Early in 2001 the department of Health gave the National Institute for Clinical Excellence (NICE) the task to appraise both the clinical effectiveness and cost effectiveness of Human Growth hormone (GH). That appraisal was divided into separate appraisals for children and for adults. The Foundation was invited to participate, and we embarked on this task with some trepidation. We sought a positive outcome from the NICE appraisal that would help to overcome the post-code lottery for funding of GH.

We had no idea how much time and energy would be required.

Our Submission

In the June 2001 Patsy Perrin, a Trustee, devised and analysed a questionnaire completed by 360 members which, at the time, represented 23% of all GH adult patients. The Foundation’s submission, promoting the clinical and cost effectiveness of GH for adults was based on both evidence from endocrinologists and the findings of this questionnaire.

The next stage was a meeting in November 2001 with the Appraisal committee at which Patsy was joined by Dr Janet Harbour and many eminent endocrinologists representing the professional bodies. The NICE committee considered the Assessment report commissioned by NICE and the input from the participants. The experience was not good. As a result of our protests a second Assessment report was commissioned and the Appraisal meeting reconvened.

The Initial Rocky Appraisal Process

In April 2002 NICE published its first provisional findings, saying that

“based on evidence of Random Control Trials the effectiveness of Somatotrophin treatment in adults with GH deficiency in terms of quality of life remains unproven.”

Together with the clinicians we took due issue with many points. The Appraisal Committee stuck to their views and when the FAD (Final Appraisal Determination) was published saying that no-one could have GH we determined to Appeal.

One of the worse aspects of the appraisal was the blanket of confidentiality NICE imposed which limited our access to patients and their carers, to tell them what was happening and to enable them to take a wider role in the appraisal. It caused an indeterminate amount of stress for many people. (Appraisals that started after ours were not subject to this restriction.)

The Appeal

We were allowed to appeal against the decision, under categories determined by NICE, and produced a very robust appeal against the FAD.

At the same time we...

• Encouraged our members to contact their MPs, and hundreds responded,
• Took advantage of the recent review of NICE carried out by the Parliamentary Health Select Committee. We quoted parts of the Health Select report in our appeal document,
• Reported progress to members of the Health Select Committee,
• Were delighted that Mr Roger Berry, MP for Kingswood, Bristol tabled an Early Day Motion No 1683 concerning the negative view of adult GH taken by NICE.

The Appeal hearing held in August 2002 and chaired by Sir Michael Rawlings the Chairman of NICE, was an interesting meeting.

A healthy number of the appeal points raised by ourselves and our fellow appellants were successfully upheld.

The appraisal was referred back to the Appraisal Committee for reconsideration of several points, including the all-important selection criteria.

To our great delight the confidentiality restriction was lifted and information could be published.

We realised that we were the first technology appraisal to have won their appeal and to have a ‘second round’.

Creating new ground – a second round.

So we embarked on another round of documents, arguments and meetings to determine three key points. These were:

1. The identification of a group of GH deficient adults who would derive the most benefit from hormone replacement therapy (in terms of cost-effectiveness), and to develop selection criteria for this group if possible.
2. Guidance relating to the continuation of treatment into adulthood for children who have been receiving GH therapy, and
3. To ensure that any particular needs of survivors of childhood cancer with ‘iatrogenic’ (caused by people) pituitary failure are adequately considered.

The confidentiality restrictions were lifted, so the appraisal was at last being treated in the same way as other appraisals.

Together with the Society for Endocrinology, we proposed to NICE that a preparatory meeting might be held with them to map out how the remainder of the appraisal might be conducted. That historic meeting was held in late November 2002 and involved ourselves, the professionals and the pharmaceutical companies.

So the process started again with an Appraisal meeting, a provisional and the final decision (FAD). Again we identified issues and prepared for a second appeal meeting in July 2003.

The second Appeal

Despite the fact that the appeal panel members should “have no prior involvement in the appraisal in question”, this was chaired again by Sir Michael Rawlins. We fought to win the right for existing adult patients to be able to stay on treatment, and not to have to ‘pass the tests’ – although this has not been acknowledged as an ‘upheld’ point in the appeal documentation. In fact we are deemed to have misinterpreted this point.

Situation at the end of August 2003

The guidelines were published on NICE ’s website on 27th August 2003.

Together with the endocrinologists we believed that we could live with the proposals and urged NICE to produce a swift outcome so that there could be clarity about the provision of GH for adults.

In turn we hoped that this will turn out to be a powerful tool in combating post-code funding of GH by Area Health Authorities, Primary Care Trusts and general hospitals.