ALLIANCE FOR HUMAN RESEARCH PROTECTION
(AHRP)
re: Pediatric Research Equity
Act of 2003 (S 650).
Tel.
212-595-8974
Fax: 212-595-9086
142 West End Ave. Suite 28P
New York, NY 10023
Co-founders:
Vera Hassner Sharav, President
John H. Noble, Jr., PhD, Treasurer
David Cohen, Ph.D., Secretary March 27, 2003
March 27, 2003
Senate Committee
on Health, Education, Labor, and Pensions
Dear Senator Judd
Gregg and Senator Edward Kennedy:
The Alliance for
Human Research Protection (AHRP) is a grassroots organization
of professional and lay citizens, one of whose missions is to
protect children from harmful medical experiments.
We are writing
about the Pediatric Research Equity Act of 2003 (S 650). We
are in full agreement that children should have the same
safeguards as adults; that medications used in children be
thoroughly tested for safety, effectiveness and appropriate
dose. However, S 650 does not do this. Instead, it requires
that children be used to test all drugs as a condition of
licensure by FDA — unless a waiver is obtained from the
Secretary of the department of Health and Human Services under
section 505B.
As you know, no
drug is perfectly safe. Drugs are prescribed by physicians
using a risk-benefit equation. Certain drugs are highly toxic,
for example some antiarrhythmics and chemotherapy agents.
Other drugs lead to severe, idiosyncratic reactions in a small
percentage of recipients. These reactions may include bone
marrow failure or life-threatening anaphylaxis.
Furthermore, many
drugs are unlikely to be used in children, and these are often
the most toxic drugs, for instance, chemotherapy agents for
tumors that only occur in adults, or certain cardiac drugs.
There is no reason to test these drugs in children,
particularly as they have significant toxicities. S650,
however, requires that this be done.
Instead, in
accordance with The Belmont Report and existing federal
regulations that had been adopted in 1983 for the protection
of children — 45 CFR 46 Subpart D — The Alliance for Human
Research Protection recommends that drugs be tested in
children only when a role for the drug in the pediatric
population is anticipated by the manufacturer, and only
children who have the condition for which this drug is a
treatment may be used in clinical trials.
Using children as
subjects in clinical research is currently limited for a very
good reason: they are unable to give voluntary, informed
consent. Current regulations do permit children to serve in
clinical trials that investigate treatments for conditions the
children have. We believe this legal regime balances the need
to investigate new therapies with the need to protect
vulnerable members of society who cannot consent to being
experimental subjects.
It allows drug
manufacturers to test pediatric drugs in the appropriate
population. But there is no benefit to either children or
society when children are used to test drugs for illnesses
they do not have, or to test drugs that will be used solely
for adult conditions.
If a physician
wishes to use an adult drug for a child, he/she still may use
the drug off-label, using available information on the drug in
adults, and his/her professional judgment regarding its
benefit in children.
We wish to
further express our concern about the lack of adequate
safeguards to protect children from research that is likely to
put their health at risk of harm.
Children are
incapable of exercising the adult right to informed
consent—they should not be made to bear the burden and risks
of research, unless they have a condition that would stand to
benefit from a specific drug trial.
It is surprising
that you need training and a license to drive a car, but no
comparable training or licensing requirements exist for
researchers who conduct medical experiments on people. It is
surprising as well that Congress could pass legislation that
would subject children to risk without a commensurate benefit.
By the FDA's calculation, the law will expose as many as 30%
of the child subjects to needless risk.
Instead, The
Alliance for Human Research Protection proposes the following
guidelines for all clinical research in which children are
experimental subjects:
- Restricting the use of children in research to
studies involving no greater than minimal risk — unless
the potential benefit to their condition justifies the
risk.
- Prohibiting conflicts of interests, such as paying a
fee to physicians who recruit children.
- Establishing a registry of all pediatric clinical
trials and requiring mandatory reporting of serious
adverse effects.
- Imposing stiff penalties when foreseeable risks have
not been disclosed or informed consent requirements have
been violated.
- Assuring every child who participates in clinical
trials will be protected by no-fault insurance coverage
against possible adverse effects that may arise from or
in the course of participation in such research.
- Assuring every child who participates in clinical
trials no greater risks than the child would incur if
given the currently available "best standard" of medical
treatment.
- Prohibiting use of financial enticements to induce
parents or guardians of children to enroll them in
research.
- Mandating long-term monitoring for adverse effects.
- Requiring all researchers who conduct research on
human subjects to be trained and certified as proficient
in the knowledge of medical ethics and the best
standards of medical care.
- Restricting children from being used as subjects in
trials of therapies that are not intended for children.
Testing,
unfortunately, does not always assure drug safety.
Experimental subjects can be harmed, and patients who receive
approved drugs can also be harmed. Rare, but severe adverse
effects will often not emerge in pre-marketing studies, but
only later, when the drug is in wide use. We agree with the
Association of American Physicians and Surgeons that adults,
not children, should bear the brunt of unavoidable drug
testing risks whenever possible.
Sincerely yours,
Meryl Nass, MD,
AHRP board member
Vera Sharav,
President, AHRP
John H. Noble,
Jr., Ph.D., Treasurer, AHRP
Cc: Congressional
Human Rights Caucus ;
Senate Children's Caucus
