Friday, September 28, 2012

Letter to a Friend- Ibrutinib Access and Other Unmet Needs

Here is an edited excerpt of a letter that I sent to a long time friend who lives near the Canadian border in northern Washington state. I cut out the personal stuff. 

Since my dad's illness and passing, I have not been up to visit much so I wanted to bring him up to pace.

It gives a decent executive summary of my last year with CLL. I have cut some corners with the data and details, but it paints the picture, and it at least for me, it is a prettier picture these days than it has been in a long time. 

After the letter is my commentary of what I believe are the significant unmet needs as related to ibrutinib and other similar drugs. I talk  mostly about ibrutinib because it is what I know best, and believe at least for me was the best possible choice.


It’s been too long since I last brought you up to date on my latest “trials” with leukemia in particular and life in general.

It has been a wild ride for sure. Your parting wish to go with gusto was certainly prophetic.

Despite my best efforts to make peace with my cancer with a calming diet and a calmer lifestyle, my evil clone has mutated to a more aggressive form. Fatigue, huge nodes, and malaise.

In one of my more prescient and courageous acts, I was fortunate to win a fight with my HMO to get authorized to grab one of the last openings in clinical trial at Ohio State with what now appears to be a game changing medication. I have been commuting to Columbus for months now. My nodes melted to half their size in the first 30 days while stamina and sense of well being rebounded.

My new miracle pill, ibrutinib doesn’t kill the cancer, but it controls it- similar to insulin in diabetes. Stop taking it and the cancer can jump right back at you.

You would appreciate the way it works. It blocks critical communications with other blood cells. Think a whole cell condom. Cancer birth control. The cancer cells lose their support network, become unanchored from their safe niches in the lymph nodes and bone marrow and float aimlessly in the more exposed and vulnerable reaches of the blood stream. At first, the white count shoots up as the cells depart on mass from their lards, suggesting disease progression, but a few months later the counts falls as the clone dies of loneliness (actually lack of pro-survival and pro-proliferative signaling). And the more aggressive the cancer, the more it likes the crosstalk, and so the pills may be most potent on the worst leukemias.

Response rates are > 90% in tough refractory and relapsed patients. And because it is not a killer, there are few side effects. It’s still an early story, it’s probably not a cure, and it’s only available in trials to the many who need it. And many more need it than can get it.

Be well


Access is the big problem now. And it may persist into the future as well. 

I am doing great, and so are many others I know that are in clinical trials at OSU or the NIH or elsewhere. But thousands are not doing so well and might benefit from ibrutinib and others like it. For some, there time is running out. The many emails and posts about how to get into a trial or compassionate use are testimony to this tragic unmet need.

There are other needs too. Less pressing today, but maybe more significant in the long run.

We need robust data. For how long and for how many will it work?  Are some combos better than others? Will new side effects emerge down the line?  Who will relapse and how will those relapses look? These are only some of the many unanswered questions. The answers will take time, often many years. All of it will take carefully planned and conducted trials. That means strict inclusion and exclusion criteria. That means that too many patients who have exhausted their options will have no ibrutinib or similar trial to turn to.

We need to get this and other similar drugs FDA approved as soon as possible. That starts by filling all the spots in the pivotal trials, even when the chances to get ibrutinib are only 50/50. The phase III trial is the acid test, the potential proof that this BTK inhibitor meets, at a predetermined significant level, an unmet need or that it exceeds what is out there in terms of an agreed surrogate markers for what we all really want - longer progression free survival. Right now no-one has been on it much more than two years, and only a few hundred people in the whole world have ever swallowed the battleship grey pills. It’s a small and short cohort. We have so much to learn and the phase III data will be a huge boon.

Once it is approved, probably in a few years, the restrictions then would be from the payors (Medicare and private insurers) who might not limit the use of the drug, but who could refuse to pay for any therapy that even slightly deviates from those for which it was licensed. If it is priced anything like Revlimid and other new oral cancer drugs, that could effectively put the drug, out of reach for most of us. We would either have to match the exact indication on the package insert and perhaps even more age and health restrictions demanded by the insurance company, or we would have to be very rich. Accordingly, we may need to push to improve access again, even after it hits the market.

And that is not the end of the story. When it on the market we will still need to ask our friends in the pharmaceutical companies and the research institutes to keep looking for better drug combos and new compounds so we can cure our disease once and for all. 

We all need to work together- patients, providers, advocates, payers, academics, government, foundations, and pharma to get this done.

We always know it would be a long journey. And the pace is frustratingly slow, but for the first time ever, we just might be seeing the finish line. And that is a very pretty picture indeed.

Please let me know what you think, what needs you see, and your ideas on how we might meet them.


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