Estimating the value of novel interventions for Parkinson's disease: An early decision-making model with application to dopamine cell replacement

Hjelmgren, Jonas; Ghatnekar, Ola; Reimer, Johan; Grabowski, Martin; Lindvall, Olle; Persson, Ulf; Hagell, Peter

doi:10.1016/j.parkreldis.2006.04.006

Cited by 17 publications

(56 citation statements)

References 38 publications

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“…A previous study on Parkinson’s disease has illustrated the value of early health-economic modeling for the clinical application of a future cell replacement therapy [26]. Despite the caveats, our current results indicate that SCT may be a cost-effective therapy for stroke patients under a wide range of assumptions.…”

Section: Discussionmentioning

confidence: 70%

Societal Value of Stem Cell Therapy in Stroke – A Modeling Study

Svensson

Ghatnekar²,

Lindgren³

et al. 2012

Cerebrovasc Dis

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Background: Stroke is one of the major causes of disability in the adult population and represents a heavy social and economic burden. Currently available therapeutic tools to support the recovery of impaired brain functions are quite limited. Animal studies have demonstrated that neuronal replacement and partial reconstruction of neural circuitry or modulation of the recovery process is possible with cell transplantation in the damaged adult brain. Stem cell therapy (SCT) may promote functional recovery also in stroke patients, thereby improving quality of life and reducing costs. Our aim was to estimate the potential societal value of SCT in stroke patients. Methods: We created a decision-analytic model in Microsoft Excel 2010 to assess life-long costs and quality-adjusted life years (QALYs) of SCT versus standard care for stroke patients from a societal perspective. The model structure consisted of 7 health states in accordance with the modified Rankin Scale (mRS). We modeled for age (55, 65, and 75 years), functional status at discharge (mRS 2, 3, and 4), effectiveness of SCT (50 and 25% increase in the probability to improve 1 mRS grade), mode of stem cell administration, risk of recurrent stroke, complications of intervention, and use of immunosuppressive drugs. The difference between an assumed societal willingness to pay for a QALY gain in Sweden (110,400 USD) and the cost per QALY gain resulting from the model was interpreted as the value of SCT. Results: Increased survival (1.06 life years) and improved functional status gave rise to an estimated gain of 1.34 QALY in a cohort of patients aged 55 with mRS 2 at hospital discharge. Although the SCT intervention increased costs by 64,014 USD (excluding cost of stem cells), the costs of intervention were offset mainly by decreased productivity losses. In total, the intervention saved 19,055 USD, i.e., at a price of 19,055 USD for stem cells, the SCT would be cost neutral. The societal value of SCT was 166,500 USD. Conclusions: The application of the health-economic model to Sweden shows that in younger stroke patients with moderate disability, the societal value of SCT given a zero price of stem cells is 166,500 USD. Although the transplantation itself is more costly, SCT offers potential for cost offset and cost savings in a long-term perspective by reducing the disability after stroke. The therapy appeared cost effective under a wide range of assumptions. Hence, further research and development in stem cells suitable for stroke therapy could potentially produce great value to society.

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Section: Discussionmentioning

confidence: 70%

Societal Value of Stem Cell Therapy in Stroke – A Modeling Study

Svensson

Ghatnekar²,

Lindgren³

et al. 2012

Cerebrovasc Dis

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“…Goulionis and Vozikis, [49] Greece (independent study) Hjelmgren et al, [50] Sweden (independent study)…”

Section: Evaluations Of Surgical Optionsmentioning

confidence: 99%

“…Target population: pts with PD (HY III+IV) aged <64 y Research question/objective: ICER of an intrastriatal graft of dopaminerich human embryonic ventral mesencephalic tissue vs standard therapy as an example of early evaluation of a novel/hypothetical Disease progression: data from original study (79 pts; characteristics published in the article [50] ) in routine clinical practice Mortality: Swedish life tables, excess mortality from published study [51] HR-QOL: published studies [44,52] Costs: published studies [26] in [53] (for adverse event costs) Efficacy and adverse event rates: data [54] from ongoing nonrandomized clinical and published reports h38 000-70 000 per QALY maximum price premium of h12 000-64 000 would be possible…”

Section: Evaluations Of Surgical Optionsmentioning

confidence: 99%

Modelling the Cost Effectiveness of Treatments for Parkinsonʼs Disease

Dams

Bornschein²,

Reese

et al. 2011

PharmacoEconomics

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The objective of this review was to assess models of cost effectiveness for Parkinson's disease (PD) published after July 2002 and to derive recommendations for future modelling. A systematic literature search was performed in the databases PubMed, Current Contents, EMBASE, EconLit, the Cochrane Database of Systematic Reviews, and DARE (Database of Abstracts of Reviews of Effectiveness), NHS EED (Economic Evaluation Database) and HTA (Health Technology Assessment) of the UK NHS Centre for Review and Dissemination (July 2002 to March 2010). Only fully published studies using decision trees, Markov models, individual simulation models or sets of mathematical equations were included. Most of the 11 studies identified used Markov models (n = 9) and two employed were based on decision trees. Based on the Hoehn & Yahr (HY) scale, authors evaluated the cost effectiveness of drug treatments (n = 6), surgical approaches such as deep brain stimulation (n = 1) or striatal cell grafting (n = 1), and diagnostic procedures such as single photon emission computed tomography (SPECT) testing (n = 3) over a time horizon of 1 year to lifetime. Costs were adapted to address a societal and/or healthcare provider/third-party payer perspective. All but one of the interventions investigated were considered cost effective or cost saving. Cost-effectiveness modelling in PD between 2003 and 2010 showed only minor improvement when compared with our earlier review of models published from 1998 up to 2003. Cost-effectiveness modelling recommendations were complied with to only a limited extent, leaving room for quality improvement. More advanced modelling approaches may, so far, be under-represented, but may be used in the future, driven by the research question. Adverse events of treatment, co-morbidities or disease complications are not yet sufficiently included in the models to adequately represent clinical reality.

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“…The timing and choice of therapy is driven by the patient’s individual circumstances, meaning it is not possible to precisely define the standard of care (SOC) as a single therapy. This fact is reflected in several published health economic analyses, where the treatment in the comparator arm is commonly a homogeneous group of treatments referred to as SOC or best medical therapy [33–37,43–46]. Therefore, given the lack of a single clearly defined SOC per disease stage, we apply the available groupings of therapeutic options for a given SOC and the associated clinical and economic outcomes.…”

Section: Comparatormentioning

confidence: 99%

The potential price and access implications of the cost-utility and budget impact methodologies applied by NICE in England and ICER in the US for a novel gene therapy in Parkinson’s disease

Jørgensen

Servos

Kefalas

2018

Journal of Market Access & Health Policy

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Background: NICE in England, and ICER in the US both use cost-utility analyses (CUA) and budget impact analyses (BIA) to assess value for money and affordability, however the thresholds used differ greatly.Objective: To perform a cross-country comparison of the results of the CUA and BIA and detail the implications for reimbursed price and volumes, for a novel gene therapy for Parkinson’s disease (PD).Methods: A Markov model was built to perform country-specific CUAs and BIAsFindings: The US ceiling price identified through CUA is ~ 1.8 times higher than in England (aligning to our previous US/UK price comparison analysis of high-cost drugs). However, the net budget impact corresponding to these price levels would limit number of patients treated in order not to exceed the BIA threshold. Performance-based annuity payments can increase patient access at launch without exceeding the thresholds while reducing payers’ data uncertainty.Conclusion: Our cost-utility analysis in PD shows a difference in price potential between the US and England that aligns with what is observed in practice for other high-cost drugs. Furthermore, the budget impact threshold operational in England imposes a greater downwards pressure on price and/or volumes than the one applied by ICER in the US.

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Estimating the value of novel interventions for Parkinson's disease: An early decision-making model with application to dopamine cell replacement

Cited by 17 publications

References 38 publications

Societal Value of Stem Cell Therapy in Stroke – A Modeling Study

Societal Value of Stem Cell Therapy in Stroke – A Modeling Study

Modelling the Cost Effectiveness of Treatments for Parkinsonʼs Disease

The potential price and access implications of the cost-utility and budget impact methodologies applied by NICE in England and ICER in the US for a novel gene therapy in Parkinson’s disease

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