Genetic Alliance UK in collaboration with members petition for review of 'exceptionality criteria' underpinning Individual Patient Funding Request (IPFR) process in Wales
Genetic Alliance UK have been involved in a number of recent reviews in Wales relating to the appraisal of orphan and ultra-orphan medicines as well as the Individual Patient Funding Request (IPFR) process. Recently, Genetic Alliance UK was invited to represent the patient perspective on the national group conducting the review of the IPFR process. Unfortunately, the review did not consider the fundamental issue that patients with rare diseases face in accessing treatments via this process. The 'exceptionality' criteria on which the IPFR process is based.
Together with the Tuberous Sclerosis Association and Association for Glycogen Storage Disorders, we are calling for the National Assembly for Wales to review the use of the 'exceptionality rule' in determining whether a patient can access a treatment through the IPFR process.
The petition will be open for the next few weeks and we hope to generate as much support as possible during this time to ensure that the Petitions Committee agrees to our request to review the IPFR criteria.
We would encourage all member organisations to circulate details of the petition to their networks of supporters.
For further details about this campaign, please contact our Development Officer in Wales, Emma Hughes.
The IPFR process denies patients with rare diseases access to treatments
To access treatments through the IPFR process, a patient population must demonstrate its exceptionality. For common illnesses, it may be possible to identify a subset of patients within the larger population who are more likely to respond to a particular therapy. For rare disease patients, demonstrating that you are a unique patient when you are part of a small group of patients whose condition is considered rare is practically impossible.
The exceptionality criteria place an onus on clinicians to provide evidence that the patient’s clinical condition is significantly different to the general population of patients with the same condition and is likely to gain significantly more benefit from the intervention than might normally be expected. This evidence requirement is too onerous to apply to patients with rare diseases due to small patient numbers within rare disease populations. Patients with great clinical need are prevented from accessing life-changing/life-saving treatments.