How are drugs and vaccines discovered and developed?. Dr. Jeffrey L. Sturchio President & CEO, Global Health Council Journalist-to-Journalist Program National Press Foundation Cape Town, South Africa July 18, 2009 . Agenda. 22 years of innovation in HIV/AIDS treatment
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Dr. Jeffrey L. Sturchio
President & CEO, Global Health Council
National Press Foundation
Cape Town, South Africa
July 18, 2009
Kilby J and Eron J. N Engl J Med 2003;348:2228-2238
In vitro Pharmacology
In vivo Pharmacology
StatisticsScientific disciplines and development capabilities needed
Scale-Up to Mfg.
~ 5,000 – 10,000
ONE FDA-APPROVED DRUG
NUMBER OF VOLUNTEERS
0.5 – 2 YEARS
3 – 6 YEARS
6 – 7 YEARSDrug discovery and development is difficult and requires significant infrastructure and resources
Developing a new medicine takes an average of 10–15 years; the Congressional Budget Office reports that “relatively few drugs survive the clinical trial process”
Sources: Drug Discovery and Development: Understanding the R&D Process, www.innovation.org; CBO, Research and Development in the Pharmaceutical Industry, 2006.
During the last decade clinical trial designs andprocedures have become much more complex, demanding more staff time and effort, and discouraging patient-enrollment and retention
Procedures: include lab & blood work, routine exams, x-rays & imaging, questionnaire & subjective assessments, invasive procedures, heart assessment, etc.
Protocol: the clinical-trial design plan
Enrollment rate: the percentage of volunteers meeting the increasing number of protocol eligibility criteria (percentage screened who were then enrolled)
Retention rates: the percentage of volunteers enrolled who then completed the study; declining retention rates mean that firms must enroll more patients initially and/or recruit more patients during the trial.
Source: Tufts Center for the Study of Drug Development, “Growing Protocol Design Complexity Stresses Investigators, Volunteers,” Impact Report, 2008.
Average R&D Investment ($202 MM)
Cumulative Returns Cover R&D investment in Year 16
Source: Grabowski and Vernon. “Effective Patent Life in Pharmaceuticals.” Forthcoming in The Journalof International Technology Assessment, 1999.
Lifetime Sales Compared to Average R&D Costs
After-Tax Present Value of Sales
(Millions of 2000 Dollars)
New Rx Drugs Introduced Between 1990 and 1994, Grouped by Tenths, by Lifetime Sales
Note: Drug development costs represent after-tax out-of-pocket costs in 2000 dollars for drugs introduced from 1990–94. The same analysis found that the total cost of developing a new drug was $1.3 billion in 2006. Average R&D Costs include the cost of the approved medicines as well as those that fail to reach approval.
Sources: J. Vernon et al., “Drug Development Costs when Financial Risk is Measured Using the Fama-French Three Factor Model,” Unpublished Working Paper, 2008; J. DiMasi and H. Grabowski, “The Cost of Biopharmaceutical R&D: Is Biotech Different?,” Managerial and Decision Economics, 2007.
No. of NMEs Approved
$ Billions R&D Spend
*NME (new molecular entity) total is through August 22, 2001. R&D spend for 2000
and 2001 are estimates. Source: Washington Analysis, LLC and PhRMA
Dealing with stochastic failures is one of the primary drivers in escalating costs of R&D to the current estimate of ~ US$1 BB to bring a drug to market
How to Control
Basic ResearchFederal and Industry Roles in Research and Development
Government and biopharmaceutical industry research are complementary
Private Sector – $65.2B1
There is an ecosystem of science and biotechnology. Public organizations, patient organizations, universities, Congress, FDA, all of this is an ecosystem that is envied in the rest of the world.
– E. Zerhouni, Director of NIH
NIH3– $29.4B total
– $20.1B research
Sources: 1Burrill & Company, analysis for PhRMA, 2005–2009 (Includes PhRMA research associates and nonmembers) in PhRMA, “Profile 2008, Pharmaceutical Industry;” PhRMA, “PhRMA Annual Membership Survey,” 1996-2009; 2Adapted from E. Zerhouni, Presentation at Transforming Health: Fulfilling the Promise of Research, 2007; 3NIH Office of the Budget, “FY 2009 President’s Budget Request Tabular Data”, http://officeofbudget.od.nih.gov/ui/2008/tabular%20data.pdf
Synthesis Examination & Screening