All Good Studies Must Come To An End

Way back in the 1960's the National Cancer Institute, as part of their larger program for finding and evaluating anti-cancer agents, found that an extract of a particular sea squirt was capable of killing cancer cells.

That extract was licensed and a means of synthesizing it (instead of crushing up a shitload of sea squirts) was developed. It was brought to testing and eventually to market. It's called Trabectedin, brand name: Yondelis.

In Europe, Russia, and South Korea, it is approved for treatment of folks with soft tissue sarcomas. Like my wife, they've either received other treatments that have failed, or are too sick or weak to receive. For some, Trabectedin is the last, best hope for a working treatment. In the U.S., the FDA did not approve the drug under its original study (based on treatment of ovarian cancers) and requested additional Phase III studies be done first. Currently there is a "making available" study which provides access to it for those who have exhausted other treatment options.

Unfortunately, insurance (even the gold-plated insurance I have as a gummint employee) will often say they will refuse to pay for study-related care. This means that the patient will usually end up bearing the cost burden of infusion services and pre-medications associated with the study among other things, though the drug is provided by the study free of charge. Catch-22, right? Choose a paid-for treatment unlikely to work, or take a chance on a study treatment and pay out-of-pocket for everything except the drug?

How's that for a health care gap? And to make it even more interesting, look at the prices. Somewhere around ~$3500 for nurse's services (accessing the patient's mediport, checking vitals over infusion period, etc), infusion services (infusion pump fee, iv tubes and bags), and facilities fees (paying the rent, supplying sundries like alcohol wipes, etc.) If the chemotherapy is on a 21 day schedule, you're looking at over (365/21)*$3500 = $60,833 a year. Don't sweat it too much, though, by the time a patient is participating in clinical trials, survivability is often well below a year. This isn't a fault of the studies or the drugs, but just a hat tip to human nature. People don't choose to participate in studies until they are desperate. They (and their doctors) prefer to work through the approved and non-study drugs unless there is a really compelling reason to do otherwise. More often than not, the compelling reason is that everything else has failed, although rarely there is a breakthrough for a particular cancer that justifies it.

Want to hear something interesting, though? In oncology, if the chemotherapy drug is FDA approved, usually the insurance company will pay for it, even if it is being used for a cancer other than those for which it was approved. From an oncologist's standpoint this is often justifiable because there will be other protocol studies after a drug is a approved, testing the delivery processes, intervals, and dosages for other types of disease. If a study shows early results that a drug is particularly effective against a certain cancer, oncologists will share that information so they can offer their patients better survivability and quality of life. My wife's oncologist is one such person. He stays very connected to other oncologists and conferences. Georgetown University Hospital is (obviously) a learning hospital and the Vincent Lombardi Cancer Center affiliated with it has staff and doctors that are absolutely stellar people. Unfortunately, Georgetown doesn't have access to the Trabectedin study, so her oncologist referred her to Fox Chase Cancer Center in Philadelphia, PA for it.

She's on her third course of the stuff and while it might have been marginally effective at stabilizing her disease (some tumors shrank, some grew a bit, and a couple of new ones showed up,) it is proving to be too dangerous for her to continue treatment with it. Her blood counts drop too low for her to make it between 21-day cycles without frequent transfusions and fluid retention (could possibly cause congestive heart failure.) Additionally, while the medical staff, nurses, and doctors at Fox Chase Cancer Center are very nice and knowledgeable, we encountered so much red tape from their administration staff that it has been difficult to build a strong and trusting bond with their organization. Trust is key in the patient-hospital relationship and she just hasn't been able to trust them the way she does Georgetown.

Here's an example: we've been married almost three years, but in that time she's either been too sick or we've been too busy to go to the DMV for an ID card with her new name on it. Georgetown just put in her married name, no problem, back when we first visited them. Whereas Fox Chase absolutely would not, could not make that change without a picture ID with her new name, even if we provided the marriage certificate. It didn't matter where the request came from, doctor, nurse coordinator, administrator, without a picture ID, they refused to put in her married name. No big deal, you say? Well, that might be true, if they weren't requesting records and lab results continually under her maiden name. This causes the patient and the medical staff no end of grief as they end up trying to tie together loose ends and reconnect missing links. Utterly unacceptable, from a business process and patient relations standpoint. Rules are made to be bent and/or broken when given a well-considered justification.

It would be as easy as this: 

If an organization is so straitjacketed by their rules that a reasonable request to change the contents of a single field in a system doesn't provide enough CYA for the acting bureaucrat to feel comfortable doing so, it is no wonder that humans will not trust it. That's how we feel about Fox Chase Cancer Center. They have very good medical people, very friendly staff, and a miserable corporate culture. One possible reason is that Fox Chase underwent some financial restructuring and is working to become part of the Temple Health System, but that's purely speculation. FCCC may have had a miserable corporate culture well before any of that.

So, long story short, our patient is still in the hospital, with an expectation of discharge Sunday or Monday, depending on whether she'll need to carry oxygen home with her. She is ending her Trabectedin study participation. Her oncologist at Georgetown is working to find another study, a Phase I, appropriate to her disease and condition. She is taking a nap.