If a pharmaceutical
company wants to sell a drug, it first has to prove that it works and is safe by doing a
double-blind controlled trial. Everything else is quackery.
At least that is
what I used to think, but now I am not so sure any more. A major reason for my new-found uncertainty has been Peter W. Huber's book The Cure in the Code.
Huber is a senior fellow of the Manhattan Institute,
a free market think tank. Whilst his book is political, it raises points that should transcend the political divide. His central aim is to show that the top-down drug licensing process, as it is currently implemented in
the United States and other developed countries, is not in the long-term
interest of patients.
Consider the rare
diseases that we study at the Sanger Institute, where I am a postdoc. In many cases, they are caused
by a single mutation disrupting a single gene. Because the mutation is rare,
not enough people have the disease to make it profitable for anyone to invest
in developing a drug.
Nevertheless,
sometimes doctors are able to repurpose a drug that has originally been
developed for a different disease. For example, if the causal mutation disrupts
a gene in a particular biochemical pathway, the doctor may know of a drug that
upregulates the expression of another gene in the same pathway, therefore
compensating for the mutated gene. That is great when there is a drug that can
be repurposed, which in most cases there is not.
This issue is not limited to rare disorders. Complex diseases such as autism or diabetes have a large genetic component, but rather than a single causal variant there are tens or even hundreds. Each patient has a different combination of variants, and it is therefore unlikely that there will be a single drug that works for everyone. This makes a personalised medicine approach necessary, where the drugs that are prescribed are adjusted based on the patient's genotype. As with rare diseases, this means that patients with less common genotypes may lose out because there are not enough of them to warrant the development of a new drug, and because no drug that has been licensed for another disease is likely to help.
The higher the level
of proof that is required before a drug can be sold, the lower the number of
drugs that pass this threshold will be. Always requiring the highest level of
proof may seem like the safest option, but is not necessarily in the public's interest
if it means that many drugs will ultimately be unavailable. There is
legislation such as the US Orphan drug act and a number of accelerated approval
rules, but they only partly address the problem. There are still plenty of
examples of drugs that may work in a subset of patients, or that may in the
future be repurposed, but that are killed off in the licencing process and are
therefore not available to anyone.
What is to be done?
Creating legislation that allows the use of any compound whatsoever for the
purpose of treating people with rare diseases or rare genetic variants could be
one option. Of course, such a laissez-faire approach to drug licencing would be
controversial and could encourage unscrupulous practices.
Right now, it is unclear to me what the balance between the two extremes of complete permissiveness and complete top-down control of drug licensing should be, but I doubt it is the status quo. I would welcome any views readers of this post may have.