Genetic Alliance UK in collaboration with the Tuberous Sclerosis Association and Association for Glycogen Storage Disorders recently submitted a petition to the National Assembly for Wales. We called for a review of the use of the "exceptionality rule" in determining whether a patient can access a treatment through the Individual Patient Funding Request (IPFR) process. When the petition closed, it had over 1000 signatures.
From recent meetings, we know a number of you have had experience of the IPFR process in order to access services or treatments. Genetic Alliance UKhave included information below which outlines why this criteria is difficult to evidence for rare disease patient populations which means that those individuals are less likely to be successful in having their application approved compared to other groups of patients.
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.
Genetic Alliance UK will be presenting the petition to Assembly Members on the steps of the Senedd in Cardiff Bay at 1pm tomorrow. We would welcome support from anyone who is able to join us. Please let me know if you intend to come down to the Senedd tomorrow. firstname.lastname@example.org