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An alternative Regulatory Framework for medical technology

Attending ‘The Real Deal on Stem Cell Therapies” at Science World this week, I was fascinated to hear the industry perspective on the future of Regenerative Medicine (presented by Gregory Bonfiglio). We heard about the obstacles that hamper medical technology as a viable investment for many, as 10-15 years of clinical trials are required before an intervention, such as stem cell based technologies, are approved and allowed onto the market. Only then can investors see a return. This long process, the translation of therapies from bench to bedside affects patient outcomes too. We then heard of an alternative Regulatory approach (see Hara, Sato & Sahara, 2014 for full details). As a Neuroethics researcher, currently immersed in the world of clinical trials, hearing about the Japanese approach to regenerative medicine regulations got me thinking…

At present, in the USA, Canada and Europe, all health technologies (including medications) must provide evidence from 3 phases of clinical trial before Regulatory approval. Only then can it be released to the market (see Figure1 for a rough summary). This process can take up to 15 years, and during this time only participants in the trials have access to the experimental intervention.

Figure 1: Current Regulatory Model

Clinical Trial summary

The new Japanese regulation, coming into effect November 1st, 2014, will allow for Conditional Approval earlier in the regulatory process. This will be granted to a regenerative medicine after safety has been established, and if indications of efficacy are present. Full evidence of efficacy is not required. Looking at the traditional model in Figure 1, this Conditional approval is based on evidence similar to that from a Phase 2 clinical trial. Conditional approval allows the intervention onto the market, and can be used by physicians according to their judgment, similar to fully approved treatments. In order to gain Full approval, there is a requirement for evidence of efficacy (akin to Phase 3) to be presented within 7 years.

So what does this might this mean in practice?

For patients who are waiting for new treatment options, particularly those who do not meet the criteria for clinical trial participation, they can try new things sooner, and from their own doctor in a regulated manner. This may reduce medical tourism, and ensure that patients receive experimental interventions that are safe, if not necessarily effective.

For Industry, it shortens the lengthy time schedule from investment to market, so investors can see a financial return earlier. This could open the market to more investors, more alternative research endeavors and more options on the market in future.

Yet, as one audience member pointed out, this might not be ideal for patients. Participants in clinical trials do not pay for the experimental treatment. In this new regulatory system patients/the health care system would pay for conditionally-approved interventions i.e. interventions that might not work. Is this ethical? And what would this mean for placebo-controlled trials? Who would volunteer to perhaps be assigned to a placebo group if they have access to the intervention outside of a clinical trial?

Public acceptance can offer legitimacy to a health technology despite a lack of evidence. We live in a world where homeopathy is ‘approved’ by Health Canada (due to it’s safety, and not, as one would assume, due to it’s efficacy), where stem cell tourism thrives on a public belief that the cures are being withheld from them, and a world where internet diagnostic sites are consulted more than one’s own family doctor.

Is it then reckless to allow ‘conditionally-approved’ medications onto the market where they can cultivate a legitimacy far removed from scientific evidence-based medicine?

Or is it more damaging to maintain a cumbersome and slow regulatory process that delays the translation of science, thus losing the public’s trust as they turn to a market of readily available, yet unscientific ‘quick fixes’?

Does allowing earlier ‘conditional’ access to treatments undermine the integrity of a scientific evidence base?

One hopes that conditional approval is regulated and clarified as such so that patients are fully informed of the status of their intervention options. And, on a policy level, one hopes that there are mechanisms in place to prevent abuses, that there are disincentives to marketing a product that later proves ineffective.

However it turns out, I am intrigued to see how the new regulations in Japan impact the face of regenerative medicine globally. While it may go lengths to address the tangible hunger for immediate access to experimental interventions, I hope that the integrity of the scientific method can withstand the enormous market pressures to provide ‘miracle’ cures.

Hara, A., Sato, D. & Sahara, Y (2014). New Governmental Regulatory System for Stem Cell–Based Therapies in Japan. Therapeutic Innovation & Regulatory Science.