Access to Orphan Drugs and Therapies

Access to Orphan Drugs and Therapies

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At a recent EURORDIS (European Rare Diseases) conference CF Ireland were invited to take part and speak about the role of patient advocacy in the reimbursement of new, expensive therapies – using Kalydeco as an example. Having attended the conference and spoken with people representing a myriad of rare diseases, it is very evident that the limitation of health economic assessment for rare diseases is a common worry for all disease groups. As medical treatment moves to a more personalised medicine approach, there is a rise in the number of new innovative therapies for rare diseases. This is great news for people living with rare diseases where there are often no alternative treatments available; however, access to these therapies causes a great deal of stress and anxiety. Current health economic assessments are limited in their ability to accurately and fairly assess the 'cost-effectiveness' of orphan drugs. Some of the limitations include:

  • Numerous limitations with the QALY (Quality Adjusted Life Years gained) Ñ no consideration for wider, long-term savings to be made (joining the workforce, prevention of other treatments being needed, etc)
  • Presumption that health economics can accurately value the quality of a person's life
  • Lack of understanding of implications of living with a particular illness, such as QoL, levels of suffering, etc.
  • The utilitarian view – "the greatest good for the greatest number"
  • Lack of comparators in the Health Technology Assessment
  • Placing a price on a person's life is degrading for people living with illness

These limitations have been recognised by national Health Technology Assessment (HTA) agencies and communication between patient groups and national and international bodies are improving. There is a gradual move towards assessing 'patient-reported outcomes' more formally and incorporating these views into the assessment process. However, we are still have a way to go to find a fair, transparent process to assess the 'cost-effectiveness' of orphan drugs. CF Ireland is working alongside other rare disease groups to ensure our voices are heard and to push for the reshaping of health economic assessments for rare diseases.