This is the first of a multi-part series on the challenges of finding the right patients for clinical trials. It’s written for an audience of non-experts in trial design and conduct, but who are interested in this important part of getting new medicines to patients (hopefully including an effective vaccine to the virus causing Covid-19). I’ll try to answer the questions: why does it take a long time to bring new medicines to patients and what part does enrolling patients into the study have in this challenge?  

The series will touch (lightly) on topics including trial design, key steps in conducting the trial, and why enrollment can often be the longest part of doing a study.

My background includes over 3 decades in biotechnology and pharmaceuticals developing new therapies, so I have some familiarity with the challenges of conducting high-quality trials.

Introduction

Clinical trials are studies assessing the safety and efficacy of potential medicines for human diseases. They are the centerpiece of the complicated effort to develop new therapies. At least in theory, the medicine must prove itself in these studies to be allowed for human use. If the medicine works as expected, patients will be allowed to use it. If it fails – and most potential medicines don’t make it through clinical trials – then patients have been spared exposure to a useless or harmful drug.

The therapies put into clinical trials can be entirely new and untried against any disease or they may be an existing medicine repurposed to address a disease they weren’t originally intended for. Of great current importance, these trials can assess potential medicines against serious and mortal illnesses including cancer, heart disease, stroke, and diabetes as well as infections such as Covid-19. Trials may also determine the safety and efficacy of medical devices ranging from insulin pumps to ventilators.

The main audience for the trial results is usually not the public or even medical doctors, but rather regulatory agencies including the FDA (the US Food and Drug Administration), the EMA (European Medicines Agency, the EU counterpart to the FDA), SFDA China (the Chinese counterpart to the FDA), and other global authorities who review the results and either approve – or not – drugs to be given to patients. Trial results may also be published in scientific journals. The public is free to read these, but finding and accessing the journals may be tedious and the articles are written for experts rather than a lay person. Nonetheless, for a diligent member of the public, they can provide real insight into the qualities of a new medicine.

The next posting in this series will address who does these trials and how they’re set-up.

(Image above by Arek Socha from Pixabay)