Download Drug Development Pipeline

Drug Development
and Discovery
Access to Essential Medicines De-Cal
Spring 2007- Week 3
Roadmap
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Evolution of the pharmaceutical industry
Research & development pipeline:
Identifying compounds
 Pre-clinical and clinical trials
 Production and distribution
 Marketing and sales
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
The reality of Pharma
Evolution of Pharma

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Previously
unregulated
Huge gains in basic
scientific
understanding the
1950s and 60s
Modern drug industry
is centralized in about
ten major companies
Then
Now
R&D Pipeline
The Players:
Basic research
institutions
Contract
Research
Organizations
FDA
Small Biotech
firms
Big Pharma
R&D Pipeline
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Step 1: Basic laboratory research
Step 2: Preclinical testing after a promising
compound is found
Step 3: Investigational New Drug filing with
FDA
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Three phases of clinical trials
Step 4: New Drug Application (NDA)
Step 5: Sale
Clinical Trials
Phase I
Phase II
Phase III
Non-blind
experiments for
dosing
Single blind
experiments for
efficacy
Double blind
experiments
for efficacy
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Less of a pipeline, more of a
funnel . . .
Production and Distribution
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Developing
production
methods and
creating facilities
Distribution
Enormous
marketing
expenditures
Biologics- What are they?
Biological products include a wide range of products such as
vaccines, blood and blood components, allergenics, somatic
cells, gene therapy, tissues, and recombinant therapeutic
proteins. Biologics can be composed of sugars, proteins, or
nucleic acids or complex combinations of these substances,
or may be living entities such as cells and tissues. Biologics
are isolated from a variety of natural sources - human,
animal, or microorganism - and may be produced by
biotechnology methods and other cutting-edge technologies.
Gene-based and cellular biologics, for example, often are at
the forefront of biomedical research, and may be used to treat
a variety of medical conditions for which no other treatments
are available.
And that means . . .
Traditional organic
compound = Relatively
simple
Biologic compound =
Significantly more
complex
Thalidomide
Hemoglobin
The $800 million pill?
Important concepts

Generic drug manufacturers
New Molecular Entities (NMEs)
 Bioequivalency
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Me-too drugs
Lifestyle drugs
Second indications
Pharma companies are ruthless profit
maximizers
The Politics of Pharma
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Organized into PhRMA (Pharmaceutical
Research and Manufacturers of America
Huge political lobby:
In 2001 Name-brand pharma spent $75.4
million on lobbying
 That same year generics spent $2.4 million
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The providers for a society that
increasingly relies on drugs
The Good

In 2004 name-brand pharmaceutical firms
spent approximately $53 billion on R&D

New medicines generated 40% of the twoyear life expectancy gain in 52 developed
countries*
* F.R. Lichtenberg, The Impact of New Drug Launches on Longevity, National Bureau of
Economics Research Working Paper No. 9754 (June 2003).
The Bad

In 2001 an estimated 1.9 to 2.2 million
Americans were driven in personal
bankruptcy by health care costs*

In 2004 pharma spent approximately
$1.25 billion per NME; in 1995 that
number was $317 million+
* Health care costs main cause of bankruptcy, study finds, The New Standard, at
http://newstandardnews.net/content/?action=show_item&itemid=1439
+R&D spending per NME Declines, Drugresearcher.com, at
http://www.drugresearcher.com/news/ng.asp?id=61610-r-d-nme-spend
The Questionable
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Decline in the efficiency of R&D (i.e. less
NMEs per R&D $ spent)
Mergers of big companies hurting R&D
The cost of marketing of pharmaceuticals
Direct to consumer advertising
The pipeline isn’t sustainable
Arrr! That be questionable,
matey