A prominent attorney, Frederick Baron, who is dying of bone marrow cancer, has been granted his wish, to receive the drug Tysabri. After significant wrangling, The FDA and the Mayo Clinic found a legal basis to give him the drug in an emergency situation, even though Tysabri is not approved for any cancers.

This decision by the FDA and the Mayo Clinic culminates after a frantic time which included very prominent friends of Baron, including former President Bill Clinton, cyclist Lance Armstrong, and U.S. Senators Ted Kennedy and John Kerry, all urged Biogen Idec, the manufacturer of Tysabri, to provide the drug. Prior to this decision, Biogen refused to provide the drug, because Tysabri is not approved for multiple myeloma, Baron’s disease. Biogen’s refusal was based on their concern that providing the drug outside of a controlled clinical trial might create additional risks that the drug might be restricted for patients who need it for multiple sclerosis and Crohn’s disease for which it is currently FDA approved.

Biogen, despite receiving many phone calls from Baron’s politically connected friends as well as from many other concerned citizens, never re-considered its decision not to provide Baron with Tysabri. As pressure increased, the FDA informed Biogen that it was supporting Baron’s request and trying to resolve the situation directly with the Mayo Clinic.

Although Baron has now been supplied with Tysabri, it remains unclear how the drug is being provided, or who is assuming liability in case something goes wrong.
What makes this situation unusual is that normally, with an approved drug like Tysabri, physicians are free to prescribe it for any disease, or any dose, they see fit, under what is known as “off-label” use. However, Tysabri presents a more complication situation as it is currently available only under a strict prescribing and monitoring program called Touch; the program was instituted when Tysabri was brought back to the market in July 2006 after having been withdrawn in 2005 because of its link to a rare and often fatal brain infection called PML. Despite these additional controls, an estimated 32,000 patients worldwide who suffer from multiple sclerosis and Crohn’s disease now take Tysabri.

Samples of Baron’s cancer cells carry a molecular marker that Tysabri, an antibody drug, is designed to seek out and block. So, some of his physicians thought that it could be an effective drug to treat his cancer. Additionally, according to a letter that Andrew Baron wrote this past Monday, Biogen has done some animal tests that suggest Tysabri might be an effective treatment for multiple myeloma, his aliment. There is also some evidence that Biogen has begun treating the first patient in a clinical trial of Tysabri against multiple myeloma.

The issue of liability has been a key question in this case. Andrew Baron, Fredrick Baron’s son, has claimed that the FDA had assured Biogen that it would be protected from legal liability if it gave Baron the drug. Andrew claims, “The FDA said that whatever happened in this particular case with my father would not be counted against them. This is similar to a case which becomes expunged.”

An FDA representative, in a written response, said it never offered Biogen a liability shield. “The FDA has provided no such assurance to the company. Our policy is to consider all available information when evaluating the safety and effectiveness of drugs,” the spokesman told Pharmalot, a blog “We recognize and appreciate that expanded access programs involve less-controlled use of experimental treatments than the well-controlled environment of a clinical trial, and thus we would consider information that might be obtained in this instance within that context.”

This dispute raises many issues. Clearly, the most obvious is that is Mr. Baron being treated differently by getting Tysabri because of who are his friends. Can just regular people like us expect to get this type of special treatment? The answer is no, as an example, try to get Provenge, which has demonstrated safety and efficacy for advanced prostate cancer, outside of a trial. You cannot. If I had the same friends that Mr. Baron has and they advocated for me, I do believe I could obtain Provenge outside a clinical trial.

The FDA does have a legal justification for granting compassionate use of unapproved drugs, but it cannot require pharmaceutical companies (pharms) to release the drugs to patients. Our current society is just to “sue crazy” and the pharms are rightfully gun shy. What makes this situation even more interesting is that Fredrick Baron has been a very active attorney who has sued many pharms for injuries caused by the off label use of drugs. Now, he is clamoring for the right to use a drug off label as it might save his life. You cannot have it both ways. On the other hand, is Biogen’s resistance to giving Baron access to Tysabri a form of pay back for his prior litigation against the pharms?

Maybe, the solution lies with our legislators. There needs to be legislation that would put some teeth into the compassionate use regulations, so that we could actually see the program used. It has come time to protect pharms from liability when doctors off label prescribe their drugs. It should be the physician’s obligation, when prescribing off label drug use, to make it very clear to the patient that the drug being prescribed has not been approved for the particular illness. The patient needs to understand all of the known potential risks, including the fact that there has not been any peer reviewed evidence that the drug in question has demonstrated any efficacy for the ailment being treated.

Why is this issue more than just an abstract discussion for us prostate cancer survivors? Most of the treatments we use in what we think of as the normal course are actually off label! How many of us take Proscar or Adovart for prostate cancer? What about DES? These are just a few of the drugs we regularly take that are not FDA approved for the treatment of prostate cancer.

Joel T Nowak MA, MSW