Clinical Trials

Clinical Trials: A Large Pharmaceutical Company Perspective

Clinical trials, at first glance, would appear to be an unhurried exercise conducted calmly by lab-coated researchers in sanitised laboratories. The proverbial tip-of-the-iceberg more than adequately describes this situation.

In the first place, the planet’s 7.5 billion people are potential consumers of the drugs that undergo clinical trials. Primary targets they may be, but they are by no means the only interested party in the outcome of these trials. The medical community (especially doctors and pharmacists) may be regarded as co-primary recipients (together with potential patients) of the outcome of clinical trials. Industry regulators also have a large stake in the process and outcome of clinical trials.

Clinical Trial Process

The entire exercise of a clinical trial for a drug involves far more personnel than is immediately apparent, with practitioners outside core science disciplines easily outnumbering the actual researchers. The truth is that trial sites (for any clinical trial) are located in several countries and these trials take place simultaneously.  Each local regulator must be satisfied as to the results produced and ensure that the drug does indeed do what it claims.  These sites may sometimes number in the hundreds and each site must of necessity exercise a high degree of semi-autonomy.

Each site then, as a starting point, must have the core scientific team that has, not only in-depth, but broad expertise in the sciences related to the therapeutic area being tested. In addition statisticians, protocol designers, clinical investigators and independent experts are necessary to an operation of this nature, magnitude and potential impact.

The involvement of institutional review boards, quality controllers, safety-monitoring committees, compliance officers, manufacturers and suppliers cannot be overlooked. Project managers (to keep it all together) and their attendant administrative staff are crucial to any enterprise with a specific goal in mind, not just to ensure that timelines are realistic and respected but also to see that everyone is rowing in the same direction, so to speak.

Indeed (and perhaps paradoxically) one of the biggest hindrances to successful trials is intellectual curiosity. The attitude of ‘what if we try this’ and ‘wouldn’t it be nice to know’, etc can easily compromise success. The more the variables that are thrown up increase (and the need to test them) so does the potential for results that are not authentic, or even relevant, increase. The more complex a trial is (that is the greater the number of variables) the greater the possibility of everything spiralling out of control on all fronts: the high probability of said variables clashing, which brings in the scientific risk of experiment, and the operational risk of implementation with the inevitable delays and added financial costs.

A High Risk Business

For the stated reasons, pharmaceutical research and development is considered a high-risk business and routinely displays the highest failure rate for new product candidates of any industry. In addition when the focus of clinical trials centres around chronic diseases, the time involved (several years at least to accrue clinically meaningful information) will inevitably have a multiplier effect on the variables that emerge along the way.

The conclusion is that, at least in the foreseeable future, clinical trials will remain the domain of giant pharmaceuticals, effectively locking out medical providers and putting at serious disadvantage patients with chronic disease who must wait for years for new drug advances because only a few players can participate in this research.

Source: The New England Journal of Medicine