The issues that arise are big and very important. A recent discussion at LinkedIn's Cell Therapy Industry Group examines some of these issues, and it got me thinking. The Nobel laureate Dr. Ralph Steinman blazed a lonely new trail, and one that is worth examining. There's an excellent writeup in the New York Times ("Is the Cure for Cancer Inside You?") on how Dr. Steinman courageously used his own pancreatic cancer to further explore his treatment options. I'd highly recommend that you read that article before delving into the issues raised on LinkedIn.
As it stands, a terminally ill patient will have a very difficult time emulating Dr. Steinman's example. The regulations in most countries, and especially in the US, simply forbid an extremely individual approach to treatment. There are the standard, tested courses of treatment available to all, but stepping outside of approved treatment protocols & regimens is mostly prosecutable, could well result in a physician losing her license, and probably stiff monetary consequences.
But that leaves the terminally ill patient in a very unenviable position, for it is also acknowledged that following these "standard, approved", treatment options will result in fairly certain and fairly quick mortality. In other words, it's because the treatments are failures that patients are thought of as 'terminal'. Technically, it is possible for a patient in consultation with his physician to try to get approval for an experimental treatment, but that is a lengthy and laborious process (perhaps by design), and when time is of the essence...
What many patients opt for instead, is to enroll themselves in one of the many clinical trials revolving around their particular illness. Again, not a terribly exciting option, for there's a very big chance that you will end up as a 'control' and receive a placebo perhaps with the nonfunctional standard treatment. Even if the particular experimental treatment works for some of the patients in the trial, that might not be you. Even if it worked well in the trial, there's then no telling how long it will take for it to come through the regulatory processes and become an available option for you. One is left with the dubiously satisfying notion that by participating, one has perhaps pushed the cause of science a little further, and very likely at the cost of one's own life!
When we think of what Dr. Steinman achieved, he can be seen to have blazed a trail through the impenetrable jungle of federal regulation. His drive, focus, and stubbornness speak volumes and he achieved the near impossible. We are amazed, for even though he was in a unique position, able to direct his own well-funded research, and to request, and get, an incredible degree of cooperation from eminent specialist researchers from all over the world, he not only got away with it, he indeed seems to have succeeded in prolonging his own life well beyond the norm for the kind of disease that he was suffering from. His example is both inspiring and inspires despair in those who have not his level of influence and are not researchers in their own right.
And this brings up the question of ways and means. In short, at present there are none. The regulations are stultifying. Some of the very rich may be able force their way to their own experimental treatment (one thinks of a Steve Jobs), but for all the rest there are neither the funds, nor acceptable methodologies. Funds would probably be available if there was a clear path to commercialization, but when we are talking about individualizing therapy for the many millions who may be considered 'terminal' it is precisely the lack of any business model that is a big problem. One could go further, for if our cures are primarily found within ourselves, the days of 'big pharma' are probably numbered anyway.
Ideally of course, wherever business fears to tread should precisely be where the government steps in. Unfortunately, the whole picture of drug/treatment approvals has been hopelessly skewed to cater to the major business interests, and the entire regulatory mechanism simply sidesteps the needs of the hopelessly uncommercial 'terminally ill.' The poor patient, and that could be you or I too, just down the road - are left to the tender mercies of the licensed to kill 'standard, approved' treatment options!
The regulators are not just being stubborn. From the standpoint of regulating authorities like the FDA, one problem is accountability, as they are ultimately responsible for each life lost in a medical setting. A failed, poorly tested, poorly understood experimental treatment attempt, even when wholeheartedly entered into by a patient-physician combo, does not let them off the hook. It's also easy to see that the terminally ill patient population is particularly susceptible to quackery. Any hint of hope offered, can become irresistible, and can very easily be a conduit for parting the desperate patient from whatever funds they may have or be able to raise. Folks do need protection against such quacks.
What really is a federal regulatory authority's main purpose? An FDA is first and foremost tasked with safeguarding the health of an entire population of many millions - and that is what they are structured to do. They are simply not in a position to handle individual requests for individual therapies. The very nature of the highly individual immunology-physiology of most intractable diseases, the genetic variations that affect outcomes, and the immense number of potential treatments that could be opted for, means that blunt regulatory tools that work well for protecting large populations are completely (woefully) inadequate at an individual level.
I think it's possible to closely regulate the workup and counseling of patients with terminal illnesses, and still allow the patient and their physician to be the deciders on how to best proceed and with which particular treatment, however experimental. After ensuring a complete History and Physical is taken, and all previous medical records are on file, the data to be gathered and generated during the treatment can be rigorously specified so that each patient's progress can be carefully monitored while a meaningful database is developed which can be openly accessed and studied by researchers.
What it should boil down to, is that apart from setting some overall boundaries and demanding rigorous clinical records to be maintained, the regulatory authorities can safely stay out of fiddling with an approval process for each individual case. Certainly counseling can be required, both in the beginning and at regular intervals during the process of treatment.
Where does big pharma fit itself into this picture? Well, here is a troubling problem. For one thing, the present process by which drugs gain approval is heavily tilted towards a big pharma R&D to clinical trials scenario. Eventually, a drug that has gone through this process will be released for use by a huge medical community, and big pharma gets to recoup their costs and then make a healthy profit.
In spite of this, big pharma could well participate at an individual level and even help with providing funding and/or material support in the form of therapies under development. Unfortunately, and understandably too, most companies are leery of allowing their drugs under development to be used in situations that might just earn the drug a bad name while it is still in the midst of an involved and costly approval process. Any false step may well kill off a promising drug candidate early. Some creative thought on a thorny issue is warranted. The facilities available to big pharma for developing individualized therapies are unparalleled, but without a payoff, these will remain unutilized.
The combination of millions of cancer subtypes, evolving cancers, and the individuals' own incredible variability, is mind boggling. The same is true of other common terminal illnesses like multiple sclerosis. So many irreversibly progressive illnesses have strongly individual characters and require individually tuned therapies. One person's illness, even when apparently classified and staged in common with other 'similar' patients, can still have a stubbornly unique progression. It's only natural then that the treatment too has to be individually designed.
Clinical trials, as now commonly set up, are geared to large numbers where individual responses are unimportant. Results instead have to attain statistical significance when compared with the placebo groups (effectively the untreated 'control') in order to count for anything. Of course, with the advent of big data, as well as the deeper analysis of results, we are increasingly seeing subgroups and subtypes identified, and there is now more appreciation of individual responses. When a system is designed to test overall statistical trends, it's not surprising to note that it has resulted in a regulatory framework that's almost impossible to adapt to an individual's needs. The system has fed off itself and spiraled into an inflexible megalith. The system is also what 'big pharma' is happy with and is very happy to maintain the status quo.
I have only touched on some of the very many issues surrounding this discussion. It should be obvious that new ways of funding individual therapies need to be worked out. Only a tiny proportion of people will be able to work on their own disease as effectively as Dr. Steinman could. He had at his disposal a large, well funded research establishment, and there will be none who are similarly blessed. Again, I think creative solutions may occur to those both in and outside of 'big pharma' who may see an opportunity to bridge their own transitions through the upcoming changes in how medicine is to be done. It follows that it will behoove the insurance sector to follow suit!
Dr. Steinman has shown us the tremendous potential of allowing the terminally ill patient to explore individually tailored therapy. If we properly organize and carefully gather the data that results and then study it properly, it will eventually prove a broad and useful base for increasingly meaningful 'terminal' decision making. What looks from a scientific standpoint to be hugely messy, may eventually prove to be the path to a new way of doing medicine, not only for the terminally ill, but for every patient, and indeed for every single individual.
In one (critically important) sense we are all terminal, we just don't generally choose to face our own mortality in such stark terms.
You may also want to look at these updates:
1. Autologous cell therapies: challenges in US FDA regulation:
Discusses why autologous cell based therapies (i.e. stem cells from our own bodies) are yet to make the kind of impact expected of them. Again the FDA is the main culprit. Read it for yourself...