Marty Makary left behind an FDA families learned not to trust



With so much bad news in the world, it is worth pausing for one encouraging development: Marty Makary finally resigned as commissioner of the Food and Drug Administration last week.

Makary’s tenure at the FDA was marred by internal scandals, forced resignations, dreadful morale, and record staff turnover. More important, he actively sandbagged President Trump’s push to expand clinical trials for rare diseases through the aptly named “right-to-try” framework.

Trump’s next appointee should restore the spirit of right to try and make safe, effective treatments available to children as quickly as possible.

The idea behind right to try is straightforward. Patients with rare conditions, especially those for whom conventional medicine has failed, should have the freedom to pursue experimental treatments that have not yet received full FDA approval. Families fighting the clock have little left to lose. Government should not stand between them and a potentially lifesaving breakthrough.

Makary did.

Members of the MPS community sent more than 10 letters asking Makary for a meeting. They got a form letter in return. Sen. Ron Johnson (R-Wis.) later announced an investigation into the FDA’s denials. Makary’s agency responded by claiming approvals were already “at their peak.” The Wall Street Journal took notice of the FDA’s foot-dragging last year, yet the agency kept rejecting relevant rare-disease treatments in early 2026, including RGX-121 and drugs from Biohaven and Saol Therapeutics.

That stonewalling forced families to escalate.

In March, more than 100 mothers and other advocates staged a mock funeral outside FDA offices. Dressed in black and carrying a real coffin, they sought to draw attention to a group of rare metabolic disorders known as mucopolysaccharidoses. These disorders can show up as mild symptoms such as depression or hyperactivity, or as devastating conditions such as heart disease and skeletal abnormalities.

Many MPS disorders still have no approved treatments, even though they can severely diminish children’s quality of life or kill them outright. The FDA’s regulatory process serves a legitimate purpose. But when a bureaucracy grows so rigid, self-protective, and arrogant that it blocks desperately ill children from access to promising therapies, it stops functioning as a safeguard and starts functioning as a death sentence.

Mark Dant of the Ryan Foundation told Newsweek that some of these drugs were denied because of the FDA’s institutional “dislike” of the accelerated-approval pathway. “For decades we waited for science to find our tomorrows,” he said. “Now it has, and bureaucrats within the agency we pay for are keeping those treatments from our children. We know they are there. … We just cannot reach them.”

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Makary’s resignation will not undo the damage. But it does create an opening. We may not yet know what the FDA’s next leadership will look like, but Trump’s appointee should restore the spirit of right to try and make safe, effective treatments available to children as quickly as possible.

Across the world, in the nation of Georgia, parents have staged a protest lasting more than 500 consecutive days, maintaining a round-the-clock presence outside the main government building in Tbilisi. They are willing to risk everything to give their children the best chance at life. Americans should not have to camp outside federal offices for 500 days to get their government to listen.

The new FDA leadership must explain denials of right-to-try clinical trials with enough specificity that sponsors and families understand what evidence could change the decision. Patient and caregiver testimony should shape decisions early, not get folded in at the end as a token gesture. And Congress must demand transparency without turning each drug review into a partisan circus.

Children’s lives are not bargaining chips. The FDA exists to serve the public, not to protect its own bureaucracy from embarrassment. If Makary’s departure opens the door to that truth, families battling ultra-rare diseases may finally have reason to hope.

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Veterans shouldn’t have to worry about lawyers taking their benefits



I served in combat with the U.S. Army. Like many veterans, I know that men and women who come home carrying the physical and mental costs of war rely on disability payments to maintain mortgages and keep their families afloat. These funds help people rebuild lives that were permanently changed during their years of service and sacrifice.

Benefits are meant to help families recover from the physical and mental costs of war, yet they too often become a revenue stream for law firms that specialize in VA appeals.

Navigating the VA's disability system is rarely simple. Many veterans are already coping with serious injuries, mental health challenges, or financial stress as they transition back to civilian life. Confronting a complicated bureaucracy on top of that can feel like fighting another battle — which is why veterans should have access to a range of options for help.

The current system often leaves veterans with limited options, partly because when disability claims are delayed and pushed into drawn-out appeals, attorneys are allowed to collect a percentage of the veteran’s eventual award. The longer the process drags on, the larger the payout.

The Department of Veterans Affairs paid $394.7 million to accredited attorneys over the past year — money taken directly from veterans who fought to earn those benefits. The CHOICE Act (H.R. 3132) would help ensure that those benefits stay with the veterans who earned them, not the lawyers who see them as a payday.

Federal law limits attorney fees in most VA disability cases at 20% of a veteran’s backpay award. Those guardrails exist for a reason: Without them, veterans’ benefits risk becoming just another profit center for the litigation industry.

Organizations representing trial lawyers spend millions lobbying Congress each year on issues affecting litigation and attorney compensation. Veterans’ disability claims are no exception. When legislation like the CHOICE Act seeks to limit attorney fees and protect veterans’ benefits, the trial bar mobilizes to protect its financial interests.

This opposition raises a simple question: When the debate is about veterans’ benefits, whose side are these lobbyists really on?

Does increasing the share of benefits that go to legal fees serve those who wore the uniform?

Benefits are meant to help families recover from the physical and mental costs of war, yet they too often become a revenue stream for law firms that specialize in VA appeals.

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Veterans deserve strong advocates. The system should prioritize protecting them, not increasing the financial incentives tied to their benefits in an already strenuous process.

The complex VA benefits process can attract bad actors looking to profit from veterans navigating a complicated bureaucracy. Reputable companies that assist veterans with disability claims have been among the loudest voices calling for stronger oversight and clear rules to eliminate those abuses.

The CHOICE Act would establish guardrails that veterans deserve, including stronger consumer protections, limits on fees, and accountability for providers that violate the rules.

Congress must put veterans and their families first. The priority should not be filling trial lawyers’ deep pockets, but ensuring the system truly serves veterans’ best interests. When powerful lobbying organizations treat those benefits as a potential revenue opportunity, the system risks losing sight of whom it is meant to serve.

Our country made a promise: If you serve, and if service leaves you injured or disabled, the nation will stand behind you. The benefits belong to the veterans who earned them and not to the lawyers or lobbyists who see them as a revenue stream. Congress should pass the CHOICE Act and ensure those benefits serve the veterans they were meant for.

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MAHA is sick: RFK’s FDA is drifting the wrong way



If Health and Human Services Secretary Robert F. Kennedy Jr. wants to be true to his word and “Make America Healthy Again,” he must reform the Food and Drug Administration. Dr. Vinay Prasad, whose actions thwart medical freedom, endanger the unborn, and compromise patient choice, needs to go now, not at the end of April.

Prasad is a “Bernie Sanders acolyte” who “doesn’t think patients can be trusted to make their own healthcare decisions,” as Allysia Finley put it in the Wall Street Journal. Prasad disparages the 2018 right-to-try law, which give terminal patients access to experimental treatments, calling it “terrible” and “disingenuous,” written by people who “want to weaken the FDA.”

MAHA won’t survive as a slogan alone. Behind the facade of RFK’s rhetoric is an ideological agenda at odds with key conservative values.

Prasad claims that dying patients already have access to drugs through the FDA’s expanded-use programs and blames drug companies as the “major barrier” to unapproved drugs, downplaying the government’s role in blocking patient choice.

His personal crusade against faster drug approvals has chilled medical innovation. When Prasad originally resigned in July, months into his FDA tenure, amid backlash, the market predicted a shift toward a more patient-centric “right-to-try” approach, potentially cutting the bureaucratic red tape stifling cell and gene therapies and patient access.

Prasad’s pro-abortion record is even worse. He proudly identifies as “pro-choice” and progressive, a stance fundamentally at odds with pro-life conservatism. His appointment to the FDA’s Center for Biologics Evaluation and Research overseeing drug development that affects pregnant women and unborn children is a direct threat to the culture of life.

Prasad consistently casts abortion as a medical issue rather than a moral issue. He also fiercely defended mifepristone, the abortion pill, when a Texas judge tried to suspend its FDA approval. Prasad called the court’s intervention a “dangerous precedent,” and applauded the Supreme Court for preserving access to the drug, framing the issue purely as protecting “FDA authority” and “scientific integrity.” To pro-life voters, that posture reads less like neutrality and more like a commitment to keeping the abortion drug regime insulated from challenge.

Small-government promises are colliding with Prasad’s big-government dogma. Conservatives assumed RFK Jr. and his FDA appointees would shrink regulatory excess in support of President Trump’s innovation agenda, but they have done the opposite. Prasad came in with a “stringent regulatory mindset.” Rather than trusting patients to weigh risks for themselves, he has tightened the FDA’s grip with paternalistic, ideological rules. He has sidelined MAHA’s promise and expanded oversight instead.

Prasad’s policies have often expanded the FDA’s reach in ways that could seriously harm timely access to treatments. He is imposing tougher requirements on industry, insisting on larger trials and refusing to rely on surrogate endpoints for approvals, which means more delays and more red tape before new solutions can reach the public.

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The internal dynamics under Prasad reflect a top-down, bureaucratic rigidity and are under formal investigation, with the FDA retaining an outside investigator to examine workplace complaints alleging a toxic environment. Instead of signaling healthy reform, Prasad’s authoritarian rule of CBER is run on control and fear of pushback, where staff worry that dissent will be punished and experienced voices are pushed out or sidelined. Rather than “draining the swamp,” this approach fortified an insider bureaucracy loyal to Prasad’s agenda.

When the FDA held a meeting on a Commissioner’s National Priority Voucher drug, the voting members were top leaders like Prasad, not the scientists who reviewed the application. Career reviewers were excluded from the vote entirely, a major break from the FDA’s long-standing practice of empowering these staffers to make the final scientific call in order to shield approvals from political pressure.

The paradox for conservatives is obvious. Kennedy and Prasad earn plaudits for pulling back certain excesses, including scaling down aggressive vaccine promotion. Yet at the same time, they are building a larger, more controlling FDA bureaucracy in other domains — one that constricts medical freedom, slows innovation, and keeps pro-life concerns at arm’s length.

MAHA won’t survive as a slogan alone. Behind the facade of RFK’s rhetoric is an ideological agenda at odds with key conservative values. Conservatives who cherish medical freedom and rapid innovation find themselves at odds with Prasad’s FDA. A few welcome policy tweaks cannot obscure the reality of an expanding bureaucracy and pro-abortion policies.

With the 2026 midterms fast approaching, continuing this pattern will hurt Republicans and erode the trust of voters, handing Democrats an easy narrative about broken promises. Such an outcome would leave MAHA dead and MAGA mortally wounded. We must do better.

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FDA caution is starting to look like cruelty to sick kids



Biomedical research has produced extraordinary breakthroughs that have saved countless lives. But too many promising drugs now stall in federal review, and children with rare diseases are paying the price.

I’m a bioscientist. My work has focused on how healthy cells function and how that knowledge can be applied to therapeutic enzyme development. I’ve spent my career working inside the disciplines that move a treatment from lab bench to patient: protocol design, reproducibility, evidence standards, and layered human testing to ensure safety.

Is this simply bureaucracy doing what bureaucracy does? Or are rare pediatric therapies effectively facing a higher bar inside the system?

Standards, evidence, and process matter. But so does urgency.

Children with rare diseases do not live on regulatory timelines. They lose function month by month — speech, mobility, independence, even the ability to breathe on their own.

Of the more than 6,800 known rare diseases, about 70% begin in childhood. Better-known examples include Duchenne muscular dystrophy, Gaucher disease, and cystic fibrosis.

Developing therapies for these children is difficult, expensive, and slow even under the best conditions. Treatments such as Ultragenyx’s UX111 for Sanfilippo syndrome, Sarepta’s Elevidys for Duchenne, and Regenxbio’s RGX-121 for Hunter syndrome can take decades to develop, years to move through trials, and still more time to reach the children who need them.

That reality makes avoidable regulatory delay even harder to defend.

Too often, applications do not stall because the underlying science has failed. They stall over manufacturing or procedural concerns — in many cases, issues that are fixable and not directly tied to whether the therapy is clinically helping patients. Those delays can undermine the purpose of the FDA’s accelerated approval pathway, which exists to move critical treatments to patients faster while additional data is collected.

As a scientist, I was particularly troubled by the FDA’s recent rejection of a promising Hunter syndrome treatment and by yet another clinical hold placed on its development despite positive trial results.

That raises an uncomfortable question: Does the review process itself need review?

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The approval path for UX111 is another example. The therapy went through the rigorous biologics license application process, only to be delayed by a manufacturing hold.

Elevidys offers a similarly painful lesson. More than 1,200 Duchenne patients received the treatment over three years. Then, after two non-ambulatory patients (including one with underlying complications) tragically died, the FDA pulled the treatment from all patients, leaving families crushed and panicked.

Children are waiting too long for access to potentially life-changing therapies.

Yes, medical breakthroughs have increased. But so have regulatory burdens tied to approval and release. By the time many of these therapies reach the market, a decade or more has passed. In rare pediatric disease, that delay has a name: time children do not have.

Sometimes, it is their entire lifetime.

Manufacturing processes can be improved. Facilities can be upgraded. Paperwork can be corrected.

Lost neurons and muscle fibers cannot be replaced.

FDA leaders, along with Congress and the White House, should push for a smarter accelerated approval process — one that allows multiple requirements to be addressed simultaneously when appropriate, instead of serially dragging out timelines. If regulatory review had moved more efficiently, the Sanfilippo treatment might have cleared on its original 2025 approval timeline. Duchenne patients might not have lost access to the only available gene therapy. Hunter syndrome patients might not still be waiting.

This debate is not about abandoning safety or efficacy standards.

Ultragenyx has said manufacturing improvements are addressable and not directly related to product quality. Sarepeta responded to FDA concerns over Elevidys by requesting black-box warnings while allowing treatment to continue for ambulatory patients. In the RGX-121 Hunter syndrome case, the FDA rejected the use of a long-accepted biomarker (cerebrospinal fluid) used in the trial.

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These decisions do not help children with rare diseases. Timely, science-based approvals would.

And the stakes go beyond today’s patients. Regulatory efficiency also affects whether companies continue investing in rare-disease therapies at all. Orphan drug development requires major upfront investment, long timelines, and often poor financial returns. In many cases, these programs are closer to philanthropic science than blockbuster pharma economics.

When developers face repeated slowdowns across different diseases, sponsors, and technologies for reasons unrelated to core clinical safety or efficacy, the signal to the market is clear: Don’t take the risk.

That is how innovation gets smothered.

At some point, the pattern at the FDA becomes impossible to ignore. Is this simply bureaucracy doing what bureaucracy does? Or are rare pediatric therapies effectively facing a higher bar inside the system?

Those are scientific and ethical questions that deserve honest answers.

Accelerated approval does not mean lower standards. It means applying standards intelligently. It means allowing earlier access while confirming evidence continues to accumulate. It means recognizing that “wait and see” is not neutral. It is a choice that guarantees disease progression in children who cannot afford delay.

Good science and compassion are not competing values. We can maintain rigor and still act with urgency.

The FDA has the authority. The science is moving. The children cannot wait.

Accelerated approval is not cutting corners. It is using every tool we have to save time families do not have.

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