ARRRR! Drug Development and Discovery

1
ARRRR! Drug Development and Discovery

• Access to Essential Medicines De-Cal
• Fall 2006- Week 2

2
Happy International Talk Like a Pirate Day!
Drug companies make medicines that cost lots o'
doubloons My favorite pirate pick up line
Avast, me proud beauty! Wanna know why my Roger
is so Jolly?
3
Roadmap

• Evolution of the pharmaceutical industry
• Research development pipeline
• Identifying compounds
• Pre-clinical and clinical trials
• Production and distribution
• Marketing and sales
• The reality of Pharma

4
Evolution of Pharma

• Previously unregulated
• Huge gains in basic scientific understanding the
  1950s and 60s
• Modern drug industry is centralized in about ten
  major companies

Then
Now
5
RD Pipeline
The Players
Contract Research Organizations
Basic research institutions
FDA
Small Biotech firms
Big Pharma
6
RD Pipeline

• Step 1 Basic laboratory research
• Step 2 Preclinical testing after a promising
  compound is found
• Step 3 Investigational New Drug filing with FDA
• Three phases of clinical trials
• Step 4 New Drug Application (NDA)
• Step 5 Sale

7
Clinical Trials
Phase I
Phase II
Phase III
Non-blind experiments for dosing
Single blind experiments for efficacy
Double blind experiments for efficacy
8
Less of a pipeline, more of a funnel . . .
9
Production and Distribution

• Developing production methods and creating
  facilities
• Distribution
• Enormous marketing expenditures

10
The 800 million pill?
11
Important concepts

• Generic drug manufacturers
• New Molecular Entities (NMEs)
• Bioequivalency
• Me-too drugs
• Lifestyle drugs
• Second indications
• Pharma companies are ruthless profit maximizers

12
The Politics of Pharma

• 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
• The providers for a society that increasingly
  relies on drugs

13
The Good

• In 2004 name-brand pharmaceutical firms spent
  approximately 53 billion on RD
• New medicines generated 40 of the two-year 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).
14
The Bad

• In 2001 an estimated 1.9 to 2.2 million people
  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/?actionshow_it
emitemid1439 RD spending per NME Declines,
Drugresearcher.com, at http//www.drugresearcher.c
om/news/ng.asp?id61610-r-d-nme-spend
15
The Questionable

• Decline in the efficiency of RD (i.e. less NMEs
  per RD spent)
• Mergers of big companies hurting RD
• The cost of marketing of pharmaceuticals
• Direct to consumer advertising
• Me too and lifestyle drugs

Arrr! That be questionable, matey