Summary of key findings from surveys and interviews conducted with National TB Programmes (NTPs) and National Medicines Regulatory Authorities (NMRAs) of the 30 multidrug-resistant (MDR) tuberculosis (TB) high burden countries (October 2016 to May 2017)#
| Pre-approval access of new TB medicines¶ | Anecdotal |
| National guidelines on the use of bedaquiline and delamanid available in 24 and 15 MDR-TB high burden countries, respectively | The NTP respondent from one country stated that the current mechanism used to access bedaquiline through import waivers for patients covered by a partner's project was no longer sufficient to cover country needs (in this country, early implementation experiences and expansion of eligibility criteria have contributed to clinician's growing willingness to prescribe bedaquiline-based regimens) |
| Three high burden countries have both bedaquiline and delamanid featured in their national essential medicines lists; seven countries have bedaquiline and four have delamanid only | The NTP respondent from one country referenced its overall nascent drug registration procedures but expressed the country's willingness to discuss “the pros and cons of registration” if a dossier were to be submitted |
| The NTP respondent from one country expressed the need to continue gathering more information from in-country studies prior to the NMRA reviewing any regulatory dossier | |
| Most countries (92%, 24/26) reported having mechanisms in place for access to drugs under development and/or prior to national regulatory approval (e.g. compassionate use, expanded access programmes and the granting of import waivers) | |
| All NTP respondents of countries where either drug was successfully registered or in the process of being registered, stated that pre-approval access use strongly contributed to greater confidence in the use of the drugs | |
| Countries' perspective on the registration of bedaquiline and delamanid | |
| All NMRAs were aware of both products for the treatment of MDR-TB, and there is widespread agreement over the need for regulatory approval to ensure sustainable access | |
| Most NTP and NMRA respondents rated communication between the two bodies as neutral (67% (12/18) and 56% (10/18)) | |
| 40% (10/25) of countries reported some NTP involvement in the registration process | |
| Majority of NTP respondents (73% (19/26)) felt that their increased support in promoting the approval for these medicines would likely lead to streamlined regulatory approval; only 30% (6/20) of the NMRA respondents see a potential role of NTPs in streamlining the national regulatory approval | |
| Most NMRA respondents (65% (13/20)) stated that sole responsibility for initiating regulatory approval processes lies with manufacturers only and NMRAs are not willing to take any action prior to the submission of a manufacturer dossier | |
| Majority of NMRA respondents (89% (17/19)) confirmed the presence of legal frameworks for accelerated review of products; four countries indicated to have applied such mechanisms for bedaquiline registration | |
| 33% (10/30) countries are participating in the WHO collaborative registration procedure;+ among them, three African countries reported being part of, observing or having applied to join other regulatory harmonisation/collaborative programmes | |
| Among the countries that reported to not participate in the WHO collaborative registration procedure or any regulatory harmonisation/collaborative programme, 45% (9/20) of NMRAs expressed their interest in exploring participation | |
| High cost, pharmacovigilance and importation challenges listed as long-term challenges, even post-registration | |
#: a total of 29 responses from NTPs and 20 responses from NMRAs were received during the survey period. If a respondent did not answer a question or inconsistencies with data from desk review were noted, the denominator was adjusted and the data were not taken into account in the analysis. ¶: data from respondents of our survey were cross-checked with Médecins Sans Frontières and Stop TB Partnership Out of Step report 2017 [15] and other data obtained from extensive desk review. +: data from respondents of our survey were cross-checked and complemented with the list of participating countries available on the World Health Organization (WHO) website [25].