29 Ways to Improve Orphan Drug Evaluation

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On Monday of this week, the Genetic Alliance UK published a landmark patient charter outlining patient and advocate concern over how the UK’s National Institute for Health and Care Excellence (NICE) were determining which orphan treatments to make available on the publicly funded National Health Service (NHS). the charter is endorsed by a staggering 77 patient advocacy groups from across the UK.

There has been much debate in the UK over the public funding of rare disease treatments by the NHS. A number of government ministers recently announced their continued support for the funding of rare disease treatments, and this is a theme that is often echoed by the public. But in the wake of a number of rejections by NICE, and the current climate of heavy funding cuts in England, there is suspicion that NICE are not fairly considering potential orphan treatments.

the patient charter lists a number of recommendations and who should be dealing with them. whilst of course this list is aimed at the UKs national health service, the recommendations made are by no means unique to the UK. With the rolling out of Obamacare and increasing debate over the role of payers and insurers role in the prescribing of orphan drugs, these recommendations have world wide appeal.

No. Recommendation Action by
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 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

Download the full report here, and view the press release here.

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