Personalized Medicine  A New Treatment Paradigm

Personalized Medicine A New Treatment Paradigm

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Description: What is Personalized Medicine? Personalized medicine, sometimes referred to as precision or individualized medicine, is an emerging field of medicine that uses diagnostic tools to identify specific biological markers, often genetic, to help assess which medical treatments and procedures will be best for each patient. Personalized medicines are benefiting patients across many different diseases. Cancer is on the leading edge of personalized medicine.

Advances in personalized medicine improve the outlook for patients with blood cancers.

 
Author: Emma Van Hook  | Visits: 284 | Page Views: 457
Domain:  Medicine Category: Biotech/Pharma 
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Contents:
Personalized Medicine
A New Treatment Paradigm
Emma Van Hook, Director, Policy & Research

What is Personalized Medicine?

Personalized medicine, sometimes referred to as
precision or individualized medicine, is an emerging
field of medicine that uses diagnostic tools to identify
specific biological markers, often genetic, to help assess
which medical treatments and procedures will be best
for each patient.

Why is Personalized Medicine so Important?

A New Treatment Paradigm
Without Personalized Medicine:

With Personalized Medicine:

Some Benefit, Some Do Not

Each Patient Receives the Right Medicine For Them

Patients

Patients

Biomarker
Diagnostics
Therapy
Therapy

Benefit

No benefit

Adverse
effects

Each Patient Benefits From
Individualized Treatment

Personalized Medicines Are Benefiting
Patients Across Many Different Diseases
FDA Approvals with Biomarker information in the approved labeling
Rheumatology
4%
Gastroentrology
5%

Other
7%

Neurology
6%

Hematology/
Oncology
38%

Endocrinology
6%
Cardiology
7%
Infectious
Diseases
10%

Psychiatry
17%

Source: U.S. FDA, “Paving the Way for Personalized Medicine,”
http://www.fda.gov/downloads/ScienceResearch/SpecialTopics/PersonalizedMedicine/UCM372421.pdf, Oct 2013.

Pop Quiz!
Q: What was the first
personalized medicine?

Pop Quiz!
Q: What was the first
personalized medicine?

A: Herceptin, first
approved in 1998 to treat
HER2-positive breast
cancer.

“Development and approval of Herceptin marked the dawn of a new era of cancer treatment by
bringing an emerging understanding of cancer genetics out of the laboratory and to the patient’s
bedside. The story of Herceptin also emphasized a profound lesson: not all cancers are the same.
Breast cancer – as well as other cancers – cannot be viewed as a single disease, but rather as a
group of several subtypes, each with its distinct molecular signature.”

Source: U.S. FDA, “Paving the Way for Personalized Medicine,”
http://www.fda.gov/downloads/ScienceResearch/SpecialTopics/PersonalizedMedicine/UCM372421.pdf, Oct 2013.

Molecular Sub-typing in Breast Cancer

Cancer is on the Leading Edge of
Personalized Medicine
Breakdown of Oncology Treatment Modalities,
Global Market share 2003-2013*
2003

2013

11%
24%
48%

26%

15%
Targeted

Cytotoxics

46%
Targeted
Medicines

10%

46%
20%

Supportive Care

Hormonals

*Definitions: Targeted therapies - identify and attack specific types of cancer cells with less harm to normal cells; Cytotoxics – agents that kill rapidly
developing cells (as in chemotherapy); Supportive care - care given to improve quality of life by preventing or treating the symptoms of a disease or
the effects of its treatment; Hormonals - treatments that add, block, or remove hormones to slow or stop the growth of certain cancers.

Sources: IMS Institute for Healthcare Informatics, “Innovation in Cancer Care and Implications for Health Systems: Global Oncology Trend Report,” May 2014
(accessed May 2015); National Cancer Institute, “NCI Dictionary of Cancer Terms” (accessed May 2015).

Advances in Personalized Medicine Improve
Outlook for Patients with Blood Cancers
A greater understanding of the molecular basis of disease has transformed what was once known
collectively as “disease of the blood,” into multiple subtypes of leukemias and lymphomas.

60 YEARS
AGO

50 YEARS
AGO

40 YEARS
AGO
Chronic
Leukemia

Leukemia

Acute
Leukemia
Preleukemia

“Disease of
the Blood”

Lymphoma

Indolent
Lymphoma

TODAY
~ 40 Unique
Leukemia

5 year survival

types
identified

70%

~ 50 Unique
Lymphoma
types
identified

Aggressive
Lymphoma
Source: M Aspinal, former President Genzyme Genetics (cited at
http://www.comtecmed.com/biomarker/2014/Uploads/Editor/PDF/ppt/Edward%20Abrahams_Key%20Note%20Lecture.pdf)l; National Cancer Institute,; SEER
Cancer Statistics Review, 1975-2011, http://seer.cancer.gov/csr/1975_2011/, based on November 2013 SEER data submission, posted to the SEER web site, April
2014; PhRMA, “Medicines in Development for Leukemia & Lymphoma,” April 2015 (all cites accessed May 2015).

rates have
grown to

There are nearly

250

medicines
in development
for blood
cancers

Personalized Medicine Can Create
Efficiencies in the Health Care System
Breast Cancer

34%

Reduction in chemotherapy
use would occur
If women with breast cancer
receive a genetic test of their tumor
prior to treatment

Metastatic
Colorectal Cancer

$

Stroke

$604 Mil

17,000

In annual health care cost
savings would be realized

Strokes could be
prevented each year

If patients with metastatic colorectal
cancer receive a genetic test for the
KRAS gene prior to treatment

If a genetic test is used to properly
dose blood thinners

Sources: Personalized Medicine Coalition, “Personalized Medicine by the Numbers,” 2014 (accessed Oct 2016).

Pop Quiz!
Q: How many personalized
medicines are available
today?

Pop Quiz!
Q: How many personalized A: More than 140!
medicines are available
today?

Source: U.S. FDA, “Paving the Way for Personalized Medicine,”
http://www.fda.gov/downloads/ScienceResearch/SpecialTopics/PersonalizedMedicine/UCM372421.pdf, Oct 2013.

2015: Banner Year for Personalized
Medicine
2015 Personalized Medicine Highlights:
• 35% of new cancer treatments were
found to be personalized medicines,
including:
• Two new medicines for patients
with different forms of non-small
cell lung cancer
• A new targeted therapy for
melanoma
• A new combination therapy for
patients with cystic fibrosis
• Two new medicines to help patients
with a difficult-to-treat form of high
cholesterol

Source: U.S. FDA, “Table of Pharmacogenomic Biomarkers in Drug Labeling,”
http://www.fda.gov/drugs/scienceresearch/researchareas/pharmacogenetics/ucm083378.htm (accessed May 2016), PMC , “2015 Progress Report: Personalized Medicine
at FDA,” http://www.personalizedmedicinecoalition.org/Userfiles/PMC-Corporate/file/2015_Progress_Report_PM_at_FDA1.pdf.

Where have we seen personalized
medicine lately?

Researchers Have Made Great Progress but
Challenges Remain
We now know that cancer is not a single disease, but rather more than 200 unique diseases, many of
which are caused by genetic mutations. Identifying these mutations has led to tremendous advances
against many cancers, but the complexity of each disease presents great challenges for researchers, as
they explore still yet unknown alterations.

Selected Genomic Alterations Known to Drive Disease Progression
in Common Cancers
Lung Adenocarcinoma
RET
ROS
NRAS
MEK1
ERBB2
MET
PIK3CA
BRAF

Breast Cancer

Unknown
ERBB2

Unknown

ALK

EGFR
PK3CA

FGFRI amp

PTEN

KRAS
AKT

Sources: LA Garraway, “Genomics-Driven Oncology: Framework for an Emerging Paradigm,” J Clin Onc. 2013; 31(15):1806-1814; American Society of Clinical Oncology,
“Studies Reveal Potential New Targeted Therapies for Common, Hard-to-Treat Cancers,” http://www.asco.org/press-center/studies-reveal-potential-new-targetedtherapies-common-hard-treat-cancers, May 2014 (accessed May 2015).

Pop Quiz!
Q: What portion of the
pipeline has the potential
to be personalized
medicines?

Pop Quiz!
Q: What portion of the
pipeline has the potential
to be personalized
medicines?

A: 42%!

Source: PhRMA Innovation Hub, “Biopharma Companies Are Driving Personalized Medicine Forward,”
http://innovation.org/gallery/biopharma-companies-are-driving-personalized-medicine-forward.

The Biopharmaceutical Industry is
Committed to Personalized Medicine

Source: PhRMA Innovation Hub, “Biopharma Companies Are Driving Personalized Medicine Forward,”
http://innovation.org/gallery/biopharma-companies-are-driving-personalized-medicine-forward.

Ensuring Future Innovation in Personalized
Medicine
At a time when the scientific promise is greater than ever, thoughtful policies are necessary to
accelerate advances in targeted therapy for patients.

Discovery

Development

Delivery

 Align regulatory policy with rapid pace of science
 Ensure that payment and delivery models recognize patients’ differences, both
scientifically and in terms of their own preferences

Resources
• Innovation Hub
– www.innovation.org

• PhRMA Personalized Medicine Chartpack
– http://chartpack.phrma.org/personal-medicines-in-development-chartpack

• Personalized Medicine Coalition
– http://personalizedmedicinecoalition.org/)
Emma Van Hook
Director, Policy & Research, PhRMA
EVanHook@phrma.org
@EmmaHokie

The Impact of Genetics:
Beyond the Bench and Bedside

https://youtu.be/6ymoPRWxZl8

Personalized Medicine in Practice
A mini case study.

Personalized Medicine in Practice
Scientific Name Crustulumitis
Symptoms

Irritability, abdominal cramping, growling stomach

Causes

May flare up between meals, and often following a
salty or savory meal. Symptoms may also be more
pronounced during times of increased stress or
emotional instability.

Risk Factors

Similar to irritable bowel syndrome (IBS), many
people experience symptoms of mild Crustulumitis
as a result of inconsistent daily dietary habits or as
a response to stressful or emotional situations.

Personalized Medicine in Practice
Scientific Name Crustulumitis
Diagnosis

Crustulumitis can often be detected by conducting
a thorough Q&A assessment with a
knowledgeable professional. Verbal confirmation
of symptoms often can indicate the level of
severity of a case of Crustulumitis, though it can
sometimes be confused with Glacies crepitosis and
Fructusitis.

Treatment

While mild Crustulumitis may be treated by
preliminary administration of easily available oral
glucose supplements, a simple lab test can be
conducted on any available deoxyribnucleic acid
sample, which will indicate the mechanism of
treatment most likely to relieve symptoms for
patients.

Results
Lab result
CHO
Regular, wild type
(55% of patients)

CHO+
Gene amplification
(30% of patients)

CHOGene deletion
(10% of patients)

CHO~
Missense mutation
(5% of patients)

Results
Lab result

Treatment

CHO

Oatmeal chocolate chip
cookie

Regular, wild type
(55% of patients)

CHO+
Gene amplification
(30% of patients)

CHO-

Chocolate brownie
cookie
Sugar cookie

Gene deletion
(10% of patients)

CHO~

Carrots

Missense mutation
(5% of patients)
*1 to 3 doses, depending on the severity of the flare-up.
FYI: Crustulumitis means imaginary cookie disease in Latin. Glacies crepitosis is ice cream disease and
Fructusitis is fruit disease.