Sequestration as government malpractice
Breakthrough medicines that could save lives may not reach patients as fast as possible, in part because FDA funding has been cut by $209 million — or more than 5 percent. That figure includes $85 million in user fees already paid by industry, but frozen by Congress.
Those affected are people who have run out of options for other treatments. There is a process to expedite FDA approval for breakthrough drugs. But because of sequester cuts, the FDA won’t have the staff resources to get the lifesaving drugs to patients.
This is government malpractice — every bit as deadly as the medical kind. Here’s why.
Last year, Congress passed a law creating a new FDA designation for “breakthrough” drugs to treat life-threatening diseases where “preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies.”
The designation triggers direct participation by high-level FDA officials in helping devise creative research plans to expedite review and possible approval. Because these are by definition revolutionary drugs, the FDA and drugmakers meet more frequently and work together more closely to speed the benefits to patients.
But this kind of engagement — directed by Congress — requires increased FDA resources at a time when the sequester is taking money away from an already underfunded agency.
Who loses? Thousands of people every year. People of all ages who have run out of options to treat terrible diseases like mantle cell lymphoma, chronic lymphocytic leukemia and multiple myeloma. Men, women and children who wait for a drug on the verge of approval that could allow them to live months or years longer.
For example, Duchenne muscular dystrophy is a form of muscular dystrophy affecting around one in 3,600 boys. This terrible disease attacks the muscles beginning around age six. Deterioration follows quickly, often leaving these boys wheelchair-bound before they are teenagers. Their average life expectancy is only 25. There is no cure.
Right now a promising new treatment for DMD is in development. The drug, drisapersen, has received a “breakthrough” designation, and researchers are hopeful it will prove effective.
It is not certain that drisapersen will, or even should, get final FDA approval. But it is certain that the benefits of the breakthrough designation will not be realized if the FDA doesn’t have the resources to do the work. We need to give this, and all of the other potential breakthroughs, the attention they need.
Patient groups, drug companies and the FDA are all worried about the impact of slowing down breakthrough work — especially since there are currently 24 drugs designated as breakthrough. The FDA has already frozen hiring for hundreds of scientists it needs to assure safe and effective treatments are making their way to patients. Due to sequester cuts, FDA reviewers often can’t even attend major research conferences where data on drugs they will review are presented.
Making matters worse, sequestration has meant reduced spending on basic research at the National Institutes of Health (NIH) and institutions all across the country. Less spending by NIH leads to fewer lifesaving drugs in the pipeline at the FDA.
In the end, it’s all quite personal for those of us on the frontlines of the fight against disease. We see people we know whose lives go on because of new treatments. But we also watch people die for want of an effective treatment. The sequester is stalling medicines that could save their lives.
Government malpractice in the form of sequestration is a problem of our own making, and Washington can fix it. We must not pay our debts with the lives of our mothers, fathers, sons and daughters. Budgets for the FDA and for basic research that leads to breakthrough medicines must be restored.
PHOTO: Various medicine pills in their original packaging in Ljubljana, Feb. 14, 2012. REUTERS/Srdjan Zivulovic