By Jed Weiner
\niBIO News Brief Contributor<\/p>\n
One of iBIO\u2019s 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\u2019s Drug and Therapeutic Advisory Board (DTA) boards. <\/p>\n
iBIO believes that the state\u2019s 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\u2019s life. Case in point: four-year-old Lily Parlier of Galesburg, 45 miles northwest of Peoria.<\/p>\n
During the first year and a half of her young life, Lily\u2019s parents and her doctors recognized that she was dealing with a serious condition. The cause of her problems was the puzzle. \u201cThe doctors said Lily had \u2018low muscle tone\u2019 and that some kids \u2018just get it.\u2019 They\u2019d say that she\u2019s just little and eventually she\u2019ll catch up,\u201d recalls Lily\u2019s mom Tracy.<\/p>\n
It wasn\u2019t 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).<\/p>\n
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\u2019s condition.<\/p>\n
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. \u201cShe had more energy, she was overcoming a weak swallow and was trying to pull herself up onto the couch,\u201d Tracy said. \u201cWe 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\u2019t think she would have reached this point without the drug.\u201d<\/p>\n
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. \u201cI was surprised by the support I received,\u201d Tracy said. \u201cI 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.\u201d<\/p>\n
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\u2019s treatments, Biogen is committed to paying for the therapy. The company also has a \u201cfamily access manager\u201d that ensures patients can receive life-saving Spinraza.<\/p>\n
iBIO\u2019s 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\u2019 financial assistance programs.<\/p>\n
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,\u201d said John Conrad, president & ceo of iBIO.<\/p>\n
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. \u201cHer progress is giving us
\ngreat hope that she will be able to use her muscles better as she gets older and have a sense of independence,\u201d Tracy said. \u201cLily feels she can do anything! We are going to keep working with her to help all of our dreams come true.\u201d<\/p>\n