deep brain stimulation for movement disorders

Enclosure J2

POLICY ON THE USE OF DEEP BRAIN STIMULATION TO TREAT ADULTS WITH MOVEMENT DISORDERS

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

Author: Kim CoxSpecialised Services Commissioning ManagerNORCOM

Correspondence to: Cathy EdwardsDirector of Yorkshire and Humber SCG and Head of Collaborative ServicesNORCOMC/o Barnsley PCTHillder HouseBarnsleyS75 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 Leeds

National Institute for Health and Clinical Excellence

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

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CONTENTS

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 1APPROPRIATE OUTCOME MEASUREMENT TOOLS 12

REFERENCES 13


ABBREVIATIONS

ADL Activities of Daily Living

DBS Deep Brain Stimulation

EBCC Evidence Based Commissioning Collaborative

FT Foundation Trust

NICE National Institute for Health and Clinical Excellence

NSCAG National Specialist Commissioning Advisory Group

MDT Multi-disciplinary Team

MRC Medical Research Council

QALY Quality Adjusted Life Year

SCG Specialist Commissioning Group

DEFINITIONS

Appropriate Medical Treatment

Dopaminergic drugs such as Sinemet, Madopar, bromocriptine, pergolide, pramipexole, ropinirole and apomorphine

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 adequately to, or be unable to tolerate, maximal medical therapy

Intolerable response fluctuations, dyskinesia or psychotic adverse effects of medication


1 AIM OF THE PAPER

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 MOVEMENT DISORDERS

2.1 PARKINSON’S DISEASE1

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 TREMOR AND DYSTONIA3

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

2.2.2.1 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 posture or intentional movement.


2.2.2.2 Severe tremor can be disabling because it affects fine movement control.

2.2.2.3 Tremor can be treated by rehabilitation and drug therapy. Appropriate treatment can minimise functional disability.

2.2.2.4 Anti-tremor drugs occasionally reduce the amplitude, of tremor, but this does not always translate into functional improvement and medication may be poorly tolerated.

2.2.2.5 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.

2.2.2.6 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

2.2.3.1 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).

2.2.3.2 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.

2.2.3.3 Deep brain stimulation is one form of surgery for dystonia. Pallidotomy and thalamotomy are other surgical procedures that may be used.

2.2.3.4 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.


3 DEEP BRAIN STIMULATION

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 current.

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 condition.

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 REVIEW OF THE EVIDENCE

4.1 EFFICACY

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 COST EFFECTIVENESS

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 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 available.

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 SERVICE PROVIDERS

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


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 CRITERIA FOR TREATMENT

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 PARKINSON’S DISEASE

6.2.1 All patients considered for DBS should:

6.2.1.1 Have an established diagnosis of idiopathic Parkinson’s disease and6.2.1.2 Have no evidence of significant cognitive decline and6.2.1.3 Be in good general health and be considered to have a reasonable life

expectancy and6.2.1.4 Have received and failed to respond adequately to, or be unable to tolerate

appropriate medical therapy and6.2.1.5 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)

6.3.1.1 Patients should have severe medically refractory essential tremor causing disability, despite the use of appropriate medical therapy.

6.3.1.2 Functional disability must be severe enough to significantly compromise quality of life and activities of daily living as measured using an appropriate tool.

6.3.1.3 Treatment of tremor should be likely to produce a functionally useful improvement in disability.


6.3.1.4 All other medical and surgical interventions need to have been considered and exhausted.

6.3.2 Cerebellar Tremor (Abnormal Cranial Anatomy)

6.3.2.1 Tremor should have an established aetiology and be significantly disabling.

6.3.2.2 Functional disability must be severe enough to significantly compromise quality of life and activities of daily living as measured using an appropriate tool.

6.3.2.3 All other medical and surgical interventions need to have been considered and exhausted.

6.3.2.4 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 appropriately.

6.4 DYSTONIA

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 threatening.

6.4.6 Patients with psychogenic dystonia are not appropriate for DBS.


7 COMMISSIONING IMPLICATIONS

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 5Bassetlaw 0 0 0 0Bradford & Airedale 0 1 0 1Calderdale 0 0 0 0Derbyshire County 0 3 2 5Doncaster 0 0 1 1East Riding 0 0 1 1Hull 1 0 0 1Kirklees 0 1 0 1Leeds 0 2 2 4North East Lincolnshire

1 0 1 2

North Lincolnshire 0 1 1 2North Yorkshire & York

2 2 2 6

Rotherham 1 0 1 2Sheffield 4 6 6 16Wakefield 1 0 2 3Total 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) 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 POLICY STATEMENT

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 adults.

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.


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

1 NICE Interventional Procedure Guidance number 19 Deep Brain Stimulation for Parkinson’s disease November 2003

2 2001 Census Population data

3 NICE Interventional Procedure Guidance number 188 Deep Brain Stimulation for Tremor and Dystonia (excluding Parkinson’s disease) August 2006

4 Deep Brain Stimulation for Movement Disorders other than Parkinson’s Disease Evidence Based Commissioning Collaborative Sept 2004

5 A prevalence study of primary dystonia in eight European countries Journal of Neurology, vol. 247, no.10, October 2000, pages 787-7921

3

6 PDSurg Trial Protocol ISRCTN 34111222 October 20034


References


deep brain stimulation for movement disorders

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