Biomarkers and personalised medicine in rheumatoid arthritis: a proposal for interactions between academia, industry and regulatory bodies.

in Annals of the Rheumatic Diseases (2011), 70(10), 1713-8

Rheumatoid arthritis (RA) is one of the most appropriate conditions for the application of personalised medicine as a high degree of heterogeneity has been recognised, which remains to be explained. Such ... [more ▼]

Rheumatoid arthritis (RA) is one of the most appropriate conditions for the application of personalised medicine as a high degree of heterogeneity has been recognised, which remains to be explained. Such heterogeneity is also reflected in the large number of treatment targets and options. A growing number of biologics as well as small molecules are already in use and there are promising new drugs in development. In order to make the best use of treatment options, both targeted and non-targeted biomarkers have to be identified and validated. To this aim, new rules are needed for the interaction between academia and industry under regulatory control. Setting up multi-centre biosample collections with clear definition of access, organising early, possibly non-committing discussions with regulatory authorities, and defining a clear route for the validation, qualification and registration of the biomarker-drug combination are some of the more critical areas where effective collaboration between the drug industry, academia and regulators is needed. [less ▲]

Health-related quality of life after total knee or hip replacement: a 7-year prospective study

in Annals of the Rheumatic Diseases (2010, June), 69(Suppl.3), 469

Potential Clinical and Economic Impact of Nonadherence with Osteoporosis Medications.

in Calcified Tissue International (2010), 86

This study aims to estimate the potential clinical and economic implications of therapeutic adherence to bisphosphonate therapy. A validated Markov microsimulation model was used to estimate the impact of ... [more ▼]

This study aims to estimate the potential clinical and economic implications of therapeutic adherence to bisphosphonate therapy. A validated Markov microsimulation model was used to estimate the impact of varying adherence to bisphosphonate therapy on outcomes (the number of fractures and the quality-adjusted life-years [QALYs]), health-care costs, and the cost-effectiveness of therapy compared with no treatment. Adherence was divided into persistence and compliance, and multiple scenarios were considered for both concepts. Analyses were performed for women aged 65 years with a bone mineral density T-score of -2.5. Health outcomes and the cost-effectiveness of therapy improved significantly with increasing compliance and/or persistence. In the case of real-world persistence and with a medical possession ratio (MPR; i.e., the number of doses taken divided by the number of doses prescribed) of 100%, the QALY gain and the number of fractures prevented represented only 48 and 42% of the values estimated assuming full persistence, respectively. These proportions fell to 27 and 23% with an MPR value of 80%. The costs per QALY gained, for branded bisphosphonates (and generic alendronate), were estimated at <euro>19,069 (<euro>4,871), <euro>32,278 (<euro>11,985), and <euro>64,052 (<euro>30,181) for MPR values of 100, 80, and 60%, respectively, assuming real-world persistence. These values were <euro>16,997 (<euro>2,215), <euro>24,401 (<euro>6,179), and <euro>51,750 (<euro>20,569), respectively, assuming full persistence. In conclusion, poor compliance and failure to persist with osteoporosis medications results not only in deteriorating health outcomes, but also in a decreased cost-effectiveness of drug therapy. Adherence therefore remains an important challenge for health-care professionals treating osteoporosis. [less ▲]

Clinical en economic implications of non-adherence with osteoporosis medications

in Osteoporosis International (2009, March), 20(S1), 16

Development and Validation of a Markov Microsimulation Model for the Economic Evaluation of Treatments in Osteoporosis.

in Value in Health (2009), 12(5), 687-696

ABSTRACT Objective: Markov models are increasingly used in economic evaluations of treatments for osteoporosis. Most of the existing evaluations are cohort-based Markov models missing comprehensive memory ... [more ▼]

ABSTRACT Objective: Markov models are increasingly used in economic evaluations of treatments for osteoporosis. Most of the existing evaluations are cohort-based Markov models missing comprehensive memory management and versatility. In this article, we describe and validate an original Markov microsimulation model to accurately assess the cost-effectiveness of prevention and treatment of osteoporosis. Methods: We developed a Markov microsimulation model with a lifetime horizon and a direct health-care cost perspective. The patient history was recorded and was used in calculations of transition probabilities, utilities, and costs. To test the internal consistency of the model, we carried out an example calculation for alendronate therapy. Then, external consistency was investigated by comparing absolute lifetime risk of fracture estimates with epidemiologic data. Results: For women at age 70 years, with a twofold increase in the fracture risk of the average population, the costs per quality-adjusted life-year gained for alendronate therapy versus no treatment were estimated at Euro 9105 and Euro 15,325, respectively, under full and realistic adherence assumptions. All the sensitivity analyses in terms of model parameters and modeling assumptions were coherent with expected conclusions and absolute lifetime risk of fracture estimates were within the range of previous estimates, which confirmed both internal and external consistency of the model. Conclusion: Microsimulation models present some major advantages over cohort-based models, increasing the reliability of the results and being largely compatible with the existing state of the art, evidence-based literature. The developed model appears to be a valid model for use in economic evaluations in osteoporosis. [less ▲]

Cost-utility of calcium and vitamin D supplementation in the treatment of postmenopausal osteoporotic women

in Osteoporosis International (2008, December), 19(S2), 363

Efficiency of calcium and vitamin D in the management of osteoporosis

Conference (2008, July)

Efficiency of calcium and vitamin D supplementation in the management of osteoporosis

in Osteoporosis International (2008, April), 19(S1), 113

Cost-utility of strontium ranelate in the treatment of postmenopausal women

in Osteoporosis International (2008, April), 19(S1), 6

Lifetime absolute risk of hip and other osteoporotic fracture in Belgian women.

in BONE (2008), 43(6), 991-4

OBJECTIVES: To estimate the lifetime absolute risks of hip and other osteoporotic fracture in Belgian women aged 60 years and to examine the effect of changes in baseline population fracture risk and ... [more ▼]

OBJECTIVES: To estimate the lifetime absolute risks of hip and other osteoporotic fracture in Belgian women aged 60 years and to examine the effect of changes in baseline population fracture risk and changes in life expectancy. MATERIALS AND METHODS: Estimates were performed using a Markov microsimulation model and were based on the incidence of first fracture as well as life expectancy. Baseline scenario included projected mortality rates and increasing fracture incidence by 1% per year. Alternative scenarios were performed on age, life expectancy and trends in fracture incidence. Lifetime fracture risk for osteoporotic population (T-score <or= -2.5) was also estimated. RESULTS: In the baseline scenario, lifetime absolute risks of hip fracture and of any major osteoporotic fracture (hip, clinical vertebral or wrist) were respectively 24.8% and 44.3%. Alternative scenarios showed that when assuming no change of age-specific fracture rates over time, these lifetime risks were 18.3% and 35.2%, while these values were 20.0% and 38.3% assuming no future mortality reductions. For osteoporotic women, these values were respectively 34.5% and 51.5%. CONCLUSION: We conclude that absolute lifetime fracture risks are substantial and that trends in fracture incidence and changes in life expectancy have a marked impact on absolute lifetime fracture risks. [less ▲]