Pharmacogenomics: The Future of Pharmacy

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Pharmacogenomics The Future of Pharmacy

• Alan McKay, PhD.
• Professor and Dean

AMCP Southeastern Leadership Retreat (November 5,
2006) http//pharmacy.su.edu/AMCP/(pharmacogenomic
sAMCP.ppt)
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Popular Headlines
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A look back and a glimpse of the future

• Biopharmaceutical Marketplace
• Pharmaceutical pipeline
• Biotechnology versus Traditional Pharmaceutical
• Pharmacogenomics
• Lactose Intolerance
• Genetic Testing
• Herceptin the magic bullet for cancer
• New definitions of a drug
• Specialty Pharmacy
• Pharmaceutical pipeline
• Projected growth
• Evolution of specialty pharmacy from infusion to
  biotech
• New market realities

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Biotechnology Impact - USA

• 418 Biotech drug products and vaccines currently
  in clinical trials targeting over 200 diseases
• 30 of all new medicines undergoing clinical
  trials and 80 of entities in the RD pipeline
  are from biotechnology
• Most compounds are focused on oncology,
  infectious disease, CV and neuro

Source Bibby, K., Davis, J. Jones, C.,
Biopharmaceuticals Moving to Centre Stage,
BioPeople, North American Biotechnology Industry
and Suppliers Guide (2003)
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Biotechnology Medicines Under Development (2006)
Source Biotechnology Medicines in Development
2006 PhRMA Report
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Approved Biotech Drugs and Vaccines
Source BIO 2005-2006 Guide to Biotechnology,
Biotechnology Industry Organization
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Industry Segmentation

• Pharmaceuticals
• Very large, multi-national firms
• Top 10 average 15 billion in sales
• Major assets are products, distribution and
  manufacturing expertise
• Very profitable ( 12 19 ROI)

• Biotechnology
• Small, mostly single product firms
• Top ten average 700 million in sales
• Major assets are intellectual property, nimble
  research teams and loads of potential
• Lose money high venture capital burn rate

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Pharmacogenomics

• The study of how a patients genetic makeup
• affects their response to drugs.

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How can we relate to a concept that most of us
cant even pronounce - Pharmacogenomics

• Lactose Intolerance
• Genetic Testing
• Herceptin the magic bullet for cancer

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Lactose Intolerance

• 50 million Americans are lactose intolerant
• Lactose intolerance is genetic in origin
• Most people of European origin are tolerant
• Most people of African or Asian origin are
  intolerant (75 98)
• American Indians are almost 100 intolerant
• Lactose intolerance is the normal condition

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Genetic Testing - DNA Home Test Kits

• Cystic Fibrosis
• Alzheimers Disease
• Colon Cancer
• BRCA 1 and 2 genes associated with increased
  risk of breast and ovarian cancer

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Genetic Tests in Cancer
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Genetic Testing new frontiers

• 15 year old boy conceived through in-vitro
  fertilization sought information on his
  biological father by submitting a DNA sample to a
  genealogical database ( 295)
• Nine months later he received two hits
• With information provided by his mother on the
  sperm donors birth date and birthplace the boy
  sought the services of an online locator service
  (DNA hit birth date)
• Ten days later he contacted his biological
  father there was no report on the fathers
  reaction

Source Washington Post (November 13, 2005)
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Herceptin the magic bullet
Overall, Herceptin boosted the chances of
disease-free survival by 52 percent compared
with the control group. Forbes
Magazine October 19, 2005
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Evolution of the Pharmacogenomics

• 2000 First draft of human genome sequence
  published
• 2000 2005 Improved use of existing drugs (ADRs
  and Toxicity)
• 2006-2010 Identification of biomarkers
  associated with patient response to drugs
• Development of diagnostics for genetic-based
  disease
• 2011 2015 Creation of a new generation of
  biopharmaceuticals
• gt 2016 Personalized medicine

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Drug and Diagnostic

• Herceptin (Genetech) was the first biologic that
  required approval of both the drug and the
  diagnostic
• FDA has recently announced their intent to
  regulate gene- and protein-based tests (in vitro
  diagnostic multivariate index assays)
• A major step in the direction of requiring
  manufacturers to include genetic screening data
  even if the company does not want to market the
  diagnostic device

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Specialty Pharmacy

• High unit cost (gt 5,000/pt/year)
• Complex care
• Injectables
• Storage and inventory control issues
• Close clinical supervision required to achieve
  desired outcome

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Pharmaceutical Pipeline (2006)
Source RxPipeline Insider (Feb. 2006)
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Specialty Pharmacy Growth (2009)
Source Spending on Specialty Drugs on the Rise,
Expected to Double by 2009, Express Scripts
Says Medical News Today (10 June 2006)
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Specialty Pharmacy - Disease States

• Share Growth
• Cancer 35 20
• HIV/AIDS 15 10
• Transplant 9 na
• Hepatitis 7 30
• Hemophila 6 10
• Infertility 5 20
• Multiple Sclerosis 5 15
• Other diseases 18 19

Source Dean Erhardt, Specialty Pharmacy in
Community Pharmacy the time is now and how,
NACDS Pharmacy Technology conference, San
Diego, CA August 2006
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Evolution of Specialty Drugs

• Historically treated rare genetic diseases
• Growth has been into chronic medications
• 60 of biotechs nearing market are for chronic
  conditions
• Excluding chemotherapy most specialty drugs today
  are self-administered new biotech drugs require
  intravenous admin.

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Gene Mutation May Play Role in Crohns Disease
An international scientific team has pinpointed
an unexpected gene mutation that appears to
confer protection against Crohns disease in some
patients. The finding potentially paves the way
for new diagnostic tools and drugs to treat the
debilitating intestinal condition, which affects
more than 500,000 people in the
U.S. Michael Stroh The Baltimore
Sun October 2006
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Business Model

• Limited distribution with special storage
• Exclusive contracts
• Tight inventory control including shipping
• Clinical pharmacy oversight to insure adherence
• Patient education
• Patient monitoring
• Utilization review

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Source Preventing Medication Errors,
Pre-publication copy, Institute of Medicine, p.63
(2006)
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Management Challenges

• Growth of biopharmaceuticals
• Life threatening diseases become chronic
• Targeted interventions
• Pressure to demonstrate both economic and
  clinical value pharmacoeconomics
• Documentation of outcomes (effectiveness and
  efficacy)

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Management Strategies

• High out-of-pocket expense by patient (20 40)
• Prior authorization to encourage adherence to
  evidence-based guidelines
• Move patients from medical benefit (buy and bill)
  to pharmaceutical benefit (20 30 difference)
• Shift distribution to specialty pharmacy networks
• Vertical integration (PBM Wholesaler Network)
• Pay for performance

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Intersection of Pharmacogenomics and Specialty
Pharmacy

• Drug targets a life-threatening disease for which
  there are few treatment alternatives
• Clinical trials demonstrate a clear survival
  benefit
• Clear relationship between a biomarker and drug
  response
• An affordable diagnostic test exists

Source Phillips, K.A., The Intersection of
Biotechnology and Pharmacogenomics Health Policy
Implications Health Affairs 25(5) 1271-1280
(Sep./Oct. 2006).
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Predictions

• Pharmacogenomics will move into the mainstream of
  education and practice
• Clinical trials will adjust to different
  parameters (FDA Critical Pathway)
• Drug industry will adjust to new market realities
  (niche busters)
• Medicine will adopt new diagnostic and data
  procedures focused on genetic patient profile
• Specialty pharmacy will move from relatively
  small patient populations to a much wider
  percentage of diseases and to a broader
  representation of practice

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Pharmacogenomics
Pharmacogenomics holds great promise to shed
scientific light on the often risky and costly
process of drug development, and to provide
greater confidence about the risks and benefits
of drugs in specific populations.
Mark B. McClellan, M.D., PhD. FDA
Commissioner November 3, 2003
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Resources

• Personalized Medicine Coalition
  (www.personalizedmedicinecoalition.org)
• BIO Biotechnology Industry Organization
  (www.bio.org)
• Hampton, T., Researchers Draft Guidelines for
  Clinical Use of Pharmacogenomics, JAMA 296(12)
  1453-1454 (September 27, 2006).
• Biotech 2006 Life Sciences A Changing
  Prescription, Burrill Company (March 26, 2006)
• Namovicz-Peat, S., Specialty Pharmacy
  Stakeholders, Strategies and Markets Third
  Edition, 2007
• NACDS, Biopharmaceuticals in Pharmacy Care
  Positioning Community Pharmacy for Future Patient
  Services, Philadelphia, PA (Oct. 19 20, 2006)