| It's bad to analyze this issue through the medium of a long-form article, which wraps the story up in emotional narrative. Here is a better write-up in the MIT Technology review: https://www.technologyreview.com/s/601165/the-worlds-most-ex.... The gist of the problem is this: > The Amsterdam company spent more than $100 million testing the drug and carving a path through Europe’s medical rules and regulations, which weren’t geared to consider a new technology like gene therapy. Initially, for instance, regulators said they expected a clinical trial of 342 patients. Executives wryly noted that there were only 250 people with the disease in all of Europe. > Nor has Glybera convinced the national regulators in Europe who decide what drugs get reimbursed. Last year, French authorities said they would not pay for the drug. Germany judged Glybera’s benefits “non-quantifiable.” It leaves doctors and insurers to make decisions on a case-by-case basis. Here's a drug that cost $100 million to make, and will require even more money to move through clinical trials in the U.S. And the pool of potential patients is a less than 1,000 people, and oh many of then can't pay either because they lack insurance (U.S.) or the cost/benefit panel math doesn't work out (Europe). Viewing this after-the-fact leads one astray, into thinking there is some problem with the system that leaves this potential cure out of the hands of patients. Patents, money, it's all just a proxy for effort in, benefit out. Even if you had the government make this drug, you're talking huge amounts of money per patient, which few governments would pony up. (And rightfully so, because you could save a lot more lives with that amount of tax dollars.) The answer is probably that this drug doesn't make economic sense, and the abandonment of it by the maker is just an indication that it was a misguided effort to begin with. |
In a world where you could just put a drug on the market (where "the market" is, say, hospitals), the drug would have cost far less, and so the inventor would need to recoup far less.
Note that I'm not arguing for complete deregulation of drug manufacturing. Testing for product integrity through factory audits and the like is the original—and still core—purpose of bodies like the FDA and its EU equivalent(s). You want to know that your "ibuprofen, 20mg" indeed contains 20mg of ibuprofen in each pill, and nothing else.
But that's cheap! That doesn't cost a hundred million dollars to do. Proving product integrity is something that any food processing plant manages on the regular, not to mention manufacturers of generic/non-patented drugs.
If we stop with the need to prove—with overwhelming statistical power—the safety-and-efficacy of a molecule that we're planning to dispense to fewer than 1000 people (and even then only through doctors who can carefully monitor them for adverse reactions), the pharma industry would go back to being just another quiet industry that makes stuff, instead of the crazy behemoth it is.
Yes, certainly, there should be a stringent process before you stick something on a store shelf for OTC purchase. But most drugs never seek to be OTC anyway. Prescription drugs require an interaction with a prescribing doctor, a dispensing pharmacist, and possibly (in in-patient settings) a bunch of watchful nurses. Why, also, must they require an interaction with a standards body regulating the molecule they're composed of? The patients who were in the clinical trial itself got effectively the same treatment, minus the standards body, and they were all fine.
Maybe that's what I'm saying: why not just allow any random chemical to be given to people (without intention of standards-body registration) as long as the prescribing doctor is willing to do clinical-trial-like monitoring for adverse side-effects?