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.

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 registration objectives.

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.

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 amount.”

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 lets 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.

First published on the Cancer Blog at

Further reading on STAMPEDE



Quality Standard for Sarcoma creates new opportunities


I found the publication of the NICE Quality Standard for Sarcoma (link at bottom of page) quite personally rewarding. The process of developing it was fairly intense and took into account a lot of views, some quite trenchantly held. To be a patient in a process at NICE is always one of the better patient involvement experiences there is. I wrote about the Quality Standard a few weeks ago and I have had time to think more about it.

This simple set of six statements could generate a big evolution in the way that sarcoma treatment is delivered in England and I hope the opportunity is not missed. The statements are aimed at Commissioners, Networks and service providers. They set standards for improving the outcomes for patients built upon recent evidence and real experience. They are also intended to help patients understand what to expect.

Sarcoma treatment structures have been reorganised over the past eight years following the NICE guidance on which I also worked. Improving Outcomes for People with Sarcoma has been instrumental in helping us arrive at the 12 treatment specialist centres we have today. Five of them treat bone and soft tissue sarcoma, the other seven soft tissue alone. All of them have a Clinical Nurse Specialist within the multi-disciplinary team (MDT), alongside surgeons, oncologists, radiologists and pathologists.

Some of these centres are multi-site – hospitals linking together to provide the total service. Many of these treatment teams also undertake outreach. Some have outlying diagnostic and treatment units with clinicians who are “extended members” of the MDT. Some also hold clinics in hospitals other than their home centres – recognising that patients often live far from the main site.

Sarcoma specialist MDTs all have a character of their own, reflecting the host organisations and the individual doctors who work in them. Some have special interests (eg younger patients, people with retroperitoneal tumours), or additional qualifications and skills (eg specialist surgical techniques), many are involved in research, and some have oncologists who have an interest in supporting sarcoma patients with gynae or gastrointestinal tumours.

Now the Quality Standard is opening the way for greater networking.

One of the new standards requires retroperitoneal sarcomas to only be treated where there is specific expertise. It is for the NHS England Sarcoma CRG to determine which centres have expertise but the NICE committee was clear that such centres should have a high patient volume and thus be few in number.

All sarcoma MDTs are required to make public the pathways they have with other treatment teams eg for metastectomy and the specific special skills which they have within their own team. This should open up a better understanding of how individual patient experience can be improved. Could this lead to referral between sarcoma MDTs to maximise patient outcomes?

Gynae and GI MDTs are also required to collaborate with sarcoma MDTs on patients they diagnose with sarcoma. This is already a requirement but it is rather patchy and it is questionable whether it adds value for many patients. The Quality Standard puts it onto a new footing. GI and Gynae cancer teams will have to reach out to Sarcoma MDTs because they must agree a care plan for every sarcoma patient. This should build stronger links with Gynae and GI centres which can benefit patients (eg access to trials) but will only do so if the sarcoma teams take active involvement in their care.

There is the opportunity for new guidelines. Bringing practitioners together to share knowledge and experience to create consensus guidelines and discuss treatment protocols is a huge opportunity, if only the cost of doing it can be found. I hope that the British Sarcoma Group can rise to this challenge.

So the next three years will be very interesting. We have a new government promising that the NHS will have new funding. NHS England is starting to look better organised. NICE will remain largely unchanged and with questionable influence. CQC is making waves. Cancer Peer Review is being revitalised. Yes, we might (hopefully) have a practicable process for evaluating and funding new drugs. In all this I want so see sarcoma MDTs working more closely together with some more formal networking and I do believe that we will see an improvement in outcomes for sarcoma patients.

The NICE Quality Standard for Sarcoma can be found here: