Patient Advocates Urge FDA Disclosure
Stung by the sudden rejection of ImClone System’s highly touted cancer drug Erbitux, patient advocacy groups have begun a push to force the government to disclose more details about its decisions regarding drug approvals.
Existing law prevents the Food and Drug Administration from publicly discussing its findings, but at least two advocacy groups are seeking to change that and are pressing their case with the House Energy and Commerce Committee, chaired by Louisiana Republican W.J. “Billy” Tauzin.
The committee is investigating complaints that ImClone misled investors about prospects for Erbitux. It also told the FDA it was concerned with protecting patients from deception.
The panel, which also is investigating Enron Corp., has signaled its concern about the secrecy surrounding the drug approval process. In a letter to the agency, it asked the FDA to provide a legal analysis on whether it can provide confidential information to the Securities and Exchange Commission.
Advocacy groups maintain that interagency communication, even if permitted, would not go far enough to protect their interests. FDA documents, they argue, can alert seriously ill patients to problems or delays that drug makers might be reluctant to reveal.
“The FDA has become the shield behind which companies hide” bad news, said Bob Erwin, a biotechnology executive who also is director of the Marti Nelson Cancer Research Foundation, an advocacy group based in Vacaville, Calif., that has lobbied the committee.
But even as the Enron scandal pushes corporate disclosure issues to the forefront, advocates for cancer patients face an uphill battle. The pharmaceutical industry opposes any change, which would take an act of Congress. The industry maintains that confidentiality is needed to protect competition and promote honest communication between drug makers and the FDA.
“Communication would be much less candid,” said Carl Feldbaum, president of the Biotechnology Industry Organization. Correspondence between the FDA and drug makers would be sanitized for public consumption, he said. “It would not be helpful.”
Feldbaum said he had talked informally with committee members “in an education vein” about the regulatory process. Among his concerns, he said, was that investors would overreact to FDA disclosures, adding to the volatility of biotechnology stocks.
Alan Goldhammer, associate vice president for regulatory affairs for Pharmaceutical Research and Manufacturers of America, said safeguards are already in check to prevent the spread of misinformation. He said the federal securities laws require public companies to make timely disclosures about such material events as drug approvals.
“You’re putting the FDA in the position of policing disclosures under the act” that created the SEC. “I don’t think that’s the FDA’s job.”
But patient advocates contend that the increasingly messy controversy over Erbitux points to a need for reforms.
The FDA rejected ImClone’s application to market the drug Dec. 28, an unexpected setback that ImClone first attributed to record-keeping mistakes that it said would take a short time to resolve.
A week later, however, an industry newsletter published lengthy excerpts from the FDA’s confidential rejection letter that cast doubt on ImClone’s explanation. The Cancer Letter account said the agency had serious concerns about Erbitux that would require the company to perform additional, time-consuming tests.
Investors dumped the stock, which closed off 58 cents on Thursday at $14.87 on Nasdaq, down from a high of $75 in early December.
ImClone, which faces inquiries from the Justice Department and the SEC, has not challenged the newsletter’s disclosures. But the company has denied that it acted to deceive investors.
“What I find troubling is that if it had not been for the leak of the letter, patients would not understand that the drug is a long way from approval,” Erwin said. “This is important information to patients who must choose from among alternative treatment options.”
Erwin, chief executive of Large Scale Biology, a biotechnology company, said very little confidential information is contained in FDA approval or rejection letters. “There has to be a level of trust,” said Nancy Roach, a founder of the Colon Cancer Alliance in Kirkland, Wash. “You have to trust the drug you get; you have to trust what the drug company says about the drug. I think that trust has been damaged, and we are looking for the FDA to get that trust back together.”
In addition to the Marti Nelson Cancer Research Foundation and the Colon Cancer Alliance, the Abigail Alliance for Better Access to Developmental Drugs also is lobbying the committee.
ImClone and its estranged marketing partner, Bristol-Myers Squibb, predicted last fall that Erbitux would receive FDA approval by spring. Bristol-Myers acknowledged recently that the drug would be delayed; some analysts do not expect it to reach the market until 2004.
The two companies, meanwhile, are locked in a power struggle stemming from ImClone’s legal and regulatory problems. Bristol-Myers, which has invested $1.2 billion in ImClone, wants to restructure its commitment to invest up to $800 million more in the company. And it is demanding that the management of ImClone step aside, at least until regulatory matters regarding Erbitux are resolved.
ImClone said it was planning a response to Bristol-Myers, which also insists on taking the lead on Erbitux with the FDA.
The companies had heralded the drug as a financial blockbuster, with sales of $1.5 billion by 2005. Although those predictions attracted investors, cancer patients were drawn by well-publicized results of a clinical trial in which 22.5% of 121 patients responded to the combination of Erbitux and a chemotherapy drug. ImClone received as many as 400 calls a day on behalf of patients seeking the experimental medication.
The FDA, however, told ImClone that its drug application did not sufficiently document that Erbitux, rather than the chemotherapy drug, produced the results. ImClone Chief Executive Samuel Waksal has said the documentation sought by the agency does not exist.
