Gene therapy: Hopes, hurdles and AI

Gene therapy: Hopes, hurdles and AI

With Carmen Paun and Megan R. Wilson

A RARE ROLE FOR AI — Experts told our Robert King at POLITICO’s Next Generation of Health Care Therapies event Thursday that they’re excited about the potential for artificial intelligence to help develop and test gene therapies to treat rare diseases.

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said AI could be a “game changer” in helping manufacture complex treatments, adding that the agency has already used it to detect potential safety issues — though he stressed the technology isn’t a silver bullet.

“Some of this is not going to be through AI. Some it will be through well-chosen endpoints that can be used for accelerated approval,” Marks said.

Another panelist, Carla Rodriguez-Watson, with the Reagan-Udall Foundation, cautioned that AI is only as good as the data fed into it and warned that unless tech developers make sure they’re using data representative of the population, they risk worsening health disparities.

The discussion also covered ways to overcome barriers to patients accessing gene therapies, better incentivize companies to develop them and get the government and private insurers to cover them.

WELCOME TO FRIDAY PULSE, where your author remains skeptical as D.C.’s swampy summer drags on that the power of mosquito bites can be harnessed for good. Send itch cream, tips and stories to [email protected] and [email protected].

TODAY ON OUR PULSE CHECK PODCAST, I talk with Kelly Hooper, who delves into what’s behind the spike in overdose deaths involving both cocaine and opioids over the past decade.

TEXAS COURT DELIBERATES ON ABORTION BAN — A Texas state judge said she’ll likely take “several weeks” to rule on the fate of the medical exemptions to the state’s abortion ban.

Judge Jessica Mangrum’s announcement came at the end of two days of testimony from patients who suffered medical harm after being denied abortions and doctors who said the law prevented them from providing the proper standard of care to pregnant people experiencing a complication. The doctors and patients, represented by the Center for Reproductive Rights, are asking the court to broaden the state’s exemption to allow abortions when there’s a diagnosis of a fatal fetal anomaly or when continuing with the pregnancy would cause long-term damage to the patient’s mental or physical health.

Attorneys for Texas didn’t dispute that the women suffered injuries but argued the doctors were at fault for failing to interpret the state’s law correctly and asked the judge to leave it as-is.

MO COURT RULES ON ABORTION BALLOT MEASURE — The Missouri Supreme Court ordered the state’s Republican attorney general Thursday to stop interfering with efforts to put a measure to restore abortion access in the state on the 2024 ballot.

AG Andrew Bailey has for weeks refused to approve summaries and cost estimates for different versions of the ballot measure, delaying activists from collecting signatures. Bailey has claimed that legalizing abortion in the state would cost tens of billions of dollars, arguing that fewer births will lead to lower tax revenue for the state in the future.

The ACLU of Missouri, which brought the challenge, said after the ruling that “the courts saw the Attorney General’s effort to derail the initiative process for what it was: a deliberate threat to the direct democracy.”

Bailey’s spokesperson, Madeline Sieren, told Pulse: “We disagree with the court’s decision, as we believe Missourians deserve to know how much this amendment would cost the state, but we will respect the court’s order.”

RFK JR. TESTIMONY SPARKS FIGHTS OVER COVID, VACCINES — Democratic presidential candidate Robert F. Kennedy Jr. appeared before the House Judiciary Committee on Thursday to testify on what he sees as government “censorship” of speech on social media, reports POLITICO’s Eric Bazail-Eimil.

Though the hearing wasn’t ostensibly about Covid-19 or vaccines, lawmakers grilled Kennedy about his recent claims that Covid could be an “ethnic bioweapon … targeted to attack Caucasians and Black people” but not “Ashkenazi Jews and Chinese.”

Under questioning from Democrats, Kennedy acknowledged that his comments were akin to the centuries-old “blood libel” conspiracy about Jews and his past comparisons of Covid lockdowns to the Holocaust were inappropriate — though he insisted his remarks were misinterpreted. Democrats also blasted their GOP counterparts for inviting Kennedy as a witness, arguing the hearing dangerously elevated his conspiracy theories.

Some Republicans used the hearing to air concerns about vaccine safety — beyond Covid — and argued the benefit of “natural immunity” acquired through infection instead of vaccination has been censored.

TOTES APPROPS — Work on annual spending bills is inching along. The Senate Appropriations Committee announced a markup next week of what are expected to be the most contentious bills — those that control spending for the Departments of Defense, Homeland Security, the Interior, Labor, Health and Human Services and Education.

The upper chamber will mark up the bills on July 27 at 10:30 a.m.

On Thursday, the panel advanced its version of the State and Foreign Operations spending bill with large increases in funding for international family-planning programs and efforts to halt fentanyl trafficking. It departs significantly from the House version, setting up a clash when Congress will have to reconcile the bills later this year to avoid a government shutdown.

PBMS BRACE FOR LEGISLATION — Members of the Senate Finance Committee are rolling out more measures targeting pharmacy benefit managers, dropping two new bipartisan bills on Thursday, Megan reports.

Sens. John Thune (R-S.D.) and Sherrod Brown (D-Ohio) would force PBMs — which manage prescription drugs for health insurers — to disclose information about how they define what medicines are “limited access drugs” that only certain specialty pharmacies can distribute. It’s an attempt by lawmakers to stop PBMs from steering patients to specialty pharmacies they’re affiliated with and away from independent pharmacies.

Another bill, introduced by Sens. Tom Carper (D-Del.) and Chuck Grassley (R-Iowa), would give CMS the authority to provide oversight over PBMs and compel more transparency into how they select which medications are covered under a prescription drug plan, known as a formulary.

The Senate Finance Committee will hold its first markup on some of the measures Wednesday.

PANDEMIC FUND AWARDS FIRST GRANTS — Thirty-seven low- and middle-income countries will receive $338 million in grants to improve their capacity to prevent and prepare for disease outbreaks from the Pandemic Fund, hosted by the World Bank.

The Bank announced the first grantees of the Fund on Thursday.

Countries ranging from Burkina Faso to India and Paraguay will benefit from the money to strengthen disease surveillance systems and lab capacity, train more health care staff to respond to health emergencies and improve animal health.

The grants will mobilize $2 billion in additional resources, according to the Bank.

POLITICO’s Natalie Fertig reports that a new bipartisan bill would give the FDA the power to regulate hemp in food and beverages, an issue that could become part of the next Farm Bill.

The Atlantic reports that overdose deaths in Oregon have gone up since the state decriminalized possession of hard drugs three years ago, though advocates say more time and resources are needed to steer people to treatment programs before declaring the policy a failure.

CNN reports that infant deaths rose in Texas between 2021 and 2022, reversing years of decreases. Some experts attribute the change to the implementation of the state’s near-total abortion ban.

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