Science supports new dietary guidelines limiting alcohol consumption
Cancer patients left in the lurch when some drugmakers stop clinical trials
Every year, tens of thousands of people participate in clinical trials of cancer drugs, some of which lead to FDA approvals - 61 of which occurred in 2018 alone. What happens, though, to those patients after the trial has ended, and the drugs have been approved?
In many cases, after an arduous battle with cancer, trial patients are told by the drug manufacturer that they must get their life-saving treatment from a "commercial supply" - meaning pay out of pocket or hope their insurance covers it.
This moment is devastating to many of my patients. After devoting themselves to the trial requirements and jeopardizing their own health for the benefit of others, they are faced with the realization that access to their life-saving solution is being taken away.
I first met one such patient a decade ago, when she was rushed to the hospital with a diagnosis of leukemia.
She had gone to her primary care physician for an annual checkup that went south in a hurry. Her liver, which should have just poked its nose out from under her ribcage, was gluttonously occupying a large part of her abdomen. This led to the discovery that her white blood cell count was 50 times the normal count.
Her doctor had to make one of those phone calls that my colleagues and I dread making, and our patients never anticipated that they would ever receive.
The one that ends with "You have leukemia."
It wasn't the first time she was told that she had cancer. Fourteen years earlier, she had been handed a diagnosis of Hodgkin lymphoma and the reassurance that it was at an early stage and could be cured with radiation.
And in fact, she was cured. She returned to her family, and her office, and went on with life. She underwent regular scans to confirm her remission, and then just yearly X-rays.
But radiation therapy can cause second cancers, like leukemia.
Lying in her hospital bed, she had a "Here we go again" look on her face. She had been waiting for the other shoe to drop on her cancer course for over a decade, and had prepared for it to happen, as best she could.
Additional testing uncovered a genetic mutation in her leukemia cells indicative of what's called the Philadelphia Chromosome, which defines the diagnosis of chronic myeloid leukemia (CML).
CML is the equivalent of winning the bad news lottery because a miracle drug that targets the genetic mutation had been approved by the FDA just seven years earlier.
We discussed treatment options. In addition to the approved drug, two other drugs had been approved the year before for people who could not tolerate the first drug, or for whom it didn't work. We discussed if she'd be interested in participating in a clinical trial comparing one of those to the first drug.
This patient, like many who selflessly agree to participate in these studies, was a believer in how medical advances could help people. She felt she had benefited from such advances herself. Now, she wanted to be a benefactor, and contribute to science that might help others. She agreed to enroll in the trial.
It did not go smoothly. After starting her pills, which were provided to her by the drug manufacturer, she developed gastrointestinal bleeding, which led to a hospital admission and blood transfusions. Later, she had nausea, headaches, infections, and even developed congestive heart failure. But she soldiered on, for the good of herself, and of others. Her liver shrank back to its usual size, her blood counts normalized, and she achieved remission.
What's more, she maintained a positive attitude, throughout the trial. She never skipped her pills, and never missed a study-mandated appointment, procedure, or laboratory test, even after she moved hundreds of miles away from our cancer center.
It was this type of participation and commitment to the trial requirements - from all trial participants - that ultimately led to a change in the approved use of the drug to include people newly diagnosed with CML. Quite simply, these clinical trial participants risked their lives to confirm the drug was safe and effective for others fighting CML.
This joyous moment was followed by sobering news. My patient would no longer receive the pills from the study sponsor. She would have to obtain "commercial supply." Either her insurance would have to pay for them, or she'd have to buy her own, at a cost of tens of thousands of dollars per year.
My patient devoted years of her life to participating in this trial yet, after the study achieved the sponsor's goal, her chemotherapy was unceremoniously pulled away, and she was left alone to treat her leukemia.
In 2018, the company that manufactures her treatment enjoyed over $1.8 Billion in sales. They could easily afford to continue providing the pills to the study participants, but they chose not to. In fact, their position statement on post-trial access refers to the ethical obligation they have to provide the drug until it is commercially available.
I guess that's when the ethics ends. It's a rotten thing to do to people who have jeopardized their own health in hopes of helping others. Because that's the kind of altruism that motivates many people to join clinical trials.
Through great effort and steep co-pays, my patient was able to continue her cancer treatment. She took it all in stride and felt fortunate that her illness could be treated with a pill, which she credited for restoring her to a cancer-free state. Not all of my patients are so lucky.
I just wish she could have felt equally valued by the manufacturer of that pill.
Mikkael A. Sekeres M.D. is a professor of medicine and director of Leukemia Program, vice-chair of clinical research at Cleveland Clinic.