More efficient ways of assessing treatments for neglected tropical diseases are required: innovative study designs, new endpoints, and markers of effects.
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ABSTRACT:
SUBMITTER: Olliaro P
PROVIDER: S-EPMC3362612 | biostudies-literature | 2012
REPOSITORIES: biostudies-literature
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More efficient ways of assessing treatments for neglected tropical diseases are required: innovative study designs, new endpoints, and markers of effects.
Olliaro Piero P Vaillant Michel T MT Sundar Shyam S Balasegaram Manica M
PLoS neglected tropical diseases 20120529 5
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Project description:The health, economic, and social burden of neglected tropical diseases (NTDs) in Africa remains substantial, with elimination efforts hindered by persistent sociopolitical instability, including ongoing conflicts among political and ethnic groups that lead to internal displacement and migration. Here, we explore how innovative technologies can support Africa in addressing NTDs amidst such instability, through analysis of WHO and UNHCR data and a systematic literature review. Countries in Africa facing sociopolitical instability also bear a high burden of NTDs, with the continent ranking second globally in NTD burden (33%, 578 million people) and first in internal displacement (50%, 31.6 million people) in 2023. Studies have investigated technologies for their potential in NTD prevention, surveillance, diagnosis, treatment and management. Integrating the evidence, we discuss nine promising technologies-artificial intelligence, drones, mobile clinics, nanotechnology, telemedicine, augmented reality, advanced point-of-care diagnostics, mobile health Apps, and wearable sensors-that could enhance Africa's response to NTDs in the face of persistent sociopolitical instability. As stability returns, these technologies will evolve to support more comprehensive and sustainable health development. The global health community should facilitate deployment of health technologies to those in greatest need to help achieve the NTD 2030 Roadmap and other global health targets.
| S-EPMC11603293 | biostudies-literature
Project description:Global regulatory agencies have transformed their approach to approvals in their processes for formal review of the safety and efficacy of new drugs. Opportunities for innovation have expanded because of the coronavirus disease 2019 (COVID-19) pandemic. Several regulatory-led initiatives have progressed rapidly during the past year, including patient-focused drug development, model-informed drug development, real-world evidence, and complex innovative trial designs. Collectively, these initiatives have accelerated the rate of approvals. Despite demands to focus on urgent needs imposed by the COVID-19 pandemic, the number of new drug approvals over the past year, particularly for rare diseases, has outpaced expectations. Advancing therapeutics for nervous system disorders requires adaptive strategies that align with rapid developments in the field. Three relentlessly progressive diseases, amyotrophic lateral sclerosis, Duchenne muscular dystrophy, and Parkinson's disease are in urgent need of new treatments. Herein, we propose new regulatory initiatives, including innovative trial designs and patient-focused drug development that accelerate clinical trial conduct while meeting critical regulatory requirements for therapeutic approval.
| S-EPMC9305159 | biostudies-literature
Project description:BackgroundNeglected tropical diseases (NTDs) represent a diverse group of 20 communicable diseases that occur in tropical and subtropical areas in 149 countries, affecting over 1 billion people and costing developing economies billions of dollars every year. Within these diseases, those that present lesions on the skin surface are classified as skin NTDs (sNTDs). Mobile health interventions are currently being used worldwide to manage skin diseases and can be a good strategy in the epidemiological and clinical management of sNTDs.ObjectiveWe aimed to analyze existing evidence about mobile health interventions to control and manage sNTDs in low- and middle-income countries (LMICs) and make recommendations for what should be considered in future interventions.MethodsA systematic review was conducted of the MEDLINE, Embase, and Scopus databases over 10 years up to April 30, 2020. All types of clinical studies were considered. Data were synthesized into evidence tables. Apps were selected through a comprehensive systematic search in the Google Play Store and Apple App Store conducted between March 20 and April 15, 2020.ResultsFrom 133 potentially relevant publications, 13 studies met our criteria (9.8%). These analyzed eight different interventions (three SMS text messaging interventions and five app interventions). Six of the 13 (46%) studies were community-based cross-sectional studies intended to epidemiologically map a specific disease, mainly lymphatic filariasis, but also cutaneous leishmaniasis, leprosy, and NTDs, as well as sNTDs in general. Most of the studies were considered to have a high (5/13, 39%) or moderate (4/13, 31%) risk of bias. Fifteen apps were identified in the Google Play Store, of which three were also in the Apple App Store. Most of the apps (11/15, 73%) were targeted at health care professionals, with only four targeted at patients. The apps focused on scabies (3/15, 20%), lymphatic filariasis (3/15, 20%), cutaneous leishmaniasis (1/15, 7%), leprosy (1/15, 7%), yaws and Buruli ulcer (1/15, 7%), tropical diseases including more than one sNTDs (3/15, 20%), and NTDs including sNTDs (2/15, 13%). Only 1 (7%) app focused on the clinical management of sNTDs.ConclusionsAll mobile health interventions that were identified face technological, legal, final user, and organizational issues. There was a remarkable heterogeneity among studies, and the majority had methodological limitations that leave considerable room for improvement. Based on existing evidence, eight recommendations have been made for future interventions.
| S-EPMC7808891 | biostudies-literature
Project description:Although more than one billion people live at risk of neglected tropical diseases (NTDs) in areas of Asia, sub-Saharan Africa, and Latin America, the degree to which they burden countries like the U.S. is unclear. Even though many NTDs such as dengue, leishmaniasis, and Chagas disease are typically not endemic to the U.S., the possibility of their emergence is noteworthy, especially in states like Texas with high levels of poverty, large immigrant populations, geographic proximity to endemic areas, and a climate amenable to the vectors for these diseases. Despite the health threat that emerging NTDs may pose, little is known about the prevalence of risk factors for NTDs in the U.S. We tested the Community Assessment for Public Health Emergency Response (CASPER) method to assess the prevalence of risk factors for NTDs in Brazos County, Texas.Results: We found relatively low prevalence of risk factors related to travel (5.2% of respondents visited an endemic area in the previous 3 months); however, few respondents reported adherence to mosquito prevention, such as wearing long sleeves and long pants (14.1%, 95% CI: 13.9,14.4) and repellant containing DEET (13.5%, 95% CI: 13.2,13.7). Between 5.4% and 35.8% of respondents had a visible container (e.g., pet water dishes, flower pots, bird baths) that could support mosquito breeding. CASPER findings present public health authorities with potential avenues for implementing health education and other interventions aimed at reducing exposure to risk factors for NTDs among Texas residents.
| S-EPMC5693448 | biostudies-literature
Project description:BackgroundIn 2017, Liberia became one of the first countries in the African region to develop and implement a national strategy for integrated case management of Neglected Tropical Diseases (CM-NTDs), specifically Buruli ulcer, leprosy, lymphatic filariasis morbidities, and yaws. Implementing this plan moves the NTD program from many countries' fragmented (vertical) disease management. This study explores to what extent an integrated approach offers a cost-effective investment for national health systems.MethodsThis study is a mixed-method economic evaluation that explores the cost-effectiveness of the integrated CM-NTDs approach compared to the fragmented (vertical) disease management. Primary data were collected from two integrated intervention counties and two non-intervention counties to determine the relative cost-effectiveness of the integrated program model vs. fragmented (vertical) care. Data was sourced from the NTDs program annual budgets and financial reports for integrated CM-NTDs and Mass Drug Administration (MDA) to determine cost drivers and effectiveness.ResultsThe total cost incurred by the integrated CM-NTD approach from 2017 to 2019 was US$ 789,856.30, with the highest percentage of costs for program staffing and motivation (41.8%), followed by operating costs (24.8%). In the two counties implementing fragmented (vertical) disease management, approximately US$ 325,000 was spent on the diagnosis of 84 persons and the treatment of twenty-four persons suffering from NTDs. While 2.5 times as much was spent in integrated counties, 9-10 times more patients were diagnosed and treated.ConclusionsThe cost of a patient being diagnosed under the fragmented (vertical) implementation is five times higher than integrated CM-NTDs, and providing treatment is ten times as costly. Findings indicate that the integrated CM-NTDs strategy has achieved its primary objective of improved access to NTD services. The success of implementing an integrated CM-NTDs approach in Liberia, presented in this paper, demonstrates that NTD integration is a cost-minimizing solution.
| S-EPMC10308665 | biostudies-literature
Project description:It has been 100 years since the life-saving discovery of insulin, yet daily management of type 1 diabetes (T1D) remains challenging. Even with closed-loop systems, the prevailing need for persons with T1D to attempt to match the kinetics of insulin activity with the kinetics of carbohydrate metabolism, alongside dynamic life factors affecting insulin requirements, results in the need for frequent interventions to adjust insulin dosages or consume carbohydrates to correct mismatches. Moreover, peripheral insulin dosing leaves the liver underinsulinized and hyperglucagonemic and peripheral tissues overinsulinized relative to their normal physiologic roles in glucose homeostasis. Disease-modifying therapies (DMT) to preserve and/or restore functional β-cell mass with controlled or corrected autoimmunity would simplify exogenous insulin need, thereby reducing disease mortality, morbidity, and management burdens. However, identifying effective DMTs for T1D has proven complex. There is some consensus that combination DMTs are needed for more meaningful clinical benefit. Other complexities are addressable with more innovative trial designs and logistics. While no DMT has yet been approved for marketing, existing regulatory guidance provides opportunities to further "de-risk" development. The T1D development ecosystem can accelerate progress by using more innovative ways for testing DMTs for T1D. This perspective outlines suggestions for accelerating evaluation of candidate T1D DMTs, including combination therapies, by use of innovative trial designs, enhanced logistical coordination of efforts, and regulatory guidance for expedited development, combination therapies, and adaptive designs.
| S-EPMC9911317 | biostudies-literature
Project description:BackgroundThe neglected tropical diseases (NTDs) cause significant morbidity and mortality worldwide. Due to the growth in international travel and immigration, NTDs may be diagnosed in countries of the western world, but there has been no specific focus in the literature on imported NTDs.MethodsRetrospective study of a cohort of immigrants and travelers diagnosed with one of the 13 core NTDs at a Tropical Medicine Referral Unit in Spain during the period April 1989-December 2007. Area of origin or travel was recorded and analyzed.ResultsThere were 6168 patients (2634 immigrants, 3277 travelers and 257 VFR travelers) in the cohort. NTDs occurred more frequently in immigrants, followed by VFR travelers and then by other travelers (p<0.001 for trend). The main NTDs diagnosed in immigrants were onchocerciasis (n = 240, 9.1%) acquired mainly in sub-Saharan Africa, Chagas disease (n = 95, 3.6%) in immigrants from South America, and ascariasis (n = 86, 3.3%) found mainly in immigrants from sub-Saharan Africa. Most frequent NTDs in travelers were: schistosomiasis (n = 43, 1.3%), onchocerciasis (n = 17, 0.5%) and ascariasis (n = 16, 0.5%), and all were mainly acquired in sub-Saharan Africa. The main NTDs diagnosed in VFR travelers were onchocerciasis (n = 14, 5.4%), and schistosomiasis (n = 2, 0.8%).ConclusionsThe concept of imported NTDs is emerging as these infections acquire a more public profile. Specific issues such as the possibility of non-vectorial transmission outside endemic areas and how some eradication programmes in endemic countries may have an impact even in non-tropical western countries are addressed. Recognising NTDs even outside tropical settings would allow specific prevention and control measures to be implemented and may create unique opportunities for research in future.
| S-EPMC2910704 | biostudies-literature
Project description:BackgroundSubcutaneous (deep) mycoses are a chronic infectious disease of the skin and underlying structures endemic in tropical countries. The disease has serious medical and socioeconomic consequences for patients, communities and health services in endemic areas. The inclusion of mycetoma and other subcutaneous mycoses in the list of Neglected Tropical Diseases by WHO highlights the need to assess the burden of these diseases and establish control programs where necessary. In Ethiopia no strategies can be devised because of a lack of epidemiologic information. To address this evidence gap, we performed a national rapid assessment of the geographic distribution of subcutaneous mycoses.MethodologyWe conducted a rapid retrospective assessment using hospital records to identify all suspected and confirmed cases of subcutaneous mycoses in 13 referral hospitals across the country between 2015 and 2022. In each hospital the logbooks were reviewed for diagnoses of subcutaneous mycosess, as diagnosed per routine practice. Descriptive analysis was done.ResultFrom 13 hospitals we extracted 143 cases of subcutaneous mycoses, registered from July 2018 to September 2022. 118 (82.5%) patients were diagnosed as mycetoma, 21 (14.7%) as chromoblastomycosis and the remaining 4 (2.8%) as sporotrichosis. The mean age of patients was 35.8 years (SD = 14.5). 101 (70.6%) patients were male and 96 (67.1%) patients were farmers. 64 (44.8%) cases were from the Tigray regional state. 56 (65.9%) patients had information on diagnostic microscopic evaluation: for mycetoma histopathologic evaluation and fine needle aspiration cytology had a higher positivity rate while for chromoblastomycosis potassium hydroxide (KOH) staining had a better yield. The main clinical presentations were nodules, sinuses and infiltrative plaques on the skin. Radiologic findings of bone involvement was present in some.ConclusionsMycetoma and other subcutaneous mycoses are endemic in Ethiopia, with cases reported from almost all regions with the highest cases numbers reported from the northern part of the country. A routine program and systems should be developed to identify and document the burden of subcutaneous fungal infections in the country. Diagnosis and treatment guidelines should be developed.
| S-EPMC10561858 | biostudies-literature
Project description:Mycetoma is considered a neglected tropical disease globally. However, data on its burden and the associated complications in Uganda are limited. Hence we aimed to estimate its burden in Uganda. Firstly, a systematic PubMed search for all studies of any design on mycetoma in Uganda without restriction to the year of publication was conducted. A retrospective review of all the biopsy reports at the Pathology Reference Laboratory, Department of Pathology, Makerere University, Kampala, Uganda from January 1950 to September 2019 was conducted to identify any reports on mycetoma histological diagnosis. During the 70-years study period, 30 cases were identified by the literature review, with 249 additional cases identified by review of biopsy reports (total of 279 cases). The average incidence was estimated at 0.32/100,000 persons and prevalence of 8.32/100,000 persons per decade. However, there was a general decline in the number of cases detected recently. Males and the age group of 21-30 years were the most affected by mycetoma in Uganda, and only 7% of the cases were children. The highest number of cases was recorded from Kampala (n = 30) and Jinja (n = 19) districts. The majority of the cases (68%) were referred from surgical units. The foot was the most affected part of the body (72%). Ten per cent of the cases had bone involvement of which 58% required amputation. Fungi were the most common causative agents (89%) followed by Nocardia species (5%) and Actinomycetes (4%). The index of clinical suspicion of mycetoma was low (45%) with a very large differential diagnosis. Mycetoma is a relatively rare disease in Uganda, mostly caused by fungi, and there is a big gap in data and epidemiological studies. More systematic studies are warranted to define the true burden of mycetoma in Uganda.
| S-EPMC7190103 | biostudies-literature
Project description:Smooth tubercle bacilli (STB) including "Mycobacterium canettii" are members of the Mycobacterium tuberculosis complex (MTBC), which cause non-contagious tuberculosis in human. This group comprises <100 isolates characterized by smooth colonies and cordless organisms. Most STB isolates have been obtained from patients exposed to the Republic of Djibouti but seven isolates, including the three seminal ones obtained by Georges Canetti between 1968 and 1970, were recovered from patients in France, Madagascar, Sub-Sahara East Africa, and French Polynesia. STB form a genetically heterogeneous group of MTBC organisms with large 4.48 ± 0.05 Mb genomes, which may link Mycobacterium kansasii to MTBC organisms. Lack of inter-human transmission suggested a yet unknown environmental reservoir. Clinical data indicate a respiratory tract route of contamination and the digestive tract as an alternative route of contamination. Further epidemiological and clinical studies are warranted to elucidate areas of uncertainty regarding these unusual mycobacteria and the tuberculosis they cause.
| S-EPMC4707939 | biostudies-literature