When the Outcome of an Appraisal Is Not NICE: An Overview of the Appeal Process

HTA QUARTERLY | SPRING 2017

When the Outcome of an Appraisal Is Not NICE: An Overview of the Appeal Process

Author: David Campbell, PharmD, MS

The National Institute for Health and Care Excellence (NICE) was established in 1999 with the goal of achieving the best possible outcomes and making the most effective use of the resources available in England’s National Health Service (NHS). NICE has gained notoriety for its evidence-based appraisal guidance on health technologies which include medicines, medical devices, diagnostic techniques, surgical procedures, and health promotion activities. Decisions are based on clinical and economic evidence, and procedures are detailed in the guide as to the methods of technology appraisal, most recently updated in 2013. By law, the NHS is obligated to fund and resource medicines and treatments recommended by NICE’s technology appraisals, so its decisions are very important to both industry and patients.

While the agency has sought to advance transparency and efficiency in healthcare through evidence-based guidance, some appraisal decisions have been criticized. In an effort to be fair to all stakeholders and to ensure that evidence has been properly considered, NICE decisions can be appealed. In this article, we provide a brief overview of the procedures for technology appraisal by NICE, and we explore the appeals process when a decision is challenged.

Overview of the Technology Appraisal Process

One of the roles of NICE is to provide guidance on new health technologies within England’s NHS. Technology appraisals take the form of either: 1) a single technology appraisal (STA), which covers a single technology for a single indication; or 2) a multiple technology appraisal (MTA), which normally covers more than 1 technology, or 1 technology for more than 1 indication. Each appraisal by NICE can have 1 or more recommendations and can contain more than 1 type of recommendation.

Five Types of Decisions by NICE

Recommend: The treatment is recommended for use as described by the European Medicines Agency (EMA) and Medicines & Healthcare Products Regulatory Agency (MHRA) or how it is to be used in clinical practice in the NHS (or both).
Optimised: The technology is recommended for a smaller subset of patients than originally stated by the marketing authorization.
Only in Research: The treatment is recommended only for use in research (clinical trial) and generally recommended when more clinical evidence is needed.
Not Recommended: There is a lack of evidence for clinical effectiveness of the technology, or it is not considered a cost-effective use of NHS resources compared with current practice.
Recommended for Use in the Cancer Drugs Fund (CDF): Only an option for cancer appraisals; this decision occurs when there is early evidence to support a clinical benefit and it is believed that evidence can be generated to support the cost-effectiveness.

NICE Decision Appeals

Following issuance of the draft final guidance for a technology appraisal, all consultees (which include the manufacturer, patient organizations, and others involved with the technology appraisal) have the right to submit an appeal within 15 working days on the basis of 2 strictly limited grounds:

Ground 1: In making the assessment that preceded the recommendation, NICE has: a) failed to act fairly; or b) exceeded its powers.
Ground 2: The recommendation is unreasonable in light of the evidence submitted to NICE.

Note: Originally, parts a and b of Ground 1 were classified individually, and there were 3 grounds for appeal: 1) the Institute failed to act fairly and in accordance with its procedures; 2) the Institute prepared guidance which is perverse in light of the evidence submitted; or 3) the Institute exceeded its legal powers.

When developing an appeal letter, it is important that the format and content adhere to NICE’s guidance, which includes providing the evidence to support each point of the appeal in a clear and succinct manner. The appeal process is not a way to present new evidence or information, and it is not an opportunity to reopen arguments and issues that the committee has decided previously. Similar to NICE appraisal meetings, limited seating is available for members of the public to observe the appeal hearing.

The Vice Chair of NICE is responsible for reviewing the appeal, assessing the arguability and validity of each of the appeal points, and determining if an appeal hearing (oral or written) will be held. The response is provided in an initial scrutiny letter; if an appeal is granted in this letter, the appeal will then be heard by the Appeal Panel. The Appeal Panel consists of 5 members including: the Appeal Panel chair; a non-executive director of NICE; a person with life science industry experience; a lay member who may have experience being a patient, caregiver, or member of an organization that represents patients and caregivers; and a person with experience with the NHS.

Within 15 days after the oral/written appeal hearing, the Appeal Panel must send its decision in writing to NICE to be considered by NICE’s Guidance Executive. The Guidance Executive is a team comprising the Executive Directors and Centre Directors at NICE who are responsible for approving the final appraisal determination (FAD) before publication. If 1 or more points of the appeal are upheld, the Appeal Panel’s decision and the revised final guidance may be published on the NICE website within 15 working days of the Guidance Executive meeting. Additional time may be required if it is necessary for the Appraisal Committee to reconsider the FAD.

If the appeal is considered by the Appeal Panel and the panel decides all points of the appeal are dismissed, and there are no changes to the FAD, the Appeal Panel will notify the Guidance Executive of the decision, and NICE will publish the Appeal Panel’s decision and the final guidance within 10 working days of the Guidance Executive meeting. In some cases, the appeal is dismissed but the Appeal Panel asks for minor revision to the FAD or asks NICE for clarification on the process or methods used in its decision.

Figure 1. NICE Appeal Process

Examination of Past NICE Appeals

A retrospective review of technology appraisal appeals and their associated outcomes from 2000 to 2011 provides insight into the substance of appeals, stakeholders involved in the appeal process, and outcomes. In this study, a quarter of appraisals resulted in an appeal, and 41% of these appeals were upheld (Figure 2). The most common ground for appeal was perversity of the decision with 59%, based on misinterpretation of the clinical or cost-effectiveness evidence (now classified under Ground 2, “cannot be reasonably justified”). While stakeholders from industry were the most common appellant type, professional organizations and patient groups had the highest percentage of appeals upheld (Figure 3).

Figure 2. NICE Appeal Decisions of the 25% That Pass Initial Scrutiny of Appeal

Figure 3. Rate of NICE Appeals Upheld by Submitting Stakeholder

While these previous examinations of NICE appeals provide important insights, crucial questions remain. Appeal guidance has been periodically updated, with new guidance issued in August 2010 and again in February 2014. Appeals from the past 5 years were not included in the review, and significant changes in the frequency and outcomes of appeals may have occurred with guidance updates.

Conclusion

The appeal process for STAs and MTAs is an important and effective mechanism of ensuring that appraisal criteria are consistently and fairly applied to all assessments and that clinical and cost-effectiveness evidence is appropriately considered. Previous research suggests the appeal process is being successfully utilized by various stakeholders to lead to substantive changes in decisions, but new research is needed to assess the impact of updated guidance and successful utilization of the appeal process by stakeholders. Given the short time period for submitting an appeal, stakeholders need to understand the appeal process and guidance so they may act quickly.

The article should be referenced as follows:

Campbell D. When the outcome of an appraisal is not NICE: an overview of the appeal process. HTA Quarterly. Spring 2017. April 1, 2017.

Sources

NICE. NICE technology appraisal guidance. https://www.nice.org.uk/about/what-we-do/our-programmes/nice-guidance/nice-technology-appraisal-guidance. Accessed April 14, 2017.
NICE. Guide to the processes of technology appraisal. September 2014.
https://www.nice.org.uk/process/pmg19/chapter/appeal. Accessed April 14, 2017.
NICE. Guide to the technology appraisal and highly specialised technologies appeal process. February 2014. Last updated April 2017. https://www.nice.org.uk/process/pmg18/chapter/Foreword. Accessed April 14, 2017.
Drummond M, Sorenson C. Nasty or nice? A perspective on the use of health technology assessment in the United Kingdom. Value Health. 2009 Jun;12 Suppl 2:S8-13.
Eaton JN, Hawkins N. A review of the NICE appeals process. Value Health. 2011 Nov;14(7):A343.
NICE. NICE and the life sciences industries. 18 January 2017. https://www.nice.org.uk/Media/Default/News/NICE-and-the-life-sciences-industry.pdf. Accessed April 14, 2017.