Month: January 2024

The most costly and time demanding process of the drug discovery timeline is the clinical trials. In fact, everything that happens before this stage is to try and get the drug to “quit early” so that pharmaceutical companies can avoid the hassle of running clinical trials for a drug that is going to fail out in the first phase. Beyond the costs of clinical trials, there are a few different ethical and civic issues surrounding them, some have partial solutions others have been left untouched.

Before we delve a bit deeper into these issues, let’s lay the groundwork for how clinical trials are usually carried out. For pharmaceuticals targeted at everyday ailments and conditions, diabetes for example, there is a very well defined procedure for conducting clinical trials. For conditions like cancer, there are some exceptions.

Phase I tests the medication in healthy individuals to prove that there are no side effects. This phase also helps researchers determine what the proper dose level is and what the lifetime of the drug is in humans. Up until this point, studies have been conducted in animals and other model systems. While researchers can predict what the dosage would be from these studies, it is impossible to tell without seeing it happen in humans. Once a safe dosage range has been determined, the study moves on to Phase II of clinical trials.

During Phase II, researchers work to determine the efficacy of their drug: does it actually work? Once this phase is over, the company responsible for developing the drug submits a Investigational New Drug Application to the FDA, and once that’s approved, they move on to a much larger and longer Phase III study.

Phase III has the goals of proving what efficacy that was observed during Phase II, on a larger scale and with a much more diverse patient population. If a drug passes through Phase III, then the company responsible then submits a New Drug Application, and waits for FDA approval to begin selling the new prescription.

This entire process has requirements beyond just time and money. It requires a “suitable” patient population. To run clinical trials, you need to have enough patients to do so, and not every condition can provide this.

In the case of rare diseases, it is difficult to develop new medicines for them because the patient populations are so small. In some cases, attempting to run a proper phase I stage of clinical trials would potentially cure the entire patient population.

While on paper, this seems like a great thing, in reality it means that few pharmaceutical companies actually get anywhere close to rare diseases. Most drug development companies work by raising money for their disease targets, and it would be near impossible to raise anywhere near the required amount of money for a disease in which the investors were guaranteed not to get their money back (especially considering the success rates are usually so low for normal drug targets).

Beyond rare diseases, there are additional ethical concerns when it comes to running a clinical trial: what do you do for pressing and fatal diseases? how do you decide who can join a clinical trial? how much/do you compensate the patients?

This blog post has already gotten quite long already, so I’ll continue it in the next one.

At some point or another, we’ve all used over-the-counter prescription drugs. Whether it was a headache, runny nose, flu, cold, most of us have made that trip to the pharmacy to find some remedy to our ailment. However, in most cases, these are one-off purchases that occur once every few months, or even once a year.

In a few cases however, some prescriptions are required at a regular frequency, every day, once a week, etc. In these cases, such prescriptions are necessary for an individuals ability to improve their quality of life or even just to live. When it comes to such prescriptions that are necessary for life and good health of an individual, how to price these prescriptions becomes an ethical conundrum for the pharmaceutical industry. Healthcare is something that we all need at one point or another, so putting a price on medicine that can save people’s lives or improve them significantly becomes a seriously controversial issue.

In the words of the World Health Organization, “healthcare is a fundamental human right,” which means that “everyone should have access to the health services they need, when and where they need them, without suffering financial hardship.” The key part of this statement is “without suffering financial hardship.” In most cases, the treatment that the individual needs exists and is technically available, but due to financial limitations and the often high pricing of newer medicines, people aren’t able to receive the treatment that they need to survive. In most cases, an individuals healthcare may be able to cover most of the costs, but when medicines become increasingly expensive, the burden on the individual increases as well.

When people learn about this disparity, often their first instinct is to then blame the drug discovery and pharmaceutical companies for putting profit over the wellbeing of individuals. This however, is not the complete narrative. What most people don’t realize is that the problem does not lie with pharmaceutical companies themselves but with the challenges involved in developing medicines that are effective, non-toxic, and consumable.

The statistic changes slightly depending on who you ask, but roughly 4% of proposed drugs ever make it to market. That means that roughly 96% of drugs FAIL before they make it to the shelf. Not only that, but the cost of developing drugs goes up exponentially the further into the process that you go.

Often reaching billions of dollars in costs only to fail before reaching clinical trials (the final test before a drug is able to be approved by the FDA).

So what is the solution? How do we ensure that patients get the care and medicines that they need, while also ensuring that drugs are able to be created to continue to create medicines for currently incurable diseases? It is a issue that stems far beyond the realm of pharmaceuticals and will require properly informed legislature to be passed in order for any meaningful impact to be made.