Question to the Department of Health and Social Care:

To ask Her Majesty’s Government what the process is for ensuring that quick decisions are made about the treatment of critically ill infants suffering from hypophosphatasia in cases where effective drug treatment is available.

National Health Service commissioners are legally required to fund drugs and treatments recommended in National Institute of Health and Care Excellence (NICE) Highly Specialised Technologies evaluations. If the drug is not recommended, and until NICE’s final guidance is issued, it would be for NHS England to decide whether to make it available to patients.

NICE is currently evaluating asfotase alfa (brand name Strensiq) for the treatment of paediatric-onset hypophosphatasia under its Highly Specialised Technologies programme, which looks at drugs for very small patient populations.

The Evaluation Committee’s preliminary recommendation, published in December 2015 for consultation, was not to recommended asfotase alfa, within its marketing authorisation, for long-term enzyme replacement therapy in paediatric-onset hypophosphatasia to treat the bone manifestations of the disease.

NICE makes its recommendations based on the available evidence and in consultation with stakeholders, including manufacturers and patient groups.

This is not NICE’s final guidance and its recommendations may change after consultation. NICE currently expects to publish its final guidance to the NHS in October 2016.