Policy of the Use of Erioietin to Treat Anaemia in Patients with

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					                                                                                 Enclosure J2

On behalf of:                                 Primary Care Trusts in the Yorkshire and Humber area

Author:                                       Kim Cox
                                              Specialised Services Commissioning Manager

Correspondence to:                            Cathy Edwards
                                              Director of Yorkshire and Humber SCG and Head of
                                              Collaborative Services
                                              C/o Barnsley PCT
                                              Hillder House
                                              S75 2PY

Date completed:                               May 2008

Review Date:                                  May 2011

Conflicts of Interest:                        None

Acknowledgements                              Tim Allison, Director of Public Health East Yorkshire
                                              and Yorkshire Wolds & Coast PCTs

                                              Tracy Denby, Research Officer, Institute of Health
                                              Sciences and Public health Research, University of

                                              National Institute for Health and Clinical Excellence

                                              Dr Richard Grunewald and the staff of the
                                              Neurosciences Service, Sheffield Teaching Hospitals
                                              NHS Foundation Trust

                                                   Page 1 of 13
ABBREVIATIONS                                                     3

DEFINITIONS                                                       3

1       AIM OF PAPER                                              4

2       MOVEMENT DISORDERS                                        4

3       DEEP BRAIN STIMULATION                                    6

4       REVIEW OF EVIDENCE                                        6

5       SERVICE PROVIDERS                                         7

6       CRITERIA FOR TREATMENT                                    8

7       COMMISSIONING IMPLICATIONS                                10

8       POLICY STATEMENT                                          11

        APPENDIX 1
        APPROPRIATE OUTCOME MEASUREMENT TOOLS                     12

        REFERENCES                                                13

                                                   Page 2 of 13
ADL                                                         Activities of Daily Living

DBS                                                         Deep Brain Stimulation

EBCC                                                        Evidence         Based       Commissioning

FT                                                          Foundation Trust

NICE                                                        National Institute for Health and Clinical

NSCAG                                                       National    Specialist       Commissioning
                                                            Advisory Group

MDT                                                         Multi-disciplinary Team

MRC                                                         Medical Research Council

QALY                                                        Quality Adjusted Life Year

SCG                                                         Specialist Commissioning Group

Appropriate Medical                     Dopaminergic drugs such as Sinemet, Madopar,
Treatment                               bromocriptine, pergolide, pramipexole, ropinirole and

Thalamotomy                             Surgical destruction of a selected part of the thalamus
                                        region of the brain

Pallidotomy                             Surgical destruction of a selected part of the globus
                                        pallidus region of the brain

Failure to respond                      Intolerable response fluctuations, dyskinesia or psychotic
adequately to, or be unable             adverse effects of medication
to tolerate, maximal medical

                                                   Page 3 of 13

1.1    This paper represents the commissioning policy for the use of Deep Brain
       Stimulation (DBS) in the treatment of movement disorders in adults for Primary Care
       Trusts in the Yorkshire and Humber area. It has been produced in the context of and
       in accordance with National Institute for Health and Clinical Excellence (NICE)
       Interventional Procedure Guidance no.19 (Deep brain stimulation for Parkinson’s
       disease) and Interventional Procedure Guidance no. 188 (Deep brain stimulation for
       tremor and dystonia excluding Parkinson’s disease).



2.1.1 Parkinson’s disease is a chronic disease of the brain characterised by gradual
      worsening tremor, muscle rigidity and difficulty in starting and stopping
      movements, resulting in poor quality of life. The condition is usually treated with
      drugs. Surgery may be considered in people who have responded poorly to drugs,
      who have severe side effects from medication or who have severe fluctuations in
      response to drugs.

2.1.2 Parkinson’s disease is common, affecting about 0.5% of people aged 65 to 74
      years and 1-2% of people aged 75 years and older. Based on the 2001 census
      data there are 493,000 people aged 65 to 74 in the Yorkshire and Humber area,
      and 436,000 aged 75 and over. It is estimated that 2465 people aged 65 to 74 and
      between 4360 and 8720 people aged over 75 in Yorkshire and the Humber may
      have Parkinson’s disease2.

2.1.3 Between 1% and 10% of people with Parkinson’s disease may be suitable for
      surgery. This means that anywhere between 683 and 1119 Parkinson’s disease
      sufferers in the Yorkshire and Humber area may be suitable for surgery.

2.1.4 Surgery for Parkinson’s disease is carried out on structures in the brain that are
      responsible for the modification of movements. Surgery alters, through either
      destruction or electrical modification, the function of brain nuclei.

2.1.5 Deep brain stimulation is one form of surgery for Parkinson’s disease. Pallidotomy
      and thalamotomy are other surgical procedures that may be used.


2.2.1 Tremor and dystonia are symptoms that can arise in a number of different
      neurological diseases. These include essential tremor, multiple sclerosis, idiopathic
      focal dystonia and primary generalised dystonia.

2.2.2 TREMOR Tremor is an involuntary rhythmic repetitive movement, most frequently affecting
        the upper limbs. It can occur at rest or can be brought on (or made worse) by

                                                   Page 4 of 13
          posture or intentional movement. Severe tremor can be disabling because it affects fine movement control. Tremor can be treated by rehabilitation and drug therapy. Appropriate treatment
        can minimise functional disability. Anti-tremor drugs occasionally reduce the amplitude, of tremor, but this does not
        always translate into functional improvement and medication may be poorly
        tolerated. Surgery is usually reserved for patients with severe disabling tremor and
        functional disability that interferes with daily living, and for tremor that is refractory
        to the highest tolerated doses of medication. Prevalence of essential tremor is estimated to be 500 per 100,000, although data
        from the United States gives a range of between 8 and 22,000 per 100,000. This
        wide range may be due to issues of diagnostic threshold, overlooked diagnosis or
        unclear diagnostic criteria4. Using the 2001 census populations, the estimated
        prevalence of significant essential tremor for the Yorkshire and Humber
        population is 26,000.

2.2.3 DYSTONIA Dystonia is a neurological disorder characterised by sustained muscle spasma
        and contractions. It may be painful and can lead to abnormal movements and
        postures. It may be limited to a particular group of muscles (focal dystonia), or
        may affect most of the body (generalised dystonia). Dystonia cannot be cured but it can be managed medically or surgically. Current
        medical management options (botulinum toxin or other drugs) may improve the
        symptoms but do not cure the underlying neurological disorder. These drugs may
        have unpleasant side effects. Deep brain stimulation is one form of surgery for dystonia. Pallidotomy and
        thalamotomy are other surgical procedures that may be used. The prevalence of focal dystonia is estimated to be 1 in 3,400, with generalised
        dystonia estimated to be 1 in 30,0005. Using the 2001 census data, it is estimated
        that there are 1529 cases of focal dystonia and 173 cases of general dystonia in
        the Yorkshire and Humber area.

                                                   Page 5 of 13

3.1    Deep brain stimulation (DBS) can be carried out on nuclei within the brain that are
       responsible for modifying movements. These structures are all bilateral and surgery
       can be performed on either one or both sides.

3.2    The function of the nuclei is altered during DBS through the application of an electric

3.3    The procedure involves inserting very fine needles into the brain through small holes
       in the skull to determine the exact nuclei to be stimulated. The procedure may be
       carried out under local or general anaesthetic as appropriate to the patient’s

3.4    Once the appropriate stimulation sites and parameters have been identified, the
       electrodes are connected to a pulse generator implanted in the anterior chest wall.

3.5    Further operations will be required over time to replace the pulse generator or if
       leads break. This will only be undertaken if there is clear evidence of clinical benefit.



4.1.1 Evidence reported by NICE in Interventional Procedure Guidance no 19 showed
      that DBS results in improved motor skills, function and movement in patients with
      Parkinson’s disease1.

4.1.2 NICE also reported in Interventional Procedure Guidance no.188 that there was
      evidence of improvement in both total tremor score and activities of daily living in
      patients with tremor treated with DBS3.

4.1.3 Significant improvements in the Burke-Fahn-Marsden dystonia rating scale and in
      global disability scores were recorded in patients with dystonia treated with DBS.

4.1.4 NICE Specialist Advisors have noted concerns over long-term efficacy of DBS as
      tremor may become resistant to stimulation.

4.1.5 The NSCAG designated MRC PDSurg trial aims to determine whether early
      surgery (either through electrical stimulation or radio-frequency lesioning) is more
      cost effective for advanced Parkinson’s disease than medical therapy alone (with
      surgery deferred)6.


4.2.1 There is a lack of evidence of the cost effectiveness of DBS. NICE did not consider
      the cost effectiveness of DBS for any form of movement disorder.

4.2.2 The EBCC review conducted a cost benefit analysis of DBS for non-Parkinson’s
      disease movement disorders, however this was not considered to be a formal
      economic evaluation as it provided ‘no information on the incremental costs and
                                                   Page 6 of 13
           benefits of DBS compared to alternative management strategies’4.

4.2.3 The review reported that the cost per Quality Adjusted Life Year (QALY) using the
      total cost of the surgery was £33,980. Whilst this figure is slightly over the
      maximum NICE recommended figure of £30,000, the review noted that the
      approach used to the calculation was ‘quite different to that taken by NICE in the
      UK.’ The evaluation did not take account of the periodic need for replacement
      pulse generators.

4.3       CONCLUSION

4.3.1 The EBCC review concluded that commissioners had five options, these being:
       Restrict the use of DBS on the grounds that the evidence of effectiveness was
         inadequate. This option does not take account of the trend in evidence
       Continue to consider requests for DBS on a case by case basis. This option
         would perpetuate inequity and commissioners would continue to face
         challenging decisions.
       Commission further research. Whilst further research is clearly needed, this
         option would not produce an interim commissioning position.
       Create clear pathways and referral criteria. This option would allow a limited
         level of activity whilst targeting those most likely to benefit. The evidence for
         which groups are most likely to benefit remains weak.
       Commission a full service. This option would potentially remove inequity but
         would be based on little evidence of effectiveness.

4.3.2 After consideration of the alternatives, this policy attempts to provide clear criteria
      for referral so as to enable a service to be provided to those considered most likely
      to benefit from DBS.


5.1       NSCAG has designated 10 centres in England as meeting the required standards
          for participation in the PDSurg trial.

5.2       The centres in England are:
           Radcliffe Infirmary, Oxford
           Frenchay Hospital, Bristol
           Queen Elizabeth Hospital, Birmingham
           Kings College Hospital, London
           Walton Centre, Liverpool
           Newcastle General Hospital
           National Hospital for Neurology and Neurosurgery, London
           Hope Hospital, Salford
           Royal Hallamshire Hospital, Sheffield
           Addenbrookes Hospital, Cambridge

                                                   Page 7 of 13
5.3    Yorkshire and Humber Primary Care Trusts wish to commission all DBS treatment
       from providers meeting the rigorous NSCAG standards.

5.4    Yorkshire and Humber commissioners have primarily commissioned services from
       Sheffield, Oxford and Birmingham. Ad hoc requests for treatment have also been
       received from the Walton Centre and Newcastle.


6.1    GENERAL

6.1.1 Indications for the use of DBS for movement disorders fall primarily into 3
      categories, these being Parkinson’s disease, tremor and dystonia.

6.1.2 All patients to be considered for DBS will be discussed by the multi-disciplinary
      team (MDT). The MDT should believe that the patient would gain significant benefit
      from DBS, i.e. regaining lost functions and/or restoring independence. The MDT
      should also have agreed what expected benefit the patient is likely to gain and how
      it will be measured.

6.1.3 All patients considered appropriate for DBS should be medically fit for surgery
      when the decision to undertake DBS is made.


6.2.1 All patients considered for DBS should: Have an established diagnosis of idiopathic Parkinson’s disease and Have no evidence of significant cognitive decline and Be in good general health and be considered to have a reasonable life
        expectancy and Have received and failed to respond adequately to, or be unable to tolerate
        appropriate medical therapy and Have symptoms severe enough to significantly compromise quality of life and
        activities of daily living. Quality of life and activities of daily living must be
        measured pre-operatively using an appropriate tool.

6.3    TREMOR

6.3.1 Essential Tremor (Normal Cranial Anatomy) Patients should have severe medically refractory essential tremor causing
        disability, despite the use of appropriate medical therapy. Functional disability must be severe enough to significantly compromise quality of
        life and activities of daily living as measured using an appropriate tool. Treatment of tremor should be likely to produce a functionally useful improvement
        in disability.

                                                   Page 8 of 13 All other medical and surgical interventions need to have been considered and

6.3.2 Cerebellar Tremor (Abnormal Cranial Anatomy) Tremor should have an established aetiology and be significantly disabling. Functional disability must be severe enough to significantly compromise quality of
        life and activities of daily living as measured using an appropriate tool. All other medical and surgical interventions need to have been considered and
        exhausted. It must be clear that there are no other co-morbidities that would prevent the
        patient from gaining significant benefit. Any other co-morbidities (i.e. those that
        will not prevent the patient gaining significant benefit) must be being treated


6.4.1 The patient must exhibit focal or generalised dystonia of sufficient severity to
      compromise quality of life and activities of daily living despite appropriate medical
      therapy. Quality of life and activities of daily living must be measured pre-
      operatively using an appropriate tool.

6.4.2 Dystonia appropriate for DBS will principally be idiopathic in nature, though it is
      accepted that, on occasion, patients with secondary dystonia may be appropriate.

6.4.3 Patients must not have significant postural defects or significant fixed joint
      deformities which would preclude useful benefit from the treatment

6.4.4 Patients must not have had an adequate response to botulinum toxin treatment;
      have failed to tolerate botulinum toxin treatment; require such large or frequent
      treatments with botulinum toxin as to make such treatment impractical; or be
      unsuitable for botulinum toxin treatment.

6.4.5 Laryngeal dystonia with significant risk of aspiration pneumonia is a particular
      indication as DBS may be the only effective treatment and the condition may be life

6.4.6 Patients with psychogenic dystonia are not appropriate for DBS.

                                                   Page 9 of 13

7.1    The following table shows the numbers of new patients who have been approved for
       DBS since 2004/05.
                                         2004/05                  2005/06   2006/07   Total

         Barnsley                             2                     1         2         5
         Bassetlaw                            0                     0         0         0
         Bradford & Airedale                  0                     1         0         1
         Calderdale                           0                     0         0         0
         Derbyshire County                    0                     3         2         5
         Doncaster                            0                     0         1         1
         East Riding                          0                     0         1         1
         Hull                                 1                     0         0         1
         Kirklees                             0                     1         0         1
         Leeds                                0                     2         2         4
         North East                           1                     0         1         2
         North Lincolnshire                   0                     1         1         2
         North Yorkshire &                    2                     2         2         6
         Rotherham                            1                     0          1       2
         Sheffield                            4                     6          6       16
         Wakefield                            1                     0          2       3
         Total                               12                     17        21       50

7.2    During the period 2004/05 to 2007/08, DBS has been commissioned on a cost-per-
       case with prior approval basis.

7.3    The treatment criteria contained in this policy now express a summary of the
       patients considered appropriate and approved by PCTs over the period. This policy,
       therefore, does not suggest a change to existing practice in terms of eligibility
       criteria. Whilst the numbers of patients approved for DBS has gradually increased,
       there are no trend increases in any one PCT. Consequently it is considered unlikely
       that patient numbers will rise significantly.

7.4    Payment for DBS, for patients with Parkinson’s Disease has previously been
       structured in relation to the PDSurg trial. Patients admitted to the trial had funding
       supplied from the national subvention fund and PCTs paid only the excess costs of
       £12,828 per patient (at 2008/09 prices). The PDSurg trial is no longer taking new
       patients. However, PCTs will need to continue to pay excess costs for any patients
       currently in the trial and who will have surgery up to 31st October 2008. All other
       patients (PD and non-PD) incur a charge to PCTs of national tariff A04 (£5,645 for
       2008/09) plus excess costs for the full implant. The excess cost at Sheffield
       Teaching Hospitals is £27,739 for 2008/09. Costs at other providers may vary.

7.5    As no new patients are now being admitted to the PDSurg trial, the expected cost of
       DBS new patients in 2008/09 is £701,064 (assuming 21 patients treated). This
       comprises £118,545 within tariff and £582,519 excess costs for the implants.

7.6    In addition the pulse generator, implanted subcutaneously in the patient’s chest wall,
       periodically requires replacement at a cost of £9,600 (at Sheffield 2008/09 prices)
                                                   Page 10 of 13
       per generator in addition to the cost of HRG A02 (£3,287 for 2008/09) totalling
       £12,887. Patients who have previously been approved for DBS will not require prior
       approval for the replacement generator. However, the appropriate PCT must be
       explicitly informed when a replacement generator has been supplied. Pulse
       generator life is difficult to determine as it is very much dependent upon the strength
       of setting required by an individual patient. On average, between 4 and 5 pulse
       generators have been replaced in Sheffield each year for the past three years. This
       equates to approximately one replacement generator for every 3 new patients
       treated. Across Yorkshire and the Humber this translates to 7 replacement pulse
       generators per year. The expected cost for 2008/09 will be £90,209. This comprises
       £23,009 within tariff and £67,200 excess costs for the generators.


8.1    The following statement sets out the position of Primary Care Trusts in the Yorkshire
       and Humber area in respect of commissioning DBS for movement disorders in

8.2    There is evidence that DBS can improve motor function and movement, reduce
       disability and improve activities of daily living in patients with Parkinson’s disease,
       tremor or dystonia.

8.3    Patients fitting the treatment criteria (detailed in section 6 of this policy) and
       considered by the MDT likely to receive significant benefit will be eligible for DBS.

8.4    DBS will be commissioned on a cost-per-case without prior approval basis from
       providers who meet the NSCAG designation requirements. Commissioners
       recognise that, under current national tariff rules, an excess cost per patient will be
       charged in addition to the national tariff. Patients undergoing DBS will be classified
       under national tariff A04. Excess costs may vary between providers

8.5    Patients previously receiving DBS (either in Sheffield or from another of the
       approved providers listed in section 5.2 of this policy) requiring replacement pulse
       generators will receive them without prior approval. Providers will inform the
       patient’s PCT as soon as a replacement generator has been supplied.

8.6    This policy will be reviewed in May 2011 or when further significant information
       becomes available, either from clinical trials, technological development, NICE or
       the Yorkshire and Humber SCG.

                                                   Page 11 of 13
Appendix 1

                             Appropriate Outcome Measurement Tools

Parkinson's Disease
Reduction in severity of symptoms as measured on the Unified Parkinson's Disease
Rating Scale
Reduction in interference in daily living- increase in independence and functionality
measured by FIM or ADLcompared to pre implantation scores
Rate of infection/ complication/ revision
Pre and Post DBS physiotherapy/occupational therapy assessment
Pre and post EuroQol
Improvement in Global Disability Score

Improvement measured on Burke Fahn and Marsden Dystonia Rating Scale
Improvement in Toronto Western Spasmodic Torticollis Rating Scale ( TWSTRS )
Improvement in Global Disability Score
Rate of infection/complication/revision

Improvement in total tremor score ( Fahn Tolosa Marin score) over baseline
Improvement in ADL over baseline
Improvement in Euroqol over baseline
Rates of complication/infection/revision

                                                   Page 12 of 13
 NICE Interventional Procedure Guidance number 19 Deep Brain Stimulation for
Parkinson’s disease November 2003
    2001 Census Population data
 NICE Interventional Procedure Guidance number 188 Deep Brain Stimulation for
Tremor and Dystonia (excluding Parkinson’s disease) August 2006
 Deep Brain Stimulation for Movement Disorders other than Parkinson’s Disease
Evidence Based Commissioning Collaborative Sept 2004

 A prevalence study of primary dystonia in eight European countries
Journal of Neurology, vol. 247, no.10, October 2000, pages 787-792

    PDSurg Trial Protocol ISRCTN 34111222 October 2003

                                                   Page 13 of 13

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