ECPC Position on RARE CANCERS

Realising the Vision of Equity:
The Optimal Care and Treatment of Rare Cancers in Europe

There is a desperate need to overcome significant barriers to scientific innovation and more effective clinical practice which would translate into better outcomes and an improved quality of life for patients with rare and less common cancers.

Due to low incidence rates and the high number of distinct pathologies, local medical expertise about rare cancers is inadequate, leading to late diagnoses and poor referral rates. Patients often need to travel long distances in order to receive appropriate therapy and care at specialised centres.

Where treatment is available, long-term clinical data is difficult to provide. This leads to an increased number of negative reimbursement decisions based on 'cost effectiveness' and access to potentially effective therapy is often denied.

Allied to this, and the fact that the patient population is small, is an absence of commercial interest in the development of novel therapies for rare cancers. Public funding for clinical trials of treatments for rare cancers is not readily available.

Patients with rare cancers would benefit specifically from the development of a Europe-wide collaborative research network for translational research, 'from bench to bedside'. This would help transform the vision of truly equitable healthcare for patients with rare cancers into a reality.

Patient groups should be included as part of decision-making bodies and work in equal and collaborative partnerships with all stakeholders in order to help prioritise research and deliver a coherent research strategy that puts the patient at its centre.

ECPC urges all stakeholders, including the European Commission (EC), Members of the European Parliament (MEPs), EU Member States, the European Medicines Evaluation Agency (EMEA), national regulatory authorities, the pharmaceutical industry, professional scientific and clinical organisations and patient organisations, to take urgent action in order to improve outcomes for patients with rare cancers.

Challenges surrounding Rare Cancers

Rare Cancers are usually defined as having a prevalence of less than 50 in 100,000 or an incidence of less than 5 in 100,000 populations per year. There are specific challenges surrounding rare cancers which include:

• Delayed or lack of referral to centres of expertise. Low incidence rates lead to a general lack of awareness about symptoms, disease characteristics and pathologies of rare cancers at the primary care (GP) level. Consequently late and misdiagnosis, inadequate disease management and sub-optimal treatment outcomes are very common for patients with rare cancers.
• Few centres of expertise and reference networks exist across the EU. The lack of funding for such centres means that patients often need to travel long distances in order to receive appropriate treatment and care. This has a direct impact on the financial costs to the individual patient and family and the wider associated societal costs.
• Reluctance by health service providers to reimburse clinically effective therapy. Low patient numbers mean that it is very difficult to demonstrate the effectiveness of therapies supported by evidence-based clinical practice.
• There is inadequate provision for patient access to educative information about their disease and the therapeutic options available. Information about ways use communications technology (ICT) in order to access possible effective treatment and care is also inadequate.
• A lack of commercial interest in the development of potentially effective therapies for rare cancers.  Small patient numbers impose difficulties for researchers and clinicians to effectively fulfil the current regulatory criteria for clinical studies of innovative therapies.

Suggested Actions and solutions

• Enable collaborative research programmes through European reference networks (ERN) by the removal of national regulatory and cultural barriers in order that European reference networks are developed and facilitated by Member States.
• Enhance infrastructure to support a platform for European Centres for Translational Research to enable collaborative efforts between the basic and clinical research communities and industry with a focus on a reference network for rare disease. This would play a strategic role in improving the quality of treatment for patients with rare cancer throughout the EU
• Improve medical education for healthcare professionals. Apply the mandatory inclusion of rare cancers in the ongoing education of doctors and pathologists, in order to ensure early and accurate diagnosis
• Mandatory registration and automatic referral. Apply a mandatory requirement for doctors to register all cases of rare cancer with automatic referral to centres of expertise and suitably experienced expert clinicians, pathologists and allied healthcare professionals.
• Support for patient mobility. Provide adequate financial support for patients and their families who need to travel in order to access treatment at centres of expertise
• Improve patient education material and access to information through modern communication technology. Provide relevant up to date informative materials for patients with rare cancer. Remove legal barriers to patients' access to their medical notes. Support the sharing of information between patients, their representatives and the medical community to encourage an acknowledgement by all stakeholders that the informed patient is a valuable healthcare resource in the fight against cancer.
• Rationalise off/near-label drug use. Remove existing regulatory and legal barriers to clinical prescribing of off/near-label drugs for rare cancers. Foster a 'risk management' rather than 'risk averse' model in this treatment setting.  Owing to a lack of therapeutic options, patients with rare cancers are often willing to accept some uncertainty of outcome. Include the patient's viewpoint in the decision making process.
• Encourage innovative research and orphan drug development. Explore additional incentives at national and European levels to encourage the pharmaceutical industry to prioritise the research and development of orphan medicines in order to develop more effective and affordable therapies for rare cancers
• Where an HTA process is in use, the adoption of a 'conditional pricing' model where patients can gain access to a medicine they need, whilst at the same time further evidence is collected to demonstrate the medicine's efficacy, should be actively encouraged. All appropriate treatments for rare cancers should be considered by the relevant Health Technology Appraisal organisations in a timely manner. Patients, their representatives and the relevant expert specialist clinicians in site-specific rare cancers, should be an integral part of any appraisal process.
• Include patients as equal stakeholders in cancer research. Encourage the mandatory inclusion of patient representatives in the design of clinical trials and regulatory assessments.

ECPC welcomes the increased focus on rare cancers recently brought forward by the European Commission in terms of the Communication, a Proposal for a Council Recommendation, and an Impact Assessment on rare diseases.  We ask that the Commission address Rare Cancers in their European Partnership for Action against Cancer.
http://ec.europa.eu/health/ph_threats/non_com/rare_10_en.htm

ECPC welcomes the recommendations resulting from the 'Rare Tumours in Europe: Challenges and Solutions' Conference organised by ESMO in collaboration with the European Cancer Patient Coalition, Eurordis, Orphanet, Conticanet, the Association of European Cancer Leagues and industry.
http://www.esmo.org/fileadmin/media/pdf/policies/ESMO_Rare_Cancers_RECOMMENDATIONS.pdf

ECPC is or has been involved with the following initiatives on rare cancers:

• RARECARE Project - Surveillance of Rare Cancers in Europe
  Project co-funded by the European Commission (EC) through its Public Health and Consumer Protection Directorate (DG SANCO), PHEA programme, and contributes among other projects to the creation of networks of action for rare diseases. ECPC is a member of the advisory board. See http://www.rarecare.eu/

• EU Commission Communication on European Action in the Field of Rare Diseases
  In November 2008, the European Commission released a Communication and proposal for a Council Recommendation on Rare Diseases. Previously, ECPC informed their members about this initiative and participated in the public consultation. See http://www.ecpc-online.org/newsletter/member-updates/126-mu112008-rare-diseases.html, including ECPC background paper.

• UNESCO, Danish Cancer Society and ISE Meeting:
  World Class Infrastructure: A Platform for European Cancer Research Centres For Translational Research. Paris, October 15th 2008. ECPC was represented on a roundtable panel discussion and quoted in 'Molecular Oncology' 2008 1-4

• ESMO Conference 'Rare Tumours in Europe: Challenges and Solutions', Brussels, November 6th 2008.
  ECPC was represented on the organising committee, the taskforces for all three workshops and the steering committee. ECPC representatives gave presentations at the final plenary and one workshop session. See http://www.esmo.org/events/past-events/rare-cancers-2008.html

For further information please contact:

Sandy Craine (ECPC Secretary)
E-Mail: This e-mail address is being protected from spambots. You need JavaScript enabled to view it

Further reading

• Article in CancerWorld March/April 2009: "Europe's rare cancer community calls for a more radical approach". See also CancerWorld Portal.