How Does Drug Development Work?
The development of a new neurologic drug therapy can take decades and well over a billion dollars. How does a drug get from being an idea in a researcher’s mind to the drug store shelf? It’s a long and arduous journey that begins as just that – a thought.
The first step is Drug Discovery or the initial testing of those “thoughts”. This may come about because existing treatments are not working sufficiently or have unwanted effects or because new insight into the disease allows for a novel approach to treatment. Many molecular compounds are tested at this stage and a few promising ones are chosen.
This stage is followed by Drug Development. In this phase, those promising compounds are tested. The information gathered includes its effectiveness on its own and compared to existing drugs, the appropriate dosage, how the drug is metabolized and excreted, the best route to administer the drug, potential side effects and so forth.
The potential treatment then moves on to Preclinical Testing. This part of the journey is designed to test toxicity or whether the drug can potentially cause harm. Testing in living organisms or animal testing must follow strict regulations set out by the country’s health authority.
Once the preliminary safety of a drug is established, it moves on to Clinical Trials. In order to proceed with this stage of development, researchers must develop a plan or protocol that outlines the criteria for choosing participants, the size and duration of the study, the dosage and method of administering the drug and how the data will be collected, reviewed and analyzed. This is also the point where a study is developed to be as robust and reliable as possible from a scientific perspective. In other words developing the best study possible, one that will stand up to the scrutiny of the scientific community.
The randomized controlled blinded trial is considered the “gold standard” in study design. This design is based on randomly allocating eligible participants to one or the other treatment groups (one group may receive a placebo). After randomization, the groups are followed in exactly the same way. This reduces the amount of bias in a study. Bias can be further reduced by either “blinding” the subjects or the assessor or both (single or double blinded respectively).
Following extensive clinical testing, the new treatment presents for approval from the country’s health authority which in the US is the FDA. During this process the pharmaceutical company must present all of the data that brought the drug to this point of the development process as well as labeling and patient information. The review committee will go through all of the information presented and may ask for further study to satisfy their requirements. This part of the journey can last several years until finally the drug reaches the pharmacy shelves and ultimately becomes available to the patient community.
But the process doesn’t end there. The true safety and effectiveness of a drug comes with years of widespread use so there is ongoing monitoring once the therapy reaches the marketplace. The designated health authority may at any point put additional warnings or cautions on a drug’s use, change the prescribing recommendations or if new safety concerns arise, even take it off the market.
So the journey from lab counter to drugstore shelf is a long one but a truly necessary one. In theory the regulations put in place are for the safety and betterment of those that the therapy is being developed for although some may argue that it is one that takes too long.
"The Drug Development Process." Patient Network. FDA, n.d. Web. 13 June 2014.
"Randomized Controlled Trial." Wikipedia. Wikimedia Foundation, 12 May 2014. Web. 13 June 2014.