Contributions
Abstract: P914
Type: Poster Sessions
Abstract Category: Therapy - Long-term treatment monitoring
Background: In 2001, the National Institute for Health and Care Excellence concluded there was considerable uncertainty regarding long-term clinical and economic benefits of disease-modifying treatments (DMTs) for relapsing-remitting multiple sclerosis (RRMS) and that DMTs may not represent cost-effective use of National Health Service (NHS) resources. In response, the UK Department of Health (DoH) established the MS Risk Sharing Scheme (RSS), to provide patient access to DMTs, whilst monitoring patient outcomes in routine care, with provision, if actual outcomes fell short of expectations, for price adjustments to maintain cost-effective provision of DMTs.
Objectives: The RSS Scientific Advisory Group has provided aggregate results for all DMTs. Our aim is to present the final year 10 results of the RSS for patients treated with subcutaneous interferon beta-1a (scINF-β1a).
Methods: NHS patients meeting eligibility criteria and treated with any of the DMTs entered the RSS. Disease progression, assessed as Expanded Disability Status Scale (EDSS) was assessed at 2 year intervals. Disease course was modelled based on baseline EDSS and long-term natural history (NH) data (British Columbia). 'Target' hazard ratios (HRs) applied to the NH data allowed comparison of actual vs. target health related quality of life (utility) weighted EDSS. A shortfall in treatment benefit that exceeded 10% would trigger price adjustments to restore cost-effectiveness. HRs which would produce zero shortfall were deemed the 'implied HRs' (HRs < 1 indicate treatment benefit).
Results: For the year 10 analysis 4217 (excluding secondary progressive patients at baseline) DMT patients were included in the primary analysis. Of these, 1635 were treated with scINF-β1a, and had a mean baseline EDSS of 2.92. The observed mean year 10 EDSS was 4.12. The baseline, year 10 expected untreated, and year 10 expected with treatment, utility weighted EDSS was 0.618, 0.486, and 0.535 respectively; the actual observed utility weighted EDSS was 0.534. Thus, patients on scINF-β1a achieved RSS outcomes in line with expectation. The actual 2.6% shortfall reduced to zero with an implied HR of 0.77.
Conclusion: The UK DoH criteria for cost-effective provision of scINF-β1a were fulfilled throughout the 10 years of the RSS. The final RSS 10 year analysis provides long-term evidence, within the context of a large scale 'real-world' evaluation, of the treatment benefit provided to patients with RRMS by scINF-β1a.
Disclosure: Funded by Merck KGaA, Darmstadt, Germany. GH is an employee of EMD Serono, Inc., a business of Merck KGaA, Darmstadt, Germany. During the study GH was an employee of Merck Serono Ltd., a division of Merck KGaA, Darmstadt, Germany. SW is an employee of EMD Serono, Inc., a business of Merck KGaA, Darmstadt, Germany. AG is an employee of EMD Serono, Inc., a business of Merck KGaA, Darmstadt, Germany. AD provided consulting services to Merck as an employee of ICON plc.
Abstract: P914
Type: Poster Sessions
Abstract Category: Therapy - Long-term treatment monitoring
Background: In 2001, the National Institute for Health and Care Excellence concluded there was considerable uncertainty regarding long-term clinical and economic benefits of disease-modifying treatments (DMTs) for relapsing-remitting multiple sclerosis (RRMS) and that DMTs may not represent cost-effective use of National Health Service (NHS) resources. In response, the UK Department of Health (DoH) established the MS Risk Sharing Scheme (RSS), to provide patient access to DMTs, whilst monitoring patient outcomes in routine care, with provision, if actual outcomes fell short of expectations, for price adjustments to maintain cost-effective provision of DMTs.
Objectives: The RSS Scientific Advisory Group has provided aggregate results for all DMTs. Our aim is to present the final year 10 results of the RSS for patients treated with subcutaneous interferon beta-1a (scINF-β1a).
Methods: NHS patients meeting eligibility criteria and treated with any of the DMTs entered the RSS. Disease progression, assessed as Expanded Disability Status Scale (EDSS) was assessed at 2 year intervals. Disease course was modelled based on baseline EDSS and long-term natural history (NH) data (British Columbia). 'Target' hazard ratios (HRs) applied to the NH data allowed comparison of actual vs. target health related quality of life (utility) weighted EDSS. A shortfall in treatment benefit that exceeded 10% would trigger price adjustments to restore cost-effectiveness. HRs which would produce zero shortfall were deemed the 'implied HRs' (HRs < 1 indicate treatment benefit).
Results: For the year 10 analysis 4217 (excluding secondary progressive patients at baseline) DMT patients were included in the primary analysis. Of these, 1635 were treated with scINF-β1a, and had a mean baseline EDSS of 2.92. The observed mean year 10 EDSS was 4.12. The baseline, year 10 expected untreated, and year 10 expected with treatment, utility weighted EDSS was 0.618, 0.486, and 0.535 respectively; the actual observed utility weighted EDSS was 0.534. Thus, patients on scINF-β1a achieved RSS outcomes in line with expectation. The actual 2.6% shortfall reduced to zero with an implied HR of 0.77.
Conclusion: The UK DoH criteria for cost-effective provision of scINF-β1a were fulfilled throughout the 10 years of the RSS. The final RSS 10 year analysis provides long-term evidence, within the context of a large scale 'real-world' evaluation, of the treatment benefit provided to patients with RRMS by scINF-β1a.
Disclosure: Funded by Merck KGaA, Darmstadt, Germany. GH is an employee of EMD Serono, Inc., a business of Merck KGaA, Darmstadt, Germany. During the study GH was an employee of Merck Serono Ltd., a division of Merck KGaA, Darmstadt, Germany. SW is an employee of EMD Serono, Inc., a business of Merck KGaA, Darmstadt, Germany. AG is an employee of EMD Serono, Inc., a business of Merck KGaA, Darmstadt, Germany. AD provided consulting services to Merck as an employee of ICON plc.