The non-profit Tomorrow's Cures Today Foundation aims to open patient access to innovative drugs and treatments in advance of Food and Drug Administration approval. The group has launched an online Change.org "Free to Choose Medicine" petition to Congress urging lawmakers to create a new drug development and testing system—one that would allow patients to try not-yet-approved drugs in a more open and transparent process. Approvals could even be speeded up based on the what's learned from treatment outcomes for patients who choose to use not-yet-approved drugs.

The Foundation outlines three principles for Free To Choose Medicine:

1. Some patients and their doctors only want medicines tested and approved in the traditional way, and for them nothing would change. But others are not just willing, but eager to try not-yet-approved drugs. They should not be denied the right to choose a viable medical treatment. So, after safety testing is completed, patients should be free to choose a not-yet-approved drug if its developer is willing to make that drug available. After all, patients, under care of their physicians, and with access to detailed reports describing all prior test results, are in a better position than government bureaucrats to decide whether the risks are worth bearing. 2. To ensure patients are fully informed, developers offering not-yet-approved drugs on the Free To Choose Medicine track would have to make all prior testing data available in a publicly accessible online database. And every patient taking a Free To Choose drug would have to agree to let his or her doctor submit reports on an anonymous basis about their progress and any side effects. This "observational data" collected from real world use would provide valuable information to patients and let them make informed and meaningful choices.

3. As more and more patients use Free To Choose drugs, the value of information available to other patients will grow. Drugs that show severe side effects or little therapeutic benefit will be abandoned, although the data generated will be useful to drug developers. But drugs with positive experience will become more popular and the amount of observational data will grow even more. Technological advances in collecting and analyzing health data will make it possible for doctors and researchers to see who is benefiting the most from individual drugs and who is at higher risk. This "big data" collection will be superior to what's collected in clinical trials, where researchers try to limit enrollment to patients who are all alike. With enough patients, the FDA could even approve new drugs on the basis of observational data alone.

In my article, "Building a 21st Century FDA," I endorsed Manhattan Institute senior fellow Avik Roy's proposal…

…to permit drug companies whose new medicines have passed Phase I and Phase II trials to market them conditionally to physicians and patients. "Under conditional approval, patients in most need can benefit from a new drug, and companies can generate a modest amount of revenue that can help fund Phase III trials for full approval," argues Roy. … Given the rapid evolution of bioinformatics, it might one day be possible to roll out most new pharmaceuticals after the equivalent of Phase II trials. Since medical and prescribing records will be online, drug researchers could compare people who choose take conditionally approved new drugs with a population that does not, matched for various confounders such as age, other medical conditions, behaviors, etc. Essentially, the health care system itself might become a gigantic Phase III trial for new treatments.

Old-fashioned regulations are stalling progress and killing people.

Disclosure: I just signed the petition and made a modest donation to the Tomorrow's Cures Today Foundation.