In Establishing a Regulatory Pathway for “Biosimilar”


5 Δεκ 2012 (πριν από 5 χρόνια και 7 μήνες)

275 εμφανίσεις

A Regulatory Approval
Pathway for Biosimilars

April 2009

America’s Biopharmaceutical Companies Have Made
Significant Contributions to the Health of Americans

Companies in the vibrant biopharmaceutical sector choose to
headquarter their operations in the U.S., in part, because favorable
public policies enable their success.

Many federal and state policies help biopharmaceutical companies
achieve a greater level of innovation, including intellectual property
protections and an ability to provide returns to investors.

Emerging health threats and an aging and increasingly sicker
population drives the critical need for further innovation.

New Medicine Development is Lengthy, Costly, and Risky

The R&D process is very risky: For every 5,000 to
10,000 compounds tested, just 5 will make it to
clinical trials and, of those, only 1 will eventually
receive FDA approval.

New medicine development is a lengthy process:
The average development time has increased to
between 10 and 15 years.

R&D expenditures for each new biologic averaged
$1.24 billion in 2006.

Only 2 in 10 approved medicines bring in enough
revenue to recoup the average cost of development.

Individual company returns reflect the high risk and
long lead times inherent in drug discovery and

DiMasi, JA and Grabowski, HG.
“The Cost of Biopharmaceutical R&D: Is Biotech Different?”
Managerial and Decision Economics

79 (Jun. 2007); PhRMA.
“Drug Discovery and Development: Understanding the R&D Process.” (2007).
Pisano, GP. “Science Business

the Promise, the Reality, and the Future of Biotech.”

It is virtually impossible to

find other historical examples

[outside of the biotech sector],

at least at the industry level, for

which such a large fraction of

new entrants can be expected

to endure such prolonged periods

of losses and for which the vast

majority may never become

viable economic entities.

Gary Pisano, Harvard Business School

Biopharmaceutical R&D Creates Economic Growth and

High Value Jobs

Biopharmaceutical research companies are leaders in today’s knowledge
based economy, in which growth and productivity are driven by investments
in innovation and R&D.

Scientific & Engineering Jobs

In 2004, biopharmaceutical research
companies employed 400,000 people,
and generated economic growth
responsible for employing 2.4 million
additional people in other industries.

In the pharmaceutical industry, the
number of science and engineering
jobs increased nearly 86% from 2000

2004, compared to a 16% increase for
all manufacturing industries.

Expanding GDP by Investing in R&D

[T]he pharmaceutical and biotechnology industry,
… expands GDP by at least $27 billion annually,
on a permanent basis, for every one
time R&D
investment of $15 billion.

High Value Added per Employee

Pharmaceutical firms generated an average
$425,529 in value added per employee
compared to $130,218 for all manufacturing.


DeVol et al., “Biopharmaceutical Industry Contributions to State and U.S. Economies,” Milken Institute, October 2004;
R. Shapiro, et al.,
Economic Effects of
Intellectual Property
Intensive Manufacturing in the United States
, July 2007.

The Growing Importance of Biotechnology Medicines

Source: Biotechnology Research Continues to Bolster Arsenal Against Disease with 633 Medicines in Development. PhRMA, 2008.

Biotechnology medicines have been proven
to be safe and effective with an excellent
record of patient satisfaction and safety.

Biotechnology has produced more than 125
medicines including for some of the most
serious and intractable diseases.

In 2008, there were 633 biotechnology
medicines in development,
including 254 for
cancer and related conditions and 162 for
various infectious diseases.

Developing a biologic’s full therapeutic
potential can take time.

New treatment
advances are often realized from biologics
that have been on the market for some
time, but which were not known until
additional research was conducted.

The Complexity of Biologics Leads to Unique Challenges

for Biosimilars

Development and manufacturing process is more complex than that
for small molecule drugs:

Requires growing and harvesting the product from living cells

Can take many months to produce

Biologics are often dozens to thousands of times larger than

chemical drugs

Traditional generic drugs must be shown to be the same as the

reference drug; however, with modern science, follow
on biologics or

biosimilars can only be similar to the reference or innovator biologic

In Establishing a Regulatory Pathway for “Biosimilar” Products, It
Is Critical to Appropriately Consider Scientific and Safety Aspects

Biosimilars will be highly similar to existing, approved biologic

not the same as

Biosimilars must demonstrate safety and efficacy through clinical

America’s biopharmaceutical research companies support
the establishment of a science

approval pathway with substantial incentives for innovation
that ensures patient safety and does not hinder the
development of future medicines.

Points to Consider for Establishing a Regulatory Approval
Pathway for Biosimilars

Biologics are complex molecules with the potential for critical
medical advances.

Research and development for biologics is long, costly and risky.

A regulatory approval pathway for

must include adequate
measures for assuring patient safety.

At least 14 years of data exclusivity

or data protection must be part of

legislation to provide the certainty necessary for
continued R&D investment leading to needed medical advances.

Regulatory Process for “Biosimilars” Should Include Adequate
Measures for Assuring Patient Safety

Because follow
on biologics may be similar but not the same as the
reference product, biosimilars could have different safety and
therapeutic profiles than the innovator product.

Regulatory pathway must:

Require clinical trials to demonstrate safety and efficacy

Require clinical trials for each indication unless otherwise
scientifically justified

Recognize that current science does not permit automatic
substitution of one biologic product for another

Different biologics may have different clinical and therapeutic effects
in patients; switching should be a conscious decision made by
physicians in consultation with their patients

Innovator Products Need At Least 14 Years of Data Exclusivity

Patent protection in the context of biosimilars is less certain than for
traditional small molecule drugs.

Competitors may be able to get around patents while relying on the innovator
company’s data for FDA approval

Economic analysis of the time required for a portfolio of biologics to break
even on their R&D investment indicates data protection for biologics should
between 12.9 and 16.2 years.

Data exclusivity or data protection of at least 14 years is necessary to provide
the certainty to ensure continued R&D investment in this sector based on
economic analysis and the unique aspects of biologics, including the high
costs and time required to build specialized facilities, the higher costs of
capital and dependence on venture capital and other private sources of
capital, and the robust research and development that continues after
approval leading to medical advances in often very different disease areas.

H. Grabowski, Data Exclusivity for New Biological Entities, Duke University Economics Department Working Paper, June 2007,

Check official bios for shared affiliations.

Mention face
face encounters.

Mutual friends. (Your Government Relations team can help identify

Note personal connections

Personal testimony lifts official correspondence out of the ordinary.

It’s not about you, it’s you speaking on behalf of people you care
about (who also happen to be constituents).

Write as though you’re equipping the official to argue on your

you never know, he or she just might do it, and start a
persuasion cascade.

Explain what this means to you and your community

Writing an Effective Letter to Congress on Biosimilar Legislation

America’s biopharmaceutical companies
support health care reform that expands access
to high
quality, affordable health care for all
Americans, preserves patient and provider
choices, fights chronic disease and advances
medical innovation for the benefit of our
nation’s health and economy.