Drug Drug Development and Development and
DiscoveryDiscoveryAccess to Essential Medicines Access to Essential Medicines
De-CalDe-Cal
Spring 2007- Week 3Spring 2007- Week 3
RoadmapRoadmap
Evolution of the pharmaceutical Evolution of the pharmaceutical industryindustry
Research & development pipeline:Research & development pipeline: Identifying compoundsIdentifying compounds Pre-clinical and clinical trialsPre-clinical and clinical trials Production and distributionProduction and distribution Marketing and salesMarketing and sales
The reality of PharmaThe reality of Pharma
Evolution of PharmaEvolution of Pharma Previously Previously
unregulatedunregulated Huge gains in Huge gains in
basic scientific basic scientific understanding the understanding the 1950s and 60s1950s and 60s
Modern drug Modern drug industry is industry is centralized in centralized in about ten major about ten major companiescompanies
Then
Now
R&D PipelineR&D Pipeline
The Players:
Basic research institutions
Small Biotech firms
Big Pharma
Contract Research Organizations
FDA
R&D PipelineR&D Pipeline
Step 1: Basic laboratory researchStep 1: Basic laboratory research Step 2: Preclinical testing after a Step 2: Preclinical testing after a
promising compound is foundpromising compound is found Step 3: Investigational New Drug Step 3: Investigational New Drug
filing with FDAfiling with FDA Three phases of clinical trialsThree phases of clinical trials
Step 4: New Drug Application (NDA)Step 4: New Drug Application (NDA) Step 5: SaleStep 5: Sale
Clinical TrialsClinical Trials
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Phase I Phase II Phase III
Non-blind experiments for
dosing
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Single blind experiments for
efficacy
Double blind experiments for efficacy
Less of a pipeline, more Less of a pipeline, more of a funnel . . . of a funnel . . .
Production and Production and DistributionDistribution
Developing Developing production production methods and methods and creating creating facilitiesfacilities
DistributionDistribution Enormous Enormous
marketing marketing expendituresexpenditures
Biologics- What are Biologics- What are they?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 . . .And that means . . .
Traditional organic compound = Relatively
simple
Biologic compound = Significantly more
complex
Thalidomide
Hemoglobin
The $800 million pill?The $800 million pill?
Important conceptsImportant concepts
Generic drug manufacturersGeneric drug manufacturers New Molecular Entities (NMEs)New Molecular Entities (NMEs) BioequivalencyBioequivalency
Me-too drugsMe-too drugs Lifestyle drugsLifestyle drugs Second indicationsSecond indications Pharma companies are ruthless Pharma companies are ruthless
profit maximizersprofit maximizers
The Politics of PharmaThe Politics of Pharma
Organized into PhRMA Organized into PhRMA (Pharmaceutical Research and (Pharmaceutical Research and Manufacturers of AmericaManufacturers of America
Huge political lobby:Huge political lobby: In 2001 Name-brand pharma spent $75.4 In 2001 Name-brand pharma spent $75.4
million on lobbyingmillion on lobbying That same year generics spent $2.4 millionThat same year generics spent $2.4 million
The providers for a society that The providers for a society that increasingly relies on drugsincreasingly relies on drugs
The GoodThe Good
In 2004 name-brand pharmaceutical In 2004 name-brand pharmaceutical firms spent approximately $53 firms spent approximately $53 billion on R&Dbillion on R&D
New medicines generated 40% of New medicines generated 40% of the two-year life expectancy gain in the two-year life expectancy gain in 52 developed countries52 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 BadThe Bad In 2001 an estimated 1.9 to 2.2 In 2001 an estimated 1.9 to 2.2
million Americans were driven in million Americans were driven in personal bankruptcy by health care personal bankruptcy by health care costscosts**
In 2004 pharma spent approximately In 2004 pharma spent approximately $1.25 billion per NME; in 1995 that $1.25 billion per NME; in 1995 that number was $317 millionnumber 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 QuestionableThe Questionable
Decline in the efficiency of R&D (Decline in the efficiency of R&D (i.e.i.e. less NMEs per R&D $ spent)less NMEs per R&D $ spent)
Mergers of big companies hurting Mergers of big companies hurting R&DR&D
The cost of marketing of The cost of marketing of pharmaceuticalspharmaceuticals
Direct to consumer advertisingDirect to consumer advertising The pipeline isn’t sustainableThe pipeline isn’t sustainableArrr! That be questionable,
matey
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