Forging new paths in access to medicines research
Authors:   , Joosse Iris Rebecca 
Published: May 2024

Significant disparities in access to medicines persist. To guide effective pharmaceutical policy development for improved access, evidence from monitoring and evaluation activities is key. To bridge the gap between evidence-generation and policy-making, this thesis aimed to explore advancements to three key evidence-generation mechanisms for formulating and evaluating pharmaceutical policies: performance monitoring, health systems analysis and pharmaceutical policy analysis.
Firstly, we explored the large gap in performance data on child-appropriate medicines. This revealed that child-appropriate medicines were infrequently surveyed or not at all, and that existing instruments for collecting data on the availability or prices of medicines were limited in their ability to generate the required data for children. Accordingly, the remainder of Chapter 2 focused on monitoring performance in access to medicines for children by modifying Sustainable Development Goal (SDG) indicator 3.b.3, enabling the monitoring of access to child-appropriate medicines. Adaptations to the indicator included the selection of two core sets of child-appropriate medicines for children of different ages, and the creation of the parameter number of units needed for treatment (NUNT) to enable calculation of affordability of medicines for children. We subsequently provided proof-of concept of the adapted methodology and of its robustness.
Chapter 3 focused on novel approaches in health systems analyses of access to medicines. These approaches were applied to a case study of access to childhood oncology medicines in South Africa, with the objective to identify sources of access constraint to pediatric cancer medicines. Novel approaches included the study of alignment of several linked pharmaceutical processes – rather than in isolation from related processes – and the development of a novel analytical framework, structured as a pharmaceutical value chain, to facilitate the identification of barriers and enablers in access from a health systems perspective. This framework was applied to the case study through interviews and focus groups with professional stakeholders and caregivers of children with cancer. Finally, insights and identified barriers were triangulated with South Africa’s National Cancer Strategic Framework, leading to the identification of areas for policy strengthening.
Chapter 4 examined the pharmaceutical policy analysis landscape through the lens of pharmaceutical pricing policies, which was part of a wider systematic review on pharmaceutical pricing policies and their effectiveness in managing the prices of pharmaceutical products. The limited body of evidence suggested that public disclosure of medicine prices and policies regulating mark-ups may be effective in reducing prices of medicines and pharmaceutical expenditures. Evidence on the impact of a cost-feedback approach to prescribers was inconclusive. Additionally, common weaknesses in studies of pharmaceutical pricing policies were identified, and methodologies used were critically reviewed for their value in providing robust evidence, to guide future pharmaceutical policy analyses.
Beyond the described advancements, the following key recommendations were made in this dissertation to bridge the gap between evidence-generation and policy-making: evidence-generation must be driven by information needs of policy-makers and healthcare professionals, generated evidence must me actionable for decision-makers, and existing data and knowledge gaps must be addressed to facilitate appropriate evidence-generation, to ultimately achieve equitable access for all.