Five reasons why medical research can be a long process

1. Our bodies are complicated systems

Medical conditions, such as type 1 diabetes, are incredibly complex. As people with type 1 know all too well, so many things influence the working relationship between insulin and glucose, from your stress levels to the weather outside.

Type 1 diabetes primarily involves the insulin-producing beta cells and the immune cells that attack them. But these cells don’t exist in isolation. We have roughly 20,500 genes for around 400 different types of cells totalling 17 trillion cells in children and up to 37 trillion in adults. Our cells and the chemicals they release have intricate interactions and relationships with each other. So, our researchers have so much more than just the beta cells to consider when trying to replace, regrow or protect them.

Due to this complicated biology, researchers often face unexpected challenges and setbacks that require revisiting and refining their ideas and approaches or sometimes starting from scratch.

2. Rigorous testing and validation: the research pipeline

Before doctors can give new treatments for type 1, researchers need to thoroughly test them to make sure they are safe and effective. This extensive development and testing process is called the research pipeline and involves distinct phases.

A diagram of the research pipeline.

The drug discovery stage is when the exploratory lab work takes place, with researchers investigating new ways to prevent, treat and cure conditions like type 1. Promising ideas move forward to preclinical trials, in which researchers test new therapies using cells in petri dishes and lab animals. Treatments that prove safe and effective in animals progress to clinical trials in humans.

New treatments must undergo three phases of clinical trials. Phase I trials involve a small number of people to check the treatment isn’t toxic for humans and to find the best dose. Following this, the trial moves to phase II, recruiting more people to investigate side effects and whether the treatment works. Then, in phase III even more participants are involved to compare the new treatment to an existing therapy or a placebo. A trial can fail and be terminated at any of these three stages.

Each stage of the research pipeline involves time-consuming processes, including planning experimental methods, applying for ethical approval and writing and reviewing scientific papers, and getting those papers published in journals. Many individual research projects occur within every stage to repeat the experiments to ensure results are reproducible and to tweak methods to improve the outcomes. Only five in 10,000 prospective drugs reach clinical trials, and just one progresses all the way to securing regulatory approval.

3. Securing regulatory approval

Evidence gathered from research studies is used to apply for approval for new technologies and treatments from government regulatory bodies. Each country has their own organisation that carefully evaluates the data to ensure that the benefits outweigh any risks before granting licences for new medicines. In the UK, we have the Medicines and Healthcare products Regulatory Agency (MHRA). This step is crucial for patient safety but can add a significant amount of time to the overall process. For example, the MHRA can request more evidence of a treatment’s safety and effectiveness, so researchers must run more clinical trials.