"When I was a post-doc, I helped run a pediatric cancer trial with children who relapsed while they were receiving chemotherapy. The prognosis for every child affected was death within several months. Parents were then offered a chance at the experimental drug/protocol I was involved with. I questioned the theory behind the study and why standard animal toxicology studies had not been run but was ignored. The parents were then lied to when "informed" consent was obtained, and the untested drug combination resulted in an interaction (which likely would have been seen if the appropriate animal toxicology studies had been performed). The reaction was absolutely horrific and instead children wound up dying absolutely horrific painful deaths.
While this is an extreme case, in my opinion this proposal may actually harm rather than help as in the scenario I describe, and may delay or inhibit the development of truly effective drugs when people insist on something that may actually not work and as a consequence not enroll in studies with drugs that may actually help.
March 22, 2018 at 1:41 PM. . . ." [Do go read these comments. End, update.]
The Senate earlier passed a similar bill. As I said about a week and a half ago, neither version contains an adequate definition of what triggers the right to try -- that is simply. . . irresponsible.
Yesterday, the House passed another version of the national right to try legislation, which Mr. Pence claims is saving lives in Indiana -- at the state level. Of course, his claim is entirely without any actual evidence. The idea that someone only has two options -- take an unapproved drug candidate out of Phase I, or die -- is simply a classic false dilemma, in logic. There will never be any way to prove whether the patient was harmed or helped by the Phase I drug candidate. [End, Updated Portion.]
I suppose, if I were a truly-jaundiced skeptic, I might suggest that the failure to define what triggers the right to try, with precision, is intentional -- to allow wide latitude to (unapproved) drug makers -- in selling their wares, to often quite-desperate patients and their anguished families. But I am not such a skeptic (cough). . . .
In any event, it will be entertaining to watch what Scott Gottlieb does (he opposes the idea -- and had earlier also asked for a definition) -- once the House/Senate reconciliation measure arrives.
I'll likely be off-grid until tomorrow, now. And. . . "Go Ramblers"! Keep it spinning in good karma, down south. . . . smile.
नमस्ते
3 comments:
When I was a post-doc I help run a pediatric cancer trial with children who relapsed while they were receiving chemotherapy. The prognosis for every child affected was death within several months. Parents were then offered a chance at the experimental drug/protocol I was involved with. I questioned the theory behind the study and why standard animal toxicology studies had not been run but was ignored. The parents were then lied to when "informed" consent was obtained, and the untested drug combination resulted in an interaction (which likely would have been seen if the appropriate animal toxicology studies had been performed). The reaction was absolutely horrific and instead children wound up dying absolutely horrific painful deaths.
While this is an extreme case. In my opinion this proposal may actually harm rather than help as in the scenario I describe, and may delay or inhibit the development of truly effective drugs when people insist on something that may actually not work and as a consequence not enroll in studies with drugs that may actually help.
Thank you, Anon. -- and sadly, I can confirm the sentiment there. The hard facts are. . . deplorable.
As you well-know, clearing a Phase I study-hurdle is no guarantee of safety (from interactions, especially), on a few dimensions.
And the "compassionate use" rules -- as they now exist allow for access usually within a day -- two, tops -- if the IRB feels it will "do no harm".
SO in several ways, this is a solution hunting about vainly for a problem.
But it makes for a great political point scoring sound-bite -- with the far right base, and libertarians -- who claim the federal government has too much say over their lives. Uh-huh. Right.
And. . . I quite agree that it may well inhibit innovation (i.e., delay deeper studies), as in time, it may relieve some of the rush to get to market (get full FDA approval).
What remains unsaid, of course, is that under existing "compassionate use", the patient rarely pays anything -- and I have a strong suspicion that the omnibus spending bill being voted tomorrow ALREADY contains provisions to allow pharma companies to bill government payers near retail prices -- for these "right to try" end of life drug candidates.
I realize this is a jaundiced view -- but it is one borne of several hard. . . experiences.
Mark my words -- there will be a provision (if not tomorrow, then soon. . .) that allows for a full billing to the VA, and Medicare/Medicaid for these "experimental" dosings.
In happier news, I just touched down, in ATL. . . and. . . GO RAMBLERS!
Namaste. . . .
At 3:36... Vandy... smiling twice... namaste!
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