Periodic Reporting for period 1 - CAPACITY 2023 (CONSORTIUM FOR DEVELOPING REGULATORY CAPACITY FOR CLINICAL TRIALS USING GENE THERAPY PRODUCTS AND STRENGTHENING PHARMACOVIGILANCE IN THE CONDUCT OF CLINICAL TRIALS IN EAST AFRICA)
Periodo di rendicontazione: 2024-04-01 al 2025-09-30
Gene and cell-based therapies are reshaping global healthcare and offer curative potential for diseases such as HIV and sickle cell disease—conditions that heavily burden Sub-Saharan Africa. However, most countries in the region lack robust regulatory frameworks, safety monitoring systems, and trained personnel to oversee these advanced technologies. These gaps pose risks to clinical trial participants and limit the region’s ability to adopt nextgeneration medical innovations.
CAPACITY 2023 was established to address these challenges by strengthening regulatory, ethical, and pharmacovigilance capacity in Uganda and Tanzania—two countries leading gene therapy research in East Africa. The project is led by the University of Coimbra-Centre for Neuroscience and Cell Biology (UC-CNC) from Portugal, in consortium with the National Drug Authority (NDA); the Uganda National Council for Science and Technology (UNCST), and the Joint Clinical Research Centre (JCRC) from Uganda, and the Muhimbili University of Health and Allied Sciences (MUHAS) from Tanzania. It integrates multidisciplinary and complementary expertise in regulatory sciences, ethics oversight, clinical trials, and gene therapy.
Through four strategic pillars—Regulate, Train, Monitor, and Sensitize—the project is:
1) Developing the first evidence-based regulatory guidance for gene therapy trials.
2) Reinforcing national safety monitoring and pharmacovigilance systems.
3) Building a skilled workforce capable of regulating and conducting advanced clinical trials.
4) Engaging policymakers to support an enabling environment for innovation.
By aligning with initiatives such as AVAREF and the African Medicines Agency, CAPACITY 2023 contributes to regional harmonisation efforts. In the long term, the project aims to accelerate safe access to advanced therapies and strengthen East Africa’s position as a leader in clinical research.
CAPACITY 2023 was established to address these challenges by strengthening regulatory, ethical, and pharmacovigilance capacity in Uganda and Tanzania—two countries leading gene therapy research in East Africa. The project is led by the University of Coimbra-Centre for Neuroscience and Cell Biology (UC-CNC) from Portugal, in consortium with the National Drug Authority (NDA); the Uganda National Council for Science and Technology (UNCST), and the Joint Clinical Research Centre (JCRC) from Uganda, and the Muhimbili University of Health and Allied Sciences (MUHAS) from Tanzania. It integrates multidisciplinary and complementary expertise in regulatory sciences, ethics oversight, clinical trials, and gene therapy.
Through four strategic pillars—Regulate, Train, Monitor, and Sensitize—the project is:
1) Developing the first evidence-based regulatory guidance for gene therapy trials.
2) Reinforcing national safety monitoring and pharmacovigilance systems.
3) Building a skilled workforce capable of regulating and conducting advanced clinical trials.
4) Engaging policymakers to support an enabling environment for innovation.
By aligning with initiatives such as AVAREF and the African Medicines Agency, CAPACITY 2023 contributes to regional harmonisation efforts. In the long term, the project aims to accelerate safe access to advanced therapies and strengthen East Africa’s position as a leader in clinical research.
During the first reporting period, CAPACITY 2023 achieved significant scientific, technical, and capacity-building milestones.
Key achievements include:
Evidence generation and regulatory development
- Completed the first national study assessing knowledge, perceptions, and readiness for gene therapy in Uganda.
- Established a multidisciplinary task force of bioethicists, epidemiologists, and gene therapy experts to draft Uganda’s first regulatory guidelines for gene therapy clinical trials.
These outputs form the scientific foundation for future regulatory frameworks.
Capacity strengthening through training and placements
- Thirty-four (34) participants completed a specialised course on EU regulatory frameworks for clinical trials and advanced therapies.
- Six (6) regulatory and academic professionals participated in two-week placements at leading Portuguese institutions, gaining practical experience in regulatory review, ethics oversight, GMP processes, and clinical trial operations.
- Knowledge acquired was shared widely, reinforcing institutional learning.
Scientific capacity building and knowledge exchange
- Hosted the First Cell and Gene Therapy Symposium in Uganda, with more than 800 participants covering scientific, ethical, manufacturing, and clinical dimensions of gene therapy.
- Launched a Gene Therapy Webinar Series providing regular technical training to more than 1500 participants, mainly from East Africa but also from other countries and continents.
- Developed comprehensive Information, Education and Communication materials, targeting professionals and the general population.
- Designed a multidisciplinary Gene Therapy Course (11 modules), which has been submitted for accreditation.
Strengthening pharmacovigilance systems
- Conducted the first systematic baseline assessment of pharmacovigilance practices in clinical trials in Uganda, identifying gaps and proposing evidence-based improvements.
- Twelve (12) consortium members completed advanced Eu2P - pharmacovigilance training.
- Recruited a dedicated Safety Officer to reinforce institutional capacity.
Project management
CAPACITY 2023 successfully delivered all 18 planned Deliverables and 17 Milestones, with only minor delays. This reflects strong scientific coordination, supporting the timely execution of all technical activities, efficient and robust expert-consultation mechanisms, and effective data management structures.
Key achievements include:
Evidence generation and regulatory development
- Completed the first national study assessing knowledge, perceptions, and readiness for gene therapy in Uganda.
- Established a multidisciplinary task force of bioethicists, epidemiologists, and gene therapy experts to draft Uganda’s first regulatory guidelines for gene therapy clinical trials.
These outputs form the scientific foundation for future regulatory frameworks.
Capacity strengthening through training and placements
- Thirty-four (34) participants completed a specialised course on EU regulatory frameworks for clinical trials and advanced therapies.
- Six (6) regulatory and academic professionals participated in two-week placements at leading Portuguese institutions, gaining practical experience in regulatory review, ethics oversight, GMP processes, and clinical trial operations.
- Knowledge acquired was shared widely, reinforcing institutional learning.
Scientific capacity building and knowledge exchange
- Hosted the First Cell and Gene Therapy Symposium in Uganda, with more than 800 participants covering scientific, ethical, manufacturing, and clinical dimensions of gene therapy.
- Launched a Gene Therapy Webinar Series providing regular technical training to more than 1500 participants, mainly from East Africa but also from other countries and continents.
- Developed comprehensive Information, Education and Communication materials, targeting professionals and the general population.
- Designed a multidisciplinary Gene Therapy Course (11 modules), which has been submitted for accreditation.
Strengthening pharmacovigilance systems
- Conducted the first systematic baseline assessment of pharmacovigilance practices in clinical trials in Uganda, identifying gaps and proposing evidence-based improvements.
- Twelve (12) consortium members completed advanced Eu2P - pharmacovigilance training.
- Recruited a dedicated Safety Officer to reinforce institutional capacity.
Project management
CAPACITY 2023 successfully delivered all 18 planned Deliverables and 17 Milestones, with only minor delays. This reflects strong scientific coordination, supporting the timely execution of all technical activities, efficient and robust expert-consultation mechanisms, and effective data management structures.
The project is already delivering scientific, societal, and institutional advancements that significantly enhance regional readiness for gene and cell-based therapies. Key contributions include:
- Evidence-based regulatory development for novel therapies.
- Strengthened pharmacovigilance systems and safety culture.
- Specialised training programmes that build a long-term skilled workforce.
- Large-scale knowledge-exchange platforms connecting researchers, regulators, clinicians, and policymakers.
These outcomes lay the foundation for safer and faster access to advanced therapies, improved disease control, and stronger regional leadership in clinical research. Continued international collaboration, investment in infrastructure and workforce development, and alignment with continental initiatives will be essential for sustaining and scaling these advancements.
- Evidence-based regulatory development for novel therapies.
- Strengthened pharmacovigilance systems and safety culture.
- Specialised training programmes that build a long-term skilled workforce.
- Large-scale knowledge-exchange platforms connecting researchers, regulators, clinicians, and policymakers.
These outcomes lay the foundation for safer and faster access to advanced therapies, improved disease control, and stronger regional leadership in clinical research. Continued international collaboration, investment in infrastructure and workforce development, and alignment with continental initiatives will be essential for sustaining and scaling these advancements.