Few industries evoke an emotional response greater than the pharmaceutical industry. This cannot be surprising, since drug companies are typically viewed by the public as either providing miraculous life-altering therapies, or greedy instruments of Satan. There is little middle ground.
So, it is rather surprising that a new law, called the 21st Century Cures Act, has been overwhelmingly approved by the House, 344-77 on July 10th.
The new law, also known as H.R. 6, gives more latitude to the FDA in tailoring its safety and efficacy requirements for the approval of breakthrough medicines. Such drugs (as well as breakthrough medical devices) that are given this designation will now be able to be approved much earlier than at present, where the entire process from discovery to market usually takes between 10 and 15 years.
One way that this will be accomplished makes use of the burgeoning field of personalized medicine. For example, groups of patients that have a disease arising from a particular genetic condition will be preselected.
Dr. Josh Bloom, Director of Chemical and Pharmaceutical Science at the American Council on Science and Health says, This one is a no brainer. More and more drugs are being used to target diseases that arise from specific genetic mutations. A group of 100 patients in which only 10 have the targeted mutation will very likely give you the same information as a group of 10 people who have been pre-screened and are known to to have it. This speeds up the trial and saves money, since fewer patients need to be recruited. This law recognizes that because of new technology, one size no longer fits all. Expensive and time-consuming clinical trials that have been required by the FDA have their place, but for rare (orphan) diseases or those that have no useful therapies, exceptions are necessary. The extra flexibility given to the FDA by this law is common sense.
This sentiment is mirrored by House Energy and Commerce Committee chairman Fred Upton (R-Mich.): "This bill is about making sure our laws, regulations, and resources keep pace with scientific advances."
Of course, there were dissenters: Diana Zuckerman, president of the National Center for Health Research said, "We share Congress desire to increase funding for NIH, but there are dangerous parts of this bill that many members of Congress did not fully understand. As often happens, well-funded pharma lobbying was more effective than experts concerns about patient safety."
To which Dr. Bloom commented, Given some of the game-changing advances that have occurred in recent years, the trite profits over people dogma seems especially stale. For Zuckerman to imply that this law is nothing more than an excuse to get unsafe pills down people s throats reveals a fundamental ignorance of how drug discovery works, as well as a bias against an industry that has saved many lives by doing what it does best.
And, The Washington Post revealed a similar bias: A study published earlier this year in Health Affairs traced the origins of transformative drugs approved between 1984 and 2009 found that federal funding of basic research played an essential role in many of them; meanwhile, the fewest number of drugs originated completely within companies. Federal funding of basic research is seen as the surest path to new cures, although it takes years longer than anyone would like.
To which Dr. Bloom replies, Very cute language. The misuse of the word originated is deceptive and misleading. While a new assay or scientific discovery may be the basis of a drug that will appear in the pharmacy 20 years later, what really counts is who finished it, not who started it. The finish is where 99 percent of the effort and expense lies. This is where drug companies, not the NIH or universities, do the heavy lifting, and do so against formidable odds. This kind of statement continues to perpetuate the myth that drugs either drop out of the sky or come from a Petri dish in Bethesda. Fortunately, the House seems to recognize that this is just plain wrong.
