itwasquiteawhileago
itwasquiteawhileago t1_ixwemjx wrote
Reply to comment by FUCKYOUINYOURFACE in A novel medication for hemophilia B has just been approved by the Food and Drug Administration. The treatment is a form of gene therapy, intended to replace a dysfunctional gene that leaves people unable to control their bleeding. by Sariel007
This is where orphan drugs come in. The government (US, anyway, but I'm assuming others have similar programs) can help provide some funding and allowances for private companies to work on drugs for conditions that have a low enough prevalence as to make it economically non-viable. Should someone that has a one in a million disease be left to suffer because Big Pharma can't turn a profit? This is a primary example of why and how governments should function: to ensure people aren't left behind. And, who knows what other things may spawn off such research, so it's not like everyone else might not benefit, too.
itwasquiteawhileago t1_ixvdc9l wrote
Reply to comment by The-Fox-Says in Brain cancer vaccine succeeds at prolonging survival in Phase 3 trial by Sorin61
Some of the oncology trials I've been on compare IP to SOC. In many cases SOC is what they call "watchful waiting" (i.e., doing nothing). I'm no oncology expert, but I do work in the clinical trial space and have worked on dozens of various oncology studies over the years and see this regularly. Often times they can cross over from SOC to study drug if their condition worsens within a certain timeframe, but how they make those decisions when developing the protocol is not something to which I am involved.
I know sometimes the best results only really mean maybe a few more months before the inevitable. Those trials are rough because people just want to live the rest of their lives as comfortable as possible, so being in a clinical trial that, best case, may extend their life a few months, is not always appealing. But without these trials, we'll never get anywhere in the long run.
itwasquiteawhileago t1_ixj6ygr wrote
Reply to comment by ichuck1984 in Amazon to invest $1 billion yearly to produce movies for theaters by Familiar-Turtle
And the inevitable sequel: The Package Punter.
itwasquiteawhileago t1_ja8jr2n wrote
Reply to ‘Perry Mason’ Finds Its Mojo in Season 2 by Getting Down and Dirty by jez124
Lawyer, detective, any combination of both, I don't care. I enjoyed S1 a great deal. The performances, the sets, everything. So as long as those things remain intact, I'm down for more.