The challenges of treating and living with rare cancers were highlighted today at a major international medical meeting: “The Battle Against Rare Cancers in Europe” press conference brought together representatives from all stakeholder groups in the fight against these diseases: patients, caregivers, oncologists, pharmaceutical companies, researchers and policy makers. The media event was organised by the European Society for Medical Oncology (ESMO) during the 35th ESMO Congress in Milan, Italy (8-12 October).
“Around 20% of all cancer cases are considered to be rare, so taken collectively, these types of cancer are not rare at all”, emphasised Professor Paolo Casali, medical oncologist at the Istituto Nazionale dei Tumori in Milan and member of the ESMO Executive Committee, who chaired the press conference.
According to figures from Rarecare, over two and a half million Europeans are affected by rare cancers and each year more than 400,000 Europeans are newly diagnosed with a rare cancer.
Prof Casali added: “Even frequent cancers are now split into subgroups for therapy so they suffer from the same problems affecting other rare cancers.”
Prof Casali explained that the priority now was for existing recommendations on “Improving Rare Cancer Care in Europe” to be implemented: “ESMO is currently sponsoring a ‘Call to Action Against Rare Cancers’ which has been developed as part of the ‘European Action Against Rare Cancers’ campaign. The Call to Action is based on 39 Political Recommendations that were the result of a consensus obtained at a high-level conference on the subject in 2008 in Brussels. They have to do with improving the methodology of clinical trials, structural barriers in the organisation of healthcare and equal access to treatment options, which are the main challenges we face today.”
Top of the list of barriers to the development of new drugs for rare cancers is the funding of clinical research. A disease is considered rare if it affects fewer than five out of every 10,000 people.
“Developing drugs for small patient populations which fit within existing regulatory frameworks takes a long time, so there are few companies able to make such investments when long-term outcomes are largely unknown,” explained Dr Guido Guidi, Head of Region Europe, Novartis Oncology.
“There are two main challenges that need to be overcome,” he said. “First, we have yet to identify and specifically target the unique most important mechanism at the origin of the disease. Second, a new approach to drug development needs to be agreed with health authorities. Clinical trials for rare cancers should not require the same extensive number of patients as in other diseases which affect a broader population”.
Prof Jean-Yves Blay, President of the European Organisation for Research and Treatment of Cancer (EORTC) agreed. “In rare cancers, large numbers of patients are difficult to obtain. Clinical trials in randomised format are rare and even in this case are not considered conclusive by health authorities”.
According to Dr. Guidi: “Greater awareness of ongoing trials and of the clinical trial process should be available to patient groups and healthcare professionals”.
“We desperately need more clinical trials in Europe for rare cancers. This is crucial,” stressed Kathy Oliver, Co-Director of the International Brain Tumour Alliance (IBTA), a patient advocacy and awareness raising organisation.
“On the medical front, it is an exciting time of discovery in terms of genetic markers, targeted therapies and innovative approaches to treatment with combinations of drugs, new delivery systems and advances in surgical techniques. But these extremely difficult economic times add pressures to the rare cancer arena. For rare cancers the priorities are numerous and urgent: equitable funding for research; better support for patients who are struggling with the most appalling cancer journeys; the establishment of centres of excellence with multidisciplinary teams; more clinical trials; more flexible, transparent and fairer health technology assessment mechanisms and a range of other priorities set out in ESMO’s Political Recommendations which form part of the European Action Against Rare Cancers.”
Another problem is that when treatments do exist, they are often very expensive and not reimbursed by health plans, public or private.
“Every step in rare cancer is a challenge,” pointed out Dr. Kristina Andrékuté, Chair of the European Cancer Patient Coalition (ECPC) Rare Cancer Action Group (RCAG) and representative of the Lithuanian rare cancer group, ROLLD.
“Diagnosis is complicated and takes a long time,” she said. “Afterwards, it is hard to find a right center for treatment. The situation in Eastern Europe is even worse with very limited diagnostic and treatment possibilities for rare cancers with the usual barriers for new drug reimbursement, with patients being put on long waiting lists, selection by age or social status. Action should take place at two levels. European institutions need to facilitate the coordination, cooperation and coherence of national, regional and local initiatives addressing rare cancers. Member States should ensure access to high quality, efficient good health for all patients.”
Dr Andrékuté pleaded for the creation of a “concrete framework for cross border health care, allowing patients suffering from rare cancers the option of travelling abroad to receive treatment which their country of origin cannot offer.”
Robert Schaefer, newly appointed Senior Project Manager of ESMO’s European Action Against Rare Cancers (EAARC) explained: “There is no common status in Europe regarding the treatment of rare cancers. In many countries, especially in Eastern Europe, options are restricted due to the limited availability of treatments. With all EU countries trying to curtail healthcare spending, every new therapy that has been considered to be safe and effective by the European Medicines Agency and has obtained marketing approval by the European Commission will also be assessed at a national level mainly on the basis of cost-effectiveness.”
Mr. Schaefer said: “There are different approaches in individual countries. As a result, EU-approved therapeutic options may be readily available, in a timely fashion and reimbursed under the national health insurance scheme in one country, whereas in other countries the assessment procedure can take very long and access to previously approved treatment options can be denied altogether on the grounds of cost.” Mr. Schaefer added that EAARC is working hard for the 39 Recommendations to be implemented across Europe.
Unfortunately, this process could take a long time considering all the partners involved and patients with rare cancers do not have the luxury of time. 240.000 Europeans die each year from a rare cancer.
“The problem is of great urgency,” says Dr Andrékuté, “when talking of rare cancers, time is not an ally but an enemy.”
Kathy Oliver agrees and adds: “Patients with rare cancers also often have to deal with late diagnosis and misdiagnosis. For example, research undertaken by the Children’s Brain Tumour Research Centre at the University of Nottingham in the UK highlighted the fact that an unsatisfactory picture exists regarding diagnosis of brain tumours in children and young people up to 18 years of age. This sobering research revealed that it takes from two to three months for a child with symptoms identifiable with a brain tumour to obtain an MRI scan. This is three times longer than in the United States. (Diagnosis of brain tumours in children: a guideline to assist healthcare professionals in the assessment of children who may have a brain tumour, Archives of Disease in Childhood doi:10.1136/adc.2009.162057, Sophie Wilne et al)
“Another challenge,” she said, “is that many European governments and major cancer control organisations have prioritised prevention, screening and healthy lifestyle campaigns in the fight against cancer. These are all excellent initiatives, of course. But not every cancer can be helped by this approach. Brain tumours, as an example of a rare cancer, affect people randomly and there is no realistic screening for them. The cause of most primary brain tumours is, as yet unknown, so there can be no prevention programs for them. And there is no known preventative option by healthy living, diet or exercise.”
“Once diagnosed,” Mrs. Oliver said, “patients with rare cancers sometimes have to push hard for access to certain treatments. Sometimes if you don’t ask you don’t get. Information and support for people suffering from a rare cancer can be patchy and uneven. We need to change the landscape for rare cancers so that patients with these diseases have significantly better outcomes.”
Dr. Guidi, from Novartis summarised: “We need to ensure that the right therapy reaches the right patient at the right dose, at the right time”.
Mr. Schaefer confirmed: “ESMO understands the urgency for rare cancer patients to get the same treatment as other patients, as defined in European legislation. Therefore, together with its partners, ESMO is fully committed to putting rare cancers firmly on the European policy agenda. This reflects ESMO’s continuous efforts towards improving the prevention, diagnosis, treatment, supportive and palliative care of patients with malignant disorders, irrespective of their prevalence or incidence”.
Press Briefing Videos
Moderator: Prof Paolo G. Casali, Member of the ESMO Executive Committee, Head of the Sarcoma Medical Treatment Unit at the Istituto Nazionale dei Tumori, Italy: Existing obstacles to research and treatment of rare cancers
The researcher perspective on rare cancers
Dr James C. Yao, MD Anderson Cancer Center in Houston, Texas, USA
What the EORTC and the EU do and plan in the field of rare cancers
Prof Jean-Yves Blay, President, European Organisation for Research and Treatment of Cancer (EORTC), France
The challenges of being a rare cancer patient
Ms Kathy Oliver, Co-Director, International Brain Tumour Alliance (IBTA), UK
European Action Against Rare Cancers – A multi-partner initiative against rare cancers
Mr Robert Schaefer, Senior Project Manager, European Action Against Rare Cancers, Germany
Are pharma companies investing enough in rare cancers?
Dr Guido Guidi, Head Region Europe, Novartis Oncology, Italy