As a patient working in cancer research for over ten years now, one thing has slowly become more and more apparent.
The greatest patient benefit accrues from studies which are academically led. A
bold statement which needs a bit of unpicking.
Pharmaceutically led studies dominate our attention because of the scale of
investment and the power of the public relations team. They may well be led by
talented academic clinicians but the protocol has been driven by commercial
requirements even if it is an early phase study. The reality is that larger and
larger amounts of money are being committed to studies which benefit fewer
and fewer patients.
I am not decrying all that effort. My personal view is that if a new drug can
benefit just one patient let us have that drug. However I don’t have deep
pockets, any more than any healthcare system does, so that principle has some
weaknesses – as does the current pharmaceutical business model.
Beyond numeric significance
Academically led studies, which draw funding from government, charities or
philanthropic sources, may have a component from pharma providing free
access to drugs. These studies can also draw on treatments more widely
available in the market such as generic versions of previously patented drugs.
Academic studies will be seeking to find better ways of treating patients using
more clinically relevant outcomes and taking fuller account of the whole patient.
They are not seeking solely to identify the numeric significance of a treatment.
This sets academic studies apart from highly publicised drug studies with
The EORTC (a charity) is an important provider of academic studies. The
European Commission is funding studies through its R&D programme.
Governments support studies, often providing funding to a university hospital.
An important source for such studies is the UK, where local funding from Cancer
Research UK is a critical component. Occasionally one trial will stand out.
Two extra years from an off-patent drug
STAMPEDE is a ‘basket’ study looking at a range of drugs in treating advanced
prostate cancer. It is a 7,000 patient multi-arm study. It could not be run by
pharma. Its first results published at ASCO 2015 looked at docetaxel, which is
now off patent. The drug was given earlier than it is in current practice. An OS
(overall survival) improvement of 24% and ‘failure free’ survival of 38%
translates into significant clinical benefit, improving patient longevity by a
median of just under two years.
The Chief Investigator, Professor Nick James from University of Warwick, said
“Our headline conclusion would be that docetaxel should be considered as
routine practice in men with newly diagnosed metastatic disease. With non-
metastatic disease, there remains uncertainty as to whether there’s a survival
benefit or not but it certainly improves failure-free survival by a substantial
Pharma company researchers would give their right arms for results like that in
a cohort of patients as large as this. It will change clinical practice without
truckloads of expensive documentation being required by EMA.
So let’s pay more attention to academic studies. They can change things in big
ways and have the potential to provide patient benefit far beyond the seemingly
eternal procession of pharma company registration studies targeting smaller and
smaller cohorts of patients.
Roger Wilson blogs at http://rogerwilson.me.uk/