Drug Approvals

Definitions

Drugs deliver their effect through chemical means and need to be metabolized. There are two types of drugs:

Small molecule: generally taken by mouth, these drugs are made up of small organic molecules that are biologically active but not a polymer

Biological: generally delivered by injection, these include antibodies, proteins, blood, vaccines, cells, gene therapy and tissues (note that drugs must be metabolized, so some biologically-derived therapies may be classified as devices if they do not meet this criteria) 

Pathway Overview

Drugs are typically regulated via the New Drug Application (NDA) process. The process is similar for small molecules and biologicals, though biologicals have more emphasis on the manufacturing process. For both small molecules and biologicals, you'll need to submit an NDA if you:

  • Develop a new drug
  • Change the route of administration or dosage
  • Change the approved patient population
  • Change signficantly how you promote an approved drug

Note, if you are submitting a new generic molecule, then you can you use an Abbreviated New Drug Application (ANDA). ANDAs do not require clinical research. You only need to show bioequivalence to an existing approved drug.

Also, if your drug is approved outside the US, then your clinical data from outside the US can be used as part of your data submission to accelerate the approval process in the US, per the International Conference on Harmonization. Pre-approval meetings will determine suitability of this data and outline the US data required.

Approval Phases

The phases you must go through when developing a drug are well defined. While costs and timelines are variable, we have provided averages for each phase to give you a sense of a magnitude. 


Preclinical

In this phase, you will undergo preclinical bench testing for toxicology, pharmacokinetics, and pharmacodynamics. You will also prove manufacturability in cGMP or at a contract cGMP facility.

Time:
6.5 years
% Succeeding:
0.1%
% Reaching Market:
0.02%
Cost to Complete Phase:
$133 million
Cost to Market from Here:
$258 million

Investigational New Drug Application (IND)

In this phase you will prepare and submit your IND to the FDA. Once it is approved you can start your clinical trials.

Time:
30 days

Phase I Clinical Trial

In this phase you will demonstrate safety and some characteristics such as absorption, distribution, metabolism, and excretion (ADME).

Time:
1.5 years
% Succeeding:
71%
% Reaching Market:
21%
Cost to Complete Phase:
$15 million
Cost to Market from Here:
$125 million
Trial Participants:
20-100 Human Volunteers

Phase II Clinical Trial

In this phase you will determine efficacy and optimal dosing.

Time:
2 years
% Succeeding:
44%
% Reaching Market:
30%
Cost to Complete Phase:
$24 million
Cost to Market from Here:
$110 million
Trial Participants:
100-500 Human Volunteers

Phase III Clinical Trial

In this phase you will confirm effectiveness and monitor adverse events in long-term use.

Time:
3.5 years
% Succeeding:
69%
% Reaching Market:
69%
Cost to Complete Phase:
$86 million
Cost to Market from Here:
$86 million
Trial Participants:
1000-5000 Human Volunteers

New Drug Application (NDA) / Biologics License Application (BLA)

Once your clinical data is complete, you will submit your NDA or BLA to the FDA. Phase IV clinical trials may be a condition of approval, depending on the profile and target population for your drug.

Time:
1.5 years
Cost to Complete Phase:
$570,000 fee


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