Phenylketonuria: Medical Treatments

(asked on 29th November 2017) - View Source

Question to the Department of Health and Social Care:

To ask the Secretary of State for Health, whether he plans to allow NHS England to negotiate directly with pharmaceutical companies to improve access to treatments for phenylketonuria.


Answered by
Steve Brine Portrait
Steve Brine
This question was answered on 4th December 2017

There are two policies relating to Sapropterin (Kuvan). The first allows for access to sapropterin for women with phenylketonuria (PKU) who are pregnant and cannot maintain appropriate PKU levels with their diet. This can be found at:

https://www.england.nhs.uk/commissioning/spec-services/npc-crg/group-e/e09/

For the remaining patients, sapropterin is not routinely funded. This policy can be found at:

https://www.england.nhs.uk/commissioning/spec-services/npc-crg/group-e/e06/

This second policy is currently in the process of being reviewed, with a decision expected in 2018. This will involve evaluating any new clinical evidence that has been developed since this policy was originally formed.

NHS England has a number of mechanisms available to secure access to medicines in secondary care from pharmaceutical companies. For example, the Commercial Medicines Unit undertakes procurements through framework agreements that enable individual trusts to purchase medicines at the framework price. Where medicines have been approved by the National Institute for Health and Care Excellence, a patient access scheme may be in place to allow the National Health Service to benefit from a discounted price. The mechanism used will depend on the medicine in any given case.

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