To put it bluntly - duh.
The FDA just approved the first-ever "bionic eye" for patients with retinitis pigmentosa, a rare genetic eye condition in which the retina deteriorates over time. The Argus II Retinal Prosthesis System (Second Sight, the developer, should have hired a better marketing department) comes with a pair of eyeglasses that has a video camera attached. This camera wirelessly connects to an artificial retina implanted into the eye, which bypasses the original, deteriorated retina. Though the Argus II doesn't actually restore sight - hence, not a cure, per se - it allows patients to distinguish between light and dark in the environment, helping them get around.
This is surely a cause for celebration for patients with what is otherwise untreatable blindness, and the researchers who developed the artificial retina hope that in the future, they will be able to use the device to treat all causes of blindness including macular degeneration (affecting some 2 million people in the U.S.).
The device was approved under the FDA's "humanitarian use" pathway, which is used to rush approval for devices that that treat a condition in fewer than 4,000 people per year; the condition must also have no alternative treatment available. And the Argus II certainly qualified - RP is a condition present in less than 1 in every 4,000 Americans.
The clinical study for the device included only 30 patients - most of them were able to perform basic activities much better, and only 11 experienced serious adverse effects. This should be great news for those concerned with the FDA's regulatory burdens, but there's more that can be done. (One bit of news that's not so great, though, is that Europe has had the Argus II for a year already).
There's no reason why a "humanitarian use" pathway should apply only to such the small number of conditions that affect 1 in 4,000 Americans - surely, many patients who suffer from MS, ALS, Alzheimer's and a slew of other chronic conditions would be willing to accept certain risks to potentially treat their conditions (even though treatments exist, they are often ineffective). The humanitarian use pathway should be expanded - if a patient, with consultation from his doctor, of course, decides that the potential benefits of an untested treatment outweigh the risks (communication with pharmaceutical and biotech companies is critical here) - there is no reason why the treatment shouldn't be made available. And the results of the treatment, whether good or bad, should be recorded for use in future clinical trials.
We are at a potential watershed moment for the FDA - the organization has acknowledged that it has a problem with clinical trial designs and limited approval pathways. And to the agency's credit, a few other pathways - the accelerated approval, priority review, and fast track designations have worked very well. More recently, the FDA accepted comments on development of a new pathway geared towards life-saving drugs, with a focus on antibiotic-resistant bacteria. In short, there is progress at the agency. The FDA should take note of the successes of its existing pathways and allow more drugs and devices to receive the appropriate designations, thus expanding the designations to broader populations.