Regulatory Affairs in the Life Sciences

Earned Microcertificate in Regulatory Affairs in the Life Sciences, May 2025, which includes:

• Regulatory Science and Health Economics (May 2025)
• Introduction to Regulatory Affairs (Dec 2024)

Preamble

The complexity of regulatory systems is not a barrier to innovation—it’s what makes responsible, ethical innovation possible. The program united regulatory science, market access, and health economics with forward-looking approaches like adaptive trials and open science. This curriculum also shed light on how Investigational New Drugs (INDs) reach regulatory approval along with evidence packages for reimbursement decisions. While not exhaustive, below are key areas where my thinking and practice have evolved.

Reframing Intellectual Property: Open Science and Market Exclusivity

In a world where patents often drive access inequality, open science drug discovery presents a socially responsible alternative. One such example is regulatory exclusivity, the right to use a product dossier for market access, can replace traditional patents while encouraging affordable access. Examples like the Drugs for Neglected Diseases initiative (DNDi) show how coordinated, parallel research and development (R&D) can address market failures, especially in neglected or rare diseases.

At the same time, I now recognize that data sharing must respect Indigenous data sovereignty. Frameworks like the OCAP principles and international agreements such as the Nagoya Protocol are essential to ensure that open science does not reproduce historical patterns of extraction and exploitation.

Balancing Early Access with Evidence Standards

A critical perspective on policies like Health Canada’s Notice of Compliance with Conditions (NOC/c) allows many promising radiopharmaceuticals and biologics for early market entry. While this accelerates access, it also risks reduced evidentiary standards—often relying on single-arm Phase II trials. The ethical trade-off between speed and certainty presents challenges in patient safety and public trust.

This underpins the importance of post-market monitoring (phase IV). I learned that delayed confirmatory trials can undermine the integrity of early-access frameworks. Canada proposed reforms, such as Agile Licensing and the Advanced Therapeutic Product framework, to highlight the need for risk-based regulation with more robust enforcement and transparency.

Learning from Adaptive Trial Designs

Adaptive trial designs are appealing for their efficiency and patient-centered philosophy. By reallocating participants to effective arms or stopping early for futility, they minimize patient harm. However, they’re not always practical. Our case study focused on two landmark examples: the STAMPEDE trial, a multi-arm, multi-stage trial in advanced prostate cancer that evaluated several treatment combinations simultaneously; and BATTLE-2, a biomarker-integrated trial for non-small cell lung cancer that adaptively reassigned patients to targeted therapies based on real-time response data. Both illustrated how adaptive trials demand continuous data monitoring, rapid analysis, and seamless decision-making. This trade-off, between efficiency and implementation feasibility, has shaped how I now evaluate trial strategies in both academic and translational research contexts.

Applying Health Economics and Real-World Evidence

In the health economics module, I learned how decision-analytic models integrate real-world evidence to assess cost-effectiveness. For example, modelling the value of genetic testing strategies showed how such evaluations depend on diagnostic yield, cost variability, and assumptions about willingness-to-pay thresholds.

Another example was the Personalized OncoGenomics (POG) program in BC, Canada, which demonstrates the tension between innovation and evidence scarcity. While genomics-informed treatment has promise, managing uncertainty in outcomes means adaptive pricing and monitoring are essential for responsible reimbursement. Agencies like Canada’s Drug Agency – Agence des médicaments du Canada (CDA–AMC) increasingly welcome this type of analysis to support transparent, value-based decisions.

Patients and Regulators Joining Forces in Shaping Medicines of Tomorrow

Many view regulation as a hurdle to innovation. I now understand it as a system of checks, incentives, and ethical obligations that ensure drugs and devices meet the needs of people and the market. Increasingly, patients are not just participants but partners in regulatory science. Their lived experiences inform trial design, define outcomes, and shape pharmacoeconomic models such as discrete choice experiments (DCE). Initiatives like CDA–AMC patient input and the growing role of patient-reported outcomes (PROs) in adaptive trials and reimbursement reviews illustrate how health decisions are becoming more aligned with the values and priorities of those most affected. From multi-centre trials and international regulatory harmonization to the evolving role of patient voices in health technology assessments, the system is dynamic and evolving.