Strategic Commissioning Group West Midlands West Midlands Specialised Commissioning Team Commissioning Policy (WM/25) Targeted therapies for the treatment of pulmonary arterial hypertension Version 4 October 2010 Version: 4.0 Ratified by (name of Committee): Decision-Making Group Date ratified: October 2010 Date issued: October 2010 Expiry date: Any revisions to the policy will be based (Document is not valid after this date) on local and national evidence of effectiveness and cost effectiveness together with recommendations and guidelines from local, national and international clinical professional bodies Review date: Minimum 3 yearly Lead Executive/Director: Name of originator/author: Simon Hairsnape Dr Daphne Austin, Consultant in Public Health, West Midlands Specialised Commissioning Group Target audience: NHS Trusts, Independent Providers, GP s, patients Distribution: NHS Trusts, Independent Providers, GP s, patients, Worcestershire MP s, Public & Patient Involvement Forum CONTRIBUTION LIST Key individuals involved in developing the document Name Dr Daphne Austin Designation Consultant in Public Health, WMSCG Rebadged - West Midlands Version Of Targeted Therapies For PAH - WM25 Page 1 of 11
Commissioning Policy (WM/25) Targeted therapies for the treatment of pulmonary arterial hypertension This policy is based on the National Specialised Commissioning Group Interim Commissioning Policy Target therapies for the treatment of pulmonary arterial hypertension in Adults Version 3 dated July 2010. There are no changes to the access criteria or monitoring requirements. A local version of the policy has been produce to ensure compatibility with the West Midlands individual funding request policy, local processes and implementation. Version 4 October 2010 1. Definitions Nominated provider means a provider trust which has been approved by the Specialised Commissioning Team (West Midlands) to provide a particular specialised service without a formal assessment. This label implies that the service area and/or the provider are still awaiting formal comprehensive assessment. Designated provider means a provider trust which has been assessed and approved by the West Midlands Strategic Commissioning Group to provide a particular specialised service. The assessment is conducted against known capacity requirements and quality standards. Responsible Primary Care Trust means the Primary Care Trust which discharges the Secretary of State's functions under the National Health Service Act 2006 for an individual patient. 2. Nationally designated treatment centres London Hammersmith Hospital Royal Brompton Hospital Royal Free Hospital Cambridge Papworth Hospital Sheffield Royal Hallamshire Hospital Newcastle Freeman Hospital Rebadged - West Midlands Version Of Targeted Therapies For PAH - WM25 Page 2 of 11
3. The policy 3.1 Treatment will only be funded when initiated and supervised by a nationally designated centre unless other arrangements have been agreed by exception. 3.2 Only the following drugs will be approved. Oral Nebulised Subcutaneous Intravenous Bosentan - 125mg bd only Sildenafil Revatio 20mg tds only Viagra - 25mg-100mg tds only) Sitaxsentan - 100mg od only Ambrisentan 5-10og od Iloprost Treprostinil* Epoprostenol* Iloprost* Treprostinil* * Prostaglandins 3.3 Monotherapy 3.3.1 Monotherapy will be provided for patients as describe in table 1 at the end of this document. Other conditions are currently excluded. Any extension to the range of conditions will only be considered as a service development. These will be subject to business case assessment as set out in Section 38.8 of the 2010/11 NHS Standard Acute Contract. 3.3.2 Patients only with a functional classification of PAH of stage III of stage IV of the WHO modified New York Heart Classification will be funded. Patients with functional class I or II on baseline assessment will not normally be eligible for funding unless agreed as an exceptionality request as set out under the West Midlands Strategic Commissioning Group s policy on individual funding requests. Class I - Patients with pulmonary hypertension but without resulting limitation of physical activity. Ordinary physical activity does not cause undue dyspnoea or fatigue, chest pain, or near syncope Class II - Patients with pulmonary hypertension resulting in slight limitation of physical activity. They are comfortable at rest. Ordinary physical activity causes undue dyspnoea or fatigue, chest pain, or near syncope Class III - Patients with pulmonary hypertension resulting in marked limitation of physical activity. They are comfortable at rest. Less than ordinary activity causes undue dyspnoea, fatigue, and chest pain or near syncope. Rebadged - West Midlands Version Of Targeted Therapies For PAH - WM25 Page 3 of 11
Class IV - patients with pulmonary hypertension with inability to carry out any physical activity without symptoms. These patients manifest signs of right heart failure. Dyspnoea and/or fatigue may be present even at rest. Discomfort is increased by any physical activity It is expected that defining a patient s functional class will be a multidisciplinary team decision 3.3.3 Drug doses higher than those specified above will not normally be funded. 3.4 Dual Therapy 3.4.1 Dual therapy will only be funded in combination involving sildenafil. 3.4.2 Clinicians wishing to apply for patients not falling within the above criteria as an exceptionality request, under the West Midlands Commissioning Policy on Individual Funding Requests must use the WMSCT application form and send the request to the following secure email address: fundingrequest.wmsc@nhs.net All policies and forms can be found at the West Midlands Specialised Commissioning website. http://www.wmsc.nhs.uk/ Responsibility for demonstrating exceptionality rests with the requesting clinician Only evidence of clinical need will be taken into consideration. Factors such as gender, ethnicity, age, lifestyle or other social factors such as employment or parenthood will not be considered on grounds of equality In order to demonstrate exceptionality the patient: Must be significant different to the population of interest. AND Be more likely to benefit from this intervention than might be expected than other patients excluded from treatment. The fact that the treatment might be efficacious for the patient is not, in itself, grounds for exceptionality. If a patient s clinical condition matches certain indications which might be seen as accepted (e.g. the trial indication, the licensed indication, anecdotal or routine but unstudied clinical practice) etc) but these particular indications fall outside the commissioning policy, the patient is, by definition, not exceptional 3.5 Pre-agreed exceptions for dual therapy in combinations, not involving sildenafil. Funding will be approved through a prior approval process for the following clinical situations: Rebadged - West Midlands Version Of Targeted Therapies For PAH - WM25 Page 4 of 11
3.5.1 Dual therapy: for patient switching from one mono-therapy to an alternative monotherapy (up to a maximum of 12 weeks) 3.5.2 Dual therapy: for patients who have been listed for the following surgery may be given additional consideration: Heart-lung transplantation Double Lung transplantation Thrombo-endarterectomy (in patients with chronic thrombo-embolic disease) 3.5.3 Continuation of existing treatments (including a prostacyclin) for patients making the transition from children s services to adult services where it would be inappropriate to change treatments only to comply with the commissioning policy. 3.5.4 Continuation of existing treatments (including a prostacyclin) for adult patients (i.e. started prior to the policy being agreed) which are not in accordance with the commissioning policy is permitted until the patient and their clinician consider it appropriate to stop. 3.6 Clinical trials 3.6.1 The commissioners will not pick up the funding of patients coming off drug company sponsored drug trials / extended access programmes or compassionate funding unless prior arrangements have been made. 3.6.2 It is seen as the responsibility of those initiating therapy to ensure that there is either an exit strategy or that ongoing treatment is provided. Patients should be fully informed. 3.6.3 The commissioners will fund patients once the service development has been agreed. 3.6.4 Clinicians wishing to apply for patients to enter a trial or seek prior approval for trial pick up must apply for an individual request, under the West Midlands Commissioning Policy on Individual Funding Requests must use the WMSCT application form and send the request to the following secure email address: fundingrequest.wmsc@nhs.net Clinicians should refer to the West Midlands Strategic Commissioning Group s Commissioning Policy on Experimental and unproven treatments at the following website: http://www.wmsc.nhs.uk/ 3.7 Criteria for stopping treatment The continued use of target therapies will be reviewed on a regular basis. The key factors influencing the cessation of treatment will be:- Successful surgery Clinically relevant side-effects eg liver function Poor/no response to treatment Rebadged - West Midlands Version Of Targeted Therapies For PAH - WM25 Page 5 of 11
Drug therapies may also be withdrawn at the end of life. 4. Quality 4.1 NSCAG service specification 1 The provider is expected to comply with national standards. For those patients who are eligible for therapy, this should be commenced within 6 weeks of the first appointment with a specialist physician. The delay should be a maximum of 8 weeks. 5. Shared care protocols 5.1. All decisions relating to the drug management of this group of patient will be undertaken by designated centres. 5.2 It is expected that all patients will receive their drugs through one of the home care delivery schemes which are in operation, unless there are good reasons why this should not be the case. Emergency situations If a local clinician considers that a patient s clinical state is such that they cannot wait for a routine assessment then the clinician should contact the designated centre and agree an urgent action plan which may include inpatient transfer for urgent assessment. If the patient needs to start treatment straight away this can be done and: The designated provider will take responsibility for the clinical decision to start treatment The designated centre will seek the funding request The designated provider will ensure assessment and ongoing treatment through the usual route Ongoing treatment It may be in the interest of a patient to receive their drug on a shared care basis with a local clinician. Unless otherwise agreed in these circumstances: The designated provider will retain responsibility for prescribing decisions. The designated provider should ensure that the local hospital meets any information needs. The designated provider has the responsibility of ensuring that a satisfactory shared care arrangement is in place between itself and the local provider. The designated provider retains overall clinical responsibility for the management of the treatment. Providers should ensure that patients understand the precise nature of the shared care arrangements including whom to contact when problems arise. 1 National Specialised Commissioning Advisory Group: Service Specification for the National Pulmonary Hypertension Service London 2002 Rebadged - West Midlands Version Of Targeted Therapies For PAH - WM25 Page 6 of 11
5.3 All shared care agreements should be formally signed off by the WMSCT. 6. GP prescribing 6.1 There will be no GP prescribing of any targeted therapies for pulmonary arterial hypertension including sildenafil. 6.2 There will be no pick up of funding for any patient who does not meet the patient selection criteria. 7. Consultant prescribing in non-designated centres 7.1 The regional risk sharing scheme will not pick up funding of any prescribing undertaken by consultants in non-designated centre for this group of drugs for pulmonary arterial hypertension. Non-designated centres will be required to refer all patients to a designated centre in order to access targeted therapies. There will be no pick up of funding for any patient who does not meet the patient selection criteria. 8. Group prior approval process 8.1 Patients which meet the criteria for normally commissioned care will fall under a group prior approval process. As such there will be no requirement to seek commissioner approval prior to the commencement of treatment. BUT The release of commissioner funding is dependent on registration of the patient on the national database AND provision of the commissioner dataset. The commissioner dataset is as follows: Patient s NHS number Patient s DOB and postcode GP practice code Name of drug Primary diagnosis underlying condition WHO NYHA Functional Classification Expected maintenance dose of the drug Cost of drug Starting date of treatment Projected cost to year end 9. Individual prior approval process 9.1 Applications for pre-agreed exceptions require a clinical letter to be send to fundingrequest.wmsc@nhs.net Rebadged - West Midlands Version Of Targeted Therapies For PAH - WM25 Page 7 of 11
10. Individual funding requests 10.1 Individual funding requests, whether individual requests or exceptionality requests should be made as indicated under 3.4.2 and 3.6. 11. Monitoring information 11.1 Each centre will need to provide the WMSCT with a monthly monitoring statement covering the following. ID number Patient initials NHS number PCT/SCG codes Drug and dose Notification of changes to drugs and dosage Take off date Reason for take off Monthly cost Annual cost 12. Review This policy will be reviewed on an annual basis and/or in the light of any new clinical or costeffectiveness evidence. New treatment regimens will not be considered in year unless there is evidence of a sustainable benefit. New drugs coming onto the market may be added to the list in year via the commissioners under the following circumstances: If they have the same or greater efficacy than current drugs AND If they have an equivalent or lower cost to current treatment Rebadged - West Midlands Version Of Targeted Therapies For PAH - WM25 Page 8 of 11
Table 1 First line Second line/ Alternative* Combination Alternate combination Evidence Pulmonary Arterial Hypertension (IPAH, FPAH, Anorexogen-induced, and where associated with Portal hypertension or HIV infection) WHO III/: S WHO IV: P WHO III: B,P or X WHO IV: B B+S or X+S Patients should be entered into a clinical trial where possible P+S Patients must be entered into a clinical trial (NB trial participation previously agreed by commissioners) Few head to head studies, clinical consensus only. RCT evidence of comparable results between S and B (SERAPH) RCT and extension evidence of survival benefit of S (SUPER 1) RCT evidence of functional improvement over placebo for B (BREATHE 1) and X (STRIDE 1) RCT and extension evidence of survival benefit of P but no firm evidence of relative efficacy of different preparations in class Some small study evidence of improved results in combination of S+B PAH associated with significant venous or capillary involvement PVOD only: WHO III/IV, S, consider surgery PCH: no disease targeted treatment supported consider surgery No published data on use of B or X Case reports only on S and only in PVOD PAH associated with connective tissue disease WHO III/IV: B PAH associated with congenital heart disease WHO III: B WHO IV: B as a bridge to transplant PH due to chronic thrombotic and/or embolic disease WHO III/IV: S Consider surgery WHO III/IV: S, P or X WHO III/IV: B B+S or X+S Patients should be entered into a clinical trial where possible No RCT evidence but some evidence of functional improvement on B, X and S Long term follow-up data is poor. Evidence on P is equivocal No published evidence of benefit of combination. B+S will be considered as a bridge to surgery RCT evidence of functional improvement in WHO III with B (BREATHE-5) No published evidence of benefit of combination. B+S will be considered as a bridge to surgery Case reports on distal (inoperable) disease treated by B (BENEFIT). No published data on use of X. = Bosentan, P= Prostanoids, S= Sildenafil, X= Sitaxsentan OR Ambrisentan WHO= Functional classification of PH modified after the New York Heart Association functional classification according to the World Health Organisation 1998 IPAH= Idiopathic PAH; FPAH= Familial PAH; PVOD= Pulmonary veno-occulsive disease; PCH= Pulmonary capillary haemangiomatosis * If first line is contraindicated, ineffective in controlling symptoms or poorly tolerated Rebadged - West Midlands Version Of Targeted Therapies For PAH - WM25 Page 9 of 11
13. Documents which have informed this policy West Midlands Strategic Commissioning Group, Commissioning Policy 1: Ethical Framework to underpin priority setting and resource allocation within collaborative commissioning arrangements Department of Health, The National Health Service Act 2006, The National Health Service (Wales) Act 2006 and The National Health Service (Consequential Provisions) Act 2006. http://www.dh.gov.uk/en/publicationsandstatistics/legislation/actsandbills/dh_06 4103 Department of Health, World Class Commissioning Competencies, December 2007, http://www.dh.gov.uk/en/publicationsandstatistics/publications/publicationspolicy AndGuidance/DH_080958 Department of Health, The NHS Constitution for England, July 2009, http://www.dh.gov.uk/en/publicationsandstatistics/publications/publicationspolicy AndGuidance/DH_093419 Lead for policy Dr Daphne Austin Consultant in Public Health West Midlands Specialised Commissioning Team daphne.austin@wmsc.nhs.uk Version Fourth Policy effective from October 2010 Date of next review Following revision of the national policy Acknowledgements Rebadged - West Midlands Version Of Targeted Therapies For PAH - WM25 Page 10 of 11
Appendix 1 Approximate drug costs Basic NHS price (excluding VAT) Basic NHS Price with VAT Sildenafil (Revatio) 20mg tds 4,544 5,339 Example of contract price (including service charge Sildenafil (Viagra) 25mg tds 4,543 5,338 4,939 Sildenafil (Viagra) 50mg tds 5,822 6,842 6,081 Sildenafil (Viagra) 100mg tds 6,432 7,557 6,625 Bosentan 62.5mg - 125mg bd 19,687 23,132 20,501 Sitaxentan 100mg od 20,075 23,588 21,111 Ambrisentan 5-10mg od 20,088 23,603 21,718 Epoprostenol 31,500 37,000 35,000 Treprostinil 33,280 39,104 37,560 Iloprost (ILOfee) 35,000 41,125 36,880 Iloprost (Ventavis) 29,600 34,780 31,480 Iloprost (IV) 35,000 41,125 38,416 Appendix 2 POLICY CHANGE PROTOCOL The key steps in the policy change protocol are as follows:- (a) All requests for changes to the national policy should be made to the lead SCG Director in writing. (b) The change request should clearly set out the evidence supporting the change, the anticipated benefits, and any financial implications (c) Requests from the clinicians should come via the national clinicians group with the chair of the group writing formally to the lead SCG Directors. (d) The lead SCG Director will obtain a public health/specialist pharmacist review of the clinical and cost effectiveness and the cost implications. (e) The original request and the public health/specialist pharmacy opinion will be considered by the national PH Commissioners Forum. (f) The national PH Commissioners Forum will make a recommendation to SCGDN. Rebadged - West Midlands Version Of Targeted Therapies For PAH - WM25 Page 11 of 11