
MPs back ultra-rare disease treatment
pharmafile | March 12, 2014 | News story | Research and Development, Sales and Marketing | BIA, NHS, UK, bates, personalised, rare disease, research
Most MPs in the UK believe there should be no limit to the price per patient for treating people with very rare diseases, according to new BioIndustry Association (BIA) research.
The finding comes in a report called ‘Very rare diseases, complex issues’, which looks at how a new evaluation framework for ultra-orphan medicines in England might work – ahead of a full consultation on the issue, which is to be held later this year by NICE.
The BIA says its survey shows 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’ ability to pay – and 63% do not believe there should be a maximum price per patient for treating people with such conditions.
“It is vitally important that patients with rare diseases get access to clinically effective medicines and that there is no discrimination against such patients when it comes to making innovative treatments available,” said MP Andrew Miller, chair of the House of Commons Science and Technology Select Committee.
“Equitable access for all patients, whether the condition is rare or common, is a fundamental principle of the NHS and the new evaluation procedure for such medicines will be an important component of this,” he added.
BIA will use the NICE consultation to promote a discussion on the need for a separate evaluation process for such products, and to clarify what type of medicines are suitable for this route and which criteria should be taken into account.
The new framework should build upon the validated Advisory Group for National Specialised Services (AGNSS) one, BIA believes.
“This should bring some certainty to a period of flux since AGNSS was disbanded,” a BIA spokesman told Pharmafile. “Industry, stakeholders and most of all patients have been in limbo with a series of ad hoc arrangements to evaluate ultra-orphan products being used, such as for Kalydeco from Vertex.”
NICE is to issue final guidance this year for Alexion’s Soliris (eculizumab) for aHUS patients, after draft guidance earlier this month suggested NICE was minded to reject it unless the manufacturer provided more information on R&D costs.
BIA also believes that NICE’s new evaluation regime could point the way towards the world of more personalised medicines, where increasingly products will be developed for specific sub-sets of larger patient populations.
Although no formal link has been made between the two, BIA says this was a ‘dominating theme’ with policymakers during the BIA’s roundtable discussion on the subject in the Commons.
And NICE has made a couple of interesting noises on this subject. “For example, the product must be ‘clinically distinct’ e.g., not a subset of a larger condition, and the manufacturer must also only be using that product for that indication and no others,” the BIA spokesman went on.
“This is largely to prevent products with several indications – all of which on their own are very small – going through this route,” he said. “So policymakers are aware of the issue.”
The BIA’s report recommends that more consideration should be given to the incentives to develop orphan and ultra-orphan medicines and to avoiding the situation where medicines which work have been developed but patients cannot access them.
There is also a need to ensure that there is an integrated implementation and service delivery, BIA concludes.
Adam Hill
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