TORONTO – Whether it’s President Trump obtaining the drug Regeneron or COVID-19 test kits fast tracked in the U.S. and Canada, this has been the year of temporary emergency approvals for drugs and medical devices. What is sometimes overlooked are permanent programs like Canada’s Special Access Program (SAP) and the U.S.’s Expanded Access Program, designed to provide therapeutics to patients who have exhausted every avenue for a cure or relief from a devastating disease.

“A request to SAP for an unapproved device or therapeutic is not initiated by the companies, but by the clinician and patient,” Joel Ironstone, a medical device consultant and president of Toronto-based Ironstone Product Development Inc., explained. “The issue is why wait for long, multiphase trials when there’s absolutely nothing for these patients.”

The burden of proof

In Canada this year, several COVID-19 test kits were rushed into use, while ventilators were carved out of airplane manufacturing facilities and medical simulator assembly lines. Similar efforts have been made at General Motors assembly plants in the U.S. According to Ironstone, it helps when regulatory agencies are also teed up to aid patients in dire, life-threatening circumstances.

“Let’s say my patient has a particular type of brain aneurysm that current devices don’t address appropriately,” Ironstone told BioWorld. “I may also know that a company has developed a new one in clinical trials that could be of immediate value to this particular patient.”

Health Canada puts the onus on physicians to make the risk-benefit analysis of a particular device, Ironstone noted, raising the question of how aware doctors are about what technology is currently under development and how it works. Rick Hu is an orthopedic surgeon who ran Toronto-based medical device company Vivametrica before it was purchased this year by Toronto’s Sprout Wellness Solutions Inc.

“There are certainly instances [in which] specialists understand new technologies or therapeutics and understand the rationale for the program,” Hu told BioWorld. “But, for the most part, general practitioners are simply trying to care for as many people as possible with good quality technologies that are already available.”

Ironstone noted that Health Canada exercises particular care to avoid issuing special licenses to companies “when it looks like they are trying to use it as a replacement for appropriate licensing.” Nor can data gleaned from special access cases be used to support approval of a device downstream, he added.

Unexpected dangers

Joe Fisher, an anesthesiologist and co-founder of Ontario-based COVID-19 test kit developer Thornhill Medical Inc., has a decidedly different view on programs offering a temporary reprieve from normal regulatory processes. “One normally expects a very thorough investigation and assessment, and if you do shortcuts, all sorts of dangers may be lurking in the corners,” he told BioWorld.

Fisher cited the example of COVID-19. “Asking people who make cars to make ventilators is ridiculous. They can make something in a few weeks that pumps gas, but it takes years and great expertise to make a ventilator for humans who are sick.”

Opposed to devices chosen randomly to treat very sick patients, Fisher takes a more lenient view of technologies that are well developed, have demonstrated positive results, and are just short of entering clinical trials. “And I’d also be more lenient if the device or drug is well known, and you want to try it for a new indication because you think it might help,” Fisher said.

Ironstone’s broader concern, derived from early HIV investigations, is the impact programs like SAP might have on clinical investigations generally. SAPs, he said, have the potential to draw patients away from participating in clinical trials and losing valuable data as a result.

“Would you rather your doctor request special access where you are 100% likely to get the therapeutic, or would you rather participate in a randomized study where you have a 50% chance of getting access to a device?” Ironstone asked. “Because of this, there is a potential for too many special access cases cannibalizing our ability to obtain data from novel therapeutics.”

“It is a danger,” agreed Kullervo Hynynen, Toronto-based Sunnybrook Hospital’s vice president of research and innovation. “I mean, if I were a patient, and I had a pathway to the device, I would definitely take the 100% chance over the 50% chance.”

Conflicts of interest?

Hynynen, like other experts, stressed the importance of weeding out companies trying to do an end run around normal regulatory review processes. More likely to occur, he told BioWorld, is the failure to understand how an SAP works.

“A doctor is perhaps the inventor or part of the team developing the device, in which case there could be conflicts of interest very quickly,” said Hynynen. “In that kind of circumstance, it becomes very difficult to use this kind of an exception during regulatory reviews.”

Above all, what’s needed, said Ironstone, are entities like SAP providing greater clarity on the information manufacturers need to provide on the extent of the safety and efficacy of a medical device considered for patients in dire need.

“That would be useful for both manufacturers and clinicians,” said Ironstone. “Clearly, some Health Canada documents on that would be helpful.”

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