Page 22 - 2021 Spring CMTA Report - Special Research Edition

Clinical Trials — how the data will be reviewed and questions based on existing data.
In other cases, the FDA requires
analyzed. Clinical trials follow a stan-
The Road to dard progression, starting with early, additional studies. If the NDA doesn’t
small-scale Phase 1 studies lasting
contain sufficient data for the FDA
several months and involving 20 to
to determine the safety and effec-
Drug Approval 100 volunteers with the disease. tiveness of a drug, it may organize a
meeting of one of its Advisory
Phase 1 studies are designed to
assess safety and dosage. Committees to get independent,
The Food and Drug Administration’s Approximately 70 percent of drugs expert advice and to permit the public
drug approval process involves five move on to the next phase. Phase 2 to make comments. These Advisory
basic steps. The discovery/concept studies have up to several hundred Committees include a patient
phase comes first, as research for people with the disease/condition and representative who provides input
a new drug or device begins in the can last from several months to two from the patient perspective.
laboratory. In Step 2, the pre-clinical years. Their purpose is to examine the In cases involving serious conditions
phase, drugs and devices undergo drug or device’s efficacy and side ef- with unmet medical needs, the FDA
laboratory and animal testing to an- fects. Some 33 percent of drugs move can “fast track” the drug approval
swer basic questions about safety. on to the next phase. process in order to get important new
Clinical trials follow, then FDA review Phase 3 trials look at the drug’s drugs to patients sooner. Once a drug
and finally FDA Post-Market Safety efficacy and monitor subjects for receives Fast Track designation, early
Monitoring. Clinical trials are simply adverse reactions. Some 300 to and frequent communication between
studies, or trials, in humans. They 3,000 volunteers with the disease the FDA and a drug company is en-
occur only after researchers (or devel- participate in Phase 3 studies, which couraged throughout the entire drug
opers) complete the FDA’s Investiga- last from one to four years, with 25 development and review process. The
tional New Drug (IND) process, which to 30 percent of drugs moving on frequency of communication assures
requires them to submit animal study to Phase 4. Several thousand volun- that questions and issues are resolved
data and toxicity data, manufacturing teers with the disease take part in quickly, often leading to earlier drug
information, clinical protocols (study the Phase 4 study, which looks at the approval and access by patients.
plans) for studies to be conducted, drug’s safety and efficacy.
data from any prior human research Once the FDA receives a New Drug
and information about the investigator. Application (NDA), the review team
While preclinical research answers decides if it is complete. If not, the
basic questions about a drug’s safety, review team can refuse to file the
clinical trials study the ways the drug NDA. If it is complete, the review
will interact with the human body. team has six to 10 months to make a
They are designed to answer specific decision on whether to approve the
research questions related to a drug.
medical product and follow a specific If the FDA determines that a drug
study plan, or protocol, developed by has proved safe and effective for its
the researcher or manufacturer. intended use, it works with the
Before a clinical trial begins, research- applicant to develop and refine
ers review existing information about prescribing information, or “labeling.”
the drug, then decide who qualifies Labeling accurately and objectively
to participate (selection criteria), how describes the basis for approval and
many people will participate, how how best to use the drug.
long the study will last, whether there In many cases, issues remain to be
will be a control group, how the drug resolved before the drug is approved
will be given to patients and at what for marketing. Sometimes the FDA
dosage, how to assess the results and requires the developer to address

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