Biotechnology Industry Organization
1625 K Street, Suite 1100, N.W.
Washington, D.C. 20006
Testimony of
Charles A. Johnson, MBChB
Associate Director of Specialty Biotherapeutics
Genentech, Inc.
On Behalf of the
BIOTECHNOLOGY INDUSTRY ORGANIZATION
(BIO)
Before The
Senate Committee on
Health, Education, Labor and Pensions
April 23, 2002
Good morning, Mr. Chairman and Members of the Committee.
My name is Dr. Charles Johnson. I am Associate Director of Specialty
Biotherapeutics at Genentech, Inc., a leading biotechnology company
headquartered in South San Francisco, California. I am here today
representing the Biotechnology Industry Organization (BIO). BIO
represents more than 1000 biotechnology companies, academic
institutions and state biotechnology centers in all 50 U.S. states and 33
other nations. BIO’s members are involved in the research and
development of medical, agricultural, industrial and environmental
biotechnology products.
Most of the hard work in our industry is directed toward research on
currently unmet medical needs: new therapies and cures for various
cancers, Alzheimer’s and Parkinson’s diseases, diabetes, heart disease
and hundreds of other debilitating and life-threatening illnesses.
Thank you, Mr. Chairman, for holding this hearing on such an
important issue: how to effectively protect those who voluntarily participate
in our research while, at the same time, facilitating critical medical
research. As you and your colleagues examine this issue, I urge you to
remember two critical facts.
First, participants in research are volunteers, meaning that we must
do all we can to ensure that they have the utmost confidence that they will
be protected.
Second, medical research has and will continue to lead to cures and
treatments for millions of Americans suffering from diseases. One hundred
seventeen biotechnology products have helped a quarter billion people
worldwide thus far, and another 350 biotech medicines targeting more than
250 diseases are in late stage development. Many of these are diseases
that are currently incurable.
Much attention has been given lately to issues surrounding the
protection of the volunteers who participate in our research. As you are
already aware, Mr. Chairman, medical research is a heavily regulated
activity – our products and manufacturing processes are regulated by the
Food and Drug Administration (FDA), and our research protocols are
reviewed and scrutinized by Institutional Review Boards (IRBs) under an
extensive set of federal regulations governing research (the federal
Common Rule). Moreover, virtually all states have developed regulations
that affect research. In addition, the HIPAA privacy rule imposes a new
layer of review and oversight over our research.
Despite this extensive regulation, some have called for additional
restrictions to be instituted relating to consent, IRB accreditation and
review, and conflicts of interest.
From many different perspectives, reform of the existing system is
not only necessary and desirable, but appears inevitable. In light of this,
BIO companies have spent considerable time evaluating the existing
system of research oversight. Based on this analysis, we have identified
several key concerns and areas for improvement. They are:
Multiple and overlapping layers of review, leading to confusion and
inefficiency for participants as well as research sponsors;
New regulations that will increase the burden on an already
overwhelmed IRB system;
An existing framework for review of research involving human
participants that is inappropriate for research involving medical
archives or data;
Differing state laws govern and complicate the form of research review
and format of consent required in each state; and
A strong and persistent perception that the presence of private money in
the health care setting creates conflicts of interest in researchers that
may affect results and the quality of care provided to research
participants.
Multiple Layers of Review
The current system of research review relies heavily on IRBs.
Historically, they have filled the important role of providing independent
review of research projects. However, the current regulatory system
applies multiple overlapping layers of review for sponsors of every clinical
protocol. Specifically, FDA regulations require the sponsor to obtain review
by an IRB, and each investigator affiliated with an academic institution
must have its IRB separately review and approve every aspect of the
research protocol under federal regulations that apply to institutions that
receive federal grant money. Consequently, trials that take place in
several locations must be reviewed by several different review bodies.
Each can require changes to trial design, the informed consent form, or any
other protocol component. This adds enormous complexity and expense to
a research project.
An additional complication is the HIPAA privacy regulation governing
the use and disclosure of medical information. That regulation adds an
entirely new authorization process to the informed consent already required
from every research participant and/or data subject. It requires that
researchers get an individual’s authorization – or a waiver of authorization
from an IRB or privacy board – to access and use protected health
information for research purposes. The IRB’s review of this issue is in
addition to its consideration of the other risks present to research
participants.
Thus, two distinct assents are now required of each research subject:
informed consent to participate in research and “authorization” to disclose
and use an individual’s protected health information in research under the
HIPAA privacy regulation.
As to the overall issue of the growing multiple layers of review, BIO
believes Congress should eliminate the multiple separate legal reviews
currently required for clearance of a sponsored clinical research protocol.
Mechanisms should be developed to centralize and streamline review of
research projects. In addition, researchers should be allowed to use
patient information without authorization where researchers (1) secure
individuals’ informed consent or (2) obtain a waiver of consent by an IRB or
privacy board, in whole or in part, where waiver is warranted under existing
law. In addition, we support modifying the criteria for waiver of
consent/authorization for use of patient data and archival information both
in the privacy rule and under the current Common Rule to enhance access
to much-needed data where the confidentiality risks present to the
individual are minimal.
In this regard, we note that HHS recently proposed modifications to
the HIPAA privacy rule that would simplify and streamline the requirements
for authorization by IRBs and privacy boards. BIO supports these
proposed changes as an important first step in eliminating unnecessary
and inappropriate regulatory hurdles for the conduct of research, and we
urge HHS to adopt these modifications in its revised final rule. Without
these changes, the existing waiver of authorization standard, in particular,
is unworkable and will have a significant adverse impact on research
activities.
In addition, since IRBs play such an important role in the research
oversight system, BIO believes they should be held accountable for
meeting their responsibilities. Some have recommended that a system of
accreditation for IRBs be developed. BIO is intrigued by the concept of IRB
accreditation and would be supportive of exploring the issues involved.
Review Commensurate with Risk
Currently, research studies are reviewed using the same criteria
regardless of the type of risk faced by the research participant. For
example, a research study that entailed testing a drug on individuals will be
regulated the same way as a study that relied only on a review of medical
records. This process does not acknowledge the different types of risk
faced by the research subjects in each study. Participants in the first study
will confront safety risks, while subjects in the second study face risks
related almost entirely to confidentiality.
The regulatory structure stems from the history of our oversight
system that based federal review on factors other than the risk to the
research participant, such as presence of federal funding or regulation.
BIO believes that this paradigm is no longer appropriate – for researchers
or research participants. As we learn more about how genomic information
can be used to cure disease, medical records review and archival research
will grow in importance.
Thus, BIO supports an alternative approach that makes regulatory
oversight commensurate with the risk to the research participant. That type
of system would establish one set of requirements for research that
involves intervention or interaction with individual research participants and
a separate set of requirements tailored to the unique issues raised by
research using medical records and tissue archives. This new framework
would be applicable to all research, regardless of its funding source. It is
important to note that in a report issued last year, the National Bioethics
Advisory Commission (NBAC) made a similar observation, and endorsed
the notion that review should be commensurate with the types of risk
presented by the research.
Differing State Laws
A related problem is that researchers are subject to a patchwork of
different, and sometimes inconsistent, state laws. Although there are
extensive federal rules regarding research, state laws govern issues such
as the form of review and format of additional documentation of consent.
This is often problematic for researchers. For example, new state
laws pertaining to genetic analysis are quite restrictive, requiring additional
separate consents and imposing onerous requirements regarding the use
and retention of tissue and blood samples that sometimes are inconsistent
with FDA requirements.
A 1999 study of state health privacy laws showed the vast differences
among the states. In addition to existing differences, state laws in this area
are in flux. During the 2000 state legislative session, 26 states debated
laws concerning privacy. This turbulent environment will slow important
research efforts.
It is important to note that the differences among states do not seem
to start from differences in the level or degree of protection, but reflect
different state legislatures' views of the specific procedures or requirements
for accomplishing the same objective. Nonetheless, the requirements and
penalties are different enough to require every researcher to hire lawyers to
assure compliance with the laws of more than 50 states and local
jurisdictions in designing informed consent documents for a multi-state trial.
To remedy this problem, BIO believes that consideration should be
given to creating one national, uniform set of rules governing research.
National standards would allow researchers to create informed consent
and other procedures that will be legal in all states. These federal research
standards should preempt state laws that create conflicting obligations
regarding research participants from different states.
Conflicts of Interest
There is a strong and persistent perception that the presence of
private money in the health care setting creates conflicts of interest in
researchers that may affect results and/or the quality of care provided to
research participants. This perception has the potential to damage the
public’s trust in biomedical research.
We must take steps to maintain public confidence. However, it is
important to remember that the tremendous investment by the private
sector over the past two decades has led to remarkable medical
breakthroughs. Government policy to encourage private investment has
been a major factor in the development of a biotechnology industry in the
United States that is the envy of the world.
The best ways to both protect patients and the integrity of research is
to ensure that research protocols are independently reviewed and that all
financial interests are disclosed. We understand that the academic
institutions are in the process of carefully reviewing conflict of interest
issues and are attempting to generate a unified position and set of policies
regarding financial interests. In the meantime, BIO agrees with the
direction of the NBAC recommendations, which is to focus the discussion
in a way that encourages disclosure of financial relationships between and
among researchers, investigators and IRBs, but does not prohibit nor
otherwise impose rigid restrictions on the existence of such relationships.
Conclusion
Mr. Chairman, we believe that it is appropriate to review the existing
regulatory structure for research and urge that consideration be given to
BIO’s four key principles: (1) eliminate multiple separate levels of review;
(2) modify the regulatory framework so that review is commensurate with
the type of risk involved for the research participants; (3) preempt state
laws that create conflicting obligations; and (4) work with academic medical
centers and other affected entities and individuals to develop an approach
for addressing real and perceived conflicts of interest.
BIO companies believe that it is critical to make sure that, despite the
changes in our research infrastructure over the years, participants continue
to be protected. We firmly believe that addressing these key issues
described above will enhance the level of protections we can guarantee
participants in our research projects.
In protecting our research participants, we must also ensure the
continuation of valuable – potentially life-saving – research. We are
fortunate to live in an era of enormous promise as scientists begin to
access a vast library of genetic information with the goal of improving our
medical interventions. Decades of responsible science have shown that
protecting research participants and promoting medical research are
mutually attainable.
BIO looks forward to working with the Committee as it pursues both
goals.
Thank you.