“When you’re doing a study of rare disease, it’s a small sample size and it’s easy to manipulate the data to make it look better than it is,” said. “You don’t want an incentive to represent the company wrongly in the short-term,” to get the voucher for another larger drug.
Julia Jenkins, executive director of the EveryLife Foundation for Rare Diseases, an advocacy group pushing for drug companies to spend more on drug development, wants the pediatric drug voucher program extended. She notes that the program is still too new for officials to evaluate whether it is effective.
One problematic part of the current House version, she said, is that it only extends the program for 3 years, and drug companies generally need 10 years to scratch up investors and research a new drug. The potential reward of expedited drug review might not be enough to allow a company to make a financial plan for a drug based on the program.
The bigger cures bill covers more than 60 health issues, including a $10 billion boost in funding for the National Institutes of Health and $550 million in extra money for the FDA over the next 5 years. Other provisions include creating a database of genomic information from a million U.S. patient volunteers and allowing the FDA to approve drugs without the standard clinical trial, instead using smaller observational studies or clinical experiences.
The bill passed the House Energy and Commerce Committee unanimously in May and is expected to come up for a vote in the full House. Senators are in the early stages of working on a similar bill.
Kaiser Health News (KHN) is a nonprofit national health policy news service.