FDA Highlights It’s A Road Block To Drug Development

Posted by Jason | Posted in Government, Health Care | Posted on 10-03-2010

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If you’ve been following my blog, you already know I am no fan of the FDA. This bureaucracy costs drug developers so much time and money that many drugs that could save lives are never invested in and developed. I’ve talked about that here and here. This morning, the Wall Street Journal has an article about FDA staffers “helping” companies move their orphaned drugs through the process by holding workshops. The underlying story, if you read between the lines, is many drugs never make it to the market because the FDA is a road barrier that is not worth overcoming for rare diseases. The end result is people with rare diseases die or have to live with their disease with a lot less hope that a drug will be developed, all thanks to your all caring government.

Staff members at the Food and Drug Administration are doing something unusual. They are leaving Washington to help drug makers take a crucial step in developing drugs for rare diseases.

Only a statist would consider it a crucial step in the development of a drug to fill out government paperwork.

The staffers help administer the Orphan Drug Act, which provides incentives to create therapies for so-called orphan diseases—those that affect fewer than 200,000 Americans. There are about 7,000 such maladies, most of them serious, that have few or no drugs to treat them, from adenoid cystic carcinoma, a rare head and neck cancer, to Zollinger-Ellison syndrome, which is associated with a tumor that causes the production of high levels of stomach acid.

As a result, doctors may end up prescribing drugs developed for other diseases off-label, but not all insurers will cover this kind of use.

Getting an orphan-drug designation opens the door to incentives once the FDA approves a medicine for sale in the U.S., including seven years’ marketing exclusivity and tax breaks. Last year, just 250 requests for orphan-drug designation were filed, and 160 received it.

“We’re barely scratching the surface,” says Timothy Coté, director of the FDA’s Office of Orphan Products Development, the workshop’s sponsor. He says there are roughly 350 orphan drugs approved, covering about 150 rare diseases.

Tim Cunniff, vice president of global regulatory affairs at Lundbeck Inc., which has a number of approved orphan drugs, says most companies developing orphan drugs are small.

As I’ve stated previously, the big companies love the FDA. It’s a barrier to small companies from developing drugs to bring to market, which means less competition for Big Pharma. Small companies would develop drugs for rare diseases, because they see the whole in the market. The problem for them though is trying to find $1 billion dollars and up to 15 years time to move the drug through the FDA. This is a huge barrier to entry, and it leaves people with these rare diseases to suffer.

Big companies are starting to get more interested in rare diseases, but the key issue is the high cost of developing a drug and the typically long time it takes to move it from a lab into a clinic as a treatment that gets prescribed. Before starting down this arduous path, a company needs to feel there is a reasonable chance of making a profit.

Bing-O. Here is the proof that the FDA is a hindrance to progress. Companies have to make predictions about an extremely expensive and time consuming process, that can be swayed by politics. As we all know, everything has opportunity costs. If they decide to move forward on one drug, the money and time spent moving it through the FDA process is money and time that are not spent developing the next drug. Because of this alone, we are losing out on more drugs, but we are also making it much harder for companies to decide in favor of moving forward with a drug. It’s probably almost never going to happen for drugs for rare diseases, because the market is so small.

To help get more applications, Dr. Coté’s office put out the word: Help is available, in two workshops with on-the-spot regulatory advice. The first workshop, held last month at the Keck Graduate Institute here, drew 29 potential sponsors, from major drug companies to academic centers, small biotechs and even some patient advocates. In a follow-up survey, 74% said they had never before filed an application for orphan drug designation.

Dr. Coté said he wanted participants to understand that the workshop wasn’t providing an alternative pathway to orphan-drug designation, just regulatory advice. He said it was very important that the FDA avoid the “perception of favoritism” and even stressed that in the cover letter to an application, the sponsors shouldn’t say they had been at the workshop.

Sure wouldn’t want the “perception of favoritism”. Don’t make it easier for companies to develop drugs for rare diseases, that otherwise won’t be developed. That would be just horrible.

Barbara Fant, president and chief executive of Clinical Research Consultants Inc., attended the workshop to prepare an application for a drug-company client, and said this was her first time filing for an orphan drug designation.

An FDA staffer pointed out issues in her application that “would have come back to me as questions and delayed the designation process” if she had filed before the workshop. “I learned some nuances that I didn’t know,” said Dr. Fant, who declined to provide details about her client or the drug.

Who says you don’t have to be a rocket scientist to fill out government papers correctly. I guess being a doctor just isn’t enough. If this lady was not at this workshop, should would have probably just lost months of time before this paper went into the FDA bureaucracy and got turned down and returned, because it was missing some “nuance”.

An orphan-drug designation is no guarantee a medicine will ultimately be approved for marketing. A different FDA division reviews safety and efficacy data for approval. Upon further testing, a drug may turn out to be too dangerous or not effective. Companies may decide a product is too expensive to make, change direction, or go out of business. But Dr. Phillips and Dr. Coté hope that by increasing the pool of applicants for designation, they will increase the chances of getting more approvals.

via FDA Pushes for Cures for Rare Diseases – WSJ.com.

Did you get the underlying message of this article? Do you need more proof that the government, in this case the FDA, is a road barrier to progress? There really is no need for the FDA. There could easily be a private mechanism for testing drugs. All it would take a is change to patent laws, which have to give Big Pharma complete control of the market for a long time in order for them to make enough profit to make it worth bringing a drug to market. Because of the huge expense of the FDA, that control of the market has to be longer than it otherwise would need to be.

Also, as John pointed out in the comments section on this post, why couldn’t the FDA be optional? Why can’t we as consumers have a choice between an expensive FDA approved drug and a cheaper non-FDA approved drug? Surely, the non-FDA approved drug will go through the rigors of private testing via competitors, doctors and universities. We know the answer to that though. Government can never have something that challenges it’s claim to be our protector. If we allowed non-FDA options, the FDA would shortly be proven useless.

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Comments (2)

Oh lordy…. yes I’m sure it would out great to have a “private” mechanism for testing drugs.

What you’re proposing would be an absolute disaster.

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Why would it be a disaster? Surely a competitor would have reason to test the claims the drug maker is claiming. Also, doctors, who would be prescribing the drugs, would have an interest in testing the claims. I guess we should trust the government, right? Politicians are so honest. They are not susceptible to corruption or anything, just looking out for the people.

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