There are many diseases that were once thought incurable but are now treatable with medicines and therapies. These medical advancements are thanks to a process called drug discovery, which allows scientists to develop new medications.
Drug discovery involves several phases of research, screening, testing, and validation. Biotech and pharmaceutical companies spend years and billions of dollars on this massive undertaking, but the results go a long way towards giving people new leases on life.
What is the Drug Discovery Process?
“Drug discovery” is the process by which scientists identify and develop new medications. Researchers will test to see whether active compounds have a therapeutic effect on their targeted disease, determine dosage and delivery, and then test for safety and efficacy.
The drug discovery process takes a grueling length of time – often upwards of 10 years. Of the 5,000–10,000 drug candidates, only 250 will make it to preclinical testing. Out of those 250, only a handful might make it to market approval. Exhaustive testing and vetting is necessary to ensure the medications that reach the general public are safe and effective.
Often, researchers will outsource different phases of drug discovery to CROs – contract research organizations. CROs have the experience and resources to carry out different tasks related to drug discovery and development. Their efficiency and expertise benefits the biotech or pharma company and enables faster, better, cheaper processes.
Phases of Drug Discovery
Drug discovery has evolved significantly from the pre-modern days of clinical research. Historically, scientists would study active ingredients in traditional medicines or stumble on discoveries by chance. Now, we can start from massive pre-built libraries of billions of possible drugs. We can even use DNA to tag the drugs to keep track of which ones work by using specialized tools and machines from genomics.
This has given birth to the process of drug discovery, development, and testing. To engineer new medications, potential drug compounds must undergo meticulous research and screening before market approval.
#1 — Identification of unmet medical need
With so many diseases requiring treatments, it’s difficult to imagine where to begin. Research facilities often choose to specialize in certain fields and diseases – for example, an oncology medical research organization could focus on finding treatments for lung cancer.
Through the lens of this specialization, the researchers will identify unmet medical needs within the treatment of the disease. These can come from new insights into disease onset and progression, or existing treatments that have undesirable side effects. New technologies may also emerge that enable novel discoveries in human responses to treatment.
#2 — Target identification and validation
In this stage, researchers identify the gene or protein that plays a key role in the manifestation and development of the stipulated disease.n the initial phases of target identification, researchers use tools from biology to build evidence that a target is worth mounting an expensive drug discovery campaign.
#3 — Hit discovery and validation
Through rigorous screening and testing, researchers will land on a “hit” – a compound which produces the desired activity. They will reconfirm this activity through retesting in order to identify the molecules that interact with the drug target.
The main approaches for hit discovery and validation include
- High throughput screening
- Computational screening
- Fragment screening
- Physiological screening
#4 — Assay development and screening
This is a critical component of the discovery process. Assays are test systems that evaluate the potential drug at cellular, molecular, and biochemical levels. This assesses the way the drug will interact with and affect the disease – and the human as well.
A bioanalytical assay will allow researchers to discern the purity, performance, and potency of the chosen compound. They will then repeatedly test the compound in different biological matrices such as plasma, urine, and blood. By validating their results through repeated, reproducible tests, they ensure precise and accurate results.
#5 — Lead generation and optimization
Following the assay screening and testing, researchers will generate the “lead” compound – the primary compound or molecule that will be developed into a drug. Then begins the lead optimization process, in which researchers iteratively improve a lead compound’s chemical structure to make it safe and effective.
At this stage, researchers will also address any potential risks and safety concerns associated with the compound by modifying or reducing deficiencies in the molecular structure. Alongside that, they will investigate the efficacy, potency, drug metabolism, toxicity, stability, and bioavailability of the compound prior to preclinical and clinical trials.
Post-Discovery Phases
The drug discovery phase ends once biotech and pharma researchers have identified their lead compound. Then they can begin in vivo research, before moving onto clinical trials ahead of regulatory review.
Preclinical phase
In this stage of drug development, the substances and compounds discovered in the Discovery Phase are refined, optimized, and tested. This is also the stage at which formulation takes place. This is the process whereby the lead compound is converted into an orally available pill, often coated in a digestible polymer.
While many trials still use animal testing, there have been efforts to regulate and limit these methods. Alternatives to animal testing include biosimulation and computational modeling, cell models, and organism testing (flies, crustaceans, and zebrafish).
The aim of this stage is to assess the safety and efficacy of the substances before in-human trials can begin. It also helps researchers determine appropriate dosage and use cases.
Once this is established, researchers can produce more of the drug to meet the higher demand of clinical trials.
In preclinical research, scientists will determine
- Absorption, metabolization, and excretion routes
- Potential benefits and mechanisms
- Dosage and delivery route
- Side effects
- Effects on different demographics
- Interactions with other drugs and foods
- Effectiveness versus existing treatments
Clinical trials
With the preclinical phase over, researchers will proceed with intensive drug development. These include setting up clinical trials and volunteer studies in order to refine the drug for human intake. Researchers are responsible for conducting safe, controlled studies across diverse populations to ensure the drug works to its intended purpose.
There are four phases to a clinical trial, beginning from tolerance and safety tests to correct dosage and therapy concept. At each stage, the participant pool grows and the drug is further refined. It is essential that researchers connect with a diverse pool of patients to study the drug’s effects across different demographics.
Regulatory approval
Once the active drug compound clears the clinical trials, researchers collect the relevant data and analyze it. Then they submit the prepared data to the appropriate regulatory body for review.
No drug or vaccine can be sold without approval from a national regulatory authority. Very few compounds reach this stage, and even fewer receive approval for release to the general public.
Market monitoring
Once a drug or vaccine is released to the public, the biotech or pharma company must perform post-marketing surveillance trials (PMSTs). They track and monitor the effectiveness and safety of the drug across a large demographic, and see how it compares to currently available treatments.
PMSTs or Phase IV studies assess the long-term effects of the new medication and look out for adverse effects. This can lead to further refinement of the medication for an improved formulation.
Drug Discovery and Clinical Trials
There are more specific details and subphases involved in drug discovery and development, but this article gives a general overview of the process from target identification to market approval. Year after year, researchers all over the globe will be hard at work testing and developing new medications to treat diseases – and perhaps find cures that were once thought impossible.
Vial is a next-generation, tech-powered CRO that empowers scientists to develop treatments and cures that change lives. We deliver faster, better, cheaper clinical trials through our end-to-end technology platforms. Our team of ClinOps experts and technological innovators have developed ways to deliver more efficient results for sponsors.
If you have a proposal for Vial, contact us today!
