Putting Yourself To the Test
Volunteers for clinical medical trials are open to potential benefits - and risks

April 26, 2003
Newsday
By Jacqueline Rivkin
Jacqueline Rivkin is a freelance writer.

Hugh Doherty is independent and likes it that way. "I like to take care of myself," the retired subway inspector said.

But lately, Doherty, a diabetic, has had pain in his feet that has made it harder for him to get around. "It's the pain and your inability to get out and walk straight," the Woodmere resident said. "The burning and the tingling. Everything hurts." So when he saw an ad for a clinical trialin diabetic neuropathy, a nerve disorder caused by diabetes, he was intrigued.

Doherty had no hesitation about participating in the clinical trial at Winthrop-University Hospital in Mineola. "I think they're more careful now with all the lawsuits and everything," he said. But "I didn't just jump on the bandwagon," either. He went for an interview and asked questions, did research on the Internet and discussed the trial with his doctors.

Three months into the trial, Doherty said that while he feels "neither better nor worse" as a result of the medication, which may be a placebo, he is glad he took a chance on the program and has advised his sister, also a diabetic, to look for a trial near her home in Florida. "You have to try to do what you can to help yourself," he said.

Clinical trials are research studies used to test new forms of medical therapy for safety and effectiveness. Trials are sponsored by medical institutions, pharmaceutical or medical device manufacturers, and government entities such as the National Institutes of Health.

An estimated 2 million people participated in clinical trials last year in the United States, according to CenterWatch, a Boston- based clinical trials information Web site and publisher. Between 75,000 and 80,000 participated in trials in New York State.

CenterWatch said the number of studies involving senior citizens is on the rise, as is the number of drugs in development targeting age-related illnesses. Experts say that as the population ages, such health issues are becoming increasingly prevalent, which makes it important that senior citizens participate in clinical trials.

"We need to do a lot more research because this is an aging population and we want them to be a healthier population," said Charles Hollingsworth, chief of the clinical trials branch at the National Institute on Aging in Bethesda, Md. Hollingsworth said while it would be naive to say older participants get involved in trials only "because they want to help mankind," that aspect is also important. "As you get older, your view of life changes," he said.

Yet the decision to sign on is not a simple one. At its best, participating gives one access to state-of-the-art medical treatments, while helping to make the world a happier, healthier place. But CenterWatch estimated one in 30 volunteers will suffer a serious side effect and one in 10,000 will die. Although checks and balances make it safer to participate in trials than decades ago, there still are horror stories.

Proponents say participating is a win-win situation, in which participants are able to help themselves and others. "By and large, people participating in clinical trials get better than average care and tend to do better than people with the same condition who are not participating," said Dr. Marc Gordon, chief of neurology at The Zucker Hillside Hospital in Glen Oaks.

Dr. Louis Weiner, director of medical oncology at Philadelphia's Fox Chase Cancer Center, sounds a more cautious note. While agreeing that there are benefits, he notes that "there are excesses and mistakes, and they point out the need for vigilance by regulatory agencies." Weiner said patient safety has improved significantly but the individual must still do research, ask questions and if he or she feels it necessary, get a second opinion. "Most people wouldn't buy the first car they see," he said. "They should apply at least as much care to a clinical trial."

But while most people are wary of used-car salesmen, they are much less wary when faced by someone in a white coat with MD after his or her name. The idea that one can question or even disagree with a doctor is particularly foreign to senior citizens, but most experts agree a healthy skepticism and a well- honed list of questions should be standard equipment when considering a clinical trial.

"You really have to take the responsibility to be informed," said Kenneth Getz, president at CenterWatch and author of "Informed Consent: The Consumer's Guide to the Risks and Benefits of Volunteering for Clinical Trials" (CenterWatch, $19.95). "We advocate that before a person investigates a trial they should take the time to find out what is involved."

Yet, Getz said, astonishingly few people take the time to find out what they are signing on for.

"What we're finding is that regardless of age, very few people do any research," Getz said. "An overwhelming percentage don't even read the consent form."

Emotional state also may be an issue, Getz said. "The typical clinical trial volunteer is evaluating a trial when they have just received a diagnosis," he said. They may be dealing with the idea of a serious or even life-threatening illness, learning about their condition and thinking about the impact of their lives, leaving them scant reserves for careful evaluation.

Hospital defenders emphasize that the clinical trial form, intended for distribution to everyone, cannot reasonably disclose every possible risk.

"It doesn't look to the individual but to a reasonable standard," said David Richman, a partner and head of medical litigation at Rivkin Radler in Uniondale. "You can't stop at the form."

Even language can be a problem. The consent form should outline the issues in clear and coherent language. Yet, the document can be confusing and ambiguous, a recent study at Johns Hopkins showed. "Even educated people often come to the forms and are just daunted by them," said research fellow Michael Paasche-Orlow, who headed the study.

As a health professional, registered nurse Sarasue Cassell said she had no trouble understanding the consent form for the trial of a new Parkinson's drug she began at Winthrop-University Hospital in June 2002. But she agreed that the forms are tough for the average layperson. "The forms are very, very technical," she said.

The Rockville Centre resident said although she signed the form at once, the research staff at Winthrop insisted she take it home and discuss it with her family. While not all of her children were enthusiastic about her participation, Cassell said the care exhibited by the research staff at Winthrop gave her the confidence to proceed with the study.

"I felt it was my body and I felt it was safe, so I went ahead with it," she said, adding that she feels better and is falling less.

For Cassell, who heard of the study while still reeling from the diagnosis of Parkinson's, finding out she had been accepted "was like winning." "I figured that if it was something that could be cured, I would be right at the forefront."

Sometimes, though, "winning" one's way into a trial turns out to be a booby prize. Attorney Denise Dunleavy said her client, Catherine Abatino, also was thrilled when she was accepted into a trial for hormone- replacement therapy in 1999. "You feel like you've won a contest," said Dunleavy, a partner at Kramer & Dunleavy in Manhattan. "I think the whole process of getting into these trials adds to the allure."

The Women's Health Initiative of the National Institutes of Health halted a large national study of hormone replacement therapy in July because an increased risk of breast cancer, as well as increased risk of heart disease and stroke, had been noted in the 16,000 women participating in the study.

Abatino is now suing NewYork-Presbyterian Hospital because she developed breast cancer during the trial. She also alleges the hospital failed to do mammograms that were supposed to be part of the study protocol.

"I read about it in the news and I thought, 'I'm looking to go on replacement therapy and who better than Columbia Presbyterian?'" Abatino recalled. "Columbia Presbyterian has a reputation. I figured I would be in good hands."

But nearly a year into the study, Abatino realized she had not had a mammogram in a year and a half, although study guidelines called for one at the start of the study and at six- month intervals thereafter. "It was downhill from there," Abatino said.

Abatino had a lumpectomy in August 2000 and her cancer is in remission but she says her life will never be the same as a result of the clinical trial and its aftermath.

"It's very difficult. I live with pain in my right arm near the operation, but even more, I am always afraid of getting it again," she said. "It's always in the back of my mind - the cancer."

Myrna Manners, vice president at New York-Presbyterian Hospital, declined to comment, citing privacy issues.

Such incidents are disturbing, but experts agree many of the pitfalls of participating in a trial can be anticipated. In general, Long Islanders are better informed than the average participant and really take the time to do their homework, said Dr. Eugene Selivra, director of the Winthrop-South Nassau University Health System Clinical Trials Network.

"They do the research and they look at the Web sites," he said. "They arrive knowing a lot about their conditions and the clinical trials process."

In addition to the medical questions, there also are practical concerns. Participating in a clinical study can involve time, money and effort. Sometimes a patient may need to stay overnight or longer at the medical center for monitoring. Only about 40 percent of patients are compensated for their time or travel expenses. Such compensation is generally minimal - often not even enough to cover carfare. And in some cases, participating in a clinical trial will affect insurance coverage.

But with all the work involved and the pitfalls, CenterWatch estimated 77 percent of those completing a clinical trial consider their experience positive - sometimes, astonishingly so. Ask Ella Drucker. "I am a miracle," she said.

When Drucker arrived at the hospital just more than a year ago after suffering a massive stroke, there was no time to hit the Internet or to get a second opinion. But her case illustrates what some feel may be the best time to use an experimental drug - when there is little choice.

When Drucker and her husband, Herbert, reached Long Island Jewish Medical Center in New Hyde Park that night, the stroke team offered to administer an experimental drug that when used within three hours of the stroke is said to break up the clot before it causes permanent damage. The team encouraged Herbert Drucker to think about it and ask questions while they tested Ella to be sure the drug was appropriate, but with the prognosis grave, he said there was only one decision to be made. "My feeling was that I couldn't see her being an invalid for the rest of her life," the Fresh Meadows resident said. With just 15 minutes until the three-hour deadline, he signed the forms and the drug was administered. "Within 12 hours she was standing," he recalled. Within 24 hours, she was more or less back to normal, and today, Ella Drucker is back at the gym and is planning a cross-country driving trip to attend her grandson's bar mitzvah.

She is participating in another stroke study at Long Island Jewish, for a neuroprotectant that protects brain tissue. The study requires her to be evaluated weekly, then monthly, but she is glad to be involved. "I am just so lucky, and I want to help others," she said.

Would you be willing to participate in a clinical trial? Vote online at www.newsday.com/act2

Where to Learn More

WEB SITES

www.centerwatch.com

Online clinical trials information for patients and health care providers, with trial listings, general information on trials and links to sites that relate to specific conditions.

www.clinicaltrials.gov

A Web site sponsored by the National Institutes of Health offering information on participating in a trial; and a database listing approved studies for various health conditions.

BOOKS

Informed Consent: The Consumer's Guide to the Risks and Benefits of Volunteering for Clinical Trials, by Kenneth Getz and Deborah Borfitz. Published by CenterWatch, this one-stop guide is available at bookstores or through the CenterWatch Web site at www.centerwatch.com /bookstore/pubs_cons_infcon sent.html

Should I Enter a Clinical Trial: A Patient Reference for Adults with Serious or Life-threatening Illness. A 125-page booklet developed by ECRI, a health services research group, available for download at www.ecri.org/doc uments/prg/survey/prg.pdf

Questions To Ask Before Signing On the Dotted Line:

1. What is the purpose of this study?

2. What is the duration of the study?

3. How much of my time is likely to be involved each month?

4.Will I need to be hospitalized at any point?

5. Will I be reimbursed for travel expenses or time?

6. What are the potential benefits and risks of this treatment when compared with the treatment I am now receiving for my condition?

7. How might the study protocol interact with the medications I am taking? (Bring a complete list.)

8. What concerns were voiced by the center before they agreed to accept the trial?

9. What potential conflicts of interest do the researchers have? Are they being paid to refer patients?

10. Will my health insurance company continue to cover me if I participate in this study?

11. Do you have someone on staff who can answer coverage questions?

12. If I am injured by participating in the study, will the center pay for my medical care?

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