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Drug companies need little arm-twisting when it comes to investing their resources into diseases that afflict millions. But what about a disease that affects only a few thousand people?

To stimulate drug development for diseases with few paying customers – whether due to its sheer rarity or its sufferers’ poverty – the National Institutes of Health (NIH) has committed $120 million over five years to a new program called Therapeutics for Rare and Neglected Disease (TRND). The new trans-NIH program aims to support researchers with more money for in-house NIH studies and additional funds for external academic, advocacy and foundation collaborations.

“The federal government may be the only institution that can take the financial risks needed to jumpstart the development of treatments for these diseases,” NIH Acting Director Raynard S. Kington said in a statement.

This is not the first time the government has addressed so-called “orphan diseases.” For more than 25 years, the Orphan Drug Act has given developers of therapies for diseases afflicting less than 200,000 Americans some market exclusivity and a tax credit equal to 50% of all clinical trial expenditures.

But success has been limited. Part of the problem are steep preclinical research costs that often outweigh these incentives: An average of $10 million and 2 to 4 years spent in the preclinical phase with the vast majority of concoctions never reaching human trials, according to the NIH statement. This has led many to describe the gap between theory and application as the “valley of death.”

So will this strategy prove any more effective than Congress’ previous efforts? How far can $24 million really go to reduce the impact of rare diseases, which collectively afflict more than 25 million Americans? To get some perspective, we checked in with Iain M. Cockburn, a professor at Boston University’s School of Management who studies the drug industry.

“To bring a drug to market takes $1 billion or more,” says Cockburn, adding the global R&D budget for biopharmaceuticals is about $100 billion a year. “Against that backdrop, $24 million is rounding error.”

But this drop in the bucket, he notes, may have a relatively significant impact given just how empty the bucket is. Cockburn believes it may be enough to push some “potentially interesting molecules” through the preclinical pipeline and spark other public and private sources to put down the “really big bucks.” 

Stacie Propst, vice president of science policy and outreach at the non-profit health advocacy group, Research!America, in Alexandria, Virginia agrees. “We see this as a down-payment,” she says. “The $24 million is meant as seed money to spark investment by others.”

image by wolv via iStockphoto

5 Comments

1. 1. dbtinc 08:05 AM 5/23/09

   Simple economics at work - you got a rare disease, well it's too bad for you. With drug regulations being what they are coupled with medical liability there is no solution. Sorry.

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2. 2. solstice 03:48 AM 5/24/09

   glad to see that are gov is spending money on such things during a severe economic downturn. that's exactly what we need, more bloated research spending to keep the professors fully employed.

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3. 3. jaeteekae 08:05 PM 5/24/09

   Maybe a good publicity stunt but of dubious value. Money better spent eliminating drugs that people have been conned into buying for imagined ailments, such as RLD (restless leg syndrome) and trying to educate the consumer (I know its a hopless task, but they could try). Some surveys have indicated over 75% of health problems are self-inflicted (lifestyle) and could easily have been prevented by the patient.

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4. 4. j.quasimodo 10:43 AM 5/25/09

   Even if you take corporate profit out of the calculation, the societal issue is still "bang for the buck". We do not have unlimited resources so it's hard to justify giving any useful priority to the rare diseases when more common ones are still at large. Maybe it makes sense to allow a cheaper development path for rare fatal diseases that transfers part of the risk from the developer to the patient and/or the public.
   
   The place where the priorities are made more murky by profit is the me-too and lifestyle drugs. I don't think you can prohibit that. .
   

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5. 5. Sunfyre7896 02:22 PM 2/18/11

   The only way to find cures for diseases is to research and develop cures; and the only way to research them is through funding. How does this funding happen? Either from subsidies funded from the government or through private donations. These private donations can add up but are severely lacking. We personally began a non-profit company that is dedicated to helping researchers find cures for some of the worst diseases through the proceeds from the sale of our candles. The purchaser gets to choose which disease research their proceeds to toward. We can all do our part, but we must believe that the research will provide the answer. I believe that they will and not just "keep professors fully employed."
   Our company is Candlelight Foundation Inc. and our blog is Candles for the Cure.
   http://www.ccf1.org
   http://www.candlesforthecure.blogspot.com

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