top of page

2022 Rome Action Plan on Paediatric HIV & TB



EMA and USFDA (1)  commit to:

42 Work with other regulatory authorities within the International Council for Harmonisations of Technical Requirements for Pharmaceuticals for Human Use (ICH) 11 to advance development of supportive regulatory guidance in alignment with the Call To Action principles.


43. Encourage sponsors to consider innovative approaches (i.e., combination DART study designs, as outlined in ICH S5(R3), Section VII) to help expedite DART data collection.


44. Continue to consider the benefit and risks of new molecular entities under development to determine, on a case-by-case basis, and provided that the nonclinical safety data are supportive, whether participants who become pregnant during a clinical trial can remain in the clinical trial and continue to receive the investigational drug. The agencies will continue to proactively engage with sponsors throughout the drug development period to discuss and consider the benefit/risks of an investigational drug when used in women who become pregnant during clinical trials. In addition, to facilitate approaches to increase enrolment of certain underrepresented populations, The FDA has published a guidance, Enhancing the Diversity of Clinical Trial Populations –Eligibility Criteria, Enrolment Practices, and Trial Designs, accessible at


45. Continue to encourage sponsors to collect pharmacokinetic (PK) data in pregnant women to characterize effect of pregnancy on drug exposures (e.g., AUC, Ctrough). PK data maybe collected in pre-approval clinical trials when appropriate for pregnant participants to remain in the trial, or through post-approval clinical studies in pregnant women.


46. Continue to work with sponsors to update USPI/EMA PI as additional data become available.


47. To the extent feasible, continue to collaborate with other regulatory agencies on aspects of drug development, including trial designs, endpoints, safety monitoring plans, and patient eligibility criteria (such as pregnant status) for inclusion in clinical trials.

(1) The USFDA has committed to be an active participant of the Expert Working Group (EWG), a group tasked to develop a new ICH guidance on Clinical Trials in Pregnant & Breastfeeding People.  USFDA has published several guidance, including Pregnant Women: Scientific and Ethical Considerations for Inclusion in Clinical Trials, and Informed Consent Information Sheet, Guidance for IRBs, Clinical Investigators, and Sponsors; and encourages Sponsors, IRBs, and investigators to consider the contents of the guidance. USFDA will continue to work with Sponsors on the specifics of the Informed Consent Form (ICF) language included to communicate potential risks identified from the nonclinical data.  



All partners commit to: 


163. Address inequities by tackling the stigma and discrimination in communities, schools, and healthcare settings that prevent children living with HIV from accessing testing and treatment. 

164. Increase literacy about CD4 testing and viral load and promote a client-centred approach to support expansion of access to viral load for pregnant and breastfeeding women and children on treatment, including at the point-of-care. 

165. Review and assess emerging co-infections for immunocompromised infants and children, including those with advanced HIV disease, such as severe bacterial infections, fungal infections, and others for country consideration and implementation.

166. Engage affected communities for input and guidance on investment and programmatic priorities, provide support to in-country civil society organizations to engage in advocacy and demand creation for new tools, and ensure data is publicly available to support communities and civil society to monitor progress regarding uptake and implementation of essential diagnostic tools.




WHO-PQ and US FDA commit to:

271. WHO-PQ and US FDA to collaborate and agree on the next steps for the CRPlite.


US FDA commits to:


272. USFDA is committed to work with sponsors and investigators who may need additional guidance on drug development. USFDA encourages Sponsors to engage in the PreIND consultation program.




US Food and Drug Administration commits to: 

414. Observe the following principles in regulatory review processes: 

I. Extrapolating efficacy from adults to children is acceptable for most children and adolescents with TB. Extrapolation of adult efficacy to paediatric populations for the treatment of pulmonary TB may be appropriate for most paediatric populations, other than the very young as they can have different clinical and pathophysiologic characteristics. Pharmacokinetic (PK) and safety information in paediatric patients will be needed to support the appropriate dose for treatment of children. 

II. Adolescents with pulmonary TB can be included in phase 3 clinical trials, if appropriate. 

III. Paediatric patients can be enrolled in trials if sufficient safety and antimycobacterial activity data in adults are available and appropriate dosing regimens have been characterized. 

IV. Studies of drugs across the paediatric spectrum of ages/weights can be conducted in parallel rather than sequentially unless there are specific safety or pharmacokinetic properties that warrant a different approach. 

V. Paediatric development plans for new TB medicines could include children living with HIV and other common co-infections or conditions provided there are no safety or drug-drug interaction issues. 

VI. Cohorts for paediatric studies can be defined based on chronological age or weight-based criteria, particularly for oral drugs. 

VII. Paediatric formulation development should begin soon after adult Phase 2-b trials and dose selection. 


415. Continue to work with other regulatory authorities to seek alignment on development of products for TB in children and adolescents.


416. Remain engaged in the development of products and age-appropriate formulations for treatment of TB in children and adolescents.



bottom of page