This post is just one of many covering this topic as part of Signal’s second annual blog carnival about the theme Right to Try. Click here to read what other bloggers think.
You’ve likely come across debates about whether terminally ill people should have the right to die. The Right to Try (RTT) takes a step back and asks whether terminally ill people should have the right to try and save their own lives with experimental drugs.
Your gut reaction is probably to support the idea – at least mine was. Why shouldn’t people be allowed to take one last stab at it if they’re already dying?
It turns out, it’s just not that simple.
On the one hand, RTT is the most fundamental liberty there is: the right to fight for your life. On the other hand, as many researchers and scientists can tell you, most drugs that make it to clinical testing fail before approval. Is it really alright to test these drugs on humans who are hanging their last hope on the outcome?
Supporters of RTT claim a lack of compassion from RTT opponents, while opponents claims a lack of rationality from supporters. Both are right to some extent, as every case, every disease, and every sufferer are unique.
But there can be a more moderate approach to RTT: a reasonable compromise that answers both camps’ concerns and, if sensibly regulated and enforced, will produce the best outcome for everybody.
Current RTT legislation in the US allows patients to try any drug or treatment that has passed Phase I of clinical trials. In my opinion, this is far too liberal. Raising the bar to Phase III would produce more favourable results because:
- Since they’ve passed Phase I and II, Phase III drugs don’t pose nearly as much of a threat to the patient’s safety. Even if they don’t work, they are unlikely to harm anyone.
- The chances of the drugs working for the patient are much higher. If a drug reaches this stage of clinical trials, it means it has been successful for at least some of the test population.
- Taxpayer money or money raised by patients’ families won’t be wasted on Phase I drugs that are highly unlikely to work.
Delays mean deaths
Let’s be clear: we need an RTT Act in Canada. There is a huge amount of time needed for final regulatory approval of a drug, and in this case, delays means deaths.
Drug companies take an average of 10 years and require roughly $1.4 billion to develop new drugs, though revolutionary treatments often hit the $5 billion mark.
The FDA has a history of referencing US research in its approval process instead of considering the evidence and positions of international health bodies. Taking an unapproved drug is risky, but excessive caution can be just as dangerous.
Here are some examples from independent think tank Learn Liberty:
- Thrombolytic therapy uses drugs to dissolve blood clots that have suddenly blocked major arteries. The FDA waited a full two years to approve this therapy after its approval in other By some estimates, up to 22,000 Americans could have been saved within this time.
- Interleukin-2 is a treatment for kidney cancer. In 3.5 years, 3,500 people died while waiting for this treatment to be approved.
- Misoprostol prevents bleeding ulcers. This drug took a staggering 9.5 years to get the green light; during that time there were between 8,000 and 15,000 deaths that could have been prevented.
Of course, these are exceptional and dramatic examples, so despite their gravity, we must remember that the majority of cases are not like this – and hindsight is 20/20. The FDA’s ignorance of the international consensus on these drugs wasn’t wrong; the vast majority of clinical trials fail and they were undoubtedly doing what they considered their due diligence.
Health Canada’s approach is a little bit more relaxed. Their review process accepts some degree of influence from foreign professionals and papers, but any new drug proposed still needs to go through a good deal of Canadian testing.
Still, these procedures are a prime example of the need for liberalization of access laws.
The question is: how liberal do we make them?
A higher bar for clinical evidence
In my view, current RTT legislation in the US is too liberal in its approach. If Canada was to implement its own version, we need to be more realistic about the potential efficacy of clinical-stage drugs.
The Goldwater Institute, which spearheads lobbying for RTT in the States, claims that the destructive potential of drugs that have passed phase one of clinical trials is “extremely diminished”.
Diminished, but not eradicated. Experimental treatments that pass phase one but end up failing in later stages might not affect the patient at all, or they could be lethal. For example, the schizophrenia medication Zyprexa Relprevv from Elli Lily was identified as a serious safety risk in Phase III studies. There is potential for any number of complications or variables that researchers could not reasonably foresee. Though phase one is considered the safety phase, it is by no means the last word.
“Of 100 new drugs that go into Phase I trials, only about 20 move to Phase III. That’s because 50 per cent do not work, 20 per cent are outright harmful, and 30 per cent do not proceed for strategic reasons,” wrote André Picard in a 2015 Globe and Mail opinion piece.
Dr John Jenkins, ex-FDA Drug Development Program head, termed drugs that passed phase one a “Pandora’s box for potential harm to the patient”.
On the other hand, phase three drugs have some proven efficacy and therefore offer the patient a choice with a higher probability of success. The safety of such drugs is also more likely to be secure, given the extra rounds of testing and analysis.
This is the sweet spot: a sensible balance between compassion and rationality.
Expanded access is not enough
A common claim by opponents of RTT is that the opportunity to participate in clinical trials offers a sufficient alternative, but clinical trials, even for Canadian-developed treatments, are often not done in Canada, making opportunities to take part rare.
Canada does have the Special Access Programme (SAP), which allows doctors access to non-marketed drugs and medical devices that have not yet been approved for sale in Canada, but it has been criticized for having a review process that “uses its discretionary authority arbitrarily”. The scope of this program also extends to any experimental drug not yet approved in Canada, even those not yet passed phase one.
An RTT Act in Canada could replace SAP and become a more ethically and scientifically sound method for helping terminally ill patients.
Unfortunately, saving terminally ill patients is not the only concern when it comes to RTT; money is another issue. If publicly funded, these experimental treatments will lead to an additional burden on our healthcare system. If not covered by health insurance, RTT could lead to a huge financial burden on the families of patients that don’t make it.
Charitable organizations such as the RTT Foundation raise money to fund treatments for those terminally ill patients that are less fortunate but willing to take the plunge. Though their motives are admirable, much of their work may be wasted since the money raised could end up going to ineffective treatments instead of valuable research or support for the patients’ bereaved families.
By raising the bar for RTT to Phase III, we can ensure more of our hard-earned money has a chance to result in successful treatments.
Here in Canada, we have a right to die but not a right to try. The terminally ill can be voluntarily euthanized but they can’t try experimental treatments that could slow disease progression, improve their quality of life, or even potentially cure them, no matter how close to approval they are. Does that really make sense?
However, the hesitation of RTT opponents isn’t a callous denial; it’s based on rational anxiety over the destructive possibilities of extremely liberal RTT legislation. Setting the bar at Phase III and above will improve our level of aid while keeping patients safe.