Improved access to important new types of drugs

[Guest guest]

CAL-101 and PCI-32765 are the door to curing , or maintaining stable, CLL.

The big difference between the drugs we are used to hearing about is that

they accomplish their task by killing, sometimes selectively, cells. The new

drugs do not kill; they alter the messaging within a cell, get at the

basic genetic flaw causing the cell to misbehave, and try to cure it.

If you think about it, does this not make a lot more sense than some of the

poisons we take? I have always thought that cancer cure will involve

genetic alteration within a cell, rather than simple cell destruction with all

the horrible side effects. There is a huge difference between the two

processes: ancient thinking vs. modern thinking.

The problem is complicated by the FDA. The FDA approvals are based

primarily on not just benefit. The two criteria used are:

1)greater benefit than the currently approved therapies (requires head to

head comparisons)

2)unmet medical need (nothing approved to compare with)

The FDA has suggested that it will not allow any drugs to be further

approved for CLL using the unmet medical need criteria. This is how alemtuzumab

and ofatumumab have gained approval. Thus, a trial of drug X in patients

who have disease that is refractory to fludarabine, bendamustine,

alemtuzumab, and ofatumumab will not lead to approval. Even if this agent (like

PCI-32765 or CAL-101) has a 90% rate of clinical benefit / response.

Likewise, for some very unexplicable reason, the FDA will not allow

multiple drugs to be approved at the same indication. There are 25 different

anti-hypertensives and 10 different cholesterol lowering agents. They did not

have to prove themselves better than the currently available therapies. Just

that they were efficacious. Why not the same thing for oncology drugs?

Why can we not just show CAL-101 benefit patients and get it approved?

Additionally, the FDA will not approve an agent that is equally effective,

but less toxic over the current standard of care. This would require a

large study secondary to statistical reasons, but once done, would be a much

easier means to approve these new agents.

These are just two of the glaring issues where the FDA does not function

in a manner that is in patients' best interests. People have been

complaining or criticizing the pharmaceutical companies, physicians, etc., but

all

anyone wants is to get excellent agents to the market as quickly and safely

as possible. A less burdensome system would also encourage more novel drug

development. It could be a simple as once a drug shows efficacy and safety,

that there be programs to allow physicians to use these agents in all

patients while closely monitoring further data on safety and efficacy. Would it

not be great if anyone who needed CAL-101 or PCI-32765 could get it from

their local oncologist?

I think this is a good opportunity to build a grassroots effort to change

the way the FDA makes these decisions. Let us propose starting a campaign

to address these inadequacies in drug development. CIG could try to take the

point with LRF's advocacy help. By the way, this is Dr. Furman's strong

opinion.

Your thoughts, or actions, would be very welcome, this is important, we

need to move ahead.

[Guest guest]

,

I don't live in a highly populated area and don't know of many other CLLers

near me, but I'd be willing to help with such an effort in any way I could.

Karni

I think this is a good opportunity to build a grassroots effort to change

the way the FDA makes these decisions. Let us propose starting a campaign

to address these inadequacies in drug development. CIG could try to take the

point with LRF's advocacy help. By the way, this is Dr. Furman's strong

opinion.

Your thoughts, or actions, would be very welcome, this is important, we

need to move ahead.