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In October 2013, a review of the Individual Patient Funding Request Process (IPFR)  was commissioned by the Minister for Health and Social Services, Welsh Government, Mark Drakeford AM. Genetic Alliance UK and the Cystic Fibrosis Trust were invited to represent the patient perspective as members of the National IPFR Review Group carrying out this process.
The report fails to address the fundamental issue that clinicians and patients affected by rare diseases face when making an application to access a new medicine through the IPFR process. This relates to the continued use of the ‘exceptionality’ criteria for medicines that are developed to treat patients with rare and ultra-rare diseases.
In addition, the report omitted a series of key recommendations that had previously been agreed by members of the group. An initial draft that was circulated for comment outlined 26 recommendations which aimed to improve the IPFR process. Just 10 recommendations are contained within the published document.
To access treatments through the IPFR procedure, a patient population must demonstrate its ‘exceptionality’. The ‘exceptionality’ criteria pose one of the most challenging issues for patients, carers and health professionals affected by rare diseases. For more 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. This subset is defined as ‘exceptional’ and are eligible for the IPFR procedure. For patients with rare diseases, demonstrating that you are a ‘unique’ patient when you are already part of a small group of patients whose condition is considered rare is practically impossible.
The ‘exceptionality’ criteria places an onus on the clinician to provide evidence that the patient’s clinical condition is significantly different to the general population of patients with the same condition and as a result is likely to gain significantly more benefit from the intervention than might normally be expected. This definition of clinical ‘exceptionality’ is too onerous to apply to patients with a rare or ultra-rare condition due to the fact that they are required to demonstrate:
Due to small patient numbers within rare disease populations, it is extremely difficult to meet the above criteria. Since the report has not addressed this issue, the exceptionality rule will continue to prevent patients with great clinical need gaining access to transformational medicines which may be life-changing/life-saving.
The report makes reference to rare and ultra rare medicines specifically, stating that:
“the lack of authoritative advice on ultra rare and rare medicines was also considered problematic within the context of the IPFR process, since demonstrating exceptionality in an individual with a very rare condition and in the absence of what may be considered an acceptable evidence base is challenging.”
Despite these considerations, the report does not propose any alternative route for patients with rare diseases to access medicines that are not appraised nationally.
The report then notes the Review of the Appraisal Process for Orphan and Ultra Orphan Medicines report, published by Dr Paul Buss, Sian Evans and Professor Ceri Phillips in October 2013 , which recommends the development of a process to evaluate new orphan and ultra-orphan medicines. We support the establishment of a process which captures medicines that have not been nationally appraised  and are not suitable to be appraised via the IPFR Process (as outlined above). A nationally agreed process would deal with the situations where IPFR applications are not appropriate. This access route would be particularly important and relevant to patients with rare and ultra rare diseases as many of the applications for these groups of patients will not be able to satisfy the criteria for exceptionality.
It is important to recognise that this process will not provide a solution to the IPFR issue that is the subject of this document. The proposed All Wales Medicines Strategy Group (AWMSG) process will not routinely capture those medicines for small disease populations that are routinely available through the NHS, which should be accessible to the rare disease population through the IPFR process.
We call for the All Wales Toxicology and Therapeutics Committee (AWTTC) to lead on the development of a robust and transparent process for appraising new, innovative medicines that have not been appraised nationally and are not suitable within the criteria for an IPFR application. Guidance must be developed regarding eligibility criteria of a process for appraising these groups of medicines. Clear and robust guidance is needed for patients and clinicians which should be developed in collaboration with patient organisations and underpinned by clinical expertise.
A substantial number of recommendations supported by patient representatives on the Review Group were not present in the published report. An outline of the most significant omissions have been included below:
The Individual Patient Funding Request process is currently used inappropriately. Many applications are for medicines that have already been approved by the National Institute of Health and Care Excellent (NICE) or AWMSG. These are commissioning decisions and treatments should therefore be available across NHS Wales. Better collaboration between WHSSC, clinicians and patient organisations is needed to ensure that the IPFR process is not being utilised to re-appraise medicines that should already be available.
It should be made clear to clinicians, patient organisations and medicine manufacturers where commissioning accountability lies, i.e. with the Local Health Board (LHB) or the Welsh Health Specialised Services Committee (WHSSC). This is to ensure transparency of decision making.
The report, born out of the review into Individual Patient Funding Request process, does not address concerns that have been raised by patients and patient representatives throughout the review. A critical mistake relates to the continued use of the ‘exceptionality’ criteria within the IPFR Process. It has been acknowledged that for rare and ultra rare medicines, it is extremely difficult to satisfy the exceptionality criteria.
The review fails to address the current gap that exists between those medicines that are appraised nationally and those that are eligible to receive funding through the IPFR process. In Wales, no alternative route exists for the appraisal of medicines that cannot meet the eligibility criteria of the IPFR process and are not appraised nationally. Many patients will be left with no route of access to life-changing/life-saving treatment due to the absence of a route for appraising rare and ultra rare medicines.
We call on the Welsh Government to address this gap in the current system by developing a separate process for appraising medicines that have not been appraised nationally and that cannot satisfy these ‘exceptionality’ criteria within the IPFR process.
 The Individual Patient Funding Request Process enables clinicians to make an application for funding to enable a patient to access a medicine that is not routinely available on the NHS. The IPFR Policy can be accessed here.
 Review of the appraisal of orphan and ultra-orphan medicines in Wales (2013) Accessed here.
 National appraisal refers to appraisal by the National Institute for Health and Care Excellence (NICE) or the All Wales Medicine Strategy Group (AWMSG). NICE decisions are binding in Wales, however, where NICE are not due to make an appraisal within a 12 month period, AWMSG will appraise the medicine through their own appraisal process.