Increasingly, patient advocacy groups are emerging as influential stakeholders during the research, development, and funding of innovative, life-changing treatments. As such, it is important for patient advocates to be familiar with the standard process of drug research and development, and how their group can be involved at each step.
Research and Development
The journey of a drug begins with extensive research and development. This phase involves pre-clinical studies, where the drug’s safety and efficacy are tested in a variety of experiments. This stage often involves pharmacodynamics (i.e., what the drug does to the body; PD), pharmacokinetics (i.e., what the body does to the drug; PK), and toxicology testing, and much more. To better understand the effects of the drug on a disease before testing it in humans, animal models are often used. The most commonly used models are murine (rats or mice), canine, primate, and porcine (pigs). Typically, a single drug will be tested in two species, which are chosen based on how well they would correlate to an in-human trial – for example, genetic testing often utilizes mouse models because the mouse genome is very similar to the human genome, whereas topical treatments are often tested on pigs, whose skin is structurally similar to our own.
The pre-clinical phase traditionally relies on scientists and pharmaceutical companies to discover new compounds, understand their therapeutic possibilities, and develop formulations that are safe and effective. Patient advocacy groups are important stakeholders in this treatment development stage and are often important funders of these early-stage studies.
Clinical Trials
Once promising drug candidates emerge from the pre-clinical phase, they progress to clinical trials. These trials involve testing the drug on human subjects to evaluate its safety, dosage, and effectiveness. Clinical trials are conducted in multiple phases, each designed to provide specific insights into the drug’s performance.
- Phase 1 (First-in-Human):
- Objective: To determine the safety, dosage range, and potential side effects.
- Characteristics: Involves a small number of healthy volunteers.
- Outcome: Establishes the maximum tolerated dose (MTD) and basic safety profile.
- Phase 2 (Therapeutic Exploratory):
- Objective: To assess the efficacy and further evaluate safety.
- Characteristics: Involves a larger number of participants, often those with the target condition.
- Outcome: Provides preliminary evidence of effectiveness and identifies common side effects.
- Phase 3 (Therapeutic Confirmatory):
- Objective: To confirm efficacy, monitor side effects, and compare the intervention to standard treatments.
- Characteristics: Involves a large and diverse patient population.
- Outcome: Generates statistically significant data on safety and effectiveness, supporting regulatory approval.
The results of these trials are crucial in determining whether the drug will proceed into regulatory review.
As experts in their respective conditions, patient advocacy groups can bring valuable insight to the design of clinical trials. Their input can ensure that trials are patient-friendly and involve relevant endpoints that capture meaningful outcomes for those living with the condition. Moreover, these groups play a critical role in patient recruitment and facilitating access to clinical trials.
In conclusion, by placing patients at the forefront of the research and development process, patient advocacy groups contribute to more patient-centric, effective, and accessible therapies. As the future of pharmaceuticals unfolds, their role will continue to be essential in shaping a healthcare landscape that is responsive to the needs and aspirations of those who matter most—the patients.
