From Lab Bench to Launch: The Biotech Commercialization Journey
Biotechnology has become a familiar term in recent years, particularly because of the rapid development of COVID-19 vaccines like Moderna’s mRNA-based platform. The term biotechnology was used for the first time by Karl Ereky in the early 1900s, when he used it to describe that merging of biology and technology. This idea is applied to several different industries, including within healthcare and medicine. Today, there are many large, successful biotech companies like Genentech, and newer, aspiring companies popping up hoping to drive the next innovation in medicine.
1. Discovery & Preclinical Development
The biotech journey begins with a scientific discovery. This involves identifying a target (what the biologic will act on, such as a protein, receptor or gene) and determining whether the target is a viable option for treating.
From there, several biologic candidates are developed, and various in vitro (in cells) and in vivo (in animals) studies are completed to assess safety, efficacy, how the candidates will behave and move through the body.
This phase is the cornerstone of the journey, as some sort of novel scientific discovery needs to be made to kick off the process. It's also crucial that the right target is selected and that the proposed candidates act on that target in an effective way.
Stakeholders
Even though a scientific discovery is at the core of this process, stakeholders will range from scientific, regulatory, operational, and strategic.
Scientists from several different specialties are involved, such as biologists, biochemists and toxicologists. These could be academic researchers, or those in an established biotech company. If the company is a startup or coming out of a university, university tech transfer offices, seed investors and VCs may be involved.
Depending on the size of company and whether they need external support, a Contract Research Organizations (CROs) or Contract Development and Manufacturing Organizations (CDMOs) may be contracted to conduct studies or manufacture materials if the company lacks in-house capacity.
Regulatory Affairs typically leads the Investigational New Drug (IND) strategy, assembly and submission, with support from Quality Assurance (QA), CMC Regulatory Authors and Nonclinical Regulatory Authors. Executive Leadership will be responsible for governance decisions throughout the process, and Investors or the Board of Directors may even be key stakeholders if IND readiness is tied to funding or partnerships.
Key Information, Tooling and Databases
As you can imagine, there are several key data sources and tools out there for each stakeholder group. For example, during early stage biological research, researchers often consult a number of databases such as NCBI Gene, UniProt, GeneCards, OMIM, KEGG, Reactome, STRING, BioGRID, clinicaltrials.gov and DrugBank. There are also many different software tools developed for design and engineering, such as Rosetta for protein modelling, docking and design, and BioLuminate (Schrödinger) for biologics engineering.
Centralized data management is important as work is completed in this phase, so biotechs often leverage Electronic Lab Notebooks (ELNs) such as Benchling or Revvity, and Lab Information Systems (LIMS). Tools like Veeva Vault, or SharePoint organize documents by module for IND filing (CMC, nonclinical, clinical).
Regulatory Checkpoint
Once preclinical studies indicate a promising candidate, the regulatory affairs teams take charge of preparing a submission for human trials. In the US, that is an Investigational New Drug (IND) application to the FDA, and in the EU this is a Clinical Trial Application (CTA). IND applications to the FDA must include comprehensive documentation across three main areas: CMC (Chemistry, Manufacturing, and Controls), nonclinical (preclinical) studies, and clinical protocols, along with administrative and regulatory documents.
Legal professionals and regulatory affairs experts work together to ensure that the submission meets all necessary compliance standards to avoid costly delays or clinical holds due to incomplete or insufficient documentation.
The FDA has 30 days to review the submission, and if no concerns arise, clinical trials can commence. However, if the FDA identifies potential safety issues, it may place the application on a clinical hold, delaying the initiation of human
4-7 years
$10 million to $100 million
If the research is successful and the company passes regulatory approval, it progresses from studies in cells or animals to testing in humans. This marks the beginning of the next major hurdle: clinical trials.
2: Clinical Trials: Phases I-III
Clinical Trials tends to be the longest, most expensive phase of the biotech life cycle as patient safety and scientific rigour are paramount. It is broken into three distinct phases, each with a crucial regulatory checkpoint. Each phase is also designed to answer a specific question (safety, efficacy, dosage, etc.) before moving to the next, larger trial.
One important note is that patents are often filed during the discovery phase, so speed and efficiency during clinical trials is important to the commercial success of a biotech. Any time spent in the development process counts against the patent protection period after it goes to market, and once a patent expires, generics create competition that will inevitably reduce revenue.
Phase I: Safety and Dosage
- Goal: Assess safety, dosage range, and side effects in healthy volunteers or patients
- Timeline: up to 1 year
- Checkpoint: Safety data submission to FDA
Phase II: Efficacy & Side Effects
- Goal: Determine efficacy and side effects, and continue safety monitoring in a larger patient group
- Timeline: 1-2 years
- Checkpoint: End-of-Phase II meeting with FDA (optional)
Phase III: Large-Scale Efficacy
- Goal: Confirm effectiveness, monitor side effects, compare with standard treatment
- Timeline: 1-4 years
- Checkpoint:: Pre-submission meeting (e.g. pre-BLA meeting with FDA)
Stakeholders (All Clinical Phases)
Although research is still ongoing in the Clinical Trials phase, the research team often expands to include a new set of stakeholders, each with specific responsibilities essential to the success and integrity of the study. Clinical Investigators play a central role. They are responsible for recruiting and caring for participants, conducting trial procedures, collecting data, and reporting any adverse events or protocol deviations.
Because studies are now taking place in human, study participants (healthy individuals or patients) are critical stakeholders. Their safety and informed consent are a top priority throughout the study. The Institutional Review Board (IRB) ensures the rights, safety, and well-being of all participants. The IRB reviews and approves the trial protocol and consent forms and continues to oversee the study as it progresses.
Healthcare providers, such as doctors and nurses, support the delivery of investigational treatments and help monitor participants during the trial.
Operational support often comes from clinical operations teams, project managers, or outsourced Clinical Research Organizations (CROs), who coordinate the logistics of the trial. Biostatisticians and clinical data managers are responsible for managing trial data and performing statistical analyses to evaluate the study outcomes.
Finally, regulatory and compliance teams including Regulatory Affairs, Quality Assurance (QA), Legal, and Medical Affairs ensure that the trial complies with applicable laws and responds to inquiries from regulatory authorities
Key Information, Tooling and Databases
Clinical trials require a coordinated effort across scientific, operational, regulatory, and technological domains. This is similar to the pre-clinical phase, but a layer of complexity is added due to human participant involvement and regulatory oversight.
To collect and manage all of the patient data, several systems are used throughout the trial. Electronic Data Capture (EDC) systems (e.g. Medidata Rave) allow researchers to input and track clinical data in real-time. Clinical Trial Management Systems (CTMS) like Veeva CTMS support scheduling, documentation, and overall trial coordination. In parallel, Electronic Health Records (EHRs) (e.g. Epic) provide background medical information for participants.
Researchers may also reference clinical trial registries and databases, including ClinicalTrials.gov and the WHO International Clinical Trials Registry Platform, to review past or ongoing trials. However, access to some datasets is limited due to privacy or regulatory restrictions.
For ethical oversight, Institutional Review Boards (IRBs) often use platforms like IRBNet to manage protocol submissions and approvals.
When preparing regulatory submissions, teams use eCTD (Electronic Common Technical Document) software, such as Lorenz docuBridge or GlobalSubmit, to compile and submit documentation to health authorities. These submissions must adhere to data standards established by the Clinical Data Interchange Standards Consortium (CDISC), which helps creates a common language and format.
One challenge in this phase is data silos based on several different systems being used by different stakeholders, and integration issues (or a lack of possible integrations) between the various systems. This can be a challenge for real-time monitoring, and a holistic view of the vast amounts of information and data.
Burn Rate
Clinical trials get more expensive as candidates move through the phases, and costs can vary greatly depending on factors like the therapeutic area, study design, geography, and recruitment challenges. Estimates for cost by phase show that Phase 1 can cost between $1-7M, Phase 2 between $7-20M, and Phase 3 between $10-$100+M.
After the time, effort and money invested into making sure the biologic works effectively and safely in humas, the company now has successful Phase III results in hand. Attention turns from scientific discovery and validation to solely seeking regulatory approval.
3. Regulatory Approval
90% of candidates fail in clinical trials so if a candidate makes it through to regulatory approval it is somewhat of a unicorn. At this point, a biotech company submits a Biologics license application (BLA) to the FDA, or equivalent filings to EMA/PMDA.
You could think of this regulatory step like submitting a final thesis for your PhD, except instead of just your work, the BLA is a team effort involving years of collaborative research and combines all important information into one massive, coordinated package. The FDA first performs a filing review to ensure the application includes everything required: clinical and nonclinical data, manufacturing details, product labeling, and facility information.
Then, if the submission passes this initial check, the FDA assigns a Prescription Drug User Fee Act (PDUFA) date, which is typically 10 months for standard review and 6 months for priority review. The PDUFA is essentially a deadline for making an approval decision.
The FDA then conducts an in-depth, multidisciplinary review. This includes evaluations by clinical, statistical, and manufacturing experts. The agency may also inspect the manufacturing facilities and negotiate final product labeling with the sponsor.
The process concludes with a final decision. If successful, the FDA issues an approval, allowing the biologic to be marketed in the U.S. If issues are identified, the company receives a Complete Response Letter (CRL) detailing deficiencies that need to be addressed before approval can be granted.
Stakeholders
At this point, the primary stakeholder involved are the regulating bodies making the decision to issue a license. Regulatory Affairs teams are still heavily involved after the submission and act as the primary liaison with the regulatory bodies. They will pull in other cross-functional stakeholders as needed, depending on the nature of the inquiries from the regulating bodies.
FDA Approval or EMA Marketing Authorization, etc.
Up to 1 year for standard review - priority review may be shorter.
Burn Rate
The cost of this phase is $5M+, as the application fee alone ix $4.3M (including clinical data) for a BLA. This doesn’t include any of the costs related to preparation or regulatory consulting.
4. Commercialization & Post-Marketing
After regulatory approval, the journey isn’t over yet, it just enters a new domain. Biotech companies now shift focus to launching the product, market access, pricing, and ongoing safety surveillance.
If the manufacturing facility passed pre-approval inspections, full-scale production can begin. Companies then roll out a market access strategy, which includes determining pricing, securing reimbursement from payers, and developing materials to educate healthcare providers. This is also when sales and marketing efforts kick off in earnest.
Even though the biotech has regulatory approval, there will be ongoing regulatory requirements that have to be followed. Agencies like the FDA or EMA may require post-marketing (Phase 4) clinical trials to gather additional data on long-term safety, efficacy, or real-world outcomes. Regulatory authorities can also conduct routine inspections to ensure the company remains in compliance with quality and safety standards, and ongoing safety monitoring and adverse event reporting is mandatory. In some cases, companies must implement formal risk management strategies (e.g. Risk Evaluation and Mitigation Strategy (REMS) in the U.S., Risk Management Plan (RMP) in the EU).
In short, while approval is a major milestone, long-term success depends on effective execution, monitoring, and continuous compliance.
Ongoing.
Burn Rate
There are several upfront and ongoing costs in this phase. Manufacturing costs are significant, and can be up to a $1 billion just for new manufacturing facilities. Manufacturing costs themselves can range from $95 to $225 per gram. Post-market surveillance costs can range from $1 million to $50 million or more per year, depending on the scope and duration of the studies.
Summary
As you can see, getting a biologic to market is no small feat. It takes many years, involves dozens of stakeholders, and requires mountains of data. However, when it works and a company brings a scientific discovery from lab bench to market launch, it’s not just an end; it’s an invitation to begin the process again, fueled by the next big idea in science.
| Discovery & Preclincal Development | Clinical Trials (Phase I-III) | Regulatory Approval | Commercialization & Post-Marketing | End-to-End |
|---|---|---|---|---|
| 4-7 years | 3-7 years | Up to 1 year | Ongoing | 10-12+ years |
| $10-100 million | Phase I: $1–7 million Phase II: $7–20 million Phase III: $10–100+ million | $5 million | - | $1B+ |
References
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