J. Mark. Access Health Policy, Volume 1, Issue 1 (January 2013) – 6 articles

Objective: This study compares the performances of new-molecule (NM) launches in Turkey with those in the European Union and United States for the years 2007–2013. Methods: The Thomson Reuters Newport Horizon for Innovators Database is used to identify NMs with a launch date after 1 January 2007, worldwide and marketing authorization approval after 1 January 2007, in the European Union. The launch dates for the European Union, the United States, and Turkey were retrieved from the same database. Data for Turkey were confirmed via IMS and RxMedia. Results: Out of 183 records identified that are launched in the European Union, the United States, or both, 44 of the NMs are launched in Turkey (24%). Conclusion: The results of this study show that 24% of the NMs that are launched in either the European Union or United States were able to be launched in Turkey with a mean delay of 821 days (2.25 years). Full article

Objective: Since 2004, the National Institute of Health and Clinical Excellence (NICE) has required manufacturers to conduct a probabilistic sensitivity analysis (PSA) in their technology appraisals. The objective of this review is to assess the cost-effectiveness of different technology appraisals and compare them with the actual decision made by the NICE based on PSA. Methods: The search term ‘probabilistic sensitivity analysis’ was used on the NICE home page (25 January 2012). The appraisals identified in the search were assessed and subjected to further review, if a probability of being cost-effective was provided, regardless of the threshold indicated. If several probabilities were provided, the number provided by the evidence review group was used. If several scenarios were presented, the base case scenario was chosen. Finally, the probabilities of being cost-effective were compared with the actual decision made, which could result in two outcomes: recommended or not recommended. Results: A total of 31 assessments were included for the final review. The results were plotted on a graph to illustrate whether there was a relationship between the PSA outcomes and the final recommendation. The assessments were ranked according to their probability of being cost-effective. Conclusion: A higher probability of a technology being cost-effective was correlated with more positive decision-making. There appeared to be a clear threshold at which technologies with a 40% certainty of being cost-effective tended to be recommended, whereas those below the threshold were not recommended. The reports suggested that the incremental cost-effectiveness ratios (ICER) estimate was not a robust driver of decision-making. A NICE applicant should pay increased attention to the PSA in addition to the ICER estimate. Full article

Background: Medicinal products that have been developed and approved for one disease may be the object of additional clinical development in other disease areas or of additional pharmaceutical development for new and different formulations. The newly developed products can be named as repositioned or reformulated products, respectively. Market access of repositioned or reformulated products in Europe and the United States is an interesting object of study as it may provide clarity about which parameters are assessed and considered to bring added value, other than the molecule itself. As such, we aim to evaluate if the added value of repositioned or reformulated medicinal products can be systematically described, quantified, and predicted. As a first step toward investigating the impact of market access on drug research and development trends for repositioned and reformulated products, it is necessary to have consistency in the designations for the case studies evaluated in this project. In an attempt to achieve that consistency, the current study aims to propose harmonized definitions for the repositioning and reformulation strategies and to propose a taxonomy for the medicinal products derived thereof. Methods: A systematic literature review was conducted to collect information on existing cases of repositioning or reformulation. A search strategy was developed by defining the search objectives, targeted data sources, search keywords, and inclusion/exclusion criteria for the retrieved documents. Results: A total of 505 publications were retrieved through a search of the main data sources. The screenings and the ad hoc search led to a total of 56 publications to be used for the case study data extraction. In total, 87 repositioning and/or reformulation cases were found described in the literature, 23 of which presented different definitions and/or classifications by different authors. Conclusion: Given the disparity and inconsistency of terminologies and classifications in the literature, a harmonized nomenclature for drug repositioning, reformulation, and combination cases will allow for a robust analysis of the added value and market access conditions attributed for each strategy and case type as assessed by regulators and payors in Europe and the United States. After evaluation of the existing terminologies and given the absence of clear and consistent definitions for drug reformulation and repositioning in the literature, we propose a global terminology and taxonomy in order to cover all of the previously unclear definitions and classifications for repositioned and reformulated products. Full article

Access to the French drug market is being impacted by an ongoing dramatic shift in practice as well as by two laws that came into force in December 2011. This new environment has been described and analyzed in two separate articles. This second article analyzes how this new environment will actually impact the access to French drug market. French drug market access will be increasingly driven by comparative-effectiveness and cost-effectiveness data, and an increased role of postmarketing studies in the years to come. This access is evolving in a more complex environment for stakeholders due to the uncertainties surrounding these changes and it will be more complex and difficult for the pharmaceutical industry to address. The main issue faced by the pharmaceutical companies will be to minimize uncertainty at the time of a drug's launch to narrow the decision window. This is a major change of paradigm for the pharmaceutical business, in which pre- and postlaunch risks are directed toward the pharmaceutical industry. Full article

Access to the French drug market is being impacted by an ongoing dramatic shift in practice as well as by two laws that came into force in December 2011. This new environment has been described and analyzed in two separate articles. The first article aims to describe the recent changes in access to the French drug market. The severity of the condition being treated, which used to be the main determinant of the drug's reimbursement level in France, has now been replaced with the drugs’ efficacy criterion. Moreover, the effect size required for acknowledging drug innovation has substantially increased. Perceived evidence might also be more important than actual evidence. Comparative evidence and real-world data are considered critical conditions for marketing authorization. Cost-effectiveness studies will now be part of the market access requirements for all drugs in order to satisfy the selection criteria for medico-economic assessment. Full article