scholarly journals Ustekinumab for the treatment of moderate to severe psoriasis

2009 ◽  
Vol 13 (Suppl 3) ◽  
pp. 61-66
Author(s):  
E Gospodarevskaya ◽  
J Picot ◽  
K Cooper ◽  
E Loveman ◽  
A Takeda
Keyword(s):  
Sensitivity Analysis ◽  
Cost Effectiveness ◽  
Adverse Events ◽  
Supportive Care ◽  
Clinical Effectiveness ◽  
Severe Psoriasis ◽  
Base Case ◽  
Mixed Treatment Comparison ◽  
Single Technology Appraisal ◽  
Treatment Comparison

This paper presents a summary of the evidence review group (ERG) report into the clinical effectiveness and cost-effectiveness of ustekinumab for the treatment of moderate to severe psoriasis based upon a review of the manufacturer’s submission to the National Institute for Health and Clinical Excellence (NICE) as part of the single technology appraisal (STA) process. The submission’s main evidence came from three randomised controlled trials (RCTs), of reasonable methodological quality and measuring a range of clinically relevant outcomes. Higher proportions of participants treated with ustekinumab (45 mg and 90 mg) than with placebo or etanercept achieved an improvement on the Psoriasis Area and Severity Index (PASI) of at least 75% (PASI 75) after 12 weeks. There were also statistically significant differences in favour of ustekinumab over placebo for PASI 50 and PASI 90 results, and for ustekinumab over etanercept for PASI 90 results. A weight-based subgroup dosing analysis for each trial was presented, but the methodology was poorly described and no statistical analysis to support the chosen weight threshold was presented. The manufacturer carried out a mixed treatment comparison (MTC); however, the appropriateness of some of the methodological aspects of the MTC is uncertain. The incidence of adverse events was similar between groups at 12 weeks and withdrawals due to adverse events were low and less frequent in the ustekinumab than in the placebo or etanercept groups; however, statistical comparisons were not reported. The manufacturer’s economic model of treatments for psoriasis compared ustekinumab with other biological therapies. The model used a reasonable approach; however, it is not clear whether the clinical effectiveness estimates from the subgroup analysis, used in the base-case analysis, were methodologically appropriate. The base-case incremental cost-effectiveness ratio for ustekinumab versus supportive care was £29,587 per quality-adjusted life-year (QALY). In one-way sensitivity analysis the model was most sensitive to the number of hospital days associated with supportive care, the cost estimate for intermittent etanercept 25 mg and the utility scores used. In the ERG’s scenario analysis the model was most sensitive to the price of ustekinumab 90 mg, the proportion of patients with baseline weight > 100 kg and the relative risk of intermittent versus continuous etanercept 25 mg. In the ERG’s probabilistic sensitivity analysis ustekinumab had the highest probability of being cost-effective at conventional NICE thresholds, assuming the same price for the 45-mg and 90-mg doses; however, doubling the price of ustekinumab 90 mg resulted in ustekinumab no longer dominating the comparators. In conclusion, the clinical effectiveness and cost-effectiveness of ustekinumab in relation to other drugs in this class is uncertain. Provisional NICE guidance issued as a result of the STA states that ustekinumab is recommended as a treatment option for adults with plaque psoriasis when a number of criteria are met. Final guidance is anticipated in September 2009.

scholarly journals Infliximab for the treatment of adults with psoriasis

2009 ◽  
Vol 13 (Suppl 1) ◽  
pp. 55-60
Author(s):  
E Loveman ◽  
D Turner ◽  
D Hartwell ◽  
K Cooper ◽  
A Clegg
Keyword(s):  
Sensitivity Analysis ◽  
Cost Effectiveness ◽  
Plaque Psoriasis ◽  
Indirect Comparison ◽  
Response To Treatment ◽  
Base Case ◽  
Infliximab Treatment ◽  
Single Technology Appraisal ◽  
Severe Plaque Psoriasis ◽  
Pasi Score

This paper presents a summary of the evidence review group (ERG) report into the clinical and cost-effectiveness of infliximab for the treatment of moderate to severe plaque psoriasis, in accordance with the licensed indication, based on the evidence submission from Schering-Plough to the National Institute for Health and Clinical Excellence (NICE) as part of the single technology appraisal (STA) process. The outcomes stated in the manufacturer’s definition of the decision problem were severity [Psoriasis Area and Severity Index (PASI) score], remission rates, relapse rates and health-related quality of life. The main evidence in the submission comes from four randomised controlled trials (RCT) comparing infliximab with placebo and eight RCTs comparing either etanercept or efalizumab with placebo. At week 10, patients on infliximab had a significantly higher likelihood of attaining a reduction in PASI score than placebo patients. There were also statistically significant differences between infliximab and placebo in the secondary outcomes. In the comparator trials both the efalizumab and etanercept arms included a significantly higher proportion of patients who achieved a reduction in PASI score at week 12 than the placebo arms. No head-to-head studies were identified directly comparing infliximab with etanercept or efalizumab. The manufacturer carried out an indirect comparison, but the ERG had reservations about the comparison because of the lack of information presented and areas of uncertainty in relation to the included data. The economic model presented by the manufacturer was appropriate for the disease area and given the available data. The cost-effectiveness analysis estimates the mean length of time that an individual would respond to infliximab compared with continuous etanercept and the utility gains associated with this response. The base-case incremental cost-effectiveness ratio (ICER) for infliximab compared with continuous etanercept for patients with severe psoriasis was £26,095 per quality-adjusted life-year. A one-way sensitivity analysis, a scenario analysis and a probabilistic sensitivity analysis were undertaken by the ERG. The ICER is highly sensitive to assumptions about the costs and frequency of inpatient stays for non-responders of infliximab. The guidance issued by NICE in August 2007 as a result of the STA states that infliximab within its licensed indication is recommended for the treatment of adults with very severe plaque psoriasis, or with psoriasis that has failed to respond to standard systematic therapies. Infliximab treatment should be continued beyond 10 weeks in people whose psoriasis has shown an adequate response to treatment within 10 weeks. In addition, when using the Dermatology Life Quality Index (DLQI), care should be taken to take into account the patient’s disabilities, to ensure DLQI continues to be an accurate measure.

scholarly journals Cost-Effectiveness Evaluation for the US of Fondaparinux Compared to Argatroban in the Management of Suspected HIT

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 4727-4727
Author(s):  
Ahmed Aljabri ◽  
Mahdi Gharaibeh ◽  
Yvonne Huckleberry ◽  
Hussam Kutbi ◽  
Yuval Raz ◽  
...  
Keyword(s):  
Sensitivity Analysis ◽  
Cost Effectiveness ◽  
Adverse Events ◽  
Wholesale Price ◽  
Secondary Analysis ◽  
Case Analysis ◽  
Base Case ◽  
Base Case Analysis ◽  
Off Label ◽  
Primary Base

Abstract Introduction. The relative efficacy and safety of fondaparinux and argatroban in the management of suspected heparin-induced thrombocytopenia (HIT) have been documented. We conducted a cost-effectiveness analysis of both agents in terms of cost and adverse events averted. Methods. We developed a two-armed decision tree model to simulate a cohort of patients with suspected or confirmed HIT managed with argatroban or fondaparinux, using published efficacy data and probabilities of developing HIT-related VTE or major bleeding. The primary base case analysis considered our institutional cost while the secondary analysis used the Average Wholesale Price. Probabilistic sensitivity analysis (PSA) was performed to confirm findings and one-way sensitivity analysis to evaluate additional scenarios. Results. In the primary base case analysis, fondaparinux dominated argatroban treatment as it was less expensive in managing suspected HIT ($154 vs. $2,064) and more effective in terms of adverse events averted (0.998878 vs. 0.995739). This was confirmed in PSA in terms of both cost ($154 vs. $1,999) and adverse events averted (0.9988711 vs. 0.9957212). Sensitivity analysis showed that the total costs of argatroban would never be less than fondaparinux based on our assumptions. In the secondary analysis, fondaparinux was less expensive ($362 vs. $3722) and more effective in terms of adverse event averted (0.998878 vs. 0.995739). The PSA confirmed these findings both in terms of cost ($343 vs. $3477) adverse events averted. (0.9988711 vs. 0.9957212). Conclusions. The cost savings from a once-daily SC fondaparinux injection with limited laboratory monitoring are more advantageous than IV argatroban infusion that requires continuous titration. Disclosures Off Label Use: Fondaparinux is used as "off-label" for the management of patients with suspected or confirmed HIT.

scholarly journals Adalimumab for the treatment of psoriasis

2009 ◽  
Vol 13 (Suppl 2) ◽  
pp. 49-54 ◽  
Author(s):  
D Turner ◽  
J Picot ◽  
K Cooper ◽  
E Loveman
Keyword(s):  
Cost Effectiveness ◽  
Supportive Care ◽  
Plaque Psoriasis ◽  
Economic Model ◽  
Clinical Evidence ◽  
Single Technology Appraisal ◽  
Utility Values ◽  
Treatment Comparison ◽  
Significant Difference ◽  
Systemic Therapies

This paper presents a summary of the evidence review group (ERG) report into the clinical and cost-effectiveness of adalimumab for the treatment of moderate to severe plaque psoriasis based upon a review of the manufacturer’s submission to the National Institute for Health and Clinical Excellence (NICE) as part of the single technology appraisal (STA) process. The submission’s clinical evidence came from three randomised controlled trials comparing adalimumab with placebo, two extension studies and one ongoing open-label extension study. The studies were of reasonable quality and measured a range of clinically relevant outcomes. A higher proportion of patients on 40 mg adalimumab every other week achieved an improvement on the Psoriasis Area and Severity Index (PASI) of at least 75% (PASI 75) compared with placebo groups after 12 or 16 weeks of treatment, and there was a statistically significant difference in favour of adalimumab for the proportion of patients achieving a PASI 50 and a PASI 90. In a mixed treatment comparison, for each PASI outcome the probability of a response was greater for infliximab than for adalimumab, but the probability of response with adalimumab was greater than that with etanercept, efalizumab and non-biological systemic therapies. Adverse event rates were similar in the treatment and placebo arms and discontinuations because of adverse events were low and comparable between groups. The submission’s economic model presents treatment effectiveness for adalimumab versus other biological therapies based upon utility values obtained from two clinical trials. The model is generally internally consistent and appropriate to psoriasis in terms of structural assumptions and the methods used are appropriate. The base-case incremental cost-effectiveness ratio for adalimumab compared with supportive care for patients with severe psoriasis was £30,538 per quality-adjusted life-year. Scenario analysis shows that the model was most sensitive to the utility values used. Weaknesses of the clinical evidence included not undertaking a systematic review of the comparator trials, providing very little in the way of a narrative synthesis of outcome data from the key trials and not performing a meta-analysis so that the overall treatment effect of adalimumab achieved across the trials is unknown. Weaknesses of the economic model included that the assumptions made to estimate the cost-effectiveness of intermittent etanercept used inconsistent methodology for costs and benefits and there were no clear data on the amount of inpatient care required under supportive care. The NICE guidance issued as a result of the STA states that adalimumab is recommended as a treatment option for adults with plaque psoriasis in whom anti-tumour necrosis factor treatment is being considered and when the disease is severe and when the psoriasis has not responded to standard systemic therapies or the person is intolerant to or has a contraindication to these treatments.

scholarly journals Cost-Effectiveness of Remdesivir Plus Supportive Care for Patients with COVID-19 in Iran

2021 ◽  
Author(s):  
Mohammad Tasavon Gholamhoseini ◽  
Reza Goudarzi ◽  
Vahid Yazdi-Feyzabadi ◽  
Mohammad Hossein Mehrolhassani ◽  
Meysam Yousefi
Keyword(s):  
Sensitivity Analysis ◽  
Cost Effectiveness ◽  
Supportive Care ◽  
Purchasing Power Parity ◽  
Transition Probabilities ◽  
Cost Effective ◽  
Sensitivity Analyses ◽  
Base Case ◽  
Power Parity ◽  
System Perspective

Abstract Background: Remdesivir is a medication used for moderate to severe Coronavirus disease 2019 (COVID-19) patients with favorable effects. However, it is an expensive medication. Therefore, the present study aimed to assess the cost-effectiveness of remdesivir plus supportive care (SC) for COVID-19 patients in Iran.Methods: Markov model was used to compare costs and quality-adjusted life-days (QALDs) of remdesivir+SC and SC for patients with COVID-19. The model simulated a cycle length of one day and a 30-day time horizonin TreeAge 2020 software. The costs from the healthcare system perspective were obtained from Afzalipour hospital as a referral hospital for the hospitalization of COVID-19 patients in Kerman, Iran. All the costs were converted to 2018 purchasing power parity (PPP) US dollars. Utility values were derived from published sources. The results were presented as an incremental cost-effectiveness ratio (ICER) at a willingness-to-pay (WTP) threshold of three times the Gross Domestic Product per capita of Iran. Both deterministic and probabilistic sensitivity analyses were performed.Results: The base-case results showed that the treatment of COVID-19 patients with remdesivir+SC had a cost of 8795 PPP US dollars for 21.13 QALD gained. The SC alone cost 8637 PPP US dollars with a gain of 20.20 QALD. Our findings demonstrated that at a WTP threshold of 159 PPP US dollars per QALD, remdesivir+SC was not cost-effective with an ICER of 168 PPP US dollars per QALD. Deterministic sensitivity analysis indicated ICER to be sensitive to the transition probabilities and costs. Probabilistic sensitivity analysis revealed that remdesivir+SC was cost-effective at a WTP of 159 PPP US dollars per QALD in 47% of iterations.Conclusions: Our findings demonstrated thatremdesivir+SC is not cost-effective, compared to SC alone. Considering the lack of studies on the effectiveness of remdesivir, the findings should be interpreted with caution. Further evaluations are recommended to determine the efficacy and effectiveness of remdesivir in COVID-19 patients.

scholarly journals Omalizumab for the treatment of severe persistent allergic asthma

2009 ◽  
Vol 13 (Suppl 2) ◽  
pp. 31-39
Author(s):  
J Jones ◽  
J Shepherd ◽  
D Hartwell ◽  
P Harris ◽  
K Cooper ◽  
...  
Keyword(s):  
Sensitivity Analysis ◽  
Cost Effectiveness ◽  
Allergic Asthma ◽  
Standard Therapy ◽  
Sensitivity Analyses ◽  
Base Case ◽  
Life Questionnaire ◽  
Exacerbation Rate ◽  
Single Technology Appraisal ◽  
Significant Difference

This paper presents a summary of the evidence review group (ERG) report into the clinical effectiveness and cost-effectiveness of omalizumab for the treatment of chronic severe persistent allergic asthma, in accordance with the licensed indication, based upon the evidence submission from Novartis to the National Institute for Health and Clinical Excellence (NICE) as part of the single technology appraisal (STA) process. The clinical evidence comes from a randomised controlled trial comparing omalizumab as an add-on to standard therapy with placebo and standard therapy over a 28-week treatment period. For the primary outcome of the rate of clinically significant asthma exacerbations, there was no statistically significant difference between treatment groups. However, after making a post hoc adjustment for a suggested ‘clinically relevant’ imbalance between trial arms in baseline exacerbation rate, the difference became marginally statistically significant. In terms of secondary outcomes, there were statistically significant differences favouring omalizumab over placebo in total emergency visits, Asthma Quality of Life Questionnaire scores, total symptom scores and lung function. Adverse events appeared to be similar between the trial arms. Results from three other publications are included in the manufacturer’s submission as supporting evidence for the effectiveness of omalizumab, despite not meeting the inclusion criteria which adhere strictly to the licensed indication. The ERG checked and provided commentary on the manufacturer’s model using standard checklists as well as undertook one-way sensitivity analysis, scenario analysis and a probabilistic sensitivity analysis. The cost-effectiveness analysis estimates the incremental costs and consequences of omalizumab as an add-on to standard therapy. The base-case analysis of the trial’s primary intention-to-treat population estimates a cost per quality-adjusted life-year of £30,647. The ERG conducted one-way sensitivity analyses for parameters omitted from the manufacturer’s submission sensitivity analysis. The results were most sensitive to variation in the utility values for omalizumab responders, and the unit cost of omalizumab. The guidance issued by NICE in November 2007 as a result of the STA states that omalizumab is recommended as a possible treatment for adults and young people over 12 years with severe persistent allergic asthma when their asthma meets certain conditions. Omalizumab treatment should be given along with the person’s current asthma medicines. It should be prescribed by a doctor who is experienced in asthma and allergy medicine at a specialist centre. If omalizumab does not control the asthma after 16 weeks, treatment should be stopped.

scholarly journals Varenicline in the management of smoking cessation: a single technology appraisal

2009 ◽  
Vol 13 (Suppl 2) ◽  
pp. 9-13
Author(s):  
D Hind ◽  
P Tappenden ◽  
J Peters ◽  
K Kenjegalieva
Keyword(s):  
Sensitivity Analysis ◽  
Smoking Cessation ◽  
Cost Effectiveness ◽  
Meta Analysis ◽  
Clinical Excellence ◽  
Clinical Effectiveness ◽  
Pooled Analysis ◽  
Net Benefit ◽  
Single Technology Appraisal ◽  
Quit Attempt

This paper presents a summary of the submission’s evidence for the clinical effectiveness and cost-effectiveness of varenicline for smoking cessation included four studies of varenicline (one of which was commercial-in-confidence) and a meta-analysis of varenicline versus nicotine replacement therapy (NRT), bupropion and placebo. Two controlled trials of 12 weeks of varenicline versus sustained-release bupropion and placebo suggested that varenicline results in a statistically significant improvement in the odds of quitting at 12 weeks [odds ratio (OR) for quit rate during last 4 weeks of the study: 1.90–1.93 (p < 0.001) varenicline versus bupropion; 3.85 (p < 0.001) varenicline versus placebo). The ORs for sustained abstinence (weeks 9–52) for varenicline versus bupropion were 1.77 (p = 0.004) and 1.46 (p = 0.057), and for varenicline versus placebo were 2.66–3.09 (p < 0.01). A placebo-controlled maintenance trial examined whether a further 12 weeks of varenicline would maintain the rate of abstinence among those successfully treated on one 12-week course [OR = 2.48 at week 24 for varenicline versus placebo (p < 0.001)]. The meta-analysis suggested that varenicline was superior to placebo and bupropion at 1 year and 3 months. Based on indirect comparisons, varenicline was reported to be superior to NRT when compared with placebo or all controls at 1 year and 3 months. The submission presented a state transition model to estimate the incremental cost-effectiveness of varenicline compared with bupropion, NRT and placebo. The model suggests that varenicline dominates bupropion, NRT and placebo.Treatment efficacy was based on a pooled analysis of 1-year quit rates from the varenicline clinical trials. Assuming a willingness-to-pay threshold range of £20,000–30,000 per quality-adjusted life-year gained, the probabilistic sensitivity analysis suggests that the probability that varenicline produces the greatest amount of net benefit is 0.70. Weaknesses of the manufacturer’s submission include the assumption that only a single quit attempt using a single smoking cessation intervention is made, the presence of multiple computational errors and a limited sensitivity analysis. In conclusion, varenicline is likely to be clinically and costeffective for smoking cessation assuming that each user makes a single quit attempt. The key area of uncertainty concerns the long-term experience of subjects who have remained abstinent from smoking beyond 12 months. The guidance issued by the National Institute for Health and Clinical Excellence in July 2007 states that varenicline is recommended within its licensed indications as an option for smokers who have expressed a desire to quit smoking and that varenicline should normally be prescribed only as part of a programme of behavioral support.

scholarly journals Gemcitabine for the treatment of metastatic breast cancer

2009 ◽  
Vol 13 (Suppl 2) ◽  
pp. 1-7
Author(s):  
J Jones ◽  
A Takeda ◽  
SC Tan ◽  
K Cooper ◽  
E Loveman ◽  
...  
Keyword(s):  
Breast Cancer ◽  
Sensitivity Analysis ◽  
Metastatic Breast Cancer ◽  
Cost Effectiveness ◽  
Indirect Comparison ◽  
Metastatic Breast ◽  
Base Case ◽  
Health State ◽  
Single Technology Appraisal ◽  
Quality Adjusted Life

This paper presents a summary of the evidence review group (ERG) report into the evidence for the clinical effectiveness and cost-effectiveness of gemcitabine with paclitaxel for the first-line treatment of metastatic breast cancer (MBC) in patients who have already received chemotherapy treatment with an anthracycline, compared with current standard of care, based upon the manufacturer’s submission to the National Institute for Health and Clinical Excellence (NICE) as part of the single technology appraisal (STA) process. The clinical evidence for gemcitabine as a treatment for MBC comes from the unpublished JHQG trial (some data commercial-in-confidence): overall survival was 3 months longer for the gemcitabine/paclitaxel arm (18.5 months) than for the paclitaxel arm (15.8 months) (p = 0.0489); gemcitabine/paclitaxel also improved tumour response and time to documented progression of disease compared with paclitaxel monotherapy, but haematological serious adverse events were more common. In the absence of any formal methods of indirect comparison there is insufficient robust evidence to compare the relative effectiveness of gemcitabine/paclitaxel with docetaxel monotherapy or docetaxel/capecitabine combination therapy. The manufacturers used a Markov state transition model to estimate the effect of treatment with five different chemotherapy regimes, adopting a 3-year time horizon with docetaxel monotherapy as the comparator. Health state utilities for different stages of disease progression and for patients experiencing treatment-related toxicity are used to derive quality-adjusted life expectancy with each treatment. The base-case cost-effectiveness estimate for gemcitabine/paclitaxel versus docetaxel is £17,168 per quality-adjusted life-year (QALY). When longer survival with docetaxel is assumed in a sensitivity analysis, the incremental cost-effectiveness ratio (ICER) is £30,000 per QALY. Probabilistic sensitivity analysis estimates a 70% probability of gemcitabine/paclitaxel being cost-effective relative to docetaxel at a willingness-to-pay threshold of £35,000. There is considerable uncertainty over the results because of the lack of formal quality assessment or assessment of the comparability of the 15 trials included in the input data, and the questionable validity of the indirect comparison method adopted. An illustrative analysis using a different method for indirect comparison carried out by the ERG produces an ICER of £45,811 per QALY for gemcitabine/paclitaxel versus docetaxel. The guidance issued by NICE in November 2006 as a result of the STA states that gemcitabine in combination with paclitaxel, within its licensed indication, is recommended as an option for the treatment of MBC only when docetaxel monotherapy or docetaxel plus capecitabine is also considered appropriate.

scholarly journals Clinical effectiveness and cost-effectiveness of beta-interferon and glatiramer acetate for treating multiple sclerosis: systematic review and economic evaluation

2017 ◽  
Vol 21 (52) ◽  
pp. 1-352 ◽  
Author(s):  
GJ Melendez-Torres ◽  
Peter Auguste ◽  
Xavier Armoiry ◽  
Hendramoorthy Maheswaran ◽  
Rachel Court ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Systematic Review ◽  
Sensitivity Analysis ◽  
Cost Effectiveness ◽  
De Novo ◽  
Meta Analysis ◽  
Clinical Effectiveness ◽  
Cost Effective ◽  
Base Case ◽  
The Mean

Background At the time of publication of the most recent National Institute for Health and Care Excellence (NICE) guidance [technology appraisal (TA) 32] in 2002 on beta-interferon (IFN-β) and glatiramer acetate (GA) for multiple sclerosis, there was insufficient evidence of their clinical effectiveness and cost-effectiveness. Objectives To undertake (1) systematic reviews of the clinical effectiveness and cost-effectiveness of IFN-β and GA in relapsing–remitting multiple sclerosis (RRMS), secondary progressive multiple sclerosis (SPMS) and clinically isolated syndrome (CIS) compared with best supportive care (BSC) and each other, investigating annualised relapse rate (ARR) and time to disability progression confirmed at 3 months and 6 months and (2) cost-effectiveness assessments of disease-modifying therapies (DMTs) for CIS and RRMS compared with BSC and each other. Review methods Searches were undertaken in January and February 2016 in databases including The Cochrane Library, MEDLINE and the Science Citation Index. We limited some database searches to specific start dates based on previous, relevant systematic reviews. Two reviewers screened titles and abstracts with recourse to a third when needed. The Cochrane tool and the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) and Philips checklists were used for appraisal. Narrative synthesis and, when possible, random-effects meta-analysis and network meta-analysis (NMA) were performed. Cost-effectiveness analysis used published literature, findings from the Department of Health’s risk-sharing scheme (RSS) and expert opinion. A de novo economic model was built for CIS. The base case used updated RSS data, a NHS and Personal Social Services perspective, a 50-year time horizon, 2014/15 prices and a discount rate of 3.5%. Outcomes are reported as incremental cost-effectiveness ratios (ICERs). We undertook probabilistic sensitivity analysis. Results In total, 6420 publications were identified, of which 63 relating to 35 randomised controlled trials (RCTs) were included. In total, 86% had a high risk of bias. There was very little difference between drugs in reducing moderate or severe relapse rates in RRMS. All were beneficial compared with BSC, giving a pooled rate ratio of 0.65 [95% confidence interval (CI) 0.56 to 0.76] for ARR and a hazard ratio of 0.70 (95% CI, 0.55 to 0.87) for time to disability progression confirmed at 3 months. NMA suggested that 20 mg of GA given subcutaneously had the highest probability of being the best at reducing ARR. Three separate cost-effectiveness searches identified > 2500 publications, with 26 included studies informing the narrative synthesis and model inputs. In the base case using a modified RSS the mean incremental cost was £31,900 for pooled DMTs compared with BSC and the mean incremental quality-adjusted life-years (QALYs) were 0.943, giving an ICER of £33,800 per QALY gained for people with RRMS. In probabilistic sensitivity analysis the ICER was £34,000 per QALY gained. In sensitivity analysis, using the assessment group inputs gave an ICER of £12,800 per QALY gained for pooled DMTs compared with BSC. Pegylated IFN-β-1 (125 µg) was the most cost-effective option of the individual DMTs compared with BSC (ICER £7000 per QALY gained); GA (20 mg) was the most cost-effective treatment for CIS (ICER £16,500 per QALY gained). Limitations Although we built a de novo model for CIS that incorporated evidence from our systematic review of clinical effectiveness, our findings relied on a population diagnosed with CIS before implementation of the revised 2010 McDonald criteria. Conclusions DMTs were clinically effective for RRMS and CIS but cost-effective only for CIS. Both RCT evidence and RSS data are at high risk of bias. Research priorities include comparative studies with longer follow-up and systematic review and meta-synthesis of qualitative studies. Study registration This study is registered as PROSPERO CRD42016043278. Funding The National Institute for Health Research Health Technology Assessment programme.

Sign in / Sign up

Export Citation Format

Share Document