BIO chair: U.S. has a ‘crisis' on drug costs
Driving The Day
NEW BIO CHAIR'S POLICY TAKE — The biotech industry faces a mix of longstanding and novel political challenges amid President Donald Trump's second term. One of its chief lobbying arms is gearing up to take them on.
Ahead of the Biotechnology Innovation Organization's annual convention this week in Boston, Genentech's Fritz Bittenbender — who was elected Monday as the group's board chair — chatted with Lauren about BIO's priority issues, distrust in science and medical institutions and its approach to geopolitical engagement. Pros can read the full Q&A, but here are some highlights and outtakes:
An 'existential policy crisis': Bittenbender used the term to refer to debates around 'intellectual property, how do you pay for medicines, what kind of investments are we going to make at early-stage research' — all of which bring to mind Trump administration priorities like the most-favored-nation approach to bringing down drug prices and limiting National Institutes of Health spending on indirect costs in university research.
'We have a lot of sound-bite policy happening right now, and [most-favored-nation is] a great example of that,' he said, adding that the U.S. health system works much differently from those in allied European countries, often held up as examples of cheaper markets.
How industry can combat distrust in science: Biotechs need to do a better job of communicating the benefits they bring to patients, and they need policymakers' help, Bittenbender said.
'Working with policymakers, and … bringing as much transparency to our industry as we possibly can — using real-world data and digital analytics more effectively to understand postmarketing studies of products on the market and being transparent about that,' he said.
Federal job cuts: Regulatory uncertainty is an 'existential threat' to the industry amid the massive downsizing and restructuring of the Department of Health and Human Services, Bittenbender said.
'If it's going to take 10 or 15 years to bring a product to the market and $1 [billion] to $2 billion to do that, that's a significant investment over a long-term period. And for investors to want to do that, they have to know that there's a certain regulatory environment,' he said. 'They have to know that regulatory timelines are going to be met or exceeded.'
The tariff threat: 'Our industry is a national security imperative for the country, and that means having essential medicines that patients really need manufactured here,' Bittenbender said.
'Hopefully, we won't see tariffs on the pharmaceutical industry, and we work in other ways to get manufacturing into the United States and to ensure that essential medicines that we need in times of emergency or pandemic are sourced from a place that we trust and that we know we can get them,' he added.
IT'S TUESDAY. WELCOME BACK TO PRESCRIPTION PULSE. NPR reports on how music therapy can help cancer patients manage their stress and symptoms.
Send your tips to David Lim (dlim@politico.com, @davidalim or davidalim.49 on Signal) and Lauren Gardner (lgardner@politico.com, @Gardner_LM or gardnerlm.01 on Signal).
Eye on the FDA
SECOND DMD DRUG DEATH — A second patient with Duchenne muscular dystrophy has died after taking Sarepta Therapeutics' Elevidys gene therapy, the company said, prompting a reevaluation of the drug's treatment protocol.
The death occurred in a 15-year-old nonambulatory individual enrolled in the company's randomized, placebo-controlled trial intended to confirm the drug's benefit in patients who can't walk under the FDA's accelerated approval pathway.
A company official said the latest death shares 'some similarities to the previous' death of a teenage boy earlier this year; both fatalities were due to acute liver failure, a known side effect of the viral vector gene therapy used in Elevidys. Sarepta said the signal has emerged only in patients who can't walk, which they consider a surrogate for disease progression.
Sarepta President and CEO Douglas Ingram said the company wants to meet with the FDA 'as rapidly as possible' to establish a new immunosuppressive regimen for nonambulatory patients. Until that's implemented, the company has paused drug shipments for that population, as well as dosing for its clinical trial, it said.
Response: HHS spokesperson Emily Hilliard said the FDA is treating the death 'with the highest level of concern' and 'will take all appropriate regulatory actions to protect patients during our review of gene therapy products.'
The FDA is reviewing a cell therapy candidate from Capricor Therapeutics to treat DMD. The target date for a decision is Aug. 31.
PDUFA PROBLEMS? KalVista Pharmaceuticals raised some eyebrows late last week after announcing that the FDA had disclosed it would miss its Tuesday PDUFA target for the drug sebetralstat, which is used as therapy for hereditary angioedema, 'due to heavy workload and limited resources.' The rare disorder causes episodes of swelling in various parts of the body and can sometimes be fatal.
FDA Commissioner Marty Makary has repeatedly said medical product reviews would continue apace despite thousands of job cuts at the agency, and reviewers were not among the terminated workers. But employees charged with supporting review staff by booking travel and securing supplies were impacted, and drug companies remain concerned about attrition in the remaining workforce.
HHS did not comment.
In Congress
EXPANDED ORPHAN EXEMPTION OUT — The Senate Finance Committee's reconciliation bill strips an effort by the House to expand a Medicare drug price negotiations exemption for orphan drugs to include medicines that treat multiple rare diseases or conditions.
The Congressional Budget Office estimated the policy in the House bill would cost the federal government nearly $5 billion over 10 years, a figure groups like AARP and Patients For Affordable Drugs Now used to urge senators to keep the measure out of the Senate bill.
But pharmaceutical companies argue the provision would incentivize additional investment in rare-disease drug development as the IRA exemption currently applies to orphan drugs that treat a single rare disease.
SANDERS WANTS ACIP INVESTIGATION — Senate HELP ranking member Bernie Sanders (I-Vt.) wants his counterpart to open a bipartisan investigation into the removal of 17 members of the CDC's outside vaccine committee.
In his letter to Sen. Bill Cassidy (R-La.), chair of the Health, Education, Labor and Pensions Committee, Sanders asked for 'serious oversight' of HHS Secretary Robert F. Kennedy Jr.'s actions regarding the Advisory Committee for Immunization Practices.
'Secretary Kennedy's reckless decision to fire these non-partisan scientific experts and replace them with ideologues with limited expertise and a history of undermining vaccines will not only endanger the lives of Americans of all ages, it directly contradicts a commitment he made to you before he was confirmed that he would not make any significant changes to this important Committee,' Sanders wrote to Cassidy.
A spokesperson for Cassidy did not immediately respond to a request for comment.
In the courts
PURDUE SETTLEMENT 2.0 — All 50 states and several U.S. territories have agreed to sign onto a $7.4 billion settlement with Purdue Pharma and its principal owners, the Sackler family, that will resolve state and local government claims.
'The local government sign-on and voting solicitation process for this settlement moving forward will be contingent on bankruptcy court approval,' California Attorney General Rob Bonta's office said in a news release. 'A hearing is scheduled on that matter in the coming days.'
The settlement ends the Sackler family's control of Purdue and prevents them from selling opioids in the country.
Document Drawer
The FDA's Psychopharmacologic Drugs Advisory Committee will meet on July 18 to discuss Otsuka Pharmaceutical's supplemental new drug application to approve Rexulti to treat adults with post-traumatic stress disorder in combination with sertraline.
The FDA published final guidance outlining recommendations for generic drugmakers on how to submit a pre-submission facility correspondence that can be used to help the agency begin site assessments in advance of submitting an abbreviated new drug application.
WHAT WE'RE READING
The 17 dismissed members of the CDC's vaccine advisory panel published an op-ed in JAMA, saying their abrupt dismissal last week 'undermines the committee's capacity to operate effectively and efficiently, aside from raising questions about competence.'
HHS awarded an Arizona law firm $150,000 for its expertise on the Vaccine Injury Compensation Program, NOTUS' Margaret Manto reports, suggesting it's considering policy changes to the 40-year-old system.
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