By Jed Weiner
iBIO News Brief Contributor

One of iBIO’s legislative priorities in 2019 is the passage of Illinois House Bill 2259. If signed into law, HB2259 would establish panels of experts to support the needs of the state’s Drug and Therapeutic Advisory Board (DTA) boards.

iBIO believes that the state’s reimbursement system must appropriately and adequately reflect the value of new therapies by ensuring timely and appropriate review by DTA boards; securing adequate reimbursement will facilitate patient access to life-saving and life-enhancing therapies. In many cases, the faster a child can receive safe and effective therapies, the greater the positive impact on a fragile patient’s life. Case in point: four-year-old Lily Parlier of Galesburg, 45 miles northwest of Peoria.

During the first year and a half of her young life, Lily’s parents and her doctors recognized that she was dealing with a serious condition. The cause of her problems was the puzzle. “The doctors said Lily had ‘low muscle tone’ and that some kids ‘just get it.’ They’d say that she’s just little and eventually she’ll catch up,” recalls Lily’s mom Tracy.

It wasn’t until 2016, when Lily was 17-months-old, that a blood test for genetic abnormalities revealed she has a rare and potentially deadly genetic disease called spinal muscular atrophy (SMA).

Like most people with SMA, Lily had very little physical strength, making it nearly impossible for her to walk, swallow, speak and breathe properly. Then, at the age of 23 months, Tracy and her husband, Randy, agreed to try Spinraza, a recently FDA-approved biological therapy for Lily’s condition.

Tracy remembers noticing improvements after the first few doses, and by the fifth dose (approximately six months after starting therapy), Lily was gaining overall strength. “She had more energy, she was overcoming a weak swallow and was trying to pull herself up onto the couch,” Tracy said. “We had been doing sign language because her jaw was to weak for her to speak, but she quickly became more vocal and today her speech is normal for her age. We don’t think she would have reached this point without the drug.”

Tracy and Randy also appreciate that the developer of the drug, Biogen, helped them work through the paperwork required for their insurance company to reimburse the costs of Spinraza. “I was surprised by the support I received,” Tracy said. “I was amazed, actually. I was skeptical that a pharma company would give me personal attention but they provided genuine help. They directed me to information on the internet to help us understand the paperwork that we and our doctor needed to complete.”

Tracy acknowledges that the treatment costs are extremely high, quickly adding that the company demonstrated its support in this area from the very start. She shared that if their insurance company ever denies them coverage for Lily’s treatments, Biogen is committed to paying for the therapy. The company also has a “family access manager” that ensures patients can receive life-saving Spinraza.

iBIO’s members believe that all patients who can benefit from medications and therapies should have access to them. To that point, in 2016 Illinois-based biopharma companies helped more than 400,000 patients receive the medications they need through the companies’ financial assistance programs.

The focus of the life sciences industry is to develop innovative therapies and cures for patients. This means it is imperative not only that we develop these new medicines, but that all patients have access to them, as necessary to meet their healthcare needs,” said John Conrad, president & ceo of iBIO.

According to Tracy, Lily is doing more than she and Randy ever thought would be possible. When Lily was an infant, one doctor said she would never be able to walk, or even stand. “Her progress is giving us
great hope that she will be able to use her muscles better as she gets older and have a sense of independence,” Tracy said. “Lily feels she can do anything! We are going to keep working with her to help all of our dreams come true.”

Follow Lily’s story at Longevity for Lily