Social Welfare Update: Court of Appeal held NICE was right to use the Highly Specialised Drug procedure to appraise a new drug for treatment of phenylketonuria (PKU)

Thursday 17 December 2020

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The facts

The appellant, an 11-year-old girl, appealed an earlier Administrative Court decision that NICE had not erred in its choice of assessment method when deciding whether to recommend that the NHS should fund a new drug for PKU.

The appellant, with PKU, had undertaken a trial of a new drug, Kuvan, and had been found to be responsive to it. The Secretary of State asked NICE to assess Kuvan and make a recommendation whether it should be provided under the NHS. For this NICE had to decide which of two procedures should be used: its standard procedure, the Health Technology Appraisal (HTA), or the Highly Specialised Technology (HST) procedure which is reserved for highly specialised technologies that met seven criteria set out in NICE's "Interim Process and Methods of the Highly Specialised Technologies Programme" (the 2017 guidance). 

NICE decided that Kuvan did not satisfy three of those seven criteria and decided to assess the drug under the HTA process. The appellant maintained appraisal under the HST process would significantly enhance the prospect of a favourable recommendation of Kuvan.

The judgment

This issue concerned the choice of assessment method. In the decision the three criteria which were found not to be satisfied were 1 (target patient group so small that treatment would usually be concentrated in very few centres); 2 (target patient group distinct for clinical reasons) and 4 (technology expected to be used exclusively in the context of a highly specialised service).

The court was invited to determine the meaning of the 2017 guidance and thus it was necessary to have regard to the statutory context. Nevertheless, the appellant conceded that not all technologies falling within the definition of "highly specialised health technologies" had to be appraised under the HST procedure, and NICE was entitled to establish criteria to determine which health technologies were to be appraised under that procedure. Thus the seven criteria set out in the 2017 guidance were lawful. 

As a result of this position the court held that there was no scope for any rebuttable presumption that all the highly specialised health technologies intended for use in the provision of a service listed in the NHS Commissioning Board and CCGs (Responsibilities and Standing Rules) Regulations 2012 Sch.4 should be appraised under the HST procedure. The meaning of each of the criteria to be determined in accordance with the natural meaning of the words used in context of the decision make.

The court held that the judge had not lost sight of the principle that the meaning of a passage in a policy document had to be interpreted objectively. The interpretation of "used exclusively in the context of a highly specialised service" in criterion 4 was such that the primary readership for which the 2017 guidance was designed consisted of the expert decision-making group with medical professionals and persons in the pharmaceutical industry. It was accepted that such a readership was knowledgeable about NHS terminology and practices and would read and understand the guidance in light of that knowledge. 

The Court held that when criterion 4 was viewed in such a context it was then apparent there was a well-understood distinction between "highly specialist" and "highly specialised" to inform the true meaning of the criterion.  Thus the Court of Appeal held that the judge was entitled to accept that "highly specialised" services were not merely those services commissioned by NHS England, but were a recognised subset of such services. Thus, this subset was to be commissioned through a national commissioning function and delivered at a small number of centres of excellence listed in the "Highly Specialised Services List". As such the judge was right to hold a "highly specialised service" in criterion 4 was a service listed in the Highly Specialised Services List rather than having the broader meaning of a service. As services for PKU patients did not appear on the list, the judge was right to find that Kuvan did not satisfy criterion 4.

In respect of "distinct for clinical reasons" in criterion 2 the Court of Appeal identified that the judge found that there was no clear bright-line cut-off between PKU patients who would benefit from Kuvan and those who would not. Nevertheless, the group of PKU patients responsive to Kuvan was distinct from those who were not and it was hard to see why the reason for their distinctiveness should not be regarded as clinical. The court noted the danger of over-complicating the issue on insisting on a requirement that a group was only distinct for clinical reasons if it could be identified without a trial of the drug in question. However, as criterion 4 was not met, it was unnecessary to reach a final decision on criterion 2 but merely to comment that the better view was that it was met.


It can be seen that the application of guidance referred to by the court appears to be designed to be understood to be used and understood by specialist expert decision makers. This was the key part of the focus of decision making and can thus be frustrating for families and carers, especially when taking part in a trial which seems successful.

It is noteworthy that the Court of Appeal identified that it would be preferable for all of the criteria in the guidance to be expressed in plain language which could be readily understood by patients and those caring for them even though the guidance was designed for use by specialists.

It would seem that the simplification of such guidance would be welcome throughout healthcare provision and commissioning.


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