Maine is in line to become the 6th state to pass Right to Try legislation. State Representative Richard R. W. Longstaff (D - Waterville) has introduced a bill which will have a public hearing before the Joint Committee for Health and Human Services on April 6th. The proposed bill (LD180) is titled "An Act To Allow Terminally Ill Patients To Choose To Use Experimental Treatments".
I received an email from the HHS Committee Clerk as I was watching Tweets and Facebook posts from a rally in DC by ALS patients, caregivers and advocates in support of the FDA allowing ALS patients the right to choose to try a drug named GM604. GM604 has passed a small Phase 2A trial. The results of the trial appear promising as have many of this drugs predecessors, which failed to show efficacy in later stages of research.
However, the failure or success rate of any "experimental" drug isn't the issue. In my view the growing "Right To Try" movement - especially for ALS patients and their families - is about getting the government out of the way of protecting an informed individual from himself.
The fact is, there are very few afflictions for which modern science has failed to find multiple, at least marginally effective drugs and therapies for. In the 76 years since Lou Gehrig put ALS into the public consciousness, the FDA has approved one drug for ALS. That drug is known as Rilutek or Riluzole. Research has shown it to extend life expectancy by three months. A long way from a cure.
There are very few, if any forms of cancer for which there isn't some form of treatment. I'm certain there diseases which are statistically as (or more) rare than ALS which have no cure. My point is, the diseases for which there are no effective treatment to even retard the disease process are few and far between. ALS remains a riddle.
There is much concern in the research community over the Right To Choose. I understand and respect the concerns of researchers who claim giving access an unproven drug, especially to patients who don't meet the trial criteria, will jeopardize the data that is so important to verifying efficacy. As an ALS patient, I respectfully say: Get over it.
I'm dying. I have a brain that still functions moderately well (although my wife may disagree on some days). There are no options the traditional medical community can offer me, except Rilutek and moral support. With all due respect to alternative practitioners and their patients, been there done that. In my experience if the alternative practitioners had anything of value to offer, they would be open to scientific scrutiny, doing studies and demonstrating the efficacy of their treatments as well. People certainly have the Right To Try alternative medicine. I just remain a skeptic.
So, with no options, a healthy brain and an appropriate level of information, why shouldn't I be allowed to try a drug that has shown promise in a legitimate Phase 1 (or beyond) trial? Should I not be free to risk dying sooner than I might if I didn't try?
Be careful how you answer that last question. It's a trap of logic. If you answer "No, you don't have the right to try.", then we should ban sky diving and rock climbing. Period. Those are inherently risky activities which present the risk of death. Do healthy people have more freedom to assume risk than the terminally ill among us?
The current drug approval process of the FDA is archaic. It hasn't undergone significant scrutiny or change since it started in the 1960's. Add to that an approval pipeline that is clogged with applications for new toenail fungus and erectile dysfunction drugs, because that's where the money is, and you have a process which doesn't so much protect the public as it does rubber stamp the most profitable products for the pharmaceutical industry. (And yes, I understand that the profits from popular, mass market drugs help fund R & D for other drugs. Just makes one wonder who the FDA really works for.)
Upon first read Maine's proposed legislation isn't perfect. For example, the draft bill's definition of "Terminally Ill" is: "...a disease or condition that, without life sustaining measures, is reasonably expected to result in death within 6 months." This definition most likely would not cover ALS patients. It is interesting that the 6 month timeframe aligns exactly with the requirement for a patient to qualify for Hospice care. More interesting is that most, if not all Hospice providers won't provide "life sustaining measures" to patients in their care. So...if the law passes as is, patients will be forced to choose between Hospice and Right To Try.
Regulators, researchers and drug companies have valid concerns regarding Right To Try. They should be part of the dialogue. But they should also be painfully aware that this issue won't go away. Healthcare advocates and families facing terminal disease are demanding the Right To Try. The horse has left the barn, as they say.
Regulators, researchers and drug companies are free to oppose the Right To Try. Just as I should be free to try. Let's all work together to arrive at a solution that works for all, shall we?
