The less FDA the better to my eyes. The incentives are broken, and the FDA is greatly slowing progress at a time when the potential to create new medicine is greater than ever before. Regulation is reason why we don't have the first fruits of the biotechnology revolution in the clinic today.

The much-touted ballpark estimate of a billion dollars to produce a modern pharmaceutical from start to finish is about a decade old now - that figure is adjusted for today's diminished dollar value, eroded by inflation. You can replace "pharmaceutical" with any medical technology that is going to require a fair amount of original research and further tinkering in the laboratory to obtain the first working prototypes and the result is much the same. This large round number is the kitchen sink cost across a decade of work, including failed attempts and the opportunity cost of investment. The lion's share of the direct expenditures are imposed by regulatory requirements: trials, data, and more trials.

It has long been my position that almost all of the work carried out in the US at the behest of the FDA to prove safety is unnecessary. In fact it is counter-productive, as the immense imposed costs on development shut out a great deal of the experimentation and small-scale initiatives needed for rapid progress. Much of what the FDA demands is not demanded by similar regulatory bodies in other parts of the world, and even their requirements are very onerous in comparison to the standards in place fifty years past, a time when medical development seems to me to have worked just fine. It is a question of balances and choice: it is better to err in favor of faster progress and informed patient choice, but that is very far from the present state of affairs.

The result of an excessive and growing regulatory burden is that many potential medical technologies languish, are rejected outright, or are never developed at all, and patients suffer as a result. Our future health is determined by the pace of progress, and a slowdown across the board harms all of us considerably. Unfortunately that cost is invisible to the public at large and thus bureaucrats suffer very little as a result of the harms they cause by blocking progress. Meanwhile even comparatively small harms caused by an approved treatment that turns out to be overwhelmingly beneficial save for some negative effects for some people can snowball in the media to cause great damage to a career in the FDA bureaucracy. So you can see that the incentives are very much aligned with ever greater demands for proof and ever greater costs imposed on medical research and development. This is in fact what has happened over the past few decades, with the present result that in an age of radical progress and plummeting costs in the laboratory it is nonetheless the case that medicine is ever more expensive and the introduction of new applications of medical research has slowed down. This is well known and widely commented on, but so entrenched that this detrimental trend shows no signs of slowing.

So, to return to the cost of developing a modern pharmaceutical product: estimated at a billion dollars (in today's dollars) ten years ago, the same approach results in an estimated $2.5 to $2.8 billion dollars now. Interestingly the bulk of that is apparently not due to the increased time required to run the regulatory gauntlet, but rather largely due to other increases in the demands of regulators: much larger trials and more data.

http://csdd.tufts.edu/news/complete_story/pr_tufts_csdd_2014...

http://lifescivc.com/2014/11/a-billion-here-a-billion-there-...

There is great frustration with this state of affairs, and it manifests with the creation of organizations such as Faster Cures and Tomorrow's Cures Today, none of which seem to be making much difference, sadly. The lobbying makes some people richer, and nothing changes as a result. In the end the only thing that is going to work is regulatory arbitrage - that the development goes to other parts of the world, and therapies are available there until the FDA is shamed into approving them because the people in charge look like clowns if they don't. This is exactly what happened for stem cell therapies; those available in the US today would still be held up in interminable trials and requests for more data without the fact that they have been widely available in overseas clinics for years. The pattern of activity in the FDA has nothing to do with safety and efficacy and everything to do with job security for bureaucrats.