Patient Advocacy Toolkit: Key Messages

Concentrate on certain key message to get your ideas across

Inequalities in Rare Cancers

  • Five-year relative survival rates are worse for rare cancers in adults (47%) than for common cancers (65%)
  • Survival rates differ significantly across Europe (gaps between Northern/Western Europe and Eastern Europe)
  • Patient access to new cancer drugs is not equitable across Europe


  • Rare cancers are often misdiagnosed, leading to late or inadequate treatment
  • Rare cancer diagnostics require expert opinion
  • Need for referral (second, reimbursed, opinion)
  • Experts may not be available in the country where the patient lives
  • Need for cross-border referral
  • Experts and reference centres are poorly known to patients and doctors


  • Few therapeutic options
  • Treatment based on low level of evidence
  • Frequent "off label" use of drugs
  • Patient referral is sub-optimal (long delays)
  • Biological material may not be readily available
  • Quality of pathological material not always adequate or quantity insufficient
  • Need for a system in place that would swiftly direct rare cancer patients to an expert centre together with appropriate data and good quality material


  • Rare Cancer treatment not as attractive for the pharmaceutical industry (return on investment lower than for other cancers)
  • Randomised clinical trials considered not possible for rare cancers due to small numbers
  • Poor acceptance of adaptive designs and lower level evidence by regulators
  • Randomised clinical trials are feasible (except for ultra-rare cancers)
  • Need for large international collaboration
  • Need for incentives (funding)
  • Methodology concerns/Study design
  • Enrollment issues
  • Production issues (small quantities in each country)
  • Reimbursement issues
  • Paediatric specificity (long-term safety issues)
  • Post Marketing Authorisation issues

Patient participation in clinical trials

  • Cross-border participation in clinical trials
  • Patient involvement in the design of clinical trials
  • Better information of ongoing clinical trials and their results

European Reference Networks

  • Need to implement the ‘EU Directive on Patient’s Rights to Cross-border Healthcare’ (Directive 2011/24/EU) in order to allow prompt referral of rare cancer patients for timely and accurate diagnosis
  • Provisions for transfer of tissues
  • Need for processes to allow structured collaboration between specialists and centres of expertise at a national and European level
  • Establishment of cross-border networks of clinical databases and registries on rare cancers, with a focus on tissue banks, to foster translational and clinical research


  • Of second opinion
  • Of innovative treatments (criteria should be different than for other cancers)
  • Of "off label" use of drugs

Patient consent

  • Informed consent needs to be more “patient friendly” (easy to understand wording)
  • Need for enduring (withdrawable) patient consent
  • Patients should be considered “experts” and their feedback taken into account

Centres of excellence

  • Need for Centres of Excellence where patients can be referred for second opinion and treatment
  • Provision for tele consultation and tele pathology
  • Centres should include tumour banks open to researchers

Download the complete Rare Cancers Europe Patient Advocacy Toolkit