Moving the boundaries of international collaboration on clinical trials and QoL: experiences in oncology and legislation within the European Parliament

Abstract

3rd EORTC Quality of Life, Symptom Research and Patient Reported Outcomes in Cancer Clinical Trials Conference

The European Parliament, Brussels, Belgium, 17–20 October 2012

The European Organisation for Research and Treatment of Cancer undertook another successful event with their third annual conference addressing quality of life matters in cancer clinical trials. More than 40 presentations were made over a 3-day period hosted at the European Parliament on 17–20 October 2012, in Brussels. The conference managed to get speakers and policy makers together to debate all the key issues in cancer clinical trials, design and reporting, including future policy and regulatory concerns. This meeting set the stage for future research and policy meetings to give greater visibility to quality of life as an outcome in clinical trials within the world of EU legislators.

In her conference welcome address, European Organisation for Research and Treatment of Cancer (EORTC; Brussels, Belgium) Director General, Professor Françoise Meunier welcomed more than 300 delegates from across Europe and beyond. She thanked the various conference sponsors, the European Commission’s DG SANCO, and Member of the European Parliament Marisa Matias for hosting the EORTC Conference on Quality of Life for the second year running.

Meunier explained in her speech that the conference helped highlight a very special year for EORTC, 2012, with the organization celebrating its 50th birthday. She told the audience that quality of life (QoL) has been a central theme for EORTC for more than 30 years. Indeed, one of the first QoL questionnaires was for children with leukemia in the 1970s. Meunier emphasized that, as the years have rolled by and treatments have improved, more and more people are surviving cancer, and thus QoL issues related to survivorship and treatment have assumed higher importance. Furthermore, she paid tribute to the 180,000+ patients across Europe who had placed their faith in EORTC and its mission by taking part in clinical trials run by the organisation.

While new treatments and the prospect of personalized medicine are revolutionizing cancer care, Meunier said that financial constraints and austerity across Europe were leaving many people wondering who would be able to afford these novel treatments. She added that the conference was taking place against a background of Euroscepticism with commentators regularly questioning the future of the EU itself. “In these circumstances, we have to fight really hard to keep Europe on the medical research map,” said Meunier. “We need to bring hope to the younger generation that there is a future for both health and quality of life research in Europe.”

In her introductory remarks, Matias said the conference was a great example of researchers, professionals, patients and legislators working together. “This is an opportunity to show that the European Parliament takes cancer quality of life issues seriously,” she said. She added that while member states still played a central role in dealing with QoL issues, the European Parliament was a forum to provide responses to issues affecting all EU citizens. Matias discussed the European Commission’s Horizon 2020 research program, which will replace the existing Framework Programmes for projects running from 2014 to 2020. Health is central to the societal challenges in Horizon 2020, and Matias said negotiations were ongoing to make the budget of the program as large as possible, with the largest share going to biomedical research.

Rationing of funding is becoming more important than ever before, both in terms of healthcare and health research. “We are facing questions we thought we’d never face in Portugal – which patients should we treat, because we can no longer afford to treat them all,” said Matias, adding that recent discussions in Portugal have covered whether it is economically viable to treat patients in the later stages of cancer. “Who is making these decisions?” asked Matias. “Issues such as this make it clear that we need more solidarity in Europe, not less.”

Other key talks included the EMAs proposal to create a new appendix, focusing on QoL measures, to the anticancer guidelines used to approve new cancer drugs and therapies. “This new guidance will be increasingly important in this new era of anticancer medicines. Some new therapies that lead to limited improvements in survival can cause extensive problems with a patient’s QoL. Conversely, new therapies can cause less toxicity than older therapies since they have become more targeted,” said Andrew Bottomley, Assistant Director of EORTC and Head of the organization’s Quality of Life Department.

QoL in the context of cancer assesses symptoms such as fatigue, nausea, sexual functioning or psychological problems, which a patient may experience as a direct consequence of cancer treatment. These are different from the traditional clinical trial measures commonly reported such as ‘overall survival’ and ‘progression-free survival’. Work on the new appendix is being done on behalf of EMA’s Committee for Medicinal Products for Human Use by the Oncology Working Party. “During the revision of the anticancer guidelines, it was recognized that there was enough scope to warrant a separate health-related QoL appendix so that certain issues could be considered in more depth,” said Daniel O’Connor, the presenter of this session who is based at the UK’s Medicines and Healthcare Regulatory Authority. O’Connor, who is part of EMA’s working party on this guideline but who was giving his own personal views at the conference, adds: “There is increasing recognition that HRQoL is an effective and relevant measure with which to evaluate and monitor therapeutic outcomes. health-related QoL measures may complement the range of traditional objective indicators such as survival, which could contribute to benefit–risk decisions.”

Clinical trial design, health-related QoL instruments, statistical methodology, clinical importance and patient-related outcome measures will be considered in the new appendix that is expected to be released for public consultation in 2013. Bottomley added: “It is excellent that the EMA is making significant strides to have this separate quality of life appendix added to the standard guidelines. This demonstrates to patients that their voice, wishes and needs are being taken seriously when drugs are given approval. Patients will be able to understand not only the survival benefits of the new therapies, but also the effects of new treatment on both short- and long-term quality of life. I look forward to a firm release date for this guidance.”

Patient-reported outcomes essential for the next generation of cancer therapies were one of the topics of the conference of this year, as was charting the progress made in QoL research for patients with breast cancer over the past three decades. However, in her presentation, Lesley Fallowfield (Brighton and Sussex Medical School, UK), a world expert in this field, showed that huge gaps remain in terms of data needed to properly assess QoL, and that patient-reported outcomes will be essential in future drug approvals. She also concluded that medicine must move away from its default position that patient-reported outcomes represent ‘soft’ data, whereas data reported by clinicians and other healthcare professionals is always taken much more seriously.

She pointed out that almost all new drugs approved have an excellent safety profile until they are in the real world, where side effects, reported by patients, begin to mount. Fallowfield also concluded: “We really need to get away from this idea, still prevalent in medicine, that outcomes reported by the doctor or nurse are somehow more valid than those provided by the patients themselves, even though evidence shows that patients’ reports are more accurate.”

One of the abstracts presented unpublished data from the DietCompLyf study, which aims to assess possible links between plant phytoestrogen consumption and breast cancer recurrence and survival, as well as the analysis of QoL outcomes. The research was carried out by Ruth Swann, study co-ordinator at the University of Westminster, London, UK, and colleagues. The latest analysis carried out by Swann and her colleagues on nearly 3000 breast cancer patients has produced preliminary data showing that patients with a higher QoL score had a lower BMI and exercised more than once a week. A higher QoL score was also associated with responsible levels of alcohol consumption and the feeling that the patient felt less alone. Furthermore, patients who perceived that they had the practical and emotional support they needed following a breast cancer diagnosis also had a higher QoL score.

In another part of the abstract session, new data were presented that show that women undergoing breast cancer treatment are far more likely to be concerned about their reproductive health than are healthy women. The study’s authors, led by Mirjam Garvelink, Leiden University Medical Center, The Netherlands, conclude that such women should be offered counselling about interventions such as fertility preservation treatment (freezing embryos, eggs or ovarian tissue) to help improve their QoL.

These highlights were just a few of the many excellent plenary presentations delivered at the conference. As we do not have space to describe them all in our report, EORTC have uploaded over 20 h of video showing the presenters’ speeches, and these can be found at [1].

On a final note, this conference was also special in being able to offer awards for best research for young investigators and also for advanced researchers (see the winners at [1]). Clearly, QoL research in clinical trials is not suffering from a lack of results, and while we face a challenging economic environment, it is certain that QoL research is something that continues to be critical and important for society.

Acknowledgements

The authors thank Cheryl Whittaker and Shelia Sanderson for the editorial review of this article.

Financial & competing interests disclosure

A Bottomley is Senior Editor for Expert Review of Pharmacoeconomics & Outcomes Research. A Bottomley, J Bean and J Walker are employees of the EORTC. This conference was funded in part by an EU grant. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.