Real World Health Care Blog

Tag Archives: cystic fibrosis

“Not Being Able to Afford Medication for Our Child is Devastating”

During the month of August, Real World Health Care will take a short break from focusing on medical breakthroughs and the researchers who are shaping the future of medicine. We will instead bring you a special series from our sponsor, the HealthWell Foundation, about what happens when families cannot afford the medical treatments their children desperately need. The families we will profile have turned to the Foundation for help, through the HealthWell Pediatric Assistance Fund®, the only fund of its kind.


Did You Know?

Thanks to HealthWell’s Pediatric Assistance Fund, more than 400 children suffering from 90 life-altering conditions have been able to access critical medical treatments.


Since its launch in 2013, HealthWell’s Pediatric Assistance Fund has awarded more than $850,000 in grants to help more than 400 children start or continue critical treatments covering more than 90 disease areas and conditions, including ADHD, autism, cerebral palsy, Type 1 Diabetes, epilepsy, scoliosis, seizure disorder and many more. The Fund covers family cost-shares for surgical procedures, medical devices, counseling services, prescription drug copays and other out-of-pocket costs. This week, we’d like you to meet Alejandro, who was diagnosed with Cystic Fibrosis when he was six weeks old.

In the following letter, Alejandro’s parents, Ruben and Thelma, share the challenges of affording care for their child and the big difference that HealthWell’s Pediatric Assistance Fund grant made in their lives.

Aljeandro

Aljeandro

“Our son, Alejandro, was diagnosed with Cystic Fibrosis (CF) when he was six weeks old. This was a time with many mixed emotions for our family. Alejandro is a twin and we were thrilled to welcome two new babies into our family. Along with our excitement, there were many sad times because we were scared of the unknown: CF and our biggest fear as parents came true with his diagnosis. He has a chronic illness that can be managed, somewhat, but has no cure. 

CF is a disease that requires Alejandro to take many medications and breathing treatments. As part of his treatment, his doctors prescribed medication during the respiratory syncytial virus (RSV) season. Alejandro was prescribed one shot per month for a total of five shots; one for each month from November through March. Our out-of-pocket cost is $750 [$150 per shot], which is a huge financial burden. We applied to the HealthWell Foundation for financial assistance in order to be able to pay the copayment for Alejandro’s treatment. We also contacted the manufacturer of the drug. Unfortunately, having CF does not make you eligible to receive financial support. Having a child with a chronic illness has placed a lot of stress on our family, especially on my husband and me. 

Not being able to afford medication for our child is devastating and extremely frustrating for us as parents. This is why we are so thankful for the Pediatric Assistance Fund through the HealthWell Foundation. Without a grant, we would not have been able to afford this extremely important medication for Alejandro. Receiving help was heartwarming to us because, although the contributors to the Pediatric Assistance Fund do not know our son, they were willing to donate money to ensure a child who is in need of medication receives it. On behalf of Alejandro and our family, we would like to thank all of those kind people who donate their hard earned money for children like Alejandro who need extras in order to live.”

Ruben & Thelma (Alejandro’s parents)

Companies: An average Pediatric Assistance Fund grant is $2,500. With your corporate donation, you can help children like Alejandro afford the medical treatments they desperately need. Donate today to HealthWell’s Pediatric Assistance Fund.

The State of Cystic Fibrosis and Precision Medicine

EDITOR’S NOTE: This article is reprinted with permission from BioTechPrimer.com.

During President Obama’s State of the Union address earlier this year, a cystic fibrosis patient named Bill Elder sat beside First Lady Michelle Obama. Diagnosed with the disease at 8 years old, Mr. Elder is “healthier now than ever before” at age 27, thanks to ivacaftor. As a third-year medical student, he is not only surviving but thriving. Receiving an invitation to be the guest of honor at the presidential speech of the year is the exclamation mark to an extraordinary story.

Emily Burke, BiotechPrimer.com

Emily Burke, BiotechPrimer.com

Mr. Elder is an example of the success of modern medicine. His cystic fibrosis (CF) treatment derives from an understanding of the underlying molecular causes of the disease. This approach, referred to by the President as precision medicine, is the focus of new federal investments to speed the development of targeted therapeutics — drugs designed for a subset of patients with a specific genetic defect rather than for the “average” patient.

Personalized Medicine vs. Precision Medicine

Personalized medicine implies the development of medicines for an individual, based on their unique genetic, metabolic, microbiomic and other “signatures.” Think of a breast cancer patient getting a genetic test for the BRCA gene to determine their specific genetic mutation and subsequent personalized course of treatment — not just a therapy for all BRCA-induced cancers. As large scale, full-genome sequencing becomes more efficient and common, we may start to see truly personalized medicines.

But for now, a better term is “stratified” or precision medicine — dividing patient groups into specific populations and designing new drugs for those subtypes.

What is Cystic Fibrosis?

Cystic fibrosis is a genetic disease caused by one of several possible mutations in the gene encoding the “cystic fibrosis transmembrane conductance regulator” (CTFR) protein. The CTFR protein is critical for the production of sweat, digestive fluids and mucus. It affects around 70,000 people globally and is prevalent in America, Europe and Australia.

The CTFR protein is classified as a channel protein — a category of proteins that create a channel, or tunnel, across the cell membrane. This specialized gateway allows things to pass through the cell that will otherwise be denied entry or exit.

Negatively charged chloride ions use CTFR to exit cells, and if CTFR is not functioning correctly, the chloride ion builds up inside of cells. The build-up affects the fluid balance of tissue, which results in characteristically thick mucus seen in the lungs of CF patients. This thick mucus makes CF patients vulnerable to potentially fatal lung infections.

CF is an autosomal recessive disorder, meaning if an individual has one functioning copy of the CTFR gene, they are termed “carriers” and will not develop the disease. Two copies of the malfunctioning CTFR gene, one from each parent, will equal a diagnosis. And while CF is always caused by a mutation, many possible mutation combinations have been associated with the disease — up to 1,500 mutations, maybe more, are possible.

Precision medicine plays the hero by identifying the exact effect these underlying mutations have on CTFR, and designing treatments to overcome the disease.

On the Market

Cystic Fibrosis is symptomatically managed by reducing the risks of lung infections and implementing lifestyle changes to prevent such infections. Antibiotics are taken at the slightest sign of sickness, or even prophylactically, and other medications work to thin mucus. As the disease progresses, a double lung transplant may be the only, albeit elusive, treatment.

The dire medical outlook changed for a subset of CF patients in 2012 when ivacaftor, a small molecule drug, received FDA approval. Ivacaftor works by binding to the misfolded CTFR channel protein and increasing its ability to remain open and functional on cellular surfaces. It is indicated for few than 10% of CF patients; Mr. Elder is one of the lucky ones who responds to it.

With the success of ivacaftor, the manufacturer developed another small molecule drug, lumacaftor.

Approved by the FDA in July 2015, lumacaftor is paired with ivacaftor to target the most common CF mutation responsible for about 70% of the diagnosed CF cases in U.S. Caucasians. In these patients, the channel protein is so damaged it never makes it to the cell surface. Lumacaftor corrects some of the misfolds, improving CTFR’s ability to travel to the cell surface.

******************************************************************************

A Message from our Sponsor: Are you or a loved one struggling with copay costs for your Cystic Fibrosis treatments? HealthWell Foundation may be able to help. Visit our Cystic Fibrosis Fund page to learn more.

********************************************************************************

Make Life a Little Easier for Sick Children This #GivingTuesday

Cyndi from Midland, TX didn’t know how she was going to pay for the asthma medication her son Ethan desperately needed. Feeling like nothing was going right and not knowing where to turn, she was on the verge of losing hope. Then something dramatic happened that would change everything. Cyndi discovered the HealthWell Foundation:

Cyndi didn't know how she was going to pay for her son Ethan's (right) asthma medication until she found the HealthWell Foundation.

Cyndi didn’t know how she was going to afford asthma medication for her son Ethan (right) until she applied to the HealthWell Foundation for financial assistance.

Our son was born very sick. He spent 5 weeks in the NICU, 2 of which were on a ventilator. When we finally brought him home, we knew I had to stay home with him. If we put him in daycare, we knew he would get sick and that would be hard on his lungs. With me not working, it put a huge cloud over our finances. We quickly found out that his asthma medication was not cheap, even with insurance. I also found out that there isn’t a lot of help for people who have insurance and a decent job. I spent hours and hours on the Internet and the phone trying to find some help. One day I found HealthWell. We applied thinking we would be denied. The day I got the acceptance letter in the mail, I sat on the kitchen floor and cried. I felt a sense of relief. Finally, something was going right. We cannot be more grateful for all the help HealthWell has given us. 

When you give to HealthWell’s Pediatric Assistance Fund for #GivingTuesday, you will make a difference for children who have been diagnosed with a chronic or life-altering condition, just in time for the holidays. Every penny of your donation will help us provide financial assistance to patients so they can afford the rising cost of care:

  • To date, HealthWell has awarded more than 274,000 grants to insured adults and children across all of our funds to help them afford treatments.
  • In just two months, we awarded grants of up to $5,000 to more than 20 families.

No family should ever have to wonder whether they can afford to save their child’s life. By giving to the Pediatric Assistance Fund, you will help us remain a lifeline for families so they can afford life-saving treatment for their sick children.

Your gift will help ensure that families with children who have chronic conditions – including leukemia, cystic fibrosis, or juvenile diabetes – are not forced to choose between paying the bills and affording quality care. Here’s a breakdown of how donations, large and small, will have a tangible impact:

  • $10 covers the cost of diabetes test strips for Michael for one month.
  • $45 supports Yessenia’s pituitary hormone treatment for 1 week.
  • $75 covers Quinnlian’s lesion treatment for immunosuppressive disease for one month.

Start the holidays off right by donating to HealthWell for #GivingTuesday and make life a little easier for more families like Ethan’s. Your gift will make the season brighter for children and families struggling to afford the medications or treatments they need.

Categories: Cost-Savings