Access to drugs for very rare diseases should be based on clinical need, say majority of UK MPs
There should not be a maximum price per patient, according to 63% of MPs
The majority of MPs in the UK believe there should not be a maximum price per patient for treating people with very rare diseases, finds research commissioned by the BioIndustry Association (BIA).
The research, published in a report entitled: 'Very rare diseases, complex issues', found that 68% of MPs agree or strongly agree that access to treatments on the NHS for very rare diseases should be based on clinical need and not the NHS's ability to pay.
It also found that 63% of MPs disagree that there should be a maximum price per patient for treating people with very rare and complex diseases.
Almost half (49%) of MPs who expressed a preference said that the National Institute for Health and Care Excellence (NICE) should not apply its standard mathematical methodology to evaluating very rare diseases, while just over one-fifth (22%) agreed that it should.
Ultra-orphan medicines should have a separate evaluation framework
Collectively rare diseases affect roughly 1 in 17 people, approximately 3.5 million people in the UK. The research and development of rare and very rare diseases (or orphan and ultra-orphan drugs) is therefore a critical undertaking. Given the high costs of developing a drug, and the very small patient populations found within the UK for any one rare condition (around 500 or less for ultra-orphan medicines), the report asks how the UK can ensure equitable patient access to drugs for these diseases at a cost that is acceptable to the tax payer.
The report makes a number of recommendations, including giving consideration to the incentives to develop orphan and ultra-orphan medicines and avoiding clinically effective medicines being developed that patients cannot access. It also says ultra-orphan medicines should have a separate evaluation framework because of the unique characteristics and challenges involved with evaluating treatments for very rare conditions.
There is also a need to ensure integrated implementation and service delivery in recognition that the functions are now split between NICE and NHS England.
Brendan Martin, General Manager for UK and Ireland at Genzyme Therapeutics, commenting on the report, said: 'The progress seen in the development of new therapies for rare diseases has been little short of revolutionary in recent years. Diseases which might affect only a handful of patients in the UK are now the subject of intensive research programmes and new therapies are being approved.
'For this to continue, and more patients with rare and complex conditions to benefit, it is imperative that the NHS develops appropriate mechanisms to recognise innovation and ensure patients receive new, often lifesaving, medicines.'
Steve Bates, BIA Chief Executive Officer, said the report outlines why the evaluation of very rare diseases not only has relevance to the many patients in the UK who could benefit from innovative new medicines but also has far wider implications on how stratified and personalised medicines are reimbursed in future.
'2014 will be a crucial year for rare diseases with the new evaluation procedure being consulted upon by NICE and it will be important for patients and the sector that we get this right,' he said.
