Drugs don’t grow on trees

A question to be answered...
Where, exactly, does a new drug come from?
When I started blogging about drug discovery, I
had already been in the pharmaceutical industry
for 12 years, so I thought I had a reasonably
good idea of how to answer that question. But
putting my name out in public exposed me to a
wider spectrum of opinions, some of them worth
hearing and some of them almost entirely wrong.

Discovering new drugs isn't as easy as picking

apples off a tree © Shutterstock

‘A large number of

people are convinced

that virtually all new

drugs originate in

academic labs’

Drugs don’t grow on

trees

Where, exactly, does a new drug come from?

When I started blogging about drug discovery, I

had already been in the pharmaceutical industry

for 12 years, so I thought I had a reasonably

good idea of how to answer that question. But

putting my name out in public exposed me to a

wider spectrum of opinions, some of them worth

hearing and some of them almost entirely wrong.

In that last category are (apparently) a large

number of people who are convinced that virtually

all new drugs originate from academic labs. No,

not just some of the ideas for drug targets, or

some of the mechanistic details of the

biochemistry: the compounds themselves. These,

I am not-very-reliably informed, are snatched up

for insultingly small sums by the big drug

companies, who then spend a few weeks deciding

on a brand name and colour scheme, then

introduce it to the market and rake in billions of

dollars. I’m parodying this point of view, but

unfortunately not by much. Especially in the US,

there are large numbers of people who are

convinced that all drugs come from universities

and/or the National Institutes of Health.

The truth is, of course, that a lot of valuable work

does come from such sources. But not very many

drugs themselves. Somewhere around 70% to

85% of all new drugs are, in fact, discovered and

developed by the biopharma industry – the exact

count depends on what years you examine, and

how you apportion credit in cases of

collaboration. And even when a compound that

makes it to the pharmacy shelves is one that was

first made in a university or non-profit lab, the

compound development work is done by industry.

And just to be clear, that ‘development’ involves

more than deciding on the colour of the tablet.

Even a compound dropped directly into a drug

company’s lap has to be taken through toxicity

testing in multiple animal species; evaluated in a

range of formulations for blood absorption,

stability, and so on; taken through a gauntlet of

extremely expensive human clinical trials; and

other such tasks. No university has the facilities

or expertise to do these things on their own (nor

should they, many would say), so any drug that’s

marked down as coming from academia will still

have plenty of industrial work behind it.

But any drug coming from industry will, likewise,

have plenty of academic work behind it, in a

broader sense. Much of our knowledge of cell

pathways and human biochemistry derives,

eventually, from just such academic research.

Industry contributes to this as well, but industry

also contributes a great number of drugs and

drug candidates that are used as tools in

academic labs to work out still more unknown

details. We actually play to each other’s

strengths, most of the time. But in the end,

discovering that pathway X is relevant in disease

Y is not the same as discovering a drug to treat

disease Y.

No doubt my perspective has been altered by

years of doing just this sort of work. I don’t find

any of this information to be surprising, and I

don’t see why anyone else should, either. But you

should hear people argue the contrary! The recent

hearing in the US House of Representatives on

drug pricing is an example. This featured a most

uninformative appearance by Martin Shkreli, who

revels in his recent fame as a hard-nosed price-

raising pharma executive. That alone should have

made watching the whole process difficult

enough. But the questions from members of

Congress made it clear that many of them had no

idea whatsoever about how drug research actually

works, or even the difference between a

prescription drug and a generic one.

It was a depressing spectacle for anyone with any

actual knowledge of what the committee was

supposed to be talking about. It probably did

provide plenty of campaign-commercial material

for the presidential election, though, which may

be more to the point. To try to cheer myself up, I

reminded myself that at least I make a living as a

scientist and not a politician. It worked.

Derek Lowe is a medicinal chemist working on

preclinical drug discovery in the US