Considerable variety in how patients respond to treatments, driven by differences in their geno- and/ or phenotypes, calls for a more tailored approach. This is already happening, and will accelerate with developments in personalized medicine. However, its promise has not always translated into improvements in patient care due to the complexities involved. There are also concerns that advice for tests has been reversed, current tests can be costly, there is fragmentation of funding of care, and companies may seek high prices for new targeted drugs. There is a need to integrate current knowledge from a payer’s perspective to provide future guidance. Multiple findings including general considerations; influence of pharmacogenomics on response and toxicity of drug therapies; value of biomarker tests; limitations and costs of tests; and potentially high acquisition costs of new targeted therapies help to give guidance on potential ways forward for all stakeholder groups. Overall, personalized medicine has the potential to revolutionize care. However, current challenges and concerns need to be addressed to enhance its uptake and funding to benefit patients.

Background: There are potential conflicts between authorities and companies to fund new premium priced drugs especially where there are safety and/or budget concerns. Dabigatran, a new oral anticoagulant for the prevention of stroke in patients with non-valvular atrial fibrillation (AF), exemplifies this issue. Whilst new effective treatments are needed, there are issues in the elderly with dabigatran due to variable drug concentrations, no known antidote and dependence on renal elimination. Published studies have shown dabigatran to be cost-effective but there are budget concerns given the prevalence of AF. There are also issues with potentially re-designing anticoagulant services. This has resulted in activities across countries to better manage its use. Objective: To (i) review authority activities in over 30 countries and regions, (ii) use the findings to develop new models to better manage the entry of new drugs, and (iii) review the implications for all major stakeholder groups. Methodology: Descriptive review and appraisal of activities regarding dabigatran and the development of guidance for groups through an iterative process. Results: There has been a plethora of activities among authorities to manage the prescribing of dabigatran including extensive pre-launch activities, risk sharing arrangements, prescribing restrictions, and monitoring of prescribing post-launch. Reimbursement has been denied in some countries due to concerns with its budget impact and/or excessive bleeding. Development of a new model and future guidance is proposed to better manage the entry of new drugs, centering on three pillars of pre-, peri-, and post-launch activities. Conclusion: Models for introducing new drugs are essential to optimize their prescribing especially where there are concerns. Without such models, new drugs may be withdrawn prematurely and/or struggle for funding.

Introduction: In January 2018 the European Commission published a Proposal for a Regulation on Health Technology Assessment (HTA): ‘Proposal for a Regulation on health technology assessment and amending Directive 2011/24/EU’. A number of stakeholders, including some Member States, welcomed this initiative as it was considered to improve collaboration, reduce duplication and improve efficiency; there were however a number of concerns including its legal basis, the establishment of a single managing authority, the preservation of national jurisdiction over HTA decision making and the voluntary/mandatory uptake of joint assessments by Member States. Areas covered: This paper presents the consolidated views and considerations on the original Proposal as set by the European Commission of a number of policy makers, payers, experts from pricing and reimbursement authorities and academics from across Europe. Expert commentary: The Proposal has since been extensively discussed at Council and while good progress has been achieved, there are still divergent positions. The European Parliament gave a number of recommendations for amendments. If the Proposal is approved, it is important that balanced, improved outcome is achieved for all stakeholders. If not approved, the extensive contribution and progress attained should be sustained and preserved, and the best alternative solutions found.

The economic burden of rheumatoid arthritis (RA) on society is high. Disease-modifying antirheumatic drugs (DMARDs) are the cornerstone of therapy. Biological DMARDs are reported to prevent disability and improve quality of life, thus reducing indirect RA costs. We systematically reviewed studies on the relationship between RA and indirect costs comparing biological treatment with standard care. Studies, economic analyses, and systematic reviews published until October 2018 through a MEDLINE search were included. A total of 153 non-duplicate citations were identified, 92 (60%) were excluded as they did not meet pre-defined inclusion criteria. Sixty-one articles were included, 17 of them (28%) were reviews. After full-text review, 28 articles were included, 11 of them were reviews. Costs associated with productivity loss are substantial; in several cases, they may represent over 50% of the total. The most common method of estimation is the Human Capital method. However, certain heterogeneity is observed in the method of estimating, as well as in the resultant figures. Data from included trials indicate that biological therapy is associated with improved labor force participation despite an illness, in which the natural course of disease is defined by progressive work impairment. Use of biological DMARDs may lead to significant indirect cost benefits to society.

(2008). Generic olanzapine: health authority opportunity or nightmare? Expert Review of Pharmacoeconomics & Outcomes Research: Vol. 8, No. 6, pp. 549-555.