Dataset Information

Drug repurposing in pharmaceutical industry and its impact on market access: market access implications.

ABSTRACT:

Background

Drug repurposing is a group of development strategies employed in order to overcome some of the hurdles innate to drug research and development. Drug repurposing includes drug repositioning, reformulation and combination.

Objective

This study aimed to identify the determinants of successful market access outcome for drug repurposing in the United States of America (USA) and in Europe.

Methods

The case studies of repurposing strategies were identified through a systematic review of the literature. Price information and reimbursement conditions for all the case studies were collected mainly through access of public datasources. A list of attributes that could be associated with market access outcome (price level and reimbursement conditions) was developed, discussed, and validated by an external expert group. Detailed information for all attributes was researched and collected for each case study. Bivariate regression models were conducted to identify factors associated with price change for all repurposing cases. A similar analysis was performed for reformulation and repositioning cases, in the USA and in Europe, separately. A significance level of 5% was used for all analyses.

Results

A total of 144 repurposing case studies were included in the statistical analysis for evaluation of mean price change. Combination cases (the combination of two or more individual drug components) were excluded from the statistical analysis due to the low number of cases retrieved. The main attributes associated with a significant price increase for overall repurposing cases were 'change in administration setting to hospital' (374%, p<0.0001), 'addressing unmet needs' (69%, p<0.05), 'reformulations belonging to Group 3'-that is, change in administration route (117%, p<0.001), and being a repurposed product with the 'same brand name' as the original product (65%, p<0.05).

Conclusion

We found that the ability of the repurposed product to address unmet needs, a reformulation where the target product had a different administration route than the source product, and having a similar brand name for repurposed and original products, were variables that impacted a positive price change for repurposed drugs overall. Our research results also suggested that orphan designation could have a positive impact for repositioning in the USA, in particular. Although a change of administration from ambulatory to hospital setting seemed to be significantly correlated with a price increase for the target product, only one case was retrieved for this parameter; as such, it was not possible to infer a firm correlation between this parameter and a change in price.

SUBMITTER: Murteira S

PROVIDER: S-EPMC4865758 | biostudies-literature | 2014

REPOSITORIES: biostudies-literature

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Publications

Drug repurposing in pharmaceutical industry and its impact on market access: market access implications.

Murteira Susana S Millier Aurélie A Toumi Mondher M

Journal of market access & health policy 20140225

<h4>Background</h4>Drug repurposing is a group of development strategies employed in order to overcome some of the hurdles innate to drug research and development. Drug repurposing includes drug repositioning, reformulation and combination.<h4>Objective</h4>This study aimed to identify the determinants of successful market access outcome for drug repurposing in the United States of America (USA) and in Europe.<h4>Methods</h4>The case studies of repurposing strategies were identified through a sy ...[more]

PMID: 27226833

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Project description:BackgroundMedicinal 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.MethodsA 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.ResultsA 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.ConclusionGiven 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.

Project description:BackgroundThere has been increased demand for greater public accountability and transparency of private sector-led global health partnership programs. This study critically reviews and pilot tests the World Health Organization (WHO) medicine program checklist as a framework for public reporting and assessing of programs.MethodsWe reviewed each question on the WHO checklist for clarity and usability. Next, we pilot tested the subset of checklist questions focused on program assessment. We extracted and analyzed publicly available information on one randomly selected program from each of the 20 largest research-based biopharmaceutical companies. For each program, we assessed whether publicly available information allowed for an assessment of each relevant question in the checklist.ResultsChecklist questions fit in four main categories: [1] national health and development plans, needs, capacity, laws and policies; [2] financial, performance, and public accountability; [3] risk management and mitigation strategies; and [4] long-term sustainability. Nearly all (21 of 22) questions in the checklist require information best provided by companies; one question requires information best provided by governments.Programs frequently reported on the public health needs of their programs (100%), program objectives and activities (100%) and the actual or expected program outputs (95%). There was less information on program alignment with country plans and capacity (50%), detailed program monitoring and evaluation plan (20%), risks mitigation strategies (5%), program needs assessment (5%), and additional resources required from or contributed by government (0%).ConclusionThe WHO checklist of key considerations for evaluating proposals for access to medicine programs could be a useful framework for public reporting of program information as most of checklist questions ask for data that should be available to those leading the program. Further revisions of the WHO checklist will help refine it to improve clarity and content validity.

Project description:A change in the pharmaceutical environment has occurred from previously only needing to convince regulators of a product's safety and efficacy to obtain marketing authorisation to now needing to satisfy the value perceptions of other stakeholders, including payers, to attain market access for products. There is thus the need to understand the concept of market access that may be defined as 'the process that ensures the development and commercial availability of pharmaceutical products with appropriate value propositions, leading to their prescribing and to successful uptake decisions by payers and patients, with the ultimate goal of achieving profitability and best patient outcomes'. The aim of this research therefore was to explore the understanding of market access among various stakeholders and how their understanding of this concept could improve patient access to pharmaceutical products.A literature review was conducted on MEDLINE by using the term 'market access' to find articles with explicit definitions of market access for pharmaceutical products; non-peer-reviewed and other grey literature sources were also examined. A paper-based interview survey was also conducted in three different settings. The respondents were asked about what factors they think contribute to the successful development of pharmaceutical products, as well as their definition of market access for these medicines.The peer-reviewed literature review did not reveal appropriate comprehensive definitions for market access, although several definitions were proposed from the non-peer-reviewed literature. These definitions ranged from basic to detailed. The survey of 110 respondents revealed differing levels of understanding of market access. Factors considered to influence successful market access, as described by the respondents, included unmet need/burden of disease (68.2%), clinical efficacy (47.3%), comparator choice (36.4%), safety profile (36.4%), and price (35.5%).The concept of market access is still poorly understood, and the definition varies depending on the stakeholders' perspectives. For cost-effective products to be developed and made accessible to patients, there is a need for wider understanding of market access and the value perspectives of the various stakeholders. There is also a need to determine whether and how involved payers should be in the development of pharmaceutical products.

Dataset Information

Drug repurposing in pharmaceutical industry and its impact on market access: market access implications.

Background

Objective

Methods

Results

Conclusion

Publications

Drug repurposing in pharmaceutical industry and its impact on market access: market access implications.

Similar Datasets

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