The starting point for any new treatment, whether a chemically based drug or a biologic therapeutic, is the laboratory. Scientists investigate cells and molecules, chemical reactions, and much more, seeking to discover potential solutions to pressing medical needs.
What Is Biotech?
Biotechnology is a field of health care that uses biology and biological – rather than chemical – resources for medical purposes. Some examples of biotech therapies include living cells grown from cultures, or manufactured enzymes and antibodies very similar to those that occur naturally in the body.
Once a promising drug or other therapeutic option is identified, preclinical testing performed in the lab and on animals is required by law before the treatment can be tested in humans. These studies are conducted to investigate the treatment's ability to fight against a targeted disease as well as demonstrate its safety.
The preclinical testing stage usually takes three to four years, and the success rate is very low – only a very small percentage of treatment candidates qualify to move on to the next stage.
If a treatment's efficacy and safety is successfully determined by preclinical testing, preparations begin for clinical trials – meaning testing in humans. An Investigational New Drug (IND) application is filed with the local regulatory agency (in the U.S., this is the Food and Drug Administration, or FDA) outlining the results of preclinical testing and clearly defining how future studies will be conducted. The FDA has 30 days to review the IND or to request additional information. Once the FDA approves the IND application, the clinical trials can move forward.
A clinical trial is a research study performed in human subjects to determine if a new drug or therapy is both safe and effective. All clinical trials are based on a set of rules called a protocol, which helps ensure that researchers in different locations carry out the study consistently. The protocol describes several aspects:
- Who may participate in the trial (for example, age, gender, health condition, etc.)
- The planned tests, procedures, medications, and dosages
- The testing timeframe and schedule
- The specific clinical issues the trial is evaluating, sometimes called the endpoints or outcome measures
Clinical trials are carried out in steps called phases. Patients may be eligible to participate in certain phases depending on their general condition, the type and stage of their disease, and what therapy, if any, they have already had. Throughout all phases, research staff regularly see the trial participants to monitor their health as well as assess the safety and effectiveness of the treatment.
Clinical Research Success Rate
The entire process of clinical trials averages 6-7 years – and sometimes longer. The testing is rigorous, and less than 25% of treatments that enter phase 1 trials are ultimately approved for patient use.
A phase 1 clinical trial is the first time a new treatment is tested in humans, and the primary goal is to assess its safety. The study is designed to determine how the human body reacts to the treatment and, specifically, what side effects occur as dosage levels are increased.
- Tested on a small number (20-100) of individuals who are most often healthy volunteers. Studies focused on cancer therapies are tested in patients affected with the target condition.
- Typically takes several months to a year
- 85% of experimental treatments move on to phase 2
Once safety is confirmed in phase 1 trials, phase 2 studies can begin to test effectiveness. Most phase 2 trials are randomized, meaning that one group of patients receives the experimental treatment while a second control group receives a standard treatment or placebo. Often these studies are also blinded: neither the patients nor the researchers know who is getting which treatment. This approach allows both the researchers and regulatory agencies to compare treatment results with an unbiased perspective.
- Tested on up to several hundred patients affected with the targeted condition
- May take from several months to two years
- 45% of experimental treatments move on to phase 3
In a phase 3 trial, testing is broadened to many more patients, providing more data on the treatment's effectiveness, benefits, and the range of possible adverse reactions (side effects). Most phase 3 studies are both randomized and blinded.
- Tested on several hundred to several thousand affected patients
- Usually take several years
- 70% of experimental treatments move on to request approval
Once a phase 3 trial is successfully completed – that is, when the treatment demonstrates all the desired safety and efficacy goals – the company can request marketing approval from regulatory agencies. In the U.S., this involves filing a New Drug Application (NDA, used for chemically-based drugs) or Biologics License Application (BLA, used for biologic therapeutics) with the FDA. These documents contain all the scientific data compiled from all phases of the clinical trials.
The FDA or other regulatory agency typically takes from six months to up to two years to review the NDA or BLA. Once approved, the treatment can be made available to patients. However, the company or developer must continue to comply with regulatory requirements, such as reporting adverse effects of the treatment and maintaining quality control standards.
Even after a treatment is approved and on the market, regulatory agencies will often require further studies to evaluate it over the longer term. These are called post-marketing or phase 4 studies, and they have several objectives:
- Evaluating the treatment's effects in special patient populations not previously studied (for example, children or the elderly)
- Monitoring a drug's long-term effectiveness and impact on patients' quality of life
- Determining the cost-effectiveness of a drug therapy relative to other traditional and new therapies
Genzyme's Clinical Research
Find out about Genzyme's clinical research, including our approach and policies, details of ongoing and completed trials, and information on how to enroll.
Learn more at our clinical research website
Last Updated: 4/25/2014