Our Work

NICE’s evaluation of highly specialised technologies

Last Reviewed 30/04/2014

In April 2014 Genetic Alliance UK launched its first Patient Charter, which highlighted the concerns patients have about the interim Highly Specialised Technology (HST) Evaluation Programme, the new approach within the National Institute for Health and Care Excellence (NICE) used since May 2013 to determine whether very rare disease treatments should be recommended for NHS-wide commissioning in England.

It has been shaped through collaborative discussion across the patient group community, specifically including groups that might have to participate in a future HST evaluation. As a result, the Charter represents a strong, clear and consistent statement, outlining key recommendations to be communicated back to NICE, from the perspective of patients and their families. 78 patient groups signed up to the 29 recommendations the Patient Charter made to improve NICE’s Highly Specialised Technology (HST) Evaluation Program.

The diagram below illustrates the key stages of NICE’s HST evaluation process annotated with each of the 29 recommendations made in this Patient Charter:

HST Charter Recommendations Diagram

Ensuring that HST evaluation is fit for purpose in the wider commissioning landscape

1          The HST evaluation process’ relationship with the wider commissioning landscape should be clarified (Genetic Alliance UK, NHS England, NICE)

2          Consideration should be given to whether an HST evaluation or another route to commissioning is most appropriate during topic selection (NICE)

3          Representatives of NHS England’s specialised services team should be non-voting experts at NICE’s Evaluation Committee meetings (NHS England’s specialised services team, NICE)

Enabling the patient voice

4          Patient communities involved in an HST evaluation should be supported in the formation of patient groups if they do not already exist (Department of Health, Genetic Alliance UK, NICE)

5          Mentoring and support for all patient groups should be available from a NICE-contracted third-party (NICE)

6          NICE should seek out existing and newly established patient groups for the duration of the HST evaluation process (NICE)

Scope and transparency: getting the right technologies into the HST evaluation process

7          Topic identification and selection processes, methods, mode of deliberation and outcomes should be fully transparent and publicly available (Department of Health, NICE, NIHR Horizon Scanning Centre)

8          Patient groups should be involved in the process of topic identification and selection (NICE, NIHR Horizon Scanning Centre)

9          Topic selection criteria need to be tightened to clarify their meaning as some are at risk of misinterpretation (Department of Health, NICE)

10        Repurposing of medicines should be encouraged by allowing treatments with multiple indications to qualify for multiple HST evaluations (NICE)

11        The definition of ‘clinically distinct’ requires updating so that it reflects a modern understanding of the genetic and molecular basis of disease and the frequent variation in clinical presentation common in rare diseases (NICE)

12        The number of HST evaluations carried out should be determined exclusively by the number that meets the revised selection criteria, which includes a consideration of appropriateness (Department of Health, NICE)

13        The timeline of HST evaluation should permit ‘clock stops’ to allow flexibility and enable all patient groups to participate (NICE)

Finding out where and how the patient voice is taken into account

14        The nature and format of the proposed patient group evidence submission needs clarification (NICE)

15        An explanation of ‘impact beyond direct health benefits’ and its value in an HST evaluation is required (NICE)

16        The Evaluation Committee should be able to accommodate sufficient ‘nominated experts’ so as to reliably reflect the diversity of opinion in the relevant patient community (NICE)

17        Nominated patient experts should be permitted to make a brief, formal presentation to the Evaluation Committee (NICE)

18        The patient evidence submission should be seen in its original form by the Review Group and Evaluation Committee (NICE)

19        The patient evidence submission’s use and impact should be communicated back to patient groups (NICE)

Updating the health economist’s tool box

20        All calculations made by health economists during an HST evaluation should be communicated back to patient groups (NICE)

21        For HST evaluations, a measure of health outcomes more applicable to rare diseases should be developed and used in place of the EQ-5D (Department of Health, Genetic Alliance UK, NICE (NICE International))

22        Expectations of clinical trial data must be pragmatically aligned to reflect the limitations of data derived from studies on small numbers of patients (NICE (Evaluation Committee))

23        NICE should work with medicine regulators in a coordinated way to provide advice to companies regarding clinical trial design and data collection; and guidelines for the manufacturer’s evidence submission (EMA, EUnetHTA, NICE, pharmaceutical companies)

Generating additional evidence

24        To facilitate equitable access to new treatments, research recommendations should not be subject to restrictive eligibility criteria (NICE)

25        Patients should be involved in outlining the nature and timeline of any post-evaluation research recommended by NICE (NICE)

26        Use of NICE-commissioned HSTs in the NHS should be monitored as part of routine clinical care to ensure expected patient outcomes are met (NHS England, NICE, Public Health England)

27        Research that examines dosage and clinical trials evaluating the impact of dosage should be encouraged, with the view to making HSTs more effective and more affordable NHS England

Risk versus effectiveness in a NICE evaluation

28        Re-examination of benefit/risk by NICE should be explicitly justified, involve patient consultation and refer to patient testimonies collected by the EMA        (EMA, EUnetHTA, NICE)

29        Patients should make the ultimate decision on whether the benefits of a new drug outweigh its risks, in partnership with their doctor (NICE)

Join our alliance of 180+ patient charities

Join us