Drugs now go through a three-phase testing process. Phase 1 – which tests toxicity, but not whether the drug actually works – typically involves a handful of patient volunteers; they all receive varying doses of the medicine and are closely monitored for their responses. The vast majority of new drugs never get beyond Phase 1, which takes about a year.
If a drug passes, it goes to Phase 2, in which researchers start to measure the drug’s effectiveness on several hundred volunteers over about two years or more.
Then, if the drug gets to Phase 3, more tests are done on as many as a thousand volunteers and information is gathered over at least three years about the drug’s efficacy and safety.
Phase 2 and 3 trials both rely on “randomized controlled testing,” in which volunteers are selected by lottery to get either the new drug or a placebo. To avoid bias, often neither the volunteers nor the testers know which version the volunteer is receiving.
Only after Phase 3 will regulators such as Health Canada and the U.S. Food and Drug Administration begin to assess whether the drug can be sold. And even then the drug can be rejected.
This is why the process of getting a drug from development to market can take 14 years and cost as much as $1-billion – nearly all of it funded by drug companies.
That system remained largely unchallenged until the 1980s and the advent of AIDS. As people began to die, and research went into high gear, activists mounted an aggressive campaign against the medical and pharmaceutical establishments. It was the first time people openly challenged the drug-testing system.
There were demonstrations for earlier access to drugs in development and protests against the use of placebos. Why, the advocates demanded, should terminally ill people volunteer for a trial where they only had a 50-per-cent chance of getting the new drug, especially if it showed promise?
AIDS activists “were not willing to accept doctors’ views on these matters at face value and just take it lying down,” said Udo Schuklenk, a professor at Queen’s University who specializes in bioethics. “‘Why should I not have the last say?’ they argued. ‘Why not throw the dice one last time?’”
Experts argue early access can be hazardous
Yet many medical experts resist such emotional appeals. They argue that opening more access to drugs can be dangerous, even to terminally ill people: They could end up dying faster, more miserably, enduring painful side effects. Some early recipients of artificial hearts, for example, came to regret it because of the suffering that followed.
“There is a tendency of people to simply believe that new drugs are better than the older ones,” said Trudo Lemmens, an associate professor of health law at the University of Toronto.
In the case of AIDS, the activists’ campaign had some success. Certain drugs were released during later-stage testing and the approval time for others dropped significantly. Cancer patients took note and pushed for similar measures. By the 1990s, the FDA and Health Canada developed “compassionate access” policies, allowing drug companies to release some promising medication to terminally ill people during Phase 2 and 3 testing.
But that still did not go far enough for people like Steve Walker.
Mr. Walker is a Florida-based scientist who lost his wife to colon cancer. He is also a co-founder of the Abigail Alliance, a Vermont-based organization named after 21-year-old Abigail Burroughs, who died of cancer in 2001 after her family spent years trying to get her early access to a promising drug.
The alliance sued the FDA in 2003, arguing that the agency’s compassionate-access programs were too restrictive, excluding far too many sick people and violating the constitutional right of terminally ill people to live. The group lost the legal action but the case sparked widespread debate and the organization is now pushing Congress to change how the FDA operates, by increasing early access to drugs and streamlining the overall approval process.
“Our position has always been that the data you need to make these types of decisions emerges when it emerges, and has nothing to do with phases,” Mr. Walker said. “If you have a drug that is either helping a small number of patients dramatically or a large number of patients somewhat or dramatically, then, for patients who have a terminal disease and no other options, that’s enough. And they should be allowed to make that decision.”
James Gowing, an oncologist in Cambridge, Ont., and past chairman of the Cancer Advocacy Coalition, has sympathy for people like Mr. Doherty, but he rejects the idea of allowing access to drugs as early as Phase 1.
“What are the chances that it will be effective? Almost zero,” he said. “It’s way too early.”