Dataset Information

The Impact of the Priority Review Voucher on Research and Development for Tropical Diseases.

ABSTRACT:

Background

In 2007, the priority review voucher (PRV) was implemented in the US to incentivize research and development (R&D) for tropical diseases. The PRV is issued by the US FDA and grants a quicker review to manufacturers upon successful development of a product for a disease eligible for the program.

Objective

The objective of this analysis was to assess whether the PRV has incentivized R&D (measured as clinical trial activity) for the intended tropical diseases.

Method

We used a difference-in-difference-in-differences (DDD) strategy by exploiting variation in its implementation across diseases and registries around the world. Clinical trials were retrieved from the World Health Organization International Clinical Trials Registry Platform for the years 2005-2019.

Results

We found a positive, but not statistically significant, effect of the PRV on stimulating R&D activity. Delayed effects of the policy could not be found.

Conclusion

Our findings, which were robust across a series of robustness tests, suggest that the PRV program is not associated with a trigger in innovation for neglected diseases and therefore should not be considered as a stand-alone solution. It should be supplemented with other government measures to incentivize R&D activity. To increase the value of the program, we recommend that the PRV only be awarded to novel products and not to products that have already been licensed outside the US. Doing so would restrict the number of vouchers awarded and slow down their ongoing market depreciation. Finally, we propose that product sponsors be required to submit an access plan for PRV-awarded products.

SUBMITTER: Aerts C

PROVIDER: S-EPMC9217899 | biostudies-literature | 2022 Jun

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

The Impact of the Priority Review Voucher on Research and Development for Tropical Diseases.

Aerts Celine C Barrenho Eliana E Miraldo Marisa M Sicuri Elisa E

Pharmaceutical medicine 20220519 3

<h4>Background</h4>In 2007, the priority review voucher (PRV) was implemented in the US to incentivize research and development (R&D) for tropical diseases. The PRV is issued by the US FDA and grants a quicker review to manufacturers upon successful development of a product for a disease eligible for the program.<h4>Objective</h4>The objective of this analysis was to assess whether the PRV has incentivized R&D (measured as clinical trial activity) for the intended tropical diseases.<h4>Method</h ...[more]

PMID: 35588350

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Project description:BackgroundThe World Health Organization's (WHO) Neglected Tropical Disease (NTD) Road Map for 2021-2030 was recently endorsed by all member states at the World Health Assembly in November 2020. Although only 3 of the 20 NTDs are endemic in Canada (i.e., echinococcosis, rabies, and scabies), the Canadian research community has contributed to advancing the knowledge base of all 20 NTDs. Previous research comprehensively detailed Canadian research on 11 NTDs between 1950 and 2010 using a network analysis approach. The specific objective of the present analysis was to update the publication record over the last decade (2010-2019) to include all 20 NTDs.Materials and methodsA bibliometric analysis was conducted in Scopus and Web of Science databases (for English or French articles published between January 1, 2010 and December 31, 2019) using appropriate search terms for each of the 20 NTDs and where at least 1 of the authors had a Canadian institution address. A 21st search was added to include publications including multiple NTDs or a discussion of NTDs in general. Following assessment of inclusion and exclusion criteria, 2 reviewers independently screened all abstracts, with discordant observations rereviewed to arrive at an agreement. Duplicates were removed.ResultsA total of 1,790 publications were retrieved (1,738 with a disease-specific NTD focus and 52 with a general NTD focus, resulting in 1,659 unique publications), giving an average of over 160 articles per year. Over 80% were classified as full-length research articles. The top 3 journals in terms of frequency were PLOS Neglected Tropical Diseases, PLOS ONE, and the American Journal of Tropical Medicine and Hygiene. Authors' institutions were from all Canadian provinces. While all 20 NTDs were addressed in these publications, the 5 most commonly studied were leishmaniasis, dengue fever and chikungunya, Chagas disease, soil-transmitted helminthiases, and rabies.ConclusionsCanadian researchers across the country have contributed to the evidence base of all 20 NTDs, publishing an average of over 160 publications per year between 2010 and 2019. As WHO NTD Road Map 2021-2030 rolls out globally, the Canadian research community, in collaboration with its partners and in solidarity with people living in vulnerable circumstances in endemic regions worldwide, is well positioned to meet future research challenges so that the goal of eliminating the disease burden attributable to NTDs can be achieved.

Project description:The global burden attributed to Neglected Tropical Diseases (NTDs) is 47.9 million Disability-Adjusted Life Years (DALYs). These diseases predominantly affect disadvantaged populations. Priority for NTDs has grown in recent years, which is observed by their inclusion in the sustainable development goals (SDGs). This study analyzed the process that allowed these diseases to be included on the global health policy agenda. This global policy analysis used the Shiffman and Smith framework to understand the determinants of global health political priority for NTDs. The framework comprises four categories: actor power, ideas, political contexts, and issue characteristics. Global documents and World Health Assembly (WHA) resolutions were examined, key-informant interviews were conducted, and academic publications were reviewed to understand the four categories that comprise the framework. A total of 37 global policy documents, 15 WHA resolutions, and 38 academic publications were examined. Twelve semi-structured interviews were conducted with individuals representing different sectors within the NTD community who have been involved in raising the priority of these diseases. This study found that several factors helped better position NTDs in the global health agenda. These include the leadership of actors that mobilized the global health community, the creation of a label combining these diseases as a group to represent a larger disease burden, the presence of mechanisms aligning the NTD community, and the agreement on ways to present the NTD burden and potential solutions. The process of building the priority of NTDs in the global health agenda shows that several determinants led to positive outcomes, but these diseases continue to have low priority at the global level which requires the implementation of actions to increase their global priority. These include sustaining the commitment of current actors and engaging new ones; increasing the attention given to diseases formerly categorized as "tool-deficient", including zoonotic NTDs; continue leveraging on policy windows and creating favorable policy moments to sustain commitment, as well as setting realistic targets. Findings from this study can help develop strategies to build the momentum and drive actions to implement the goals of the new Roadmap for NTDs in the pathway to universal health coverage (UHC) and sustainable development.

Project description:BackgroundThis scoping review provides a thorough analysis of how stakeholders have so far been involved in research priority setting. The review describes, synthesizes, and evaluates research priority setting projects not only for the field of health-as previous reviews have done-but does so on a much broader scale for any research area.MethodsA comprehensive electronic literature search was conducted in the databases PubMed, Scopus, and Web of Science. Reflecting the importance of grey literature, Google Scholar and relevant websites were also screened for eligible publications. A computational approach was then used for the study selection. The final screening for inclusion was done manually.ResultsThe scoping review encompasses 731 research priority setting projects published until the end of 2020. Overall, the projects were conducted within the realm of 50 subject areas ranging from agriculture and environment over health to social work and technology. Key learnings include that nearly all priority setting projects aimed to identify research priorities for the field of health (93%), particularly for nursing and care, cancer, pediatrics, and mental, behavioral and neurodevelopmental disorders. Only 6% of the projects were not health-related and 1% identified research priorities at the interface between health and a non-health area. Over time, 30 different stakeholder groups took part in research priority setting. The stakeholders most frequently asked to identify research priorities were doctors, patients, academics/researchers, nurses, allied healthcare professionals, family members, friends, and carers. Nearly two thirds of all projects have been conducted in Europe and North America. Overall, only 9% of the projects emphasized the importance of stakeholders in their goals and rationales and actively involved them. In around a quarter of the projects, stakeholders deliberated on their research priorities throughout the entire process.ConclusionBy mapping out the complex landscape of stakeholder involvement in research priority setting, this review guides future efforts to involve stakeholders effectively, inclusively, and transparently, which in turn may increase the overall value of research for society. As a practical addition to this review, the first worldwide research priority setting database was created: https://ois.lbg.ac.at/en/project-database . The database contains all the projects analyzed for this review and is constantly updated with the latest published research priority setting projects.

Project description:BackgroundUnderstanding the barriers to and facilitators of participation in research could enhance recruitment rates for biomedical research on Neglected Tropical Diseases (NTDs) and help to avoid the problems associated with poor recruitment.Methodology/principal findingsWe conducted a systematic review to identify factors related to willingness to participate in biomedical research on Neglected Tropical Diseases (NTDs). Our search included the following databases: Medline/PubMed, Embase (Embase.com), Global Index Medicus (WHO), Web of Science (Core collection), and gray literature. We included studies that analyzed or reported factors associated with willingness to participate in NTD research, using either quantitative methods (such as clinical trials, cohorts, and cross-sectional studies) or qualitative methods (such as focus group discussions, semi-structured interviews, and in-depth interviews). There were no language restrictions, but we excluded review articles, notes, case reports, letters to the editor, editor's notes, extended abstracts, proceedings, patents, editorials, and other editorial materials. Screening of citations, data extraction, and risk of bias assessment was conducted by independent reviewers, according to the study protocol registered on PROSPERO. For analyses, we assessed the frequency of barriers, enablers, and the frequency of recruitment interventions mentioned in the included studies. The protocol for this systematic review was registered under registration number CRD42020212536. (S1 Appendix) We identified 2070 citations, 1470 from the databases, and 600 from other sources. From those, eleven studies were selected for data extraction and analysis. The studies were conducted in Africa, Asia, and North America. Personal health benefits, monetary benefits, and community engagement and sensitization strategies were identified as the main reasons for participating in biomedical research on Neglected Tropical Diseases (NTDs). However, distrust in researchers, lack of knowledge about research methods among potential participants, and previous negative experiences were identified as the main barriers to participating in biomedical research on NTDs.Conclusions/significanceThis systematic review provides recommendations for improving adherence to biomedical research on Neglected Tropical Diseases, which can be applied in practice.

Dataset Information

The Impact of the Priority Review Voucher on Research and Development for Tropical Diseases.

Background

Objective

Method

Results

Conclusion

Publications

The Impact of the Priority Review Voucher on Research and Development for Tropical Diseases.

Similar Datasets

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