Science – Part 1: What is the current phase of research?

We are launching a blog series on how to understand science better

TV, social media and newspapers often report about scientific health related news. Physicians and researchers are frequently invited to comment on the news.

During the last months, for example, the predominant topic was the COVID-19 pandemic. In the handbook (“Vademecum”) on how to deal with information about the Coronavirus – which can be found in our German Latest Tips – we were discussing how to not get lost in the flood of news, i.e., how to assess the source of information and reliability of the news, how to recognize conspiracy theories, etc. One post briefly addresses the question: Does science contradict itself?

However, scientific news do not only inform about COVID-19 but also about other areas, such as spinal cord injuries. That’s why we have decided to take a closer look at some aspects in a blog series.

A new blog series to understand science better

Scientific studies are complex. Once we understand how they work, we are better able to gauge if and when the tested drug or medical product could eventually improve our lives one day. For example, we often read about the development of a new vaccine against COVID-19 or a new implant that is supposed to enable people with spinal cord injury to walk. The goal of these blog articles is to empower everyone to put a realistic amount of hope and expectations in these studies.

Obviously, reading these blogs will not make us experts of epidemiology or neurology. We will learn, however, to ask ourselves the right questions to understand, for example,

• whether a study is promising,
• whether the outcomes can soon be applied in clinical practice or whether it will take years to do so,
• whether the outcomes are applicable to our personal situation or rather not, etc.

A critical analysis of scientific findings is useful, especially for our so called “information society” in which news are commonly “packaged” to reach an audience or generate clicks. Many newspapers and online news do actually choose sensational headlines and catching sentences when reporting about scientific news. Unfortunately, this often makes us think and believe things that are not reality (yet). How often have we read about revolutionary studies that promise to “heal” spinal cord injury?

Therefore, when we receive or read information about scientific studies it is useful to ask oneself the following questions:

• What is the current stage of research?
• What kind of study are we dealing with?
• Do these outcomes apply to me?

In this first part, we deal with the question: What is the current stage of research?

It takes years to develop a drug

Research is a slow process and is advancing step by step. Developing a drug or vaccine, for example, takes years – ten years on average. This is necessary to precisely assess, by following strict procedures, whether a drug is not only effective but also safe and well tolerated by the body.

The development of a drug begins with studying the biochemistry and physiology of a disease. These studies identify a substance with a therapeutic effect called active ingredient. This active ingredient is assumed to be an effective treatment. The active ingredient must then be lab-tested, first via “in vitro” tests (“in the glass”, i.e., in test tubes) and then “in vivo” (i.e., on animals).

Only when these tests are successful, clinical trials are performed, i.e. testing the active ingredient on people. These take a very long time, even 6-7 years and consist of four phases, three of which occur before market approval and the fourth after approval.

During the first phase, the drug is administered to a small group of normally health people in order to measure adverse effects. During the second phase, the drug is given to several dozen or hundreds of volunteers who are affected by the disease. The goal is to find out which dose shows the best effects.

The third phase evaluates the efficacy of a drug. To do so, it is compared with similar drugs already available on the market to assess the benefit-risk ratio. During this phase the drug is administered to hundreds or thousands of people over several months. Monitoring of the effects of the drug can continue for several years.

When a drug passes all these tests, it is market approved. After market introduction the fourth phase begins during which less frequent side effects are monitored that occur when a very large population uses a drug.

Only one of ten drugs gets to the market

This lengthy process ensures the safety and efficacy of an active ingredient is properly assessed. Indeed, every step requires approval by the respective authorities, e.g., Swissmedic in Switzerland.

Therefore, when we read about a study that has shown excellent test results in the lab, we should keep in mind that the study is still at an early stage and many further steps are still necessary before the product or drug will be available at the pharmacies.

Furthermore, not all studies reach the stage of market approval. Even if the majority of the drugs passes the first study phase, around 90% don't make it to the market because they do not fulfill the requirements relating to safety or the benefit-risk ratio.

Since we do now know the long and complex development process of a new drug or therapy, we can conclude: Knowing in which phase the study currently is, is very important to adapt our own expectations accordingly.

However, the difference does not only lie in the stage of the study phase; the study approach also influences which conclusions we can draw and which expectations we may have. How to differentiate between various study approaches will be the topic of our next episode!