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BIA calls for separate framework to evaluate and commission medicines for rare and very rare diseases

In its response to NHS England (NHSE)’s consultation on investing in specialised services, the UK BioIndustry Association (BIA) calls for a separate framework to evaluate and commission medicines and treatments for rare and very rare diseases.

NHSE’s consultation document sets out suggested principles and processes for specialised commissioning for stakeholder feedback. Whilst the BIA welcomes the opportunity to contribute and comment on NHSE thinking on this important issue, our response raises several concerns and suggests that a different and distinct approach is required for the consideration of medicines and treatments for rare and very rare diseases.

In addition to responding to the specific questions set out by NHSE, the BIA proposes that:

  • Medicines and treatments for very rare diseases are assessed by the National Institute for Clinical Excellence (NICE) under its Highly Specialised Technologies (HST) programme.
  • National fit for purpose frameworks for the evaluation and commissioning of medicines and treatments for rare diseases are also required, which are separate and distinct, based on the impact of treatment on unmet medical need, burden of illness and impact on patients and carers rather than a blunt assessment of affordability.
  • NHSE should be clear that it is a commissioning body and not an evaluation body. Whilst NHSE should of course have fit for purpose interim commissioning policies, assessment and evaluation must only be undertaken by NICE and equivalent bodies in devolved administrations.

Steve Bates, CEO of the BIA commented:

“A separate approach for rare and very rare diseases is essential because the small numbers involved introduce uncertainty into assessment and commissioning. Almost three quarters (72%) of the public agree that patients with a very rare disease should have the same access to treatments as patients with common diseases. This demonstrates the high importance the public place on equitable patient access and the BIA believes the solution is for separate frameworks to be agreed and implemented”.

“These treatments are often at the cutting edge of innovation. Ensuring that they are taken up rapidly in the NHS not only benefits patients but sends a signal to global biotech to utilise and invest in the UK’s R&D capability.”

The full BIA response to NHSE’s consultation document, Investing in specialised services can be downloaded here

NHSE’s consultation document, Investing in specialised services, can be found here