Betaseron near formal approval.

Four months have elapsed since a special advisory panel to the federal Food and Drug Administration recommended that Interferon beta-1b --Betaseton--receive FDA approval for use in relapsing-remitting MS. As this publication goes to press, formal approval is still pending. The Society is standing by to alert chapters and branches as soon as the FDA announces its decision. At the March 19 public meeting in Washington, DC, the panel voted 7-to-2 in favor of recommending approval of the new agent. Panel members based their decision on results from two years of a threeyear, randomized, placebo-controlled study of 338 people with relapsing-remitting MS.

What the study showed

People in the study were divided into three groups -- to receive high-dose Betaseton, low-dose Betaseton, or a dummy, or "placebo", drug -- but neither doctors nor patients knew for certain who was in which group. Everyone underwent regular examinations, including an annual MRI to document changes within the brain and central nervous system.

After two years, the high-dose patients had one-third fewer exacerbations than those on the dummy drug. The episodes they had were milder and they spent less time in the hospital.

Moreover, MRI scans documented that the high-dose group incurred dramatically fewer abnormalties in the central nervous system.

These results, which were published in the April, 1993 edition of Neurology, spurred much excitement among people with MS and in the health-care community. While Betaseron is not a cure, it is the first drug in nearly twenty-five years to treat effectively the disease itself, not just its symptoms. Use of Betaseron should help people with early mild MS to stay on the job and out of hospitals.

"It should make the course of this unpredictable disease a bit more predictable," said Dr. Stephen Reingold, vice president of NMSS Research and Medical Programs. "However, the current study does not show that Betaseron either changes or fails to change accumulation of disability caused by the disease."

The next steps

In the months following the March announcements, the FDA has been conducting additional reviews to answer additional questions. The issues include what the package insert should say about efficacy, side effects, interactions with other drugs, and warnings about situations in which the drug shouldn't be used. Until formal approval is given, Betaseron is only available to people who took part in the controlled trials.

While careful review is essential to safe use of this drug, FDAred tape also contributes to the time lag. The National MS Society believes the process could be speeded up without jeopardizing safety. To keep FDA officials aware of what is at stake in human terms, the Society arranged for Patricia Redondo of Randellstown, Maryland, to testify at the March hearing. She "will always wonder" what Betaseron might have meant in her life had it been available a decade ago, when she was first diagnosed, she said. Since March, the National MS Society's "Action Alert" program has coordinated a nationwide letter-writing campaign, through our local chapters, urging the FDA to speedy action. (See "The MS Community Responds.")