The journey of medicine from the laboratory to the pharmacy is a complex process that consists of different stages and takes around 10–15 years. This process includes pre-clinical development, clinical development, and time for dealing with the regulatory authorities. Before moving to the next stage, scientists test thousands of diverse chemical compounds until they come to just a few useful candidate drugs. In this blog post, we will review the main stages of drug development in medicine and learn the ins and outs of this process.
How We Research New Medicines
The process of researching and developing new medicines is growing and becoming more complex. The average cost of developing a new drug is estimated to be $2.5 billion, including the cost of failures. Overall, the average probability of a drug turning out to be successful is less than 12%.
Just imagine, the drug discovery process includes approximately 5,000–20,000 compounds, and only 200–250 of them move to the next stage, leaving only five compounds for clinical research, and resulting in one final drug that is approved for sale. The main drug development challenges include:
- Increasing costs
- Lack of the predictive validity of current animal models
- Insufficient knowledge regarding underlying mechanisms of disease
- Patient heterogeneity
- Lack of targets and biomarkers
- A high rate of failed clinical trials
So who creates medicine? It’s the job of pharmaceutical scientists trained to discover, develop, test, and manufacture new medications. How much do medical researchers make? The average annual wage for medical scientists is $88,790. As you can see, the process of drug development is very resource-intensive and involves highly sophisticated technologies and manufacturing processes, and complex project management.
The Stages of Drug Development: What the Process Looks Like
As we have already mentioned, the drug development process consists of several stages. On average, new drugs are developed in 12.5 years; however, nowadays, the technology in the research and development industry is evolving and may accelerate this process. Let’s review the main drug development stages and learn how scientists identify and search medicines.
Phase 1: Pre-Discovery Stage
Pre-discovery is the first stage of medicine development. The drug discovery process begins with investigating how the disease impacts and changes the biological processes in the body. The average time of this stage is undefined, as the scientists need to identify what causes the disease, the proteins that take part in the process, and the valid target. After the valid target is determined, drug candidates are tested for their interaction with the drug target. After the interaction is confirmed, scientists need to validate the target. The target is validated if its activity is connected to the disease and if a drug can modify it.
Phase 2: Discovery Stage
The main goal of this stage is to find a molecule that will work as a gene or protein on a specific target. Scientists can either create a molecule from scratch or choose an existing molecule from the library. During this stage, they process over 7,000 molecules to find the ones that will stop the disease.
Phase 3: Molecule Development
During this stage, the main goal is to test the molecule and find the combination of compounds that make the molecule work effectively. The testing is usually done with the help of computer simulations (in silico studies), cell-based tests (in vitro), and animal testing (in vivo studies) to mimic its behavior in humans.
Phase 4: Clinical Trials
During this stage, the medicine is tested for safety and efficiency. Medicines & treatments are tested in humans during four stages:
- Phase 1 – 20–100 health volunteers
- Phase 2 – 100–500 patients with the disease
- Phase 3 – 1,000–5,000 patients
- Phase 4 – Ongoing monitoring during the life span of the medicine
Researchers must meet special guidelines during clinical trials to ensure patient safety. Scientists often partner with universities, hospitals, general practitioners, and the government to perform clinical trials and identify and develop new medicines.
Phase 5: Licensing Approval
After the clinical trial stage, special regulatory agencies need to confirm the safety, efficiency, and quality of the medicine and issue a medical license. Doctors can only prescribe a medication to patients after regulators have approved the medicine..
Pre-discovery Stage: The Starting Point of the Research
The pre-discovery stage is an important phase of medicines development that begins with understanding and studying the disease. Everything starts with target identification. It aims to find a molecule involved in that particular disease that can be modulated by a drug. But before finding a target, a scientist needs to determine if there is an unmet need in the industry. If there is no available medicine for this disease or there is one but with harsh side effects, the scientists will continue their research and development.
After that, scientists need to study the disease at a molecular level. This process usually includes:
- Exploration of how genes have changed
- Identification of how these changes affect the proteins encoded by the genes
- Studying how these proteins interact with each other in living cells,
- Researching how the affected cells change the specific tissue they are in
- Defining the effect of these processes on patient health
After that, the biological target for a prospective medicine is developed. The biological target is a biomolecule that interacts with a potential drug compound and produces a clinical effect such as treatment. Target validation helps scientists identify the most promising approaches for medicine development before creating potential drug candidates, which increases the efficiency and effectiveness of the R&D process.
Clinical Trials Phases in Drug Development
Drug development has a structured sequence of phases to confirm the efficiency of medicine and identify if the new biomedical or biobehavioral interventions are safe for patients and disease treatment. Clinical trials are divided into three main phases.
Phase 1 includes small trials (usually recruiting 20–80 people who have no conditions) and aims to determine the highest dose of medicine humans can take without any side effects. The scientists monitor how the patient’s body reacts to the medicine and identify the best dosage. Also, investigators decide on the best way to administer the drug: orally, topically, or intravenously. After this stage, approximately 70% of medications move to phase 2.
Phase 2 trials involve several hundred people who suffer from the disease condition. The aim of phase 2 is to identify how effective the medicine is and gather more information about side effects. After that, approximately 30% of medications proceed to the next phase.
Phase 3 focuses on comparing new treatments with the treatments that are currently available. This clinical trial stage involves around 3,000 participants and can last for several years. During this phase, the participants are divided into 2 groups. The first group receives the current treatment, and the second group proceeds with the new one. To move to the next phase, investigators have to prove that the new medication is as safe and effective as the existing medication. If this happens, the FDA approves the medication, and during phase 4 (last one), investigators find out more about the long-term safety and side effects of the drug. Phase 4 can last for many years.
Artificial Intelligence Research in Medicine: How it’s Used for Drug Development
Artificial intelligence and machine learning have contributed to advances in medicine and pharma efficiency. Here are the four main applications of AI in medicine:
- Disease diagnostics
- Faster drug development
- Treatment personalization
- Gene editing improvement
Let’s dive deeper into how artificial intelligence accelerates drug development, medicines & treatments and makes them more effective.
- AI can analyze data faster, make it easier to identify targets for intervention, and automatically identify good target proteins.
- Machine learning can predict if the compound will interact with the molecule in the desired way based on structural fingerprints and molecular descriptors.
- Machine learning can automatically identify suitable candidates for clinical trials.
- AI can help discover suitable biomarkers for a particular disease and automate this process by classifying molecules into good and bad candidates.
AI algorithms are already transforming biomedical research. For example, Google’s AI subsidiary, DeepMind, announced that its AlphaFold program could deliver computational predictions of protein structure that approach the quality of those provided by gold-standard experimental techniques such as X-ray crystallography.
“We recently developed Triage, a custom software solution for healthcare equipped with an AI-powered voice attendant. The voice-fingerprinting technology helps identify patients and locate their records. It’s one of the examples of how AI is changing healthcare.”
— Vlad Medvedovsky, Founder and CEO at Proxet (ex - Rails Reactor), a software development services company
Natural language processing (NLP) algorithms also employ deep learning to analyze genes and determine what diseases might be related to them. In recent medical studies, AI-focused startup Healx tested knowledge graphs to gain insights into rare genetic diseases.
“Our main focus is on finding new uses for old drugs. We are working with all the data on the 4,000 FDA-registered drugs, and we are building data on drugs registered elsewhere in the world.”
— Neil Thompson, Healx chief scientific officer
Read more about big data and artificial intelligence in medicine in our article “Doing big data-driven research in medicine.”
Medicines of the Future: What to Expect
So what will medicine look like in the future, and what new drugs developed will we see? Here are some jaw-dropping examples of innovations in medicine that will change the way we treat diseases in the near future.
Smart contact lenses packed with biosensors
Korean researchers have already developed contact lenses that can diagnose diabetes. These lenses can track glucose levels from tears and help people with diabetes manage diet and medications.
“The electrode and base of the wireless smart contact lens are see-through and thus do not present an obstacle to the wearer’s vision. When blood glucose levels fluctuate, the sensor is triggered, and the micro-LED display is toggled accordingly.”
— Park Ji-hoon, professor at UNIST
Virtual reality for pain relief
Recently, a study has shown that VR helps to reduce pain and creates an illusion of time acceleration, effectively shortening the length of pain episodes. In the study, patients watched VR-guided relaxation, natural environments, simulated flights, and animated games. It’s predicted that VR’s impact on pain relief will lead to future innovations.
Voice as a diagnostic tool
Scientists have found that the characteristics of the patient’s voice can reveal the state of their health and predict future diseases. Sonde Health Inc., a Boston-based company, has developed a voice-based technology platform for monitoring and diagnosing physical and mental health conditions. There will be more inventions and innovation in this industry in the future.
As you can see, artificial intelligence and other technologies have a lot to contribute to the future of medicine development. Proxet has a team of professionals, which will develop new healthcare solutions tailored to your needs. We've completed several projects for clients in healthcare, some of which utilized voice recognition. Our team possesses all the necessary skills and experience to satisfy your business needs quickly and efficiently.
