Remember - this is just an opinion piece written by someone who I do not believe has any health issues.
Author: David Kent, 04/15/14
http://www.signalsblog.ca/testing-stem-cells-in-the-clinic-a-role-for-publicly-funded-trials/
In February this year, the biggest stem cell trial for heart attack patients was begun and will involve over 3000 patients from 11 different countries in Europe.
The BAMI trial (the effect of intracoronary reinfusion of Bone marrow-derived mononuclear cells on all course mortality in Acute Myocardial Infarction) aims to demonstrate whether or not the transplantation of BM cells can help to reduce post heart attack mortality in patients. The BBC’s Fergus Walsh does a good job of breaking down for the public why the trial is being undertaken and also identifies some of the key uncertainties surrounding the trial.
I have been sitting on this article for quite some time trying to figure out which aspect of the trial was most interesting for readers. In the end, I decided to break it into two parts – one to explore the science in the trial (to come soon) and today’s to explore the idea of publicly funded trials (partial or full) to try and stir up some discussion on the Signals website. The BAMI trial, unlike the vast majority of clinical trials, is not funded by any pharmaceutical companies.
Quality of the trial’s data
Whether or not the involvement of companies in trials is a good thing is an age-old question (for example, see this article written by Tom Reynolds over a decade ago). It invariably inspires academic researchers to complain about the pharmaceutical industry biasing results towards their products and Big Pharma to complain about the lack of regulation and standardization in academia. Both are legitimate concerns as corporate interests have been known to get in the way of patient benefit (examples here and here) and academics are far from the paragons of truth they purport to be sometimes.
Size does matter
The BAMI trial is very large (3000 patients), very widespread (11 countries) and it has a relatively easy outcome to measure (mortality), which should lend it excellent statistical power. If successful, it is the kind of cell therapy that could save public health systems an incredible amount of money by avoiding costly and time-consuming post-heart attack complications. his is the scale of trial that would be difficult for a single pharmaceutical company to afford and results in a smattering of small trials that sometimes suffer from low statistical power, a correspondingly unclear picture of whether or not bone marrow derived cells are useful for cardiac patients and, in my mind, a lot of wasted money. The BAMI trial will get an answer – and at least we can move on with statistically sound data.
Funding the non-fundable
One of the big reasons why this trial needed to turn to public funding in the first place was that it involves a non-commercializable product (a patient’s own cells) – this is exactly where public funding needs to step in. In the current model, I cannot see how the private sector would get this kind of therapy into practice and that makes one worry and wonder about the number of possible treatments that are not even considered because our current system focuses on therapies and products that make money rather than save money (and maybe lives).
Overall, I see a very strong role for public funding of clinical trials and I think that Canada in particular has a strong record in regenerative medicine and publicly-funded healthcare, putting it in a position of relative strength to hit the ground running.
In the end, if we do determine that publicly-funded trials are the way to go, a large concern that remains will be addressed in my next post – how do we choose which trials get funded and what science are they based on – because I’m not so sure I’d spend my money on BAMI.
Author: David Kent, 04/15/14
http://www.signalsblog.ca/testing-stem-cells-in-the-clinic-a-role-for-publicly-funded-trials/
In February this year, the biggest stem cell trial for heart attack patients was begun and will involve over 3000 patients from 11 different countries in Europe.
The BAMI trial (the effect of intracoronary reinfusion of Bone marrow-derived mononuclear cells on all course mortality in Acute Myocardial Infarction) aims to demonstrate whether or not the transplantation of BM cells can help to reduce post heart attack mortality in patients. The BBC’s Fergus Walsh does a good job of breaking down for the public why the trial is being undertaken and also identifies some of the key uncertainties surrounding the trial.
I have been sitting on this article for quite some time trying to figure out which aspect of the trial was most interesting for readers. In the end, I decided to break it into two parts – one to explore the science in the trial (to come soon) and today’s to explore the idea of publicly funded trials (partial or full) to try and stir up some discussion on the Signals website. The BAMI trial, unlike the vast majority of clinical trials, is not funded by any pharmaceutical companies.
Quality of the trial’s data
Whether or not the involvement of companies in trials is a good thing is an age-old question (for example, see this article written by Tom Reynolds over a decade ago). It invariably inspires academic researchers to complain about the pharmaceutical industry biasing results towards their products and Big Pharma to complain about the lack of regulation and standardization in academia. Both are legitimate concerns as corporate interests have been known to get in the way of patient benefit (examples here and here) and academics are far from the paragons of truth they purport to be sometimes.
Size does matter
The BAMI trial is very large (3000 patients), very widespread (11 countries) and it has a relatively easy outcome to measure (mortality), which should lend it excellent statistical power. If successful, it is the kind of cell therapy that could save public health systems an incredible amount of money by avoiding costly and time-consuming post-heart attack complications. his is the scale of trial that would be difficult for a single pharmaceutical company to afford and results in a smattering of small trials that sometimes suffer from low statistical power, a correspondingly unclear picture of whether or not bone marrow derived cells are useful for cardiac patients and, in my mind, a lot of wasted money. The BAMI trial will get an answer – and at least we can move on with statistically sound data.
Funding the non-fundable
One of the big reasons why this trial needed to turn to public funding in the first place was that it involves a non-commercializable product (a patient’s own cells) – this is exactly where public funding needs to step in. In the current model, I cannot see how the private sector would get this kind of therapy into practice and that makes one worry and wonder about the number of possible treatments that are not even considered because our current system focuses on therapies and products that make money rather than save money (and maybe lives).
Overall, I see a very strong role for public funding of clinical trials and I think that Canada in particular has a strong record in regenerative medicine and publicly-funded healthcare, putting it in a position of relative strength to hit the ground running.
In the end, if we do determine that publicly-funded trials are the way to go, a large concern that remains will be addressed in my next post – how do we choose which trials get funded and what science are they based on – because I’m not so sure I’d spend my money on BAMI.