That's great! Kennedy's team is changing the world and how we see treatments. No more lifetime suppression of symptoms, more cures!
That's great! Kennedy's team is changing the world and how we see treatments. No more lifetime suppression of symptoms, more cures!
You're right, but because nuance isn't allowed on HN, you'll be down voted.
Primarily the focus has shifted to faster approvals with evidence for new methods and drugs coming down to one high quality trial, and removed stipulations for randomized control trials for ultra rare diseases.
> the focus has shifted to faster approvals with evidence for new methods and drugs coming down to one high quality trial, and removed stipulations for randomized control trials for ultra rare diseases
Could you share how RFK's policies helped bring this to market faster? (Not challenging you, by the way. Just need help connecting the dots.)
Seems like the CNPV used is a Trump admin project though no idea how much RFK is involved. If they Warp Speed All The Things that’s a pretty good idea.
> You're right, but because nuance isn't allowed on HN, you'll be down voted.
Nuance is fine. There's not much nuance in the parent post.
Actually I am unaware about what FDA policy changes helped this, can you elaborate ?
It’s in the OP:
> Today’s approval was issued 61 days after BLA filing, marking the sixth approval under the Commissioner's National Priority Voucher (CNPV) pilot program and the first gene therapy product approved under the program
That appears to be a late 2025 program based on the dates on the FDA page which would make it a Trump administration policy change. I don’t have any real knowledge here though. I’m just a guy clicking links. But if this is a policy change that’s exciting for approval of the other gene therapies for related conditions once the technology is solved.
0: https://www.fda.gov/industry/commissioners-national-priority...
I do agree that FDA was quite risk averse before especially in areas where the risk - reward would be better for terminally ill or other patients. Having said that this is a different kind of risk reward treatment where the patient might themselves like to try it even if the risks were elevated compared to other post Phase 3 solutions.
AFAIK critics pointed this to be a form of regulatory capture as well.
There has been a string of massive changes under the new admin.
Latest in this area: https://www.fda.gov/news-events/press-announcements/fda-laun...
“President Trump promised to accelerate cures for American families — and we are delivering, especially for children with ultra-rare diseases who cannot afford to wait,” said Health and Human Services Secretary Robert F. Kennedy, Jr. “We are cutting unnecessary red tape, aligning regulation with modern biology, and clearing a path for breakthrough treatments to reach the patients who need them most.”
“This guidance is a critical step the FDA is taking to tailor our regulatory approach to patients with ultra-rare conditions,” said FDA Commissioner Marty Makary, MD, MPH. “It is our priority to remove barriers and exercise regulatory flexibility to encourage scientific advances and deliver more cures and meaningful treatments for patients suffering from rare diseases.”
Now, you can be caught in the fun partisan civil war on emotions or simply look at outcomes. Good work being done at the FDA, just fact.
What actual regulatory changes have they made though?
Here: https://www.fda.gov/news-events/fda-newsroom/press-announcem...
Good faith, bad faith ... does not matter. Decisions and changes are public record. Read for yourself.
> or simply look at outcomes. Good work being done at the FDA, just fact.
A good outcome doesn't mean there was good work being done. You are conflating the two. You can make bad decisions and still have a good outcome.
I'm happy that this looks like a good outcome, but the current FDA is doing terrible work.
You're presumably referring to the ideologically driven cuts at the NIH and a complete misunderstanding of the efficacy-effectiveness gap in macro-level Research outcomes at the FDA? The ones that have led many prominent Professors of Medicine like Celine Gounder to conclude that “The current administration is waging a war on science.”?
RFKs deleterious impact on scientific research and its funding is well documented in the context of the NIH. 2025's Bethesda Declaration ably details the culture of 'fear and suppression' present under RFK, and the $9.5bn in grants and $2.5bn in contracts he had cut, impacting over 2,000 projects. It concludes with a chilling warning regarding plans to cut up to 40% of NIH's $48 billion budget in the future.
https://en.wikipedia.org/wiki/Bethesda_Declaration
At an FDA level, the same strategy was clearly evident last August when Trump fired CDC Director Monarez after clashing heads with RFK over vaccine policies barely a month into her role. Kennedy had demanded she fire career agency officials and commit to backing his own advisers. Four high-ranking officials resigned in support with Monarez.
In a similar vein, RFK then performed a clean sweep of the legacy 17 person vaccine panel in favour of his handpicked eight person vaccine panel – half of whom share ideologue Kennedy’s famous distrust of vaccines. Democrats on the Senate Health Committee summarised it blunty in an open-letter to RFK: “By removing all 17 of ACIP’s members and replacing them with eight individuals handpicked to advance your anti-vaccine agenda, you have put decades of non-partisan, science-backed work – and, as a result, Americans’ lives – at risk."
Moderna CEO Stephane Bancel subsequently said the company would not invest in new phase 3 infectious disease vaccine trials due to growing opposition from U.S. officials to immunizations.
This was then further compounded when the FDA RTF'd Moderna's new Flu Vaccine on spurious grounds in February, with the Alliance for mRNA Medicines calling the decision “unprecedented,” claiming the FDA was in “disarray,” and warned of a “threat to public health.”
Even last month a federal judge concluded RFKs actions re: the panel were not lawful, and that earlier votes by the panel to downgrade recommendations for hepatitis B vaccines for newborns and COVID-19 shots were invalid, blocking the Trump administration’s much publicised overhaul of the childhood vaccine schedule.
The only partisan stance at the moment would be not acknowledging the systemic dismantling of these scientific safeguards and institutions to the detriment of the American population as a whole.
Partisan, to the core.
How about https://www.fda.gov/news-events/press-announcements/fda-elim... ? Game changer for RWE. Not Science?
Or this one: https://www.fda.gov/news-events/press-announcements/fda-acce... ? A call for more research on certain compounds. Not Science?
I know the MSNOW daily hate machine is fun, so engaging and gives life meaning. But laying off the emo crack for a while would open your mind beyond partisan viewpoints.
I don't see the return of measles to be a success.
We can thank RFK for anti-vaccine leadership leading to changes to the childhood vaccine schedule which make preventable diseases harder to stop. You can celebrate more cures all you want, but as most people know, prevention is the best medicine, and the guy is lukewarm on one of our best defenses when it comes to keeping kids safe (vaccines).
And, let's not forget that RFK said: “every Black kid is now, just as a standard, put on adderall, [selective serotonin reuptake inhibitors], benzos, which are known to induce violence. And those kids are going to have a chance to go somewhere and get re-parented, to live in a community where there’ll be no cell phones, no screens, you’ll actually have to talk to people."
So yeah, according to RFK, every black kid in America deserves "re-parenting". He should resign today.