Behind every new treatment there is much more than a scientific discovery.
Identifying a promising molecule, a new therapeutic target or a technology capable of acting on a disease may be the starting point. But between that discovery and its possible arrival to patients, there is a complex process.
Drug development requires progressively generating evidence on quality, safety and efficacy, establishing appropriate manufacturing processes and meeting different regulatory requirements.
In addition, not all candidates make it to the end of the process.
What are the stages of drug development?
Although each product may follow a different path, several major stages are usually identified:
- discovery and research;
- preclinical development;
- clinical development;
- regulatory assessment;
- post-marketing follow-up.
These stages help explain the process, but they should not be understood as a completely rigid sequence. Many activities overlap, and the results obtained may require earlier decisions to be revised.
1. Discovery: finding a candidate with potential
The development of new treatments begins long before clinical trials.
Research may start from understanding the mechanisms involved in a disease, identifying a new therapeutic target or developing a technology capable of acting on a specific biological process.
From there, many candidates may be studied.
At this stage, researchers try to understand issues such as:
- how the candidate works;
- what activity it may have;
- how it behaves in the body;
- which route of administration could be used;
- what possible risks may arise.
Only some of the candidates studied will have enough supporting evidence to continue moving forward.
2. Preclinical research
Before an experimental medicine can be studied in humans, it is necessary to have enough information to assess whether there is a sufficient basis to begin clinical development.
Preclinical research may include in vitro and in vivo studies, as well as pharmacological and toxicological assessments.
The aim is not to definitively prove that the treatment will work in people, but to better understand its characteristics and reduce some of the uncertainty before moving forward.
At this stage, aspects such as possible toxic effects, exposure to the product and biological activity are studied, among others.
3. Clinical development: studying the treatment in humans
Once the previous evidence is sufficient and regulatory requirements are met, clinical research can begin.
Clinical trials study an experimental medicine in people according to previously established protocols.
In general terms, different phases are usually distinguished.
Phase I
The first studies in humans usually focus especially on safety, tolerability, how the medicine behaves in the body and dose determination.
Depending on the treatment and the disease, participants may be healthy volunteers or patients.
Phase II
At this stage, the treatment’s activity is studied in greater depth in people with the target disease, while more information on safety and dose continues to be gathered.
Phase III
Phase III studies usually include larger populations and aim to confirm the available evidence on efficacy while continuing to characterize the safety profile.
The design, size and duration of these trials can vary considerably depending on the disease and the type of medicine.
4. Manufacturing is also part of development
Drug development does not consist only of conducting scientific studies and clinical trials.
It is also necessary to define how the product will be manufactured.
This involves establishing production processes, analytical methods, quality controls and specifications that make it possible to obtain a medicine consistently.
These issues can be especially complex in biological medicines and advanced therapies, but they are important for any pharmaceutical product.
That is why manufacturing development progresses in parallel with the preclinical and clinical stages.
5. Regulatory assessment and approval
Completing a clinical program does not mean that a medicine can automatically be marketed.
Regulatory authorities must assess the information generated during development.
In Europe, this assessment mainly analyzes the available evidence on quality, safety and efficacy and determines whether the balance between benefits and risks allows the medicine to be approved for the proposed indications.
This process reviews preclinical data, clinical results, manufacturing processes and quality control systems, among other aspects.
6. Follow-up continues after approval
A medicine reaching the market does not mean the process is over.
When it begins to be used in broader populations, new data on its safety may emerge.
That is why pharmacovigilance exists: it makes it possible to continue assessing the medicine’s benefit-risk balance and to detect relevant new information.
In some cases, additional studies may also be carried out after approval.
Why do not all medicines reach the patient?
Because one of the objectives of the development process itself is to identify which candidates have sufficient evidence to continue.
A candidate may show toxicity problems, fail to demonstrate the expected activity or present manufacturing difficulties.
It may also happen that the clinical trial results do not justify continuing the program.
This does not necessarily mean that all the research was useless.
The results obtained can help rule out hypotheses, better understand a disease or guide future developments.
From the lab to the patient: a multidisciplinary effort
Turning a scientific discovery into a treatment requires the involvement of many disciplines.
Research, bioanalysis, toxicology, manufacturing, regulation, clinical research, statistics and funding are just some of them.
That is why biomedical innovation rarely depends on a single organization.
Drug development requires connecting specialized knowledge at different points in the process.
At Qube Technology Park, companies and professionals working in different areas of the Life Sciences ecosystem come together. Creating an environment where these capabilities can meet and build new connections is part of the way Qube understands innovation.
