Scientific considerations for global drug development.

Area of Science:

• Pharmacology and Global Health

Background:

• Drug regulatory processes in low- and middle-income countries (LMICs) often mandate confirmatory clinical trials.
• These trials are required even for drugs that have already received approval from regulatory agencies in high-income countries.

Purpose of the Study:

• To evaluate how scientific and technological advancements can minimize the necessity of in-country or in-region clinical trials for globally approved drugs.
• To identify limitations of these advancements that might still warrant local clinical studies.

Main Methods:

• Review of scientific literature on advancements in drug development and regulatory science.
• Analysis of existing regulatory policies regarding bridging studies and local adaptation trials.
• Discussion of case studies and expert opinions on the feasibility of utilizing global data.

Main Results:

• Technological progress, such as advanced modeling and simulation, offers potential to reduce reliance on traditional in-country trials.
• Biomarker and surrogate endpoint validation are crucial for extrapolating foreign clinical trial data.
• However, significant differences in disease prevalence, genetic factors, and patient populations can limit the applicability of foreign data.

Conclusions:

• Scientific and technological progress presents opportunities to streamline drug approval in LMICs by reducing duplicative trials.
• Careful consideration of specific limitations and the need for targeted local data remains essential for ensuring drug safety and efficacy in diverse populations.
• Balancing global harmonization with local needs is critical for timely and cost-effective medicine access.