Dataset Information

Barriers and facilitators to early access of bedaquiline and delamanid for MDR-TB: a mixed-methods study.

ABSTRACT:

Setting

Phase II trials for bedaquiline (BDQ) and delamanid (DLM) were completed by 2011 and the drugs were approved by stringent regulatory authorities for the treatment of multidrug-resistant tuberculosis (MDR-TB) between 2012 and 2014. Manufacturers established 'early access' mechanisms to provide drugs before local registration.

Objective

To inform improvements in early access, we explored experiences of providers and advocates in accessing BDQ and DLM before the end of 2015 using a mixed-methods design.

Design

We examined barriers and facilitators to early access through an electronic survey. Barriers and facilitators were classified as occurring at the manufacturer- or country-level. We identified themes using inductive content analysis and illustrated themes through case studies.

Results

We analysed 41 survey responses from 36 respondents reporting on 22 countries; early access was attempted in 30 (73%) survey responses. Eligibility restrictions (11/30, 37%) and complicated and slow processes (8/30, 27%) were manufacturer-level barriers; access to companion drugs (10, 33%) and importation difficulties (4, 13%) were country-level barriers. Previous experience with manufacturer (3/30, 10%) and country processes (2/30, 7%) facilitated access. Eight case studies show the human impact of barriers and facilitators.

Conclusion

Manufacturers and countries should develop transparent processes to permit early access, particularly for diseases that largely affect the poor, such as MDR-TB. Developers should plan for this need and rapidly register drugs with proven benefit, prioritizing high-burden settings.

SUBMITTER: Rodriguez CA

PROVIDER: S-EPMC6436488 | biostudies-literature | 2019 Mar

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

Barriers and facilitators to early access of bedaquiline and delamanid for MDR-TB: a mixed-methods study.

Rodriguez C A CA Brooks M B MB Guglielmetti L L Hewison C C Jachym M F MF Lessem E E Varaine F F Mitnick C D CD

Public health action 20190301 1

<h4>Setting</h4>Phase II trials for bedaquiline (BDQ) and delamanid (DLM) were completed by 2011 and the drugs were approved by stringent regulatory authorities for the treatment of multidrug-resistant tuberculosis (MDR-TB) between 2012 and 2014. Manufacturers established 'early access' mechanisms to provide drugs before local registration.<h4>Objective</h4>To inform improvements in early access, we explored experiences of providers and advocates in accessing BDQ and DLM before the end of 2015 u ...[more]

PMID: 30963040

Similar Datasets

Project description:BACKGROUND:At a time when programs were struggling to design effective regimens for the treatment of multidrug-resistant tuberculosis (MDR-TB), the marketing authorization of bedaquiline and delamanid was a critical development in the MDR-TB treatment landscape. However, despite their availability for routine programmatic use, the uptake of these drugs has remained slow; concerns included a lack of evidence on safety and efficacy and the need to protect the new drugs from the development of acquired resistance. As part of the endTB Project, we aimed to address these barriers by generating evidence on safety and efficacy of bedaquiline or delamanid based MDR-TB regimens. METHODS:This is a protocol for a multi-center prospective cohort study to enroll 2600 patients from April 2015 through September 2018 in 17 countries. The protocol describes inclusion of patients started on treatment with bedaquiline- or delamanid- containing regimens under routine care, who consented to participate in the endTB observational study. Patient follow-up was according to routine monitoring schedules recommended for patients receiving bedaquiline or delamanid as implemented at each endTB site. Therefore, no additional tests were performed as a part of the study. Data were to be collected in a customized, open-source electronic medical record (EMR) system developed as a part of the endTB Project across all 17 countries. DISCUSSION:The endTB observational study will generate evidence on safety and efficacy of bedaquiline- and delamanid-containing regimens in a large, extremely heterogeneous group of MDR-TB patients, from 17 epidemiologically diverse countries. The systematic, prospective data collection of repeated effectiveness and safety measures, and analyses performed on these data, will improve the quality of evidence available to inform MDR-TB treatment and policy decisions. Further, the resources available to countries through implementation of the endTB project will have permitted countries to: gain experience with the use of these drugs in MDR-TB regimens, improve local capacity to record and report adverse events (pharmacovigilance), and enhance significantly the body of data available for safety evaluation of these drugs and other novel treatments. TRIAL REGISTRATION:This study was registered on 24 August 2017 at clincaltrials.gov (Registration number: NCT03259269).

Project description:BackgroundIn November 2016, the ICH published a requirement for sponsors to develop a systematic, prioritised, risk-based approach to monitoring clinical trials. This approach is more commonly known as risk-based monitoring (RBM). However, recent evidence suggests that a 'gold standard', validated approach to RBM does not exist and it is unclear how sponsors will introduce RBM into their organisations. A first step needed to inform the implementation of RBM is to explore academic trialists' readiness and ability to perform RBM. The aim of this paper is to identify the attitudes and perceived barriers and facilitators to the implementation of RBM in academic-led clinical trials in Ireland.MethodsThis is a mixed-methods, explanatory sequential design, with quantitative survey followed by semistructured interviews. Academic clinical researchers (N?=?132) working in Ireland were surveyed to examine their use and perceptions of RBM. A purposive sample of survey participants (n?=?22) were then interviewed to gain greater insight into the quantitative findings. The survey and interview data were merged to generate a list of perceived barriers and facilitators to RBM implementation, with suggestions for, and solutions to, these issues.ResultsSurvey response rate was 49% (132/273). Thirteen percent (n?=?18) of responders were not familiar with the term risk-based monitoring and less than a quarter of respondents (21%, n?=?28) had performed RBM in a clinical trial. Barriers to RBM implementation included lack of RBM knowledge/training, increased costs caused by greater IT demands, increased workload for trial staff and lack of evidence to support RBM as an effective monitoring approach. Facilitators included participants' legal obligation to perform RBM under the new ICH-GCP guidelines, availability of RBM guidance and perception of cost savings by performing RBM in future trials.ConclusionThe results of this study demonstrate a need for training and regulatory-endorsed guidelines to support the implementation of RBM in academic-led clinical trials. The study provides valuable insights to inform interventions and strategies by policy-makers and clinical trial regulators to improve RBM uptake.

Project description:BACKGROUND:A large number of mobile health (mHealth) apps have been created to help users to manage their health or receive health care services. Many of these mHealth apps have proven to be helpful for maintaining or improving their users' health. However, many people still choose not to use mHealth apps or only use them for a short period. One of the reasons behind this lack of use is the concern for their health information security and privacy. OBJECTIVE:The goal of this study was to determine the relationship between users' characteristics and their security and privacy concerns and to identify desired security features in mHealth apps, which could reduce these concerns. METHODS:A questionnaire was designed and validated by the research team. This questionnaire was then used to determine mobile app users' security and privacy concerns regarding personal health data in mHealth apps as well as the security features most users' desire. A semistructured interview was used to identify barriers to and facilitators of adopting mHealth apps. RESULTS:In total, 117 randomly selected study participants from a large pool took part in this study and provided responses to the validated questionnaire and the semistructured interview questions. The results indicate that most study participants did have concerns about their privacy when using mHealth apps. They also expressed their preferences regarding several security features in mHealth apps, such as regular password updates, remote wipe, user consent, and access control. An association between their demographic characteristics and their concerns and preferences in security and privacy was identified; however, in most cases, the differences among the different demographic groups were not statistically significant, except for a few very specific aspects. These study participants also indicated that the cost of apps and lack of security features in mHealth apps were barriers for adoption, whereas having free apps, strong but easy-to-use security features, and clear user protection privacy policies might encourage them to use mHealth apps in their health management. CONCLUSIONS:This questionnaire and interview study verified the security and privacy concerns of mHealth app users, identified the desired security and privacy features, and determined specific barriers to and facilitators of users adopting mHealth apps. The results can be used to guide mHealth app developers to create apps that would be welcomed by users.

Dataset Information

Barriers and facilitators to early access of bedaquiline and delamanid for MDR-TB: a mixed-methods study.

Setting

Objective

Design

Results

Conclusion

Publications

Barriers and facilitators to early access of bedaquiline and delamanid for MDR-TB: a mixed-methods study.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure