A pharmaceutical insider’s take on a National Institute of Pharmaceutics

by Alan Cohen

I have a friend who works as a scientist in the pharmaceutical industry, and he recently emailed me with some thoughts on my post regarding a National Institute of Pharmaceutics. It’s clear that he knows a lot more about what he’s saying than I do, and a really interesting idea emerges at the end. The rest of this post is his email (slightly edited for context), and I respond in a comment:

“I’ve been following your blog and I read your NIP idea with a lot of interest, as someone making a living as a storm trooper for the evil big pharma empire, and as someone that has also been wondering if there is better way to develop and distribute new drugs.

“My biggest concern is how we’ll decide who will get funding. People carefully move a drug candidate from one development stage to the next, especially since it is going to cost a lot more money at the next stage than all the money spent so far combined. Those decisions are made by each company. If we now move this responsibility to the NIP, are we sure that they will do at least as good a job as current pharma CEOs do?

“The success rates of clinical trials are an abysmal 10%, and this partly explains why the pharma industry spends so much money to bring one drug to the market. Obviously the industry is not doing a great job making decisions on which ones to push forward. Many times this is because of our limited understanding of biology and medical science. But also many times, under pressure from upper management and stockholders to fill up the drug pipeline, researchers focus on the bright side of a potential drug and intentionally or unintentionally pay less attention to warning signs found in earlier (cheaper) studies. When the bar needs to be set very high, some would rather choose to move their compounds to next stage and cross their fingers, for various reasons not based on hard science (need to bring in new investors, impress stockholders, meet the company goals, desperate for good media coverage, etc.)

“The NIH funding system has worked, no doubt, but it is not flawless. The need for more funding forces a lot of academic groups to rush for publications, which leads to sloppy science and irreproducible reports even in top journals. An alarming number of ostensibly promising reports cannot be reproduced by industry researchers – lately this has bothered more and more people. Wouldn’t this happen to NIP? Especially since their paychecks are based on getting this funding, industry researchers will do their best to promote whatever they have, while completely ignoring all the red flags inside. And for outsiders it is often not easy to find those warning signs – even managers in the industry, very much insiders, end up making bad decisions, separated from their own scientists by big pharma bureaucracy.

“And spending public money has public relations issues. NIP will have to spend billions of dollars, they will not get to learn the results until at least 5 years pass, and a lot of them will turn out to be big busts. Taxpayers are often less patient than private investors. Nixon’s “war on cancer” made big bold promises which were not fulfilled, and it led to nihilism, and while we can claim that it’s not really their fault, science/medical society lost trust from public.

“At the moment research priorities are set by the free market. While it does not work perfectly, it more or less assigns what industry does with what the society needs. Society does not need another allergy drug or viagra so much – and exactly for that reason, developing another allergy drug or viagra does not make a lot of sense economically. Why would anyone buy a big-pharma-patented very-expensive-brand-new allergy drug, when there are much cheaper over-the-counter allergy drugs available? Most major big pharma, as well as a lot of small biotechs, have oncology programs, since they believe that’s where the money is. The industry has failed so far to bring a lot of promising drugs to the market not because of the lack of interest, but because cancer is very tough to understand and fight. The success rate of cancer drugs in clinical trials is even worse than other drugs – but the industry keeps trying.

“I think there are areas only public funding can take care of. There are disease areas ignored by the pharma industry – diseases serious only in poor underdeveloped countries (malaria), drugs that will not generate a ton of revenue but still are much needed by society (antibiotics), diseases with a very small market (rare diseases), or really tough ones not many dare to challenge (Alzheimer’s). Also, public funding can contribute to bringing something like dichloroacetate to market. DCA is known to have efficacy on cancer in small scale clinical trials, but it is a very common compound and not patentable, and so no pharma companies want to initiate large and expensive clinical trials. Without big clinical trials we will never know if this compound has survival benefits and no severe toxicity, and this is a case where public funding can help.

“I haven’t given as much thought on this as you have, but this is what I have thought – the society needs to share the cost of these new and expensive drugs until the patents expire. This is the same development-cost-shared-by-everyone plan, but the public won’t support every project or won’t pick who will be funded. In the meantime, governmental bodies can start funding the medical research that doesn’t interest the industry much – for example antibiotics. You can fund antibiotic studies in academic labs – this is what has been already going on. Once they come up with something good enough, you can give big money to the NIP and have them initiate clinical trials. Hopefully some drugs will survive at the end – and the program itself will survive wild political swings – and then the public will share the antibiotic. Now we can fight antibiotic-resistant strains like MRSA. If this works, now you can expand to all disease areas as you proposed.

“I am glad you brought this up. I hate worshiping free market or demonizing evil big pharma – those talks don’t get us anywhere. We need more rational conversation on medical issues.”