1.  Executive Summary

  1.1  The National Osteoporosis Society (NOS) is closely involved, as an active stakeholder, in the development process of the clinical guideline on osteoporosis and the technology appraisals on the primary and secondary prevention of osteoporotic fragility fractures in postmenopausal women since their inception. This written evidence is therefore limited to the NOS' experiences of NICE, drawing on the development of NICE guidance for osteoporosis to illustrate our points where possible.

  1.2  The development of the technology appraisals for osteoporosis has been a lengthy process and is now entering its sixth year. The NOS has some concerns about the transparency of the methodology that NICE has used in its economic modelling to reach its conclusions in this particular case. We are also concerned that since being actively involved in the development of the scope for the technology appraisals, it has changed significantly during the long development process. At the present time, much of the remit of the appraisals has been transferred to that of the clinical guideline on osteoporosis, which does not have mandatory status.

2.  Introduction

  2.1  The NOS is the only national charity dedicated to improving the prevention, diagnosis and treatment of osteoporosis. The charity provides a range of services in support of people with, and at risk of, osteoporosis. Drawing on the unrivalled expertise of the numerous health and social care disciplines involved in this disease area, together with our members, volunteers and staff, the NOS works for the improvement of treatment options and care for patients throughout the country.

  2.2  Osteoporosis is a chronic disease which can result in painful and debilitating fractures, particularly in the hip and spine (in a similar way to high blood pressure which is a chronic condition that can lead to a stroke). Almost half of all women and 1 in 5 men over the age of 50 will break a bone due to osteoporosis. A range of drug treatments exist to both prevent and treat osteoporosis, some of which can reduce the risk of breaking a bone by almost a half. The provision of these drugs on the NHS is currently being reviewed by NICE, for which the NOS is an active stakeholder organisation. However, the NOS is concerned that the final guidance will fall short of the standards required to make a difference to people with, and at risk of, osteoporosis.

  2.3  The NOS has been closely involved, as a stakeholder, with the development of NICE guidance relating to the care and treatment of people with osteoporosis. We have also inputted into the development of the clinical guideline on falls and we are an active stakeholder in a number of other clinical guideline and public health guidance programs.

  2.4  It was therefore with great interest that we read the terms of reference for the Committee's inquiry, particularly given the NOS's concerns about the ongoing development of NICE guidance for this disease area, and in light of recent developments, which we allude to in our submission. We have taken the opportunity of submitting this brief response setting out our comments on this inquiry. The comments are in order of the inquiry's terms of reference.

3.   WHY NICE'S DECISIONS ARE INCREASINGLY BEING CHALLENGED

  3.1  The NOS was actively involved in developing the scope for the technology appraisals on the prevention and treatment of osteoporosis in 2002, but since that time it has changed significantly. NICE have published a series of consultation documents, which have differed greatly despite little change in the evidence. The length and complexity of this process has led to our members becoming disillusioned and frustrated at the lack of progress.

  3.2  The NOS has some concerns about the methodology used by NICE to reach its latest draft recommendations on the use of drugs for osteoporosis. In particular we are concerned about the transparency of the economic modelling. On the basis of these concerns, the NOS is inclined to challenge the Institute's findings on behalf of our members, in order to seek justification for the conclusions they have reached in the case of guidance for osteoporosis treatments and services.

  3.3  In its draft guidance NICE has made decisions about the age of women who will be eligible to receive treatment that appear to be perverse in light of the evidence. We are therefore seeking clarification from NICE to understand fully the reasoning behind its recommendations in this particular case.

  3.4  The processes used by NICE are extremely complicated. Further to this, we are being asked more and more strongly by our members to challenge NICE's decisions on osteoporosis.

4.   WHETHER PUBLIC CONFIDENCE IN THE INSTITUTE IS WANING, AND IF SO WHY?

  4.1  As a national charity with over 24,000 members, the NOS represents patients with, and at risk of, osteoporosis around the country who are affected by the decisions made by NICE. Throughout the technology appraisal and guideline development process, the NOS has kept its members fully updated with the situation, and has received overwhelming support from both members of the charity and members of the public for the points it has taken up with NICE. During individual consultations we have received thousands of letters of concern from our members which would suggest that public confidence, in this inquiry at least, is waning.

  4.2  The NOS believes that NICE have become increasingly more conservative in their consideration of osteoporosis and that this has been perceived by our members as a questioning of the importance of osteoporosis as a disease.

  4.3  Recent examples of challenges against NICE appear to have captured the imagination of the public, encouraging some people to be more active in voicing their objections. Media exposure of challenges to NICE's decisions has opened and exposed arguments around cost effectiveness of treatments to the general public. Many of our members do not understand why an osteoporosis treatment which costs just 27 pence per day is not being made available more widely, when herceptin has been made widely available for the treatment of breast cancer.

5.   NICE'S EVALUATION PROCESS, AND WHETHER ANY PARTICULAR GROUPS ARE DISADVANTAGED BY THE PROCESS

  5.1  In section 3.1 we have referred to the significant change to the published evaluation process over the course of five years. Three separate sets of guidance on osteoporosis appear to have been allowed to be developed without NICE monitoring that they were working in parallel with each other, this being the original intention.

  5.2  The NOS is concerned that older people may be disadvantaged by the NICE process. In terms of simply accessing information, many of the NOS' members have commented that they had difficulties using the NICE website, which is the primary public access point for information on the Institute's work. We are concerned that this facility is not as "user friendly" for the older population, who, broadly speaking, do not have the same access to, or ability to use, the Internet. As a result, when the NOS encourages its members to register their views on NICE's recommendations via the website, many older people are less able to input into this process.

6.   THE SPEED OF PUBLISHING GUIDANCE

  6.1  Development of osteoporosis guidance has been ongoing for over 5 years, which is significantly longer than most other technology appraisals.

  6.2  We are concerned that the delay in publishing osteoporosis guidance may have impacted upon the levels of implementation for existing mandatory guidance. The original technology appraisal for the assessment of drugs in the secondary prevention of osteoporotic fractures (TA87) was published in January 2005. This appraisal is now under review to include a new drug, strontium ranelate, and to incorporate emerging research from the World Health Organisation with regard to risk assessment. However, our evidence suggests that the existing TA87 is not being implemented as it should be, as doctors await the outcome of the NICE review process. To this end, we believe that the length of time it has taken NICE to develop this particular guidance may have contributed to the general lack of progress in improving services for patients at risk of osteoporosis.

7.   THE APPEAL SYSTEM

  7.1  As a stakeholder, the NOS welcomes the opportunity to challenge the proposed recommendations put forward by NICE through the consultation process, and has exercised this right in relation to the development of guidance on the use of drugs for osteoporosis. We further recognise that the appeals facility is an important facet of the guidance development process.

  7.2  The NOS is currently awaiting the outcome of the consultation on the draft recommendations for the use of drugs in primary and secondary prevention of osteoporotic fractures. If there is insufficient movement on the issues of particular concern to the charity in the final recommendations we will consider mounting an appeal against them. However, we do have some reservations about the composition and independence of the Appeals Panel that will give consideration to our objections.

  7.3  More recently, over the last 12 months, it has been necessary for many of the most senior officials of NICE to become closely involved with the development of the different guidance on osteoporosis to ensure that they become realigned and work in parallel. It is our understanding that these very same officials will appoint an appeal panel and for this reason, we are concerned that the panel may lack impartiality. We would therefore question whether or not such a system could accommodate a truly independent consideration of an appellant's objections.

  7.4  The NOS would welcome the introduction of an independent, impartial panel to consider appeals.

8.   COMPARISON WITH THE WORK OF THE SCOTTISH INTERCOLLEGIATE GUIDELINES NETWORK (SIGN)

  8.1  The NOS works closely with NHS Quality Improvement Scotland as the umbrella organisation under which the Scottish Intercollegiate Guidelines Network (SIGN) and the Scottish Medicines Consortium (SMC) work as independent bodies. The NOS was represented on the guideline development group for the SIGN guidance on the Management of Osteoporosis and the Management of Hip Fracture in Older People. We have also responded to consultations on the use of osteoporosis drugs by the Scottish Medicines Consortium.

  8.2  As a nationwide organisation, the NOS campaigns to ensure that patients throughout the UK have access to the same standards of care and treatment for osteoporosis. However, it is difficult to draw direct comparisons between NICE and SIGN given that they have different roles within their respective health services.

  8.3  It is however notable that the outcome of SIGN guidance appears to gain a higher degree of buy-in from its stakeholder organisations than has been the experience of NICE.

9.  THE IMPLEMENTATION OF NICE GUIDANCE, BOTH TECHNOLOGY APPRAISALS AND CLINICAL GUIDELINES (WHICH GUIDANCE IS ACTED ON, WHICH IS NOT AND THE REASONS FOR THIS)

  9.1  We alluded to the difficulties we have observed in the implementation of TA87 in point 6.2 above. We are now extremely concerned that if TA87 has not been implemented in full it stands to reason that the recommendations made by the non-mandatory clinical guideline will also not be implemented. This is of particular concern in the field of osteoporosis, as much of the original scope of the technology appraisals has been transferred to the clinical guideline during the development process.

  9.2  We also believe that it is important that the clinical guideline and technology appraisals are fully complementary. To this end, we would argue that all guidance, both the technology appraisals and clinical guidelines, must be implemented in order for it to be fully effective. In addition, given the longevity of the development process for the osteoporosis guidelines, it would be an entirely inefficient use of NICE's time and resources for health bodies not to act on the final guidance and to ensure it is adhered to.

  9.3  We are also concerned that the implementation of NICE guidance also has consequences for the implementation of other national guidance. Once finalised, the NICE guidance will form an important part in a set of national standards for osteoporosis services, including the National Service Framework for Older People. It is necessary for all of these services to be in place in order to provide the optimal benefits for patients.

Heidi-Mai Warren

National Osteoporosis Society

March 2007