I’ll be the first to say the folks at the FDA have a hard job. It is
their responsibility to protect the public safety and certainly not
subject those of us who are ill to drugs that offer false hope or
tremendous risk. And when it comes to considering new drugs for
approval, I know how much they want to get it right the first time. The
last thing they need is to have to pull a drug off the market after
people have been hurt causing damage to the FDA’s credibility to
Congress.
I get all that, and what follows is not written disrespectfully. But my
glimpse the other day in Washington, D.C. into the process of
considering new cancer drugs left me feeling the FDA was operating too
conservatively when it comes to potentially lifesaving or
life-extending drugs.
It was also clear to me their advisors (or at least a majority of them)
were not as knowledgeable as we patients would expect. They seemed
unfamiliar with the new science being considered as well as the needs
of the community that would benefit. That was the case when the new
drug application on Genasense for CLL seemed doomed by the Oncologic
Drug Advisory Committee and the cancer leadership of the FDA.
My immediate reaction was that an injustice had been done. And the
faces of several of the nation’s leukemia super specialists confirmed
that, as the hearing ended. So what to do? Was this just a loss for
people like me with CLL, or is there a bigger concern that could make
it hard for anything but breakthrough cancer drugs to be approved? Are
congressional concerns on drug safety putting so much pressure on the
FDA that the bar is being set too high?
Am I all wet?
I called the VP of legislative affairs with the Leukemia & Lymphoma
Society (LLS) to find out. The answer was no, not at all. According to
George Dahlman, while they don’t endorse any particular drug
application, they generally feel the FDA drug advisory committees and
the FDA itself do not have enough expertise when it comes to reviewing
proposed cancer treatments that could offer incremental treatment
benefit. And they worry drug companies will not push ahead with new
drugs with the government taking such a conservative, and maybe
uninformed, approach. Drug development experts I’ve talked to
(scientists who care about saving lives) are now increasingly
discouraged. I am, as a patient and a patient advocate, discouraged too.
I’ve asked the LLS to tell me and to tell us as patients how our voices
can help. Are there congressional representatives and senators we
should talk to? Would the leadership of the FDA listen to us?
Because of my business endeavors, there are a few things you should
know. HealthTalk has, in the past, produced programs on CLL treatment
that were sponsored by unrestricted grants from Genta, the developer of
Genasense. While there are no current or planned programs, you should
know that the possibility of future programs exists. In addition, I now
run a company called Patient Power that received an unrestricted grant
from Genta to facilitate the travel of three people (myself, a CLL
patient’s family member, and another CLL patient) to the public hearing
last week. Both at HealthTalk and at Patient Power, Genta has neither
prompted nor had any control or influence over what we say or write.
Speaking personally, as a CLL patient, I have nothing to gain with
Genasense being approved. Right now, I need no medicine. Certainly,
there are other patients who do need it. But putting that aside for a
minute, what about the next cancer drug hearing? Will the panel clearly
understand the issues and have expertise in the relevant cancer area,
and will the FDA cancer drug leadership understand? If they don’t, will
the treatment you need someday (or perhaps a senator or congressional
rep may need) not be available because of the cold water being thrown
on drug development now? I am worried.
One other thing: I very much appreciated having the opportunity to
testify at the recent FDA committee hearing. They listened
respectfully, and it was a very open hearing. But there was a part of
the process that seemed improperly behind closed doors. As the hearing
ended, I questioned an FDA official about who they drew on for CLL
expertise. She said they had phone conversations with CLL consultants
but that they were under confidentiality agreements and prohibited from
revealing who they were or how the FDA was advised. She said I’d need
approval from the drug applicant company to get more information. I
checked with the company, and they said I have their permission.
So now I’ll go back to the FDA to learn more about the process and
share it with you. I am just trying to learn if the process is broken
and our hope for new medicines is at-risk. Stay tuned and let me know
what you think.