By Sian Claire Owen

The UK National Institute for Health and Clinical Excellence (NICE) has released its updated guide to their technology appraisal appeals process. Interested parties have until 22 March 2010 to comment on this document before the new version is set in stone. At 30 pages, it is dry material indeed, but important nonetheless.

Established in 1999, NICE was created to provide guidance on the use of health technologies within the UK National Health Service (NHS). For a new drug or technology to be available on the NHS, it must be approved by NICE. Technologies are assessed on clinical efficacy and cost-effectiveness.

Producing clinical guidance is a laborious process, taking up to 24 months (11 to 13 months if guidance is urgently needed), and the chance to appeal the Final Appraisal Decision (FAD) must be squeezed into this time frame. And ‘squeeze’ is the operative word – appellants have only 15 working days to appeal an FAD from the day it is published in any medium, including on the NICE website.

There is no doubt as to the importance of the appeals process. Barbara McLaughlan is the Campaigns Manager for the Royal National Institute for the Blind (RNIB) and Chair of Patients Involved in NICE (PIN), a group of patient organisations involved in NICE decision-taking. As she explains: “Having a NICE FAD appeals process is essential because the health economics that support NICE decision-making are not an exact science.”

“Decisions can give grounds to appeal and it is not in the interest of any stakeholders to forgo that option.” However, she adds that: “Any controversies about the assumptions used in the decision-making process should be ironed out during the ACD and FAD consultation process. Having to go to appeal is not in the interest of anybody, least the patient who is hoping to gain access to new treatments.”

The past year has seen some fundamental changes in the technology appraisal process to oil the wheels and make the system more efficient. Updated guides to multiple and single technology appraisal processes, and the Patient Access and Flexible Pricing Schemes were introduced, all of which have impacted the appraisal process – hence, the revised appeals manual.

Alice Law, a spokesperson for NICE says: “The aim of these changes is to ensure that the grounds of appeal are consistent with those outlined in the Secretary of State’s Directions. But the revised manual also provides greater clarification on the appeal process.”

This clarification gives us more information on the modus operandi of NICE appeals, as well as details on exactly what is acceptable and what is not. There also has been some ‘textual pruning’ to eliminate repetition between sections. This is undoubtedly useful information, saving first-time appellants precious time, and preventing them from becoming tangled in a bureaucratic quagmire.

Centred at the core of the FAD appeal process are three grounds, and any appeal is strictly limited to the scope of these grounds:

Ground One: The institute has failed to act fairly and in accordance with the appraisal procedures set out in the Institute’s Guide to the Technology Appraisal Process

Ground Two: The Institute has prepared guidance, which is perverse in the light of evidence submitted.

Ground three: The Institute has exceeded its powers.

These remain largely unchanged, with one minor revision in Ground Two, which now states:

Ground Two: The Institute has formulated guidance, which cannot reasonably be justified in the light of evidence submitted.

“This amendment was not intended to change the scope of appeals,” says Law. “We felt that ‘cannot reasonably be justified’ means the same as ‘perverse’ but was easier to understand,” which is in line with their aim of keeping it clear.

McLaughlin was involved in the NICE FAD appeals process brought forward by Derbyshire Primary Care Trust (PCT) on the decision not to approve Macugen (used in the treatment of macular degeneration) for use on the NHS. She agrees that greater clarification is needed. “Personally I would have expected one or two of the appeal grounds put forward by Derbyshire PCT and the manufacturer of Macugen to succeed. It was surprising to me that none of them did, and clarification of the process may have helped.”

The proposed appeals process also irons out any discrepancies in the time it takes to consider appeals – the final appeal decision should be considered within 10 working days of receipt, and the final guidance published on the NICE website 15 days after the Guidance Executive meeting.  New updates recommend that the Department of Health apply funding direction as soon as the NICE guidance is published, in any medium, so consistency is key.

Given her experience with this process, McLaughlin adds that if she personally could introduce further changes to the guidelines, she would allow the introduction of new evidence and arguments in cases where the appeals process becomes lengthy. “Where appraisals stretch to a year and more there may well be occasions where new evidence becomes available that has a bearing on the decision about the safety, efficacy and cost-effectiveness of a new treatment,” she says. “In these cases it should be possible for the appeals panel to refer a decision back to the Appraisal Committee.”

So will McLaughlin and colleagues be commenting on these updated guidelines? “We are in the process of putting together a response,” she says. “Our draft will then be circulated among members of PIN, and then we will submit a joint response to strengthen the patient voice in these discussions.”

All in all, these alterations are designed purely to make the appeals process quicker and slicker. When asked who would benefit most from these changes, Law says: “The appeals manual is targeted towards consultees involved in technology appraisals who would like to lodge an appeal.” In other words everyone’s a contender, time willing.

  • The updated guide to the technology appraisal appeals process is available on the NICE website [Link: http://www.nice.org.uk/]
  • Interested parties have until 22 March 2010 to comment on the revised appeals manual.

The updated guide clarifies and standardizes the acceptable grounds for appeal and provides a clearer picture of the appeal process.