Senate Passes Pediatric Research Equity Act

Senate Passes Pediatric Research Equity Act

Tue, 29 Jul 2003

Congress and the Bush Administration both are eager to test pharmaceutical products on children who cannot refuse to be test subjects.

We at AHRP are in full agreement that medications used in children be thoroughly tested for safety, effectiveness and appropriate dose. However, AHRP endeavors to protect children from harmful medical experiments.

We note that the proposed legislation does not include safeguards to protect child test subjects from harm. There are No prohibitions on the recruitment of children with financial incentives to their parents and guardians. No limits on the risks of harm or pain that child subject might be made to endure.

AHRP asks: Whose children will be conscripted to serve as “risk bearing children”? (The phrase was used by the Dept. of Health and Human Services in 2001 memo).

On October 21, 2002, AHRP proposed 10 recommendations for the protection of children in clinical trials. See below

Without such safeguards to protect children from exploitation the Pediatric Research Equity Act (PREA) is NOT equitable. Without safeguards for child subjects, the PREA is as reckless and ill-conceived as is the Pentagon’s aborted Plan for s Futures betting Market on Terror.

The difference is that children are not represented on any policy making bodies–Not in the administration and its advisory committees, Not in Congress. Children don’t have a chance vis a vis the powerful pharmaceutical / medical research lobby and their deep pockets. Congress failed, once again, to ask the Administration for a detailed report about the adverse effects suffered by children who were used as test subjects in pediatric drug trials. Congress receives such a report annually about laboratory animals who serve as test subjects. Are our legislators afraid of what that pediatric record may show?

This legislation is not equitable because those most likely to be affected are disadvantaged poor children whose parents don’t have the knowledge or resources to protect them from harm.

News Release
Thursday, July 24, 2003
Contact: HHS Press Office
(202) 690-6343

Secretary of Health and Human Services
Commissioner of Food and Drugs
Regarding Passage of S. 650, The Pediatric Research Equity Act of 2003

We commend the Senate for today’s action in passing the Pediatric Research Equity Act of 2003. This legislation is another step toward closing the gaps that have existed in guaranteeing proper testing of pharmaceutical products for children. Under the President’s leadership, HHS agencies have already taken important steps toward assuring the safety and efficacy of drugs when given to children. But an important action that is still needed is clear legislative authority for the Food and Drug Administration (FDA) to require pediatric testing for appropriate products. The Senate has taken us another step toward that goal.

Many of the innovative pharmaceutical products now being developed are likely to be used in children. But medicines to be used by children should undergo specific pediatric testing, and not simply rely on adult tests. This testing is needed both for new drugs and also for already-approved drugs that may be prescribed for children but still lack pediatric testing.

Congress took one important step toward addressing this need with enactment of the Best Pharmaceuticals for Children Act (BPCA) in January 2002. This legislation authorized HHS’ National Institutes of Health and the FDA to undertake pediatric testing of already-approved drugs that do not benefit from other types of pediatric development incentives. We are carrying out this mandate. Earlier this year, HHS identified an initial list of 12 already-approved drugs that urgently need pediatric testing. We are making up to $25 million available this year to begin this process, and the President has proposed to double the amount to be available in the coming fiscal year.

At the same time, the BPCA also extended incentives for pharmaceutical companies to provide pediatric testing for new products, and this action has also helped ensure that appropriate testing takes place for many new drugs.

However, it remains important for FDA to have clear authority to require pharmaceutical manufacturers to conduct pediatric clinical trials on appropriate drugs and biologics. The Senate’s action takes us a step closer toward enactment of a law to make that authority clear. We thank HELP Committee chairman Gregg and Ranking Member Kennedy for their leadership in shepherding this legislation, and we express the hope that the House of Representatives will also act soon on this important issue.


AHRP Recommendations for the protection of children in clinical research

(1) Federal regulations are predicated on our moral responsibility to protect children — who are not volunteers — from being subjected to medical or behavioral experiments that are not in their best interest. Thus, federal regulations — 45 CFR 46 Sub-part D — restrict the use of children in medical experiments involving greater than minimal risk, if there is no potential medical benefit for them or their condition.

(2) Inasmuch as drugs have unwanted side-effects, and medical research involves risks of harm, only children whose narrowly defined currently diagnosed medical conditions can potentially be helped, should be recruited to test drugs or other medical devices or procedures.

(3) Legislation for the protection of children’s health and welfare should put the burden of proof on those seeking to conduct research on minors under the age of eighteen (18), to establish the existence of “compelling circumstances” that justify such research on children. Investigators must provide the criteria for demonstrating that the benefits of the research outweigh severity, duration, frequency and likelihood of the risks. Children must be assured that current “best medical practice” standards of treatment will be compared to any new or experimental treatment, and that those consenting on their behalf can be held accountable for making research decisions that are in the child’s best interest.

(4) Children should not be recruited for experiments involving greater than minimal risk on the basis of vague speculations about them being “at risk” of some unproven condition that may or may not ever materialize. Rigorous standards must be established for each study involving children so that the level “of risk” can be objectively defined by demonstrable, existing factors. Investigators must demonstrate that the nature, severity, duration, and frequency of the risk is greater than the intervention proposed.

(5) All clinical trials involving the use of children, as previously defined, should provide no-fault insurance coverage for both short-term and long-term adverse effects that may arise from or in the course of participation in the stated clinical trials. [1]

(6) The pool of child subjects must not constitute an unfair burden on disadvantaged families who may not have access to current “best practice” standards of treatment in their community. Thus, care must be taken to ensure that the population from which sick children shall be recruited represents families from diverse socio-economic strata. When children are sought from a specific ethnic or socio-economic population, evidence must be provided demonstrating that the condition under study disproportionately affects that specific population.[2]

(7) The recruitment of children with financial enticements to their parents and caregivers should be prohibited.

(8) The record demonstrates that the current system of review of both the scientific and ethical components of research protocols involving sick children, have failed to protect children such as nine-month old Gage Stevens or eight year old Jennifer Munger from harmful experiments that killed them. [3] Therefore,

A. There is a need for oversight by a “Children Protection Committee” in addition to review by an institutional review board (IRB) that would serve as the child subjects’ advocates, monitoring their selection, assessing the reasonableness of their parents’ consent, the adequacy of disclosure in the informed consent documents, and monitoring their continued willingness to participate in the research. [4]

B. The majority of the Children Protection Committee (51%) should be drawn from the community, among them representatives from the same socio-economic strata as the children in the specific clinical trial.

(9) All of the members of the ethics review board and the Children Protection Committee should be vetted for complete absence of conflicts of interest.

(10) The expenses for the process of safeguarding children’s best interest in research — including community members who are involved in implementing the research review and monitoring process — should be paid from a government fund established for that purpose. The government should, in turn, be authorized to recapture its costs, including oversight of all pediatric research, by way of reimbursement from the drug or medical device manufacturers who are eventually licensed to market such drugs or medical devices that result from approved pediatric research.

[1] This is the identical phrasing of the language of state and federal workers’ compensation laws that provide such no-fault insurance coverage to virtually all employees of U.S. businesses.

[2] This requirement reflects the ethical principle articulated in the Belmont Report relating to justice; namely, equal sharing of the burden and benefit of research.

[3] Willman, D. Los Angeles Times, Dec. 20, 2000, front page; Moss, M. Wall Street Journal, June 12, 1996, front page.

[4] The recommendation for a Children Protection Committee had been proposed by the Department of Health Education and Welfare in 1973 but never adopted. See 28 Fed. Reg. 31, 738 (1973)