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New HTA Decisions in Scotland
December 2019
Drug name
ELMIRON® (pentosane polysulfate sodium)
Company
Consilient Health Ltd
Decision date
11/11/2019
Therapeutic area
Urological conditions
Therapeutic sub area
Urological conditions: general and other
Decision
Recommended
Indication
For the treatment of bladder pain syndrome characterised by either glomerulations or Hunner's lesions in adults with moderate to severe pain, urgency and frequency of micturition.
Decision Detail
Following a full submission considered under the orphan equivalent process, pentosan polysulfate sodium (Elmiron®) is accepted for use within NHS Scotland. This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of pentosan polysulfate sodium. This advice is contingent upon the continuing availability of the PAS in NHSScotland or a list price that is equivalent or lower. This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.
Summary
The key evidence to support the use of pentosan polysulfate sodium for patients with bladder pain syndrome characterised by either glomerulations or Hunner’s lesions comes from four randomised, double-blind studies. Eligible patients had symptoms of pain, frequency, urgency and/or nocturia and evidence of bladder pain syndrome with inflammation from cystoscopic examination with or without bladder hydrodistention. Other inclusion criteria varied between the studies. In patients with bladder pain syndrome and glomerulations or Hunner’s lesions, pentosan polysulfate sodium was associated with significantly more patients achieving at least moderate improvement in overall symptoms of bladder pain syndrome compared with placebo. The submitting company presented a cost-utility analysis comparing pentosan polysulfate sodium to bladder instillations for the treatment of adult patients with interstitial cystitis/bladder pain syndrome with glomerulations and/or Hunner’s lesions. The company also provided a comparison with best supportive care (BSC) in those patients considered inappropriate for, or who cannot tolerate, bladder instillations and this was presented as a scenario analysis. SMC clinical expert feedback stated that a range of treatments including oral agents, bladder instillations, and surgery are used, but expects pentosan polysulfate sodium to be used following failure to oral agents, suggesting bladder instillations is likely to be an appropriate comparator. The economic analysis was associated with several limitations and uncertainties such as weaknesses in the pentosan polysulfate sodium and bladder instillation meta-analyses; concerns over the long-run extrapolation of treatment benefit for pentosan polysulfate sodium and bladder instillations which is based on a pentosan polysulfate sodium usage study conducted in the USA over 20 years ago meaning relevance to Scottish practice is uncertain; and potential limitations with the mapping study used to estimate utilities for the model events leading to the company revising the base case analysis to remove an advantage in utility scores that the mapping had produced for responders to pentosan polysulfate sodium. The Committee also considered the benefits of pentosan polysulfate sodium in the context of the SMC decision modifiers that can be applied when encountering high cost-effectiveness ratios and agreed that the criterion for replacing an unlicensed treatment was satisfied. In addition, as pentosan polysulfate sodium is an orphan equivalent medicine, SMC can accept greater uncertainty in the economic case. After considering all the available evidence, the output from the PACE process, and after application of the appropriate SMC modifiers, the Committee accepted pentosan polysulfate sodium for use in NHS Scotland.