FDA Drug Approval Process

Alternatives

Fast Tracking Approval and Treatment/Emergency IND

The Controlled Studies

Phases II and III contain a placebo that compares the drug to the "sugar pill" which eliminates psychological bias, and is a double blind study in which doctors nor patients know who is receiving the drug for the same reason.

Postmarketing Surveillance

An ongoing safety evaluation that examines potential for drug to treat other diseases and may be combined with other drugs for a better outcome.

FDA Purpose

Ensures that all drugs are safe and effective, and is stricter than other countries' government agencies and is harder to get drugs approved here than in Europe.

FDA Meaning

Food and Drug Administration

FDA Review and Approval

Happens after clinical trials, a NDA needs to be filed, and if the drug is determined to be safe and effected, it gets approved and the patent provides 14-20 years of exclusive rights to market the drug.

IND

Investigational New Drug Application

Treatment/Emergency IND

Makes lifesaving drugs available to critically ill patients while they are still in clinical trials/before FDA approval.

NDA

New Drug Application

Fast Tracking Approval

Once clinical trials are complete, it decreases the amount of time required for FDA approval from 2 years to 6 months.

The Statistics

Only 1/1000 compounds move on from preclinical to clinical studies, only 1/5 compounds survive clinical trials, only 1/3 approved drugs ever recover the cost of their development.

Stages of the Drug Approval Process

Preclinical Research, Clinical Studies: Phases I, II, III, FDA Review and Approval, and Postmarketing Surveillance

Strategies to Get Drugs to People With Rare Diseases

The Orphan Drug Act, which provides financial incentives (tax breaks/money for clinical trials) to companies to develop drugs for rare diseases that will not generate enough profits to cover the cost of development.

Phase III

Used for more in depth analysis of the safety and effectiveness in broader human population of diseased patients, continue to monitor side effects and determine drug-to-drug interaction; tests on humans with the disease in numbers of 1,000-30,000 who may or may not be taking multiple drugs, takes 3.5 years, and costs approximately $45 million.

Preclinical Research

Used to determine potential for compounds to treat disease with the test subject of animals, takes 5+ years although it could be upwards of 20, cost is unspecified, and then need to file an IND where FDA decided if drug is safe to test in humans.

Phase I

Used to establish initial safety in humans, tests on paid healthy human volunteers in numbers of 10-100, takes 1.5 years, and costs approximately $10 million.

Phase II

Used to evaluate the safety and effectiveness in people with the disease, to determine optimal dose, and examine the side effects; tests on humans with the disease in numbers of 100-500, takes 2 years, and costs approximately $20 million.