Clinical trials and You

Often the idea of being enrolled onto a clinical trial conjures up images of being a guinea pig,with particularly nerdy white coat draped men trying out their latest discovery on a poor endangered,uninformed patient, in their hot pursuit of a coveted prize, a place in history and respect among their peers as great pioneers.

Indeed,the scientist whose discovery radically changes clinical practise,whose initiative saves lives and propels medicine and science forward will receive befitting accolades,but throughout all this,the patient’s safety comes first as enshrined in the Hippocratic oath that we as medical professionals solemnly undertake at the dawn of our medical careers and encapsulated by the phrase ”primum non nocere”(“ first do no harm”).

Clinical trials are at the very core of medicine.They dictate the way we practise and gives us confidence in the treatments we advocate.The approach to any clinical problem is backed by some degree of evidence,whether  weak or strong, based on the results of  clinical trials addressing that specific issue.
These trials are conducted within the strictest parameters,and are often  globally coordinated. Firm guidelines are set out to ensure that all participants, particularly investigators, adhere to the same,fairly high  standard of practise.
There are several levels of studies or trials.Preclinical studies are highly preliminary studies performed in the laboratory,often just to evaluate if an idea is feasible and worth pursuing any further on the clinical front.These are often performed in animals like lab rats or as test tube experiments.
The earliest “clinical” trials are Phase 1 studies. These are safety and dose finding studies.These seek to establish what the optimal dose is,and what side effects can be expected from the treatment under study.
Phase 2 studies are “efficacy” studies- Does the drug have any meaningful activity in the disease process under scrutiny?This type of study can also be used to clarify additional issues with regards to optimal doses as well.
Treatments that change clinical practise and that are recognised as the gold standard anywhere in the world,must have been tested in a Phase 3 trial.This is where a new treatment is compared with an already existing standard,to see if its use is associated with improved outcomes.For the results to be  reliable,the numbers of patients recruited must be sizeable,and often the trial must be “multi-institutional”. This  lends credibility to the study and makes it likely,after statistical analysis,that the results are reproducible and not by chance,and that they are an adequate reflection of the relevant patient population.
Without being biased,the balance of the pros and cons of taking part in a clinical trial sways almost completely to the former.The benefits far outweigh the disadvantages,which i would be hard-pressed to elucidate.
Firstly it satisfies our inherent altruism,and the need for our lives to have a lasting impact.Being part of a clinical trial allows us an opportunity to be part of a new dawn,to advance medicine and discover new treatments that may in turn save the lives of future generations.
More importantly,it helps to answer important questions,which would assist us as doctors to manage our patients better,and to gain  better insights into  the disease in question.A negative trial,which may show no difference in the old versus the new, is also equally important and is not considered a failed trial, as worthwhile lessons are learnt within  all trials regardless of the result.
Being part of a trial can also allow you to  take advantage of drugs that you would otherwise be unable to access pending actual registration of the drug by the country’s authorities .This is especially relevant in the setting of cancer treatments,where a certain treatment may already be in use in some parts of the world but not yet registered in the country,where awaiting  lengthy registration processes can have a direct impact on patient outcome.Better still,partaking in a clinical trial would allow you the use of that specific treatment at no cost to yourself,taking into account the often prohibitive costs of  newer therapies.
Patients are often  concerned about  suffering toxicities from a fairly new treatment that doctors seem vaguely familiar with.It is however worthwhile to remember that intensive research and safety profiling is undertaken before regulatory bodies allow human beings to be exposed to any substance.In addition, in the unfortunate event of an adverse event there is due process to be followed,and adequate facilities available to treat you if needed.Strict monitoring and anticipation are standard,certainly much more than in routine practise.In addition you can withdraw your consent at any stage during the clinical trial,with no consequence or penalty.
A common question is the possibility of not deriving any benefit from the treatment in question,and it is  helpful to keep in mind that such a possibility remains for any treatment,inside or outside the context of a clinical trial.The structure of any trial you are asked to participate in will always ensure that your participation does not disadvantage you in any manner.
When asked by your clinician to partake in a clinical trial,always keep an open mind.Make sure you know what question the trial seeks to answer,what the comparator arms are along with the expected toxicities of each treatment.You will get time to discuss these issues in great depth with your doctor and you will be given an opportunity to take the consent documentation home for you and your loved one’s perusal.

Participating in research is not solely an act of selflessness,it allows you,the patient to access the future,and we the medical fraternity the opportunity to perfect it.It is critical  to propel health care forward,not only for ourselves,but for future generations,our children and for strangers we will never have the privilege of meeting.