Alliance for Human Research Protection

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:

1. 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.
   
    
2. Prohibiting conflicts of interests, such as paying a fee to physicians who recruit children.
   
    
3. Establishing a registry of all pediatric clinical trials and requiring mandatory reporting of serious adverse effects.
   
    
4. Imposing stiff penalties when foreseeable risks have not been disclosed or informed consent requirements have been violated.
   
    
5. 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.
   
    
6. 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.
   
    
7. Prohibiting use of financial enticements to induce parents or guardians of children to enroll them in research.
   
    
8. Mandating long-term monitoring for adverse effects.
   
    
9. 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.
   
    
10. 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