HEALTH ISSUES

Gene Research Rule Proposed
Regulation Seeks to Increase U.S. Review, Keep Reporting Manageable

By Rick Weiss
Washington Post Staff Writer
Wednesday, December 13, 2000; Page A02

Fifteen months after the field of gene therapy was roiled by the death of a teenage volunteer and by revelations that many researchers had failed to tell regulators about complications in their own gene therapy studies, the Clinton administration yesterday proposed a new system for tracking dangerous side effects of the experimental treatments.

A proposed federal rule, published in yesterday's Federal Register, seeks to resolve conflicts between patient advocates who have called for full public disclosure of all ill effects in gene therapy volunteers, drug companies that hope to profit from the nascent field of medicine but are accustomed to keeping the details of their studies confidential, and researchers who have complained about being overburdened with regulations.

It attempts to achieve middle ground by loosening old requirements that had demanded immediate reporting of all serious complications, while at the same time beefing up federal attention to those complications when they're reported.

Representatives of groups that would be affected by the change quickly found fault with various aspects of the proposed rule. It remained unclear yesterday whether that meant the rule was a good compromise or was doomed to political oblivion.

At issue are so-called serious adverse events, including deaths, life-threatening episodes or significant incapacities that occur in volunteers getting gene therapy, an experimental approach that seeks to cure diseases by giving people new genes.

For the past 10 years the National Institutes of Health has demanded that all serious adverse events occurring in gene therapy volunteers be reported to that agency immediately, whether they seem to have been caused by the treatment itself or were probably a normal outcome of the patient's underlying condition.

That standard is more strict than the Food and Drug Administration's rule that immediate reporting is required only when researchers believe the problem may have been caused by the treatment itself--a difference that reflects NIH's longstanding concern that researchers might not be able to tell if a problem was caused by the novel treatment or not.

Three events last fall triggered a review of the NIH policy. First, several gene therapy companies submitted adverse-event reports with demands that the data be kept confidential, in violation of NIH's policy of public access. Second, a nationwide review indicated that many researchers were not even reporting adverse events to the NIH as required. And third, Tucson teenager Jesse Gelsinger became the first person to die as a clear and direct result of gene therapy.

The newly proposed rule reaffirms that all adverse events should be made public. But it calls for researchers to immediately report to the NIH only those serious adverse events believed to have been possibly caused by the treatment. That standard is in harmony with FDA's rules, a simplification that researchers said they applauded. But some patient advocates criticized the change, saying researchers cannot be expected to be unbiased when it comes to deciding whether their treatment has caused a problem.

"It's very disturbing that they are going to do something like this, where everyone can decide for themselves," said Abbey S. Meyers, president of the National Organization for Rare Disorders in New Fairfield, Conn.

Under the rule, problems deemed unrelated to the treatment must be submitted in annual summary reports--which a new NIH subcommittee would analyze, along with other reports, to watch for worrisome trends.

Paul Gelsinger, Jesse's father, said he favored the extra oversight the new committee would bring and didn't mind if some adverse-event reports got submitted annually instead of immediately--as long as it would encourage better reporting overall. "Remember," he said, "some of these guys weren't reporting adverse events at all before."

But Carl Feldbaum, president of the Biotechnology Industry Organization, criticized those aspects of the new rule, saying they would allow too much public access to commercial data he believes should be confidential. Moreover, he said, the NIH subcommittee's duties would be redundant with the FDA's.

"It usurps FDA's regulatory authority and turns the death of Jesse Gelsinger into some kind of a turf battle," he said.

The NIH is accepting comments on the proposed rule through Feb. 10.

Bush Has Pharmacy Discount Card Plan
Prescription Drug Benefit Is Part of Medicare Revamp

By Amy Goldstein
Washington Post Staff Writer
Wednesday, July 11, 2001; Page A01

The White House is devising a plan that will offer pharmacy discount cards to all older Americans by the beginning of next year in a new strategy intended to provide the elderly quick help on prescription drug costs.

President Bush is to announce the pharmacy discounts tomorrow as he also lays out a set of principles that the administration hopes will guide Congress in revamping Medicare, the nation's health insurance program for the elderly. The principles will include adding a separate, subsidized prescription drug benefit.

According to sources familiar with the pharmacy discount plan, the administration is designing a national version of an approach, adopted recently by some private insurers, that relies on companies that manage drug benefits to buy prescription drugs in bulk. The companies would sell cards to Medicare patients, who could use them at any pharmacy to purchase their medicine at a reduced rate.

The sources said the White House believes the program would not require congressional approval and could be implemented by the Department of Health and Human Services within several months.

By announcing the drug discounts, the administration is striving to simultaneously bolster the affordability of medicine for patients who need it most and to address a health care issue of acute public concern that played a prominent role in last year's campaign.

The discount cards represent the second idea for curbing drug expenses of the elderly that the administration has come up with during the six months Bush has been in office. The first idea, based on a Bush campaign proposal known as Immediate Helping Hand, would have created a temporary block grant to help states subsidize drug costs for their poor, elderly residents. It was rejected swiftly on Capitol Hill.

With the new plan, the administration can position itself as taking the lead on prescription drugs -- the most popular aspect of Medicare reform -- regardless of whether Congress acts on the issue.

The idea relies on the market-based approach the administration favors, and would fall short of the subsidized prescription drug benefit for all Medicare recipients favored by most Democrats. Sources said that the government would not dictate the size of the discount elderly patients could receive. They also said the plan would not require federal money.

In its private-sector orientation, the discount plan dovetails with the principles that Bush is preparing to set forth for a more fundamental restructuring of the 1960s-era Medicare system that is one of the nation's largest and most popular entitlement programs.

The president is scheduled to outline more than half a dozen principles publicly tomorrow morning, after he briefs a group of senators and House members.

The principles include creating more options for people in Medicare, probably by increasing reliance on private insurers to cover patients who are 65 and older. The president will say Medicare should do a better job covering preventive health care. And responding to complaints from doctors, insurers and hospitals, the president will recommend easing some of the program's regulatory requirements.

Bush also will say he wants the program to begin including voluntary prescription drug coverage, apart from the discount cards. He has not said lately what that coverage should include, but during last year's campaign, he proposed that the government pay the entire monthly premiums for eldery Americans with relatively low incomes and at least one-fourth of the premiums for other Medicare patients. He also proposed that Medicare pick up coverage for a small number of older people whose drug expenditures exceed $6,000 a year.

The strategy of outlining principles resembles one the administration used in efforts to create federal protections for Americans in managed care, when Bush last winter defined a set of broad objectives but did not craft his own detailed proposal. Instead, the president has latched onto a bill that failed in the Senate and one that is pending in the House that he has said meets his goals.

The president is stepping more firmly into the Medicare debate as Congress prepares a new round of deliberations on how to modernize the system and prevent it from running out of money once the large baby boom generation retires. Efforts at such changes have suffered in recent years because of partisan disputes.

This time, Senate Finance Committee Chairman Max Baucus (D-Mont.) has said he wants the panel to act on a Medicare proposal by the end of this month, but aides are struggling to weave together two rival bills -- one sponsored by Sen. Bob Graham (D-Fla.) and the other by Sens. John Breaux (D-La.) and Bill Frist (R-Tenn.). Their proposals differ in how generous prescription drug coverage would be, how much money would be required and how far the Medicare program should be tilted into the hands of private insurers.

Senate staffers familiar with Medicare said it is unclear whether the differences can be resolved in time for the committee to act before Congress's August recess.

As the Medicare issue intensifies, Health and Human Services Secretary Tommy G. Thompson yesterday blocked a different attempt to cope with drug costs, approved by Congress last year. It would have enabled American consumers to buy drugs that were manufactured in the United States, sold to countries in which drug prices are controlled, then imported back into this country. Reaffirming a decision reached late last year by then-HHS Secretary Donna E. Shalala, Thompson told Congress that, under such an arrangement, the safety of the drug supply could not be guaranteed.

© 2001 The Washington Post Company

HMO Execs Rake in Cash
Study Says HMO CEOs Are Making Big Bucks, Amid Massive Benefit Cutbacks

By Linda Douglass

June 20 Many of the top executives in the managed care industry were paid huge compensation packages last year according to a consumer report obtained by ABCNEWS, and the 10 highest paid executives raked in an average of $11.7 million apiece.

Some managed care executives also got stock options last year: Ten executives hold an average of $68 million in stock options apiece.
At the same time, patients in many of those health plans are being denied coverage, and the frustration level for millions of people is rising.

The report was compiled by a consumer group, Families USA, a critic of HMO cost-cutting.

"It puts the issue of cost in perspective," says Ron Pollack, executive director of Families USA. "It shows that the companies are not that concerned about costs when it lines their own pockets."

New From The Post
Senators Agree to Bill Compromise
Key senators announced a bipartisan compromise to limit health-care lawsuits against employers today as a Republican amendment to shield small business owners failed. By Helen Dewar

Index Predicts Hospitalized Seniors' 1-Year Survival

BETHESDA, MD, 26 June 2001 An elderly patient's likelihood of living one full year after discharge from a hospital can be accurately assessed using six prognostic indicators, say researchers.

For a patient 70 years or older who is hospitalized for medical, not surgical, reasons, the researchers determined that the risk of dying within 12 months of discharge is influenced by the following factors: dependence on another person for performing basic activities such as dressing oneself; the presence of cancer, congestive heart failure, a serum creatinine concentration of >3.0 mg/dL, and a low albumin level; and the patients sex.

Other factors, including age greater than 80 years, a history of myocardial infarction or dementia, and discharge to a nursing home or skilled-nursing facility, were also associated with an increased likelihood of death during the year after discharge from the hospital. But once the six main factors were taken into account, these other indicators did not influence patients survival.

These findings were reported in the June 20 Journal of the American Medical Association (JAMA).

The study involved two groups of patients 70 years or older. The first group, with 1,495 patients, provided information that was used to develop the prognostic index. A second group, with 1,427 patients, later provided data that was used to independently test the index. In all, 492 people in the first group and 398 in the second group died during the one-year follow-up periods.

Using a point-based system, the researchers divided the seniors into four risk groups. The one-year death rates ranged from 413 percent in the lowest risk group (0 or 1 point) to 6468 percent in the highest risk group (620 points).

A diagnosis of metastatic cancer added 8 risk points to a patients prognostic score; nonmetastatic cancer added 3 points. The inability of an elderly patient to bathe, dress, use the toilet, move from a bed to a chair, and eat without the assistance of another person added 5 points. The other risk factors, including male sex, added 1 or 2 points to a patients total score.

The point system was able, 7579 percent of the time, to correctly discriminate between a patient who would die within one year of hospital discharge and a person who would live. The researchers said tests that focus only on physiologic or disease factors have a lower sensitivity, 5968 percent.

An editorial (PDF) in the same issue of JAMA was mostly supportive of the prognostic index, describing it as a way to "help physicians validate their clinical impressions." The editorialists also suggested that use of the index might dissuade clinicians from heavily relying on age as a predictor of an elderly patient's short-term survival.

Despite the potential usefulness of the prognostic index, the editorialists were skeptical that the system would be widely used in clinical practice. The index, they said, is freely available and not promoted through paid advertisements, increasing the likelihood that many clinicians "will never hear about it."

Kate Traynor