Drug companies are also leery about handing out too many drugs too early. It’s already hard enough to get terminally ill people to volunteer for some cancer-drug trials and it would become even more difficult if drugs were given away.
That would delay the already lengthy approval process and cost companies more money. As a result, most drug manufacturers have their own, and differing, policies on compassionate access.
“They are not easy calls,” said Edward Lang, a spokesman for San Francisco-based Genentech Inc., a division of Hoffmann-La Roche Ltd. “It really comes down to a company decision.”
Mr. Lang explained that compassionate access is easier during Phase 3 trials, when drugs are close to being approved. Most companies will open up a special trial for some terminally ill people in Phase 3 and essentially make the drug available to them (on approval of regulators). But every company has a different policy and some do not open early access at all.
To level the field, the University of Toronto’s Prof. Lemmens and others have proposed removing drug companies from testing altogether – by creating a national drug agency. Funded by a tax on drug products, the agency would manage testing and compassionate access.
“There should be an arm’s-length relationship between companies testing the drugs and the industry itself,” Prof. Lemmens said.
Meanwhile, though, companies such as Genentech have to decide what to do when they hear public pleas for special access. One came in April from Darlene Gant, a 46-year-old Florida mother. Ms. Gant had been told by doctors that her breast cancer had returned and was spreading rapidly. She did not have long to live, but she knew Genentech was in the final stages of testing a new breast-cancer drug called Pertuzumab. Ms. Gant made a tearful 20-minute YouTube video begging Genentech to give her access to the drug.
“So many women are dying of ovarian and breast cancer and they won’t release the drugs for compassionate use,” she said on the video. “My dream is hopefully that this video would wake somebody up.”
The video sparked a public appeal and Genetech relented – it managed to include Ms. Gant in a trial, Mr. Lang said.
Meanwhile, the drug industry and regulators are facing another kind of challenge: The whole concept of randomized controlled testing for cancer drugs has begun to change because of an ethical storm that erupted in 2010 over a melanoma drug known as PLX4032.
During Phase 2 testing of the drug by Roche, half the melanoma-suffering volunteers received the new drug; the others, the control group, were treated with chemotherapy, using a drug many doctors no longer felt was effective. The idea was to compare the new drug against one that had been in use for decades.
Two California cousins in their 20s, both with melanoma, came to illustrate the controversy. One, Thomas McLaughlin, joined the trial at the University of California at Los Angeles and got the new drug. As he watched his tumour shrink, Mr. McLaughlin encouraged his cousin, Brandon Ryan, to join the trial as well. But Mr. Ryan was put into the control group, and he died.
The drug was eventually approved, but the controversy remained, fed by New York Times coverage, over the ethics of knowingly giving patients a treatment that many felt did not work. Defenders said it was the only way to properly assess the new drug. Critics called for a new way of testing.
“Those of us involved in the trial were very unhappy because we thought [the chemotherapy treatment] was worse therapy,” Princess Margaret Hospital’s Dr. Hogg said. “And we’re not going to do that again.”
Now, those selected for control groups in similar trials receive the best available therapy (aside from the drug in question).
Mr. Doherty’s case pushes some of the limits further, raising questions about how early drugs should be released.
“There’s no bad guy here at all,” Dr. Hogg said. Bristol-Myers, he said, was concerned about accidentally hastening Mr. Doherty’s death, “and there’s the potential of doing that. And Darcy [was] worried about not getting a drug that might prolong his life.”
Ms. Cumming, Mr. Doherty’s wife, knew that she was breaking new ground. “I think this is new territory for everyone and that’s why we are really pushing compassionate access,” she said in an interview before her husband died. “It’s hard to see a drug that you know your husband has a really decent chance of responding to is just outside of your grasp. We are just so close and yet can’t get it.”
The question is whether there’s any way to answer that kind of pain and frustration without putting much more at risk.
Paul Waldie is a reporter with Report on Business.Report Typo/Error