A confrontation between patients and government over access to a potentially life-saving cancer treatment is set to end with an announcement that Ontario will join other provinces and provide expanded access.
"This is the option that I think makes real sense to me," Ontario Health Minister Deb Matthews said in a telephone interview over a new program to be announced Monday. "We owe it to people who have a rare condition or a condition that doesn't fit into the established criteria."
Ontario's move to create a new program comes almost two weeks after The Globe and Mail revealed how Jill Anzarut, 35, was denied Herceptin because her tumour, at 0.5 centimetres, was deemed too small. Only tumours larger than 1 cm qualified for the drug.
In contrast, patients with small tumours were able to obtain Herceptin in British Columbia, Alberta, and Saskatchewan and on a case-by-case basis in Quebec, Manitoba and Newfoundland.
Ms. Anzarut's story elicited passionate debate: Policy experts said there was only so much money; breast-cancer activists were incensed at unequal access to treatment; and a government said its drug funding wasn't about to be determined by a newspaper story.
And late last week, Ontario's Ombudsman decided to investigate whether the government's decision to restrict the drug from such patients was informed and reasonable.
What it really came down to, however, was one question best answered by medical oncologists and scientists: Are breast-cancer patients with small HER-2 tumours at a high risk of recurrence?
"The risk is almost one-and-a-half to a twofold higher risk of recurrence," Stephen Chia, chair of the British Columbia breast-tumour group, said of HER-2 positive breast-cancer patients, compared to HER-2 negative patients with similar-sized smaller tumours. "In HER-2 positive cancers, it's not the size that drives it; it's the HER-2 gene that drives it."
When given with chemotherapy, Herceptin halves rates of recurrence within four years of diagnosis in patients with HER-2, which affects 20 to 25 per cent of breast-cancer patients. The medicine has turned one of the most deadly forms of breast cancer into one of the most curable but it is costly: about $40,000 for a course of treatment, which lasts about one year.
In Ms. Anzarut's case, her oncologist had recommended Herceptin and had made a special request to the province, which was denied. The Toronto mother of two has already undergone breast-conserving surgery and has begun chemotherapy; it was recommended she start the drug in May.
She is like an estimated 100 Ontario women each year who are ineligible for the drug for falling outside Cancer Care Ontario's treatment guidelines, which are based on well-run, large scientific studies. In the case of Herceptin, however, there was a knowledge gap: since studies only enrolled patients with tumours larger than 1 cm, oncologists were faced with a maddening dilemma on how to treat patients with smaller tumours.
That's because those designing clinical studies wanted to get their answer on whether Herceptin worked as soon as possible. So they focused on patients at the highest risk of recurrence in the five years after surgery, which were patients with cancers that had spread to the lymph nodes. Those with tumours of 1 cm or less were not able to participate.
More recently, four published retrospective studies in British Columbia, the United States, Finland and Italy revealed that small, stage 1 HER-2 positive breast cancers had an increased risk of recurrence.
And a commentary published in Lancet Oncology stated that in a survey of 530 attendees at the 2009 San Antonio Breast Cancer Conference, 74 per cent of clinicians would recommend chemotherapy and Herceptin for a patient younger than 50 with a tumour .5 cm to 1 cm.
In a telephone interview Sunday, Ms. Matthews said she hopes the province will be able to work with other provinces to share data on this new group of patients so they can be studied in larger numbers.
When asked if the case of Ms. Anzarut revealed a gap in the system, Ms. Matthews said: "For me, yes and there have been others as well that force me to look at that grey zone."
That grey zone, she said, are those patients who do not fit clinical guidelines for a given treatment, but for whom there is building medical evidence suggesting a drug may be of benefit.
"I am absolutely committed to relying on evidence," said Ms. Matthews, who herself has a research background, as someone who holds a Ph.D. in social demography. "But I've also become more aware of how there are some conditions where you have to really look at the grey areas."
Carol Sawka, Cancer Care Ontario's vice-president of clinical programs and quality initiatives, said the program will provide expanded access of as yet undetermined cancer drugs to a new group of patients, who will then be followed and studied for clinical effectiveness and toxicity.
In that way, the provincial cancer agency and government can collect data and study these patients.
"Perhaps a drug that was originally intended for one purpose might be useful in another purpose. It was tested in one group of patients but it might be useful in another group of patients," said Dr. Sawka, explaining the rationale for the new program. "A clinical trial, sponsored by a manufacturer may never be done on that new group of patients so they are in a grey zone. The evidence isn't black or white but there is a strong signal that the drug might be beneficial in that circumstance."
Since the program is so new - it is being called an evidence-building program - Dr. Sawka could not speculate on what cancer drugs or how many drugs would be in the new program, adding that "we're still working on program parameters."
However, Ms. Matthews said she hopes to have patients on new drugs as early as May, adding that: "It's important to everybody that we get this moving as quickly as we can."