  Austin and Max both have Duchenne Muscular Dystrophy, a disease that progressively robs its victims of muscle function until the day where their hearts and lungs stop working and they die, usually before their 20th birthday – and for too many, long before then.

The Duchenne community has worked tirelessly for decades for access to a safe and effective treatment. That day finally came in 2016 with the first-ever FDA approval for a Duchenne therapy, a treatment that  helps slow the progression of this disease and preserves muscle function in some Duchenne patients.  When the FDA approved Exondys51, I had no idea that the victory we had secured could become meaningless.  But now this ICER group is meeting to “vote” on whether patients like Austin and Max should have access to Exondys51, and Emflaza and a third treatment still under review by the FDA.  ICER’s recommendation could be used by insurance companies to deny access to the safe, effective, FDA-approved treatment that is improving and extending my boys’ lives.

How can that happen?  ICER has come up with its own measure of what a patient with a rare or chronic condition is worth – valuing my sons’ lives as less than their friends who were born without this fatal condition.  ICER devalues patients to make the case on behalf of the insurance industry to deny access to the very treatments we fought and sacrificed so much for. It is unfathomable that after all we have gone through, a private, non-governmental group can take away access to the only treatments that exist to help my children.

I didn’t know this was possible, and if you suffer from a rare disease or a chronic condition, this could also happen to you! ICER has already reviewed other rare diseases such as SMA, cystic fibrosis, and hemophilia A, and is planning to do the same thing to larger diseases such as Type II Diabetes, arthritis, and cardiovascular disease in the coming months. No patient who needs innovation in order to thrive is safe from ICER and their reviews.

Every person with a rare disease knows the enormity of getting FDA approval of an actual treatment.  It takes years of effort and sacrifice with no guarantee of results.  We hold bake sales and community 5Ks.  We walk the halls of Congress and the National Institutes of Health.  We knock on the doors of companies that know how to develop drugs and get them to market. And in many cases – including my sons’ – patients contribute to the cause literally with their own bodies.  Muscle tissue was cut from Austin’s body – repeatedly and over time – to determine whether the now-FDA-approved drug was safe and effective.  And only AFTER all the door knocking and bake sales, and blood, sweat, and tears, were we able to stand in front of the FDA – literally 1,000 of us strong – to present our evidence. 

The FDA is the official U.S. government entity directly responsible for protecting all Americans by evaluating  drugs to ensure that they are safe and effective.  The FDA listened to us, the patients, the researchers, the clinicians, and the experts, and reviewed all the data.  They approved a first of its kind drug for Duchenne that has given my family, and so many like mine, hope. 

The process is long and arduous. Most potential therapies fail in early clinical trials and FDA approval only comes by meeting the highest standards of safety and efficacy.  And while we may wish it would move faster, we recognize that the FDA has its charge, and importantly, we have the confidence of knowing that the FDA is accountable to all of us, as patients, as taxpayers and as citizens.  The FDA is accountable to Austin and Max.

ICER has no such charge, and has no accountability to anyone – not to patients, not to taxpayers, not to citizens.  Please join me in person or online on July 25th to ask ICER, who put YOU in charge of my sons’ health care? Click here to sign up for updates, to let us know you’re coming, or for more information on how you can get involved.

About The Author

Jennifer McNary

Jennifer McNary
Patient Advocate

Jenn McNary is a trusted voice in the rare disease community, as a mother, public speaker and fierce advocate. Her work in the rare disease space as a thought leader earned her the Ryan’s Quest Ryan’s Hero award in 2013, a nomination for the Global Genes Champion of Hope award in 2014, and the prestigious 2017 Meyer- Whalley instrument of change award. Formerly as the director of outreach and advocacy at a Massachusetts based non-profit foundation, she was responsible for the organization of the largest FDA advisory committee hearing in history, with over 1000 duchenne advocates, families, clinicians and researchers in attendance. There are currently only three drugs approved for Duchenne, Exondys51, Vyondys53 and Emflaza, though in various roles, Jenn was involved in the approval process for all three. Jenn has unique experience in the drug development field, as a parent of children enrolled in the clinical trials, an advocate engaging with the regulators and as a consultant helping to develop programing for patients. Currently, Jenn is consulting in the biotechnology space with an expertise in caregiver/patient engagement, including bringing the patient voice to drug development and solving barriers to access. Her other activities include serving as the Founder of One Rare, a non-profit formed to meet the needs of young adults with rare and chronic conditions and raising her four children in Massachusetts.

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