Court of Appeal upholds regulatory judgment in technical field

Recently, the Court of Appeal gave its decision in R (Cotter) v NICE [2020] EWCA Civ 103. The case challenged a decision by NICE which made it harder for a patient to obtain a particular medicine than would have been the case had NICE taken a different approach.

Background

The Claimant sought judicial review of NICE's decision to assess the use of Kuvan to treat phenylketonuria ("PKU") under the Health Technology Appraisal ("HTA") procedure instead of the Highly Specialised Technology ("HST") procedure. Under the HTA Procedure NICE will only recommend a health technology for use in the NHS if it will not cost more than £30,000 for each Quality Adjusted Life Year whereas under the HST procedure, NICE will recommend a drug costing up to £100,000 per QALY gained. Therefore, the test for a favourable recommendation is easier to satisfy under the HST procedure because a drug can be less cost effective than under the HTA procedure. However, the Court of Appeal agreed with the High Court that NICE was entitled to assess whether Kuvan should be recommended on the NHS using the HTA procedure.

The HST Procedure is reserved for highly specialised technologies which meet seven criteria set out in a document entitled 'Interim Process and Methods of the Highly Specialised Technologies Programme' issued by NICE in April 2017 (the "2017 Guidance"). NICE determined that four of the seven criteria set out in the 2017 Guidance were satisfied but three criteria referred to below were not satisfied. Since the requirement under the 2017 Guidance was that all the criteria needed to be satisfied for appraisal under the HST procedure, NICE concluded that Kuvan should be assessed under the HTA procedure.

The Claimant's case was that all seven criteria were satisfied in the case of Kuvan and the decision to use the HTA procedure was therefore unlawful. At first instance the Court concluded that the application of the guidance to the particular facts was a matter for the judgment of NICE and was susceptible to challenge only on irrationality grounds. The judge concluded that the criteria required the exercise of expert judgment and the use of expert knowledge and that there is always a high threshold for irrationality cases. The Court of Appeal agreed with this general approach. However, its interpretation of Criterion 1 and 2 in the 2017 Guidance and their application to the facts was different.

Grounds of Appeal and Court of Appeal’s Decision

The Claimant had five grounds of appeal.

Ground 1 – The statutory context

The Claimant argued that the judge had failed to have proper regard to the statutory context. While Males LJ accepted that it was necessary to have regard to the statutory context, he found that once it is conceded that not all technologies falling within the definition of "highly specialised health technologies" have to be appraised under the HST procedure, NICE was entitled to establish criteria to determine which health technologies should be appraised under that procedure and that the seven criteria set out in the 2017 Guidance were lawful. He saw no scope for any refutable presumption in interpreting the Guidance that all the health technologies intended for use in the treatment of rare or very rare diseases should be appraised under the HST procedure.

Ground 2 – The objective interpretation

The Claimant argued that the judge had failed to recognise that interpretation of the Guidance was an objective matter to be determined by the Court but the Court of Appeal found that the judge had not fallen into that error.

Ground 3 – "the target patient group is distinct for clinical reasons" (Criterion 2)

The Court held that PKU sufferers who are responsive to Kuvan are distinct from PKU sufferers who are not responsive and that this distinction was clinical. The fact that the licensing application recognised that not all patients with PKU would respond to Kuvan, allowed a degree of judgment to the treating physician, so that the drug was only prescribed to those who are responsive to it and this did not detract from the conclusion that patients who were responsive could be regarded as a distinct group for clinical reasons. Therefore, whilst the Court of Appeal did not reach a final decision on the point, the better view was that Criterion 2 was satisfied in the present circumstances.

Ground 4 – "the technology is expected to be used exclusively in the context of a highly specialised service" (Criterion 4)

The Court of Appeal accepted NICE's argument that the reference to highly specialised service in Criterion 4 had a specific meaning and was distinct from "highly specialist" in the context of NHS terminology. The primary readership of the 2017 Guidance was expert decision-making groups together with medical professionals and persons engaged in the pharmaceutical industry. When viewed in this context, reference to "highly specialised service" in Criterion 4 is a reference to a service which is not only commissioned nationally by NHS England but is listed in the Highly Specialised Services List.

Ground 5 – "the target patient group … is so small that treatment will usually be concentrated in very few centres in the NHS" (Criterion 1)  

The Court found that given the decision made on Criterion 4, there was no need to decide whether this criterion had been applied properly, though if Criterion 1 had been decisive, it would probably have been necessary to remit this issue to NICE so that a decision could be made on the correct legal basis.

The Court finally suggested that all of the criteria for appraisal of a "highly specialised health technology" under the HST procedure should be set out in plain language so that it is readily understood by patients and those caring for them.

Conclusion

The case is therefore an example of a patient seeking to use judicial review to make a medicine more freely available by seeking to require NICE to adopt a different procedure but also demonstrates that a Court will not lightly find that a regulator such as NICE has misapplied the statutory criteria. In this case the difficulty for the claimant was that they needed to show that NICE had been wrong on three out of seven criteria. The Court of Appeal was inclined to agree on one or two of those three but was not persuaded on all three.