A sad case all around …

I need to state a theoretical conflict of interests up front. I am a member of the Board of Directors of the International Myeloma Foundation. So, having said that …

Fred Baron, a well known Texas-based lawyer and the former finance chairman of John Edwards’s presidential campaign, has progressive multiple myeloma. I am not aware of the details of his case, but I have to assume that he has failed most forms of standard therapy currently on the market. On his behalf, his family had sought permission from Biogen Idec (the manufacturer) for him to be treated with the drug Tysabri (natalizumab).

The first question then is, why did they need anyone’s permission? Tysabri is an approved drug, indicated for the treatment of multiple sclerosis and Crohn’s disease, and is in very early-stage clinical trials for the management of myeloma. Surely his doctor could have just prescribed the drug off-label! Yes?

Unfortunately the situation with Tysabri is more complex than that. You may or may not remember that Tysabri was previously withdrawn from the market for a period of time while the company and the FDA carefully reviewed available data on the occurrence of (I believe two) cases of a rare disorder known as PML or progressive multifocal leukoencephalopathy. As one of the conditions of returning Tysabri to the market, it was agreed that Biogen Idec would be required to give explicit permission for any use of Tysabri outside its approved labeling. This may or may not have been a good decision, but it was the decision that was taken.

In reviewing Mr. Baron’s case, Biogen Idec decided, for a variety of reasons, that they could not give permission for the off-label use of Tysabri. Their reasons have been stated to include concerns that “if … Baron had bad side effects from the drug, the company would have difficulty getting approval for its use by other cancer patients. It also feared that doctors would become reluctant to prescribe the drug” for its approved indications in multiple sclerosis and and Crohn’s disease. Perhaps a more rational reason for their decision was a concern that adverse effects in Mr. Baron’s case might affect the progression of the company’s study of the drug in the management of myeloma.

Enter the heavy guns. Apparently a host of celebrities and others, from former President Bill Clinton and multi-Tour de France winner Lance Armstrong through a bevy of politicians and their staff and down into other political connections, then started to lobby Biogen-Idec on behalf of Mr. Baron and his family.

The roles of personal advocates in seeking to get specific drugs for specific patients who have exhausted other opportunities is well known and well understood, but most of us can’t call on lobbyists of this stature to assist us. Despite such advocacy, Biogen-Idec continued to say, “No.” As one report put it yesterday, “No one can accuse Biogen Idec of playing favorites.”

This morning we heard that access to Tysabri had been granted to Mr. Baron, which is good news for him and his family. And I sincerely hope that it is highly effective in his treatment.

Unfortunately, we also heard that, according to a spokesperson for Biogen Idec,
“[A]ccess to the drug was not granted by Biogen.” Rather, the FDA had authorized the company to give the drug to Baron.

This case raises a whole host of questions of an ethical and administrative nature. They are all, unfortunately, a consequence of our failure to deal well with a very fundamental issue. How can we make sure that patients, whether terminal or not (but particularly when  terminal), who have exhausted all other treatment options can get access to investigational drugs under appropriate controls through simple, easily understood mechanisms?

Now let me be clear, I do not believe that this is some sort of constitutional right, as advocated by the Abigail Alliance. Nor, apparently did the Supreme Court, when the Abigail Alliance asked the court to hear its case. However, I do believe that we need a much better and simpler mechanism to address this issue — and we need it soon. It seems grossly unfair to me that, because of Mr. Baron’s connections, pressure could be put on the FDA to do something which would probably never have been done for 99,999 patients out of 100,000. If nothing else, the unfairness of this decision should be a clear signal to the FDA and to our politicans that this problem needs to be solved — once and for all!