Sequestration as government malpractice

By Ellen Sigal
July 30, 2013

In regard to the Food and Drug Administration, the sequestration could ultimately cost lives.

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

 


 

 

 

 

4 comments

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There are hundreds, if not thousands, of government programs that provide substantial benefits to tens of millions of American citizens. Of course, the “big, bad government” advocates will fight these forever, even while acknowledging that nearly every one of them individually is of benefit. These are the same people who claim to support the Constitution, but will fight to the death to prevent the institution of “promote the general welfare”.

On top of that, they tend to vote against their own self-interest, supporting the too-big-to-fail Wall Street Casinos with taxpayer funds to cover all of their gambling losses.
It must be great to be a member of the One Percenters Club, the functional arm of the Plutocracy with its very success PR conducted through the Idiocracy.

“Let them [the Pee-Ons] eat cake!”

It’s time to accept that the United States has reverted to the feudal system of the Middle Ages. At the start of this country, one could only be consider a real citizen if one was a landowner. Now, the entry to the Club is determined by foreign bank accounts in the millions that evade, er, avoid taxes. Those corporate jets owned by one-person corporations are expensive to maintain.

Posted by ptiffany | Report as abusive

Hey Ellen Sigal what’s with the crazy pic?

Posted by Foxdrake_360 | Report as abusive

“…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.”

I respectfully disagree. America is BROKE, i.e. spending wayyyy more than it’s incoming tax revenue. What part of UNSUSTAINABLE do you not uinderstand?

Sequestration is NOT a cut in funding. It is a reduction in the rate that government spending is increasing. There will never be an end to “worthy causes” to hire government bureaucrats to administer, and no end to the money “required”.

At some point common sense (remember that?) dictates ENOUGH and it becomes necessary to PRIORITIZE expenditures such that things are funded that give the biggest bang for the fewest bucks. I think Americans can “live with that”.

Posted by OneOfTheSheep | Report as abusive

Honestly, the FDA is a joke. Sequestration has zero effect on the drug industry. They will do what ever they want, when ever they want. The only thing I have observed and experienced the FDA doing is to approve drugs that kill people and prevent medicine that would actually help from being approved or utilized appropriately. And, yes, I know what I speak. I am a psychopharmacologist. Do you want to see my degrees, proof of prescriptive privileges or my DEA number? Our entire health care system is completely corrupted. You would not believe how bad it actually is. It would blow your mind to know the truth.

Posted by 2Borknot2B | Report as abusive