Family Races Against Ticking Clock To Get Coverage Approval For $2.1M Gene Therapy
A pricey treatment offered hope to a family with a daughter with a rare defective gene that causes spinal muscular atrophy. The therapy must be administered before the age of 2, but the family is locked in a fight with its insurance company over coverage. In other pharmaceutical news: the use of PrEP in the fight against AIDS, Massachusetts' governor's drug plan, clinical trial data, and more.
The Washington Post:
‘There’s A Lot Of Screaming Into The Void’: Toddler’s Parents Battle For Coverage Of $2.1 Million Gene Therapy
When a $2.1 million gene therapy offering the chance of a cure for her daughter’s rare disease won government approval in May, Lauren Sullivan was struck by a sense of “dangerous hope.’’ But after UnitedHealthcare said it would not cover the treatment, Sullivan’s hope has given way to an anxious race against the calendar. The Food and Drug Administration said the new drug, which works by replacing the defective gene that causes Daryn’s spinal muscular atrophy with a good one, must be administered by age 2. Daryn’s second birthday arrives in early October. (Rowland, 7/10)
The New York Times:
This Drug, Underused In The U.S., May Help Make H.I.V. Very Rare In Australia
It took universal health care, political will and a health campaign designed to terrify the public, but nearly four decades into the H.I.V. crisis, Australian researchers say the country is on a path toward making transmissions of the virus vanishingly rare. The fight is not yet won, the experts caution, and the last stretch of disease eradication efforts is often the toughest. (Albeck-Ripka, 7/10)
Boston Globe:
Charlie Baker’s Plan To Curb Drug Prices Strains His Relationship With Biotechs
A proposal by Governor Charlie Baker to curb drug prices paid by the state Medicaid program has biotech leaders up in arms. As state lawmakers meet behind closed doors to hash out a final version of the plan, they are also grappling with this question: How does Massachusetts hold accountable an industry that it helped to build? (McCluskey, 7/10)
Stat:
Pharma Still Gets Poor Grades On Sharing Clinical Trial Data
Amid ongoing concerns that clinical trial data is too often kept under wraps, an analysis found that most large drug makers continue to do a poor job of sharing study data after new medicines were approved, adding further pressure on the pharmaceutical industry to improve disclosure practices. Specifically, just three of a dozen companies shared data within a reasonable time frame, according to the analysis published in BMJ. (Silverman, 7/10)
Stat:
Every Data Point Has A Face: What Michael Becker Taught Us
Michael Becker was a biotech executive before he was a patient advocate. When he discovered he had head and neck cancer, he decided to go public, and in doing so made an impact — both within the drug industry and beyond. Cancer, Becker once said, takes away a lot more than it gives. But he wanted his own experience to show people the risks of the human papillomavirus, which caused his disease, and the importance that preteens get the vaccine that prevents HPV infection. (Herper, 7/10)
