Many thousands of new drugs are developed every year. Before these can be administered by doctors and hospitals, they must undergo a thorough development and testing process.
Drug development (also known as pharmaceutical product development) is a lengthy process, involving several important steps to ensure the drug is effective, safe and beneficial. It takes several years to develop and test new drugs, and can cost millions, sometimes billions to complete.
Whilst the drug development process is long and costly, it is essential that new drugs undergo rigorous testing to ensure they are safe and do not pose serious risks.
Find out more about reducing drug development cost and increasing efficiency.
What is pharmaceutical product development?
A pharmaceutical product is a new medicine or drug that has been developed and brought to the market. Pharmaceutical product development is the process behind this, whereby the new drug has been discovered, researched, reviewed, and approved for market use.
Throughout this process, pharmaceutical products undergo extensive evaluation. The product is examined and assessed for safety, as well as compliance with all regulatory requirements when treating a particular disease or condition.
As a result, the pharmaceutical product development process is extensive and often complex. On average, it can take around 10-15 years for a pharmaceutical product to complete its journey from initial discovery to market use.
The drug development process
New drugs, or pharmaceutical products, are typically developed and tested through the following process:
- Initial drug discovery
- Preclinical research
- Partnering with a CRO
- Clinical trials
- Submitting preclinical data for approval
- Regulatory review and approval
- Post-marketing safety monitoring
New drugs are firstly developed in a laboratory setting, where researchers test on human cells and animals to ascertain basic safety. Drugs are then tested on human subjects in clinical trials to determine safety and efficacy. The drug then undergoes FDA approval and post-market safety monitoring.
1. Initial drug discovery
Initial stages of drug development begin with discovery. In this first stage, researchers have usually identified a chemical compound that can potentially be used to treat particular diseases.
There are many ways that scientists may discover promising new compounds. From systematically testing molecule compounds, to developing drugs based on new disease insights.
Testing molecule compounds
Drug discovery is a systematic process, whereby scientists test a number of molecule compounds to uncover potential beneficial effects against diseases. There are usually thousands of compounds that are tested during this stage to determine which show the most potential as medical treatments.
The compounds that show the most promising effects then undergo further research and development into new medical drugs.
Those that don’t show promising effects, or those that show potential high risks are not carried through into further drug development research.
Applying new disease insights
As well as testing compounds for potential benefits, scientists apply findings and insights from the latest disease research in order to design appropriate new drug treatments.
The aim of this is to develop new drugs that target diseases in ways that have not previously been possible or available.
For example, a new drug may be necessary to treat previously under-reported effects of a disease. Similarly, existing treatments may begin to cause unanticipated side effects, requiring a new drug development that minimises the adverse effects.
Advancements in technology
In recent years, technological advances have allowed researchers to gain deeper and more targeted insights into medicine and diseases.
As medical technology continues to develop at rapid rates, scientists can investigate drugs and diseases in ways that were not always possible. And in doing so, gain a richer understanding of how to develop new drugs appropriately.
Advancements in technology also have implications on drug testing. For instance, online systems allow for patients and researchers to report drug side effects in real-time, in a centralised database that is accessible to the contract research organisation (CRO), sponsors, and approval boards.
2. Preclinical research
Once scientists have identified compounds with potential for further development, researchers will investigate the drug in more detail through a number of preclinical research tests.
These tests are carried out to understand basic information about the drug:
- How the drug should be administered
- Potential interactions with other drugs
- The drug’s benefits
- How effective the drug is in comparison to other drugs for the same disease
- Safe dosage levels
Information from preclinical research is used to determine the best way to develop new drugs. For instance, these findings help researchers understand how to administer the development drug safely to volunteer and patient groups in clinical trials.
This research also prevents any potentially harmful compounds from reaching human clinical trials. If molecule compounds react negatively with preclinical test cells, or show no promising reactions, then the drug will not undergo further clinical development.
Preclinical research is an important step in the drug testing process, since this identifies which compounds are worth further investigation.
Additionally, this identifies and discounts any molecule compounds that present themselves as toxic to humans.
3. Partnering with a CRO
For compounds that have the potential for further development, sponsors will usually partner with a contract research organisation (CRO) in order to carry out the research with the necessary teams, infrastructure and facilities. For instance, Simbec-Orion offers clinical trial management services across phases I, II and III of investigation, alongside central laboratory facilities and other CRO services.
Sponsors usually get in touch with the CRO early on in the pharmaceutical product development process, particularly before planning phase 1 research and submitting the preclinical data for approval.
Ready to partner with a full-service CRO? Submit an RFI/RFP form with Simbec-Orion.
4. Submitting preclinical data for approval
At this point in the pharmaceutical product development process, the preclinical research shows promising data, and the CRO is in partnership with the sponsor.
Now, the sponsor must submit the preclinical data for approval. Data is submitted to the Food and Drug Administration (FDA) and/or European Medicines Agency (EMA) depending on where the clinical trial will be taking place.
FDA approval – Investigational New Drug (IND) application
In order to gain FDA approval for clinical research, the physician responsible for initiating and investigating the case submits an Investigational New Drug (IND) form.
In most cases, this type of application is submitted to study an unapproved drug or an approved drug used in an unlicensed indication or in a different patient population. The IND application is usually submitted in order to permit the investigation of an experimental drug that has shown signs of success or promise in clinical trials, or for treating serious conditions.
The FDA review the IND application whilst the final stages of clinical work are being completed. The sponsor must wait 30 days until starting the clinical trial process, as this gives FDA regulators the time to review the IND application and ensures that subjects are not subjected to unreasonable risk.
There are several areas that the IND application must cover in order to be considered, including animal model pharmacology, toxicity studies, manufacturing information, clinical study protocols and documents, and information about the investigator.
In some cases, the FDA may authorise an emergency use IND, which enables the investigator to use and research the drug in an urgent situation. In this case, there is no obligation to submit the IND in accordance with sections 312.23 and 312.20 of the FDA’s Code of Federal Regulations Title 21.
EMA approval – clinical trial application form
For clinical trials investigating treatments for human use in the European Union (EU) and European Economic Area (EEA), researchers need to submit a European Medicines Agency (EMA) clinical trial application form.
This requirement must be met in accordance with EU pharmaceutical legislation, as detailed in the EMA’s Detailed guidance on the collection, verification and presentation of adverse event/reaction reports arising from clinical trials on medicinal products for human use.
To register the trial, sponsors have to fill in an application form in the European Union Drug Regulating Authorities Clinical Trials (EudraCT) database, using a unique code provided by the Extended EudraVigilance medicinal product dictionary (XEVMPD). The code will usually differ depending on whether the investigational drug is newly developed, or has already been authorised by the EU and EEA for previous research.
Newly developed drugs that have not undergone previous clinical research must be submitted with medicine-related information into the Extended EudraVigilance medicinal product dictionary.
The EMA provide the following requirements for submitting data into the XEVMPD:
- At least one member from the sponsor organisation must complete the EMA’s XEVMPD training and pass a knowledge evaluation. This can be completed either via face-to-face or e-learning sessions. Upon successful completion, you’ll receive a notification to inform you that you have passed the knowledge evaluation.
- The sponsor has to be registered with the EMA’s Organisation Management Service (OMS).
- The sponsor has to be registered with EudraVigilance. This ensures that data and information regarding the medicine can be submitted to the EMA.
5. Clinical trials
Researchers engage with patients to develop the drugs. Following successful preclinical findings, researchers investigate how the new drug presents itself and behaves in the human body – this is done through phased clinical trial research.
In clinical trials, the development drug is tested in human subjects for the first time, often referred to as a first in human (FIH) study. Throughout clinical trial stages, the drug is tested for safety and efficacy, determining how beneficial the drug is in relation to its possible risks.
As clinical research is conducted using human participants, this is a vital step in the drug development process. A new development medicine or drug must undergo clinical trial research in order to be approved for market use.
The drug development process continues across three main phases in the clinical trial.
- Researchers investigate the safety and pharmacology of the development drug. Find out more: what is clinical pharmacology?
- Small doses of the development drug are administered to around 20 to 100 healthy volunteers (those that do not have the disease or condition).
View our phase 1 CRO services.
- Researchers investigate the efficacy of the development drug, continually monitoring its safety profile and risks.
- The drug is administered to volunteer patients who suffer from the target condition.
- The drug is usually tested on around 100 to 500 patient volunteers.
- Phase 3 clinical trials are similar to phase 2 trials, although there are several key differences.
- Researchers compare the development drug’s benefits and risks against existing drugs used to treat the target condition.
- The development drug is tested on a larger group of patients, usually around 1,000 to 5,000. This can be more challenging in rare disease clinical trials.
6. FDA review
Once a new drug’s safety and efficacy has been evidenced through clinical testing, it will undergo FDA review for market use. FDA approval is required for any drug before it enters the medical market.
Approval is usually granted when clinical and preclinical research findings prove that the drug is safe for human use, with no significant risks posed to patients. As well, FDA approval requires evidence that the drug is effective in treating the target condition.
If a drug is approved by the FDA, it can be manufactured for market use and can be administered by doctors and hospitals.
7. Post marketing safety monitoring
The drug development and testing process continues even after clinical trial research and FDA approval. When the drug has been approved for market use, it is continuously monitored by researchers to highlight any potential safety risks that weren’t present throughout clinical research.
At this point in the drug testing process, research will have evidenced that the drug is generally safe for public use.
Although in some cases, side effects may only emerge after much longer periods of time. For instance, rare side effects may only present themselves in 1 out of 10,000 patients.
Post-market safety monitoring can help identify rare side effects, acting as a vital stage in the drug testing process.
New drugs are developed under a rigorous and systematic process. This ensures that patients can take the drug safely, and that it provides beneficial effects for the disease.
The main phases of development and testing take place throughout clinical trials, which can last several years and involve a large number of volunteers.
Although from initial drug discovery through to post market safety monitoring, new drugs are heavily tested from the offset and throughout.