Here’s some Inauguration Day reading for you:
For half a century, the FDA has regulated drugs on the premise that science should show that they’re safe and effective before drug companies get to sell them. Before modern drug laws, companies filled the market with ineffective products, backed by no evidence that they worked.
As I write this week in Pacific Standard, a couple of candidates for Trump’s FDA seem to think that we should go back to that era before modern drug laws. Jim O’Neill, a venture capitalist who invests in biotech argues that as long as companies can show a drug is safe, the FDA should let patients take it “at their own risk,” regardless of whether that drug is useless.
And biomedical engineer/biotech executive Balaji Srinivasan thinks that, rather than testing drugs with clinical trials, people should just rate them the way they rate their Uber drivers. Given that people believe all sorts of insane things about what makes them healthier, this is not likely to be a way to rigorously learn whether a drug actually does something for the patients who buy – and whether its not just the drug company ripping people off.
In my piece, I explain exactly why these ideas would be bad for you. But the overarching theme is this: the problem with the two candidates, and their associate Peter Thiel (who is advising Trump on the FDA) is that they see drugs and biotech from the view of investors and startup executives. These are people who hear promising, brilliant medical ideas all the time from scientists and entrepreneurs, people who want to take fledgling ideas and turn them into therapies. That’s great, but in the end, most of these promising, brilliant ideas will in fact be wrong – and that’s why we need the FDA to protect us by weeding out the failures.
It’s going unnoticed amidst the news of the rolling disaster that is the incoming Trump administration, but our lame duck Congress has just passed a major piece of legislation called the 21st century cures act. Scientists are happy about the extra $5 billion this bill gives to the NIH – sort of. That money has to go to specific programs, like the Precision Medicine Initiative and Biden’s Moonshot program, rather than being put into the general funds of the NIH, meaning that Congress, and not the NIH, is deciding what specific research to fund. That’s generally not a good idea, but more money toward broad research and translational initiatives like cancer and precision medicine is still a net win.
More controversial are the FDA provisions of this bill. The bill pushes the FDA to take into account other, often less rigorous types of clinical studies when it decides whether or not to approve a new drug. Some worry that this means drug companies will have more leeway to push unsafe or ineffective drugs on the market. I’m more ambivalent – there are cases (drugs for rare diseases) when double blind randomized clinical trials may not be right, and the FDA should have the flexibility to demand the best evidence appropriate to each case. If – and this is a big if as we look ahead – we trust that the FDA can stand up to industry pressure, than giving them more flexibility to follow best scientific practices is the way to go.
My bigger problem with the FDA provisions are that the premise is flawed. As I write in Pacific Standard this week, the bill’s sponsors argue that, by cutting regulations and red tape at the FDA, we’ll free new cures that are just waiting to be put into the hands of patients. That’s wrong – the FDA is not the rate limiting step here. There is no backlog of effective new drugs just waiting to be approved.
Go check out my piece for the details. The rate limiting step is the science. Medical science is hard, and diseases are understood imperfectly. If you want more effective drugs faster, we need to invest more in research.
The FDA has announced a voluntary recall of the “sexual health” (ie, erectile dysfunction) dietary supplement ACTRA-Sx 500 due to “adulteration” with the active compound from Viagra, sildenafil citrate. This is yet another recall of a “dietary supplement” that happens to be adulterated with a pharmaceutical that has been proven to treat the condition the supplement claims to address. This might make the supplement “work”, but it poses a real hazard to customers who are not permitted to know what they are taking. Continue reading
Let’s say you have diabetes or heart disease and are taking nitrates for your condition. And, unfortunately, maybe you need a little more lead in your pencil, which is even more common with some of these conditions. Well your doctor might hesitate to prescribe the most common pharmaceuticals for erectile dysfunction, like tadalafil (Cialis) or sildenafil (Viagra), because these drugs taken in combination with nitrates can cause dangerously low blood pressure. So, in your desperation, you might think about reaching for one of those sexual enhancement supplements about which we all get countless emails. Continue reading