Is the priority review voucher program stimulating new drug development for tropical diseases?
Ontology highlight
ABSTRACT:
SUBMITTER: Kerr KW
PROVIDER: S-EPMC6103519 | biostudies-literature | 2018 Aug
REPOSITORIES: biostudies-literature
Publications
Is the priority review voucher program stimulating new drug development for tropical diseases?
Kerr Kirk W KW Henry Thomas C TC Miller Kathleen L KL
PLoS neglected tropical diseases 20180809 8
Congress created the tropical disease priority review voucher program to stimulate new drug development for tropical diseases. An analysis of the pharmaceutical pipeline indicates that the development of drugs for these tropical diseases has increased. However, the effects of the program are not uniform across all diseases, as malaria and tuberculosis have seen significant new drug development, while other diseases have not. ...[more]
Similar Datasets
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.
| S-EPMC7423089 | biostudies-literature
Project description:There is an urgent need to make drug discovery cheaper and faster. This will enable the development of treatments for diseases currently neglected for economic reasons, such as tropical and orphan diseases, and generally increase the supply of new drugs. Here, we report the Robot Scientist 'Eve' designed to make drug discovery more economical. A Robot Scientist is a laboratory automation system that uses artificial intelligence (AI) techniques to discover scientific knowledge through cycles of experimentation. Eve integrates and automates library-screening, hit-confirmation, and lead generation through cycles of quantitative structure activity relationship learning and testing. Using econometric modelling we demonstrate that the use of AI to select compounds economically outperforms standard drug screening. For further efficiency Eve uses a standardized form of assay to compute Boolean functions of compound properties. These assays can be quickly and cheaply engineered using synthetic biology, enabling more targets to be assayed for a given budget. Eve has repositioned several drugs against specific targets in parasites that cause tropical diseases. One validated discovery is that the anti-cancer compound TNP-470 is a potent inhibitor of dihydrofolate reductase from the malaria-causing parasite Plasmodium vivax.
| S-EPMC4345494 | biostudies-literature
Project description:Conventional patent-based drug development incentives work badly for the developing world, where commercial markets are usually small to non-existent. For this reason, the past decade has seen extensive experimentation with alternative R&D institutions ranging from private-public partnerships to development prizes. Despite extensive discussion, however, one of the most promising avenues-open source drug discovery-has remained elusive. We argue that the stumbling block has been the absence of a critical mass of preexisting work that volunteers can improve through a series of granular contributions. Historically, open source software collaborations have almost never succeeded without such "kernels".HERE, WE USE A COMPUTATIONAL PIPELINE FOR: (i) comparative structure modeling of target proteins, (ii) predicting the localization of ligand binding sites on their surfaces, and (iii) assessing the similarity of the predicted ligands to known drugs. Our kernel currently contains 143 and 297 protein targets from ten pathogen genomes that are predicted to bind a known drug or a molecule similar to a known drug, respectively. The kernel provides a source of potential drug targets and drug candidates around which an online open source community can nucleate. Using NMR spectroscopy, we have experimentally tested our predictions for two of these targets, confirming one and invalidating the other.The TDI kernel, which is being offered under the Creative Commons attribution share-alike license for free and unrestricted use, can be accessed on the World Wide Web at http://www.tropicaldisease.org. We hope that the kernel will facilitate collaborative efforts towards the discovery of new drugs against parasites that cause tropical diseases.
| S-EPMC2667270 | biostudies-literature
Project description:Today, the World Health Organization recognizes 17 major parasitic and related infections as the neglected tropical diseases (NTDs). Despite recent gains in the understanding of the nature and prevalence of NTDs, as well as successes in recent scaled-up preventive chemotherapy strategies and other health interventions, the NTDs continue to rank among the world's greatest global health problems. For virtually all of the NTDs (including those slated for elimination under the auspices of a 2012 London Declaration for NTDs and a 2013 World Health Assembly resolution [WHA 66.12]), additional control mechanisms and tools are needed, including new NTD drugs, vaccines, diagnostics, and vector control agents and strategies. Elimination will not be possible without these new tools. Here we summarize some of the key challenges in translational science to develop and introduce these new technologies in order to ensure success in global NTD elimination efforts.
| S-EPMC4774924 | biostudies-other
Project description:Drug development (DD) is a multidisciplinary process that spans the translational continuum, yet remains an understudied entity in medical schools and biomedical science institutes. In response to a growing interest and unmet need, we implemented a DD course series that details identification of viable molecular targets, clinical trial design, intellectual property, and marketing. Enrollment is open to faculty, postdoctoral trainees, and MD, PhD, and MS students. After 2 years, 37 students and 23 students completed the fall and spring courses, respectively. Pre/post-surveys demonstrated gained knowledge across course topics, with mean survey scores increased by 66% (p < 0.001) after each course. Lectures for each course were consistently rated highly, with a mean course rating of 4.1/5. Through this program, trainees will have a more innovative approach toward identification of therapeutic targets and modalities. Furthermore, they will learn to integrate technology and biomedical informatics to find creative solutions in the DD process.
| S-EPMC5350998 | biostudies-literature
Project description:BackgroundNeglected tropical diseases (NTDs) are generally assumed to be concentrated in poor populations, but evidence on this remains scattered. We describe within-country socioeconomic inequalities in nine NTDs listed in the London Declaration for intensified control and/or elimination: lymphatic filariasis (LF), onchocerciasis, schistosomiasis, soil-transmitted helminthiasis (STH), trachoma, Chagas' disease, human African trypanosomiasis (HAT), leprosy, and visceral leishmaniasis (VL).MethodologyWe conducted a systematic literature review, including publications between 2004-2013 found in Embase, Medline (OvidSP), Cochrane Central, Web of Science, Popline, Lilacs, and Scielo. We included publications in international peer-reviewed journals on studies concerning the top 20 countries in terms of the burden of the NTD under study.Principal findingsWe identified 5,516 publications, of which 93 met the inclusion criteria. Of these, 59 papers reported substantial and statistically significant socioeconomic inequalities in NTD distribution, with higher odds of infection or disease among poor and less-educated people compared with better-off groups. The findings were mixed in 23 studies, and 11 studies showed no substantial or statistically significant inequality. Most information was available for STH, VL, schistosomiasis, and, to a lesser extent, for trachoma. For the other NTDs, evidence on their socioeconomic distribution was scarce. The magnitude of inequality varied, but often, the odds of infection or disease were twice as high among socioeconomically disadvantaged groups compared with better-off strata. Inequalities often took the form of a gradient, with higher odds of infection or disease each step down the socioeconomic hierarchy. Notwithstanding these inequalities, the prevalence of some NTDs was sometimes also high among better-off groups in some highly endemic areas.ConclusionsWhile recent evidence on socioeconomic inequalities is scarce for most individual NTDs, for some, there is considerable evidence of substantially higher odds of infection or disease among socioeconomically disadvantaged groups. NTD control activities as proposed in the London Declaration, when set up in a way that they reach the most in need, will benefit the poorest populations in poor countries.
| S-EPMC4865383 | biostudies-literature
Project description:Active Kids is a government-led, universal voucher program that aims to reduce the cost of participation in structured physical activity for all school-enrolled children in New South Wales (NSW), Australia. As part of the Active Kids program evaluation, this cross-sectional study examined the Active Kids' program's reach to children in NSW and their physical activity behaviors, before voucher use. Demographic registration data from all children (4.5-18 years old) who registered for an Active Kids voucher in 2018 (n = 671,375) were compared with Census data. Binary and multinomial regression models assessed which correlates were associated with meeting physical activity guidelines and participation in the sessions of structured physical activity. The Active Kids program attracted more than half (53%) of all eligible children in NSW. Children who spoke a primary language other than English at home, were aged 15-18 years old, lived in the most disadvantaged areas, and girls, were less likely to register. Of the registered children, 70% had attended structured physical activity sessions at least once a week during the previous 12 months, whilst 19% achieved physical activity guidelines. Active Kids achieved substantial population reach and has the potential to improve children's physical activity behaviors.
| S-EPMC7460334 | biostudies-literature
Project description:BackgroundNeglected tropical diseases (NTDs) are closely related to poverty and affect over a billion people in developing countries. The unmet treatment needs cause high mortality and disability thereby imposing a huge burden with severe social and economic consequences. Although coordinated by the World Health Organization, various philanthropic organizations, national governments and the pharmaceutical industry have been making efforts in improving the situation, the control of NTDs is still inadequate and extremely difficult today. The lack of safe, effective and affordable medicines is a key contributing factor. This paper reviews the recent advances and some of the challenges that we are facing in the fight against NTDs.Main bodyIn recent years, a number of innovations have demonstrated propensity to promote drug discovery and development for NTDs. Implementation of multilateral collaborations leads to continued efforts and plays a crucial role in drug discovery. Proactive approaches and advanced technologies are urgently needed in drug innovation for NTDs. However, the control and elimination of NTDs remain a formidable task as it requires persistent international cooperation to make sustainable progresses for a long period of time. Some currently employed strategies were proposed and verified to be successful, which involve both mechanisms of 'Push' which aims at cutting the cost of research and development for industry and 'Pull' which aims at increasing market attractiveness. Coupled to this effort should be the exercise of shared responsibility globally to reduce risks, overcome obstacles and maximize benefits. Since NTDs are closely associated with poverty, it is absolutely essential that the stakeholders take concerted and long-term measures to meet multifaceted challenges by alleviating extreme poverty, strengthening social intervention, adapting climate changes, providing effective monitoring and ensuring timely delivery.ConclusionsThe ongoing endeavor at the global scale will ultimately benefit the patients, the countries they are living and, hopefully, the manufacturers who provide new preventive, diagnostic and therapeutic products.
| S-EPMC6022351 | biostudies-literature
Project description:Community drug distributors or neglected tropical disease (NTD) volunteers have played a crucial role in ensuring the success of mass drug administration (MDA) programs using preventive chemotherapy (PC) for lymphatic filariasis, onchocerciasis, schistosomiasis, trachoma and soil transmitted helminths. In recent years however, a noticeable decline in motivation of some of these volunteers has been perceived, potentially negatively impacting the success of these programs. Potential hypotheses for this change in motivation include the long duration of many MDA programs, the change in sociocultural environments as well as the changes to the programs over time. This literature review identifies factors that affect NTD volunteer performance and motivation, which may be used to influence and improve future programming.A systematic search was conducted to identify studies published between January 1995 and September 2016 that investigate factors pertaining to volunteer motivation and performance in NTD drug distribution programs. Searches from several databases and grey literature yielded 400 records, of which 28 articles from 10 countries met the inclusion criteria. Quality assessment of studies was performed using the Critical Appraisal Skills Programme(CASP) checklist. Data pertaining to motivation, performance, retention and satisfaction was extracted and examined for themes. Recurring themes in the literature included monetary and material incentives, intrinsic motivation, gender, cost to participate, and health systems and community support. Of these, community support and the health system were found to be particularly impactful. Very few studies were found to explicitly look at novel incentives for volunteers and very few studies have considered the out of pocket and opportunity costs that NTD volunteers bear carrying out their tasks.There is currently great interest in incorporating more attractive incentive schemes for NTD volunteers. However, our results show that the important challenges that volunteers face (cultural, health systems, financial and community related) may have less to do with financial incentives and may actually have a larger impact on their motivation than has previously been understood. Further integration of NTD programs into existing health systems is expected to improve the NTD volunteer working environment. Relevant community engagement related to the MDA program should also provide the supportive environment needed in the community to support NTD volunteers. Programs need to consider these issues to improve working conditions for NTD volunteers.
| S-EPMC5718409 | biostudies-literature
Project description:We studied avian development in 49 to 153 species of temperate and tropical New World passerine birds to determine how growth rates, and incubation and nestling periods, varied in relation to other life-history traits. We collected growth data and generated unbiased mass and tarsus growth rate estimates (mass n = 92 species, tarsus n = 49 species), and measured incubation period (n = 151) and nestling period (n = 153), which we analyzed with respect to region, egg mass, adult mass, clutch size, parental care type, nest type, daily nest predation rate (DMR), and nest height. We investigated covariation of life-history and natural-history attributes with the four development traits after controlling for phylogeny. Species in our lowland tropical sample grew 20% (incubation period), 25% (mass growth rate), and 26% (tarsus growth rate) more slowly than in our temperate sample. Nestling period did not vary with respect to latitude, which suggests that tropical songbirds fledge in a less well-developed state than temperate species. Suboscine species typically exhibited slower embryonic and post-embryonic growth than oscine passerines regardless of their breeding region. This pattern of slow development in tropical species could reflect phylogenetic effects based on unknown physiological attributes. Time-dependent nest mortality was unrelated to nestling mass growth rate, tarsus growth rate, and incubation period, but was significantly associated with nestling period. This suggests that nest predation, the predominant cause of nest loss in songbirds, does not exert strong selection on physiologically constrained traits, such as embryonic and post-embryonic growth, among our samples of temperate and lowland tropical songbird species. Nestling period, which is evolutionarily more labile than growth rate, was significantly shorter in birds exposed to higher rates of nest loss and nesting at lower heights, among other traits. Differences in life-history variation across latitudes provide insight into how unique ecological characteristics of each region influence physiological processes of passerines, and thus, how they can shape the evolution of life histories. While development traits clearly vary with respect to latitude, trait distributions overlap broadly. Life-history and natural history associations differ for each development trait, which suggests that unique selective pressures or constraints influence the evolution of each trait.
| S-EPMC7430732 | biostudies-literature