Ground rules for embryo edits

With help from Allie Bice, Rachana Pradhan and Victoria Colliver.

On Tap

— Edits to heritable genes will be discussed today in a public meeting of global scientists.

— HHS’ top drug pricing expert is leaving after being a “key architect” in Trump’s blueprint.

— The EU rebuffed American efforts to delay stringent changes for medical device regulations.

It’s Tuesday, welcome back to Prescription PULSE. Adam Cancryn had so much fun subbing in on Prescription Pulse that he’s back again for Friday. Send him pharma news and tips at [email protected] or @adamcancryn and keep me in the loop for my Monday return at [email protected] or@owermohle.

Around the Agencies

ON TAP TODAY: GROUND RULES FOR EMBRYO EDITS — An international group convenes its first public meeting on creating a framework for deciding when scientists might ethically make human germline edits — gene edits that can be inherited.

The International Commission on the Clinical Use of Human Germline Genome Editing isspearheaded by U.S. and U.K. science authorities along with a panel of experts from around the world including former FDA Commissioner Robert Califf. Another former FDA chief, Peggy Hamburg, will present on behalf of the World Health Organization expert advisory committee that was asked to develop global standards on gene editing. That international group in March called for aworldwide registry for genome research, but stopped short of demanding a moratorium on heritable edits, a move some had expected after outcry over a Chinese scientist’s experiment last year.

The backstory: He Jiankui shocked the world when he announced at a Hong Kong genome editing summit that he had used CRISPR to protect two embryos against HIV infection. It’s still unclear (and likely will be for a long time) whether thosechanges actually helped or hurt the twin girls who were born. But the international scientific community agrees it wasn’t a risk worth taking. Other types of edits, however, might be — which is why the WHO panel is calling for a “step-wise” approach, Hamburg said.

He Jiankui has said he followed existing guidelines when he ran his experiment — raising questions about what the international community had missed when it laid out rough standards in 2017 for the fast-evolving field (Stat’s Sharon Begley has a great breakdown).

In the U.S., House appropriators in June advanced a spending bill that blocks FDA from reviewing or even considering research involving gene editing in human embryos. Democrats had dropped that language from an earlier draft, arguing the scientific community should first weigh in.

The block was restored to get the bill through committee. “Ethics hasn’t caught up with the science, and quite frankly the science hasn’t caught up with the science,” Rep. Robert Aderholt (R-Ala.) said at the time. “There are too many unknowns.”

TOP DRUG PRICE ADVISER LEAVING ADMINISTRATION — John O’Brien, the HHS secretary’s senior adviser for drug pricing reform, will step down Aug. 22, as POLITICO was first to report. O’Brien will be replaced by John Brooks, who will take on the role in addition to his current job as principal deputy director for the Center for Medicare at CMS.

O’Brien’s nine-month tenure in the post (and before that, as adviser for health reform and drug pricing) was marked by a series of aggressive — if sometimes thwarted — efforts to flesh out President Donald Trump’s vow to slash the cost of medicines.

… He championed a sweeping effort to effectively eliminate the rebates that drugmakers pass to payers. The administration ultimately scrapped the rule amid White House concerns it would raise premiums for seniors. In an interview, O’Brien told POLITICO he’s encouraged by ongoing congressional efforts to crack down on rising costs. He predicted Congress could yet weigh in on eliminating rebates.

2020 Watch

HEALTH GIANTS POUR FUNDS INTO M4A ATTACK ADS — The Partnership for America’s Health Care Future — whose members include PhRMA, BIO and the Association for Accessible Medicines along with major insurers and hospitals is scaling up the fight against Medicare for All with an Iowa ad blitz, OpenSecrets’ Jessica Piper wrote Monday. The partnership is spending more than $200,000 on TV ads this August, according to an analysis of Federal Communications Commission filings in OpenSecrets’ political ad database. That don’t include “hundreds of thousands” spent on Google and Facebook ads bashing the idea, OpenSecrets wrote.

Eye on FDA

TRANSFORMING FDA TO ‘LIFECYCLE’ REGULATION — FDA standards for drug approvals are becoming more flexible over time but the agency isn’t restructuring itself to properly regulate those therapies once they’re on the market, writes Dalhousie University’s health policy and law expert Matthew Herder in the latest Milbank Quarterly. Some of that is due to the difficulty of collecting real-world data. But other challenges are rooted in FDA’s limited legal authority, a condition it is acutely aware of, Herder writes, based on interviews with officials.

Accelerated approval programs can get drugs to market faster but usually rely on more limited approval data, with the expectation that real world data will be collected and given to FDA. Many studies have focused on the limitations and lack of transparency tied to those requirements. But Herder writes that far less focus has been placed on the challenges the FDA faces in even enforcing them. “This study of the FDA’s shift toward lifecycle regulation reveals not simply an agency in transition, but rather an agency on guard against a set of larger political threats to its mandate,” he writes.

Coming Up in Pharma

Today: The International Commission of Clinical Use of Human Germline Genome Editing holds a meeting.

Pharma in the States

EVEN AMID LAWSUITS, OHIO TAX BREAKS FOR OPIOID MAKERS CONTINUE — Throughout the opioid epidemic and to this day, Ohio state leaders have supported a hefty tax break for companies that make or ship prescription drugs, The Columbus Dispatch reports. The tax break, 98.3 percent of which went to merchandise shipped by drug distributors in 2018, has been worth more than $4 billion since 2006 and has grown to more than $500 million a year, Doug Caruso and Marty Schladen report.

DEA data released after The Washington Post and West Virginia’s The Gazette Mail sued shows that that while distributors were shipping tax-free drugs out of state, they shipped almost 700 million opioid pills into Ohio from warehouses just across the state line.

ICYMI: DRUG IMPORTATION PLAN GETS MIXED STATE RESPONSE — HHS’ incremental step to potentially allow states to import prescription drugs from other countries includes one appealing mechanism but another that might preempt what the states are already trying to do, Rachana Pradhan reports in POLITICO’s Pulse: State Health Care newsletter.

The Trump administration’s recent notice outlines two potential pathways: The first would let states, wholesalers and pharmacists submit their own plans to import from Canada a limited set of drugs, and HHS would approve them as long as they can assure safety and tracking throughout the supply chain. The second, and more problematic one in states’ view, would let pharmaceutical companies import their own products and sell them in the U.S. at a lower price. It’s unclear whether any drugmakers would be interested in the approach.

“We want to make sure it doesn’t become a vehicle for some minimal, nominal short-term efforts to preempt wholesale importation,” said Trish Riley, executive director of the National Academy for State Health Policy.

SF’S E-CIG LEGISLATION: THE DIFFERENCE A ‘MAY’ MAKES — A San Francisco committee has finalized the language for a Juul-backed measure on the November ballot that would overturn a city ordinance suspending the sale of e-cigarettes until they undergo an FDA review process, Victoria Colliver reports.

Proponents and opponents of the measure tried to convince the city’s arcane Ballot Simplification Committee to use their preferred word choices, but Juul lost on a crucial point: the voter pamphlet will inform voters that the measure also “may” repeal the city’s flavored tobacco ban, which residents overwhelmingly affirmed last year. The measure’s proponents deny any intent to overturn the flavor ban.

… Don’t expect this issue to end here. Parker Kasmer, an attorney for Juul, told POLITICO Monday the company is “looking at options, including potential litigation” to try to change the language. “We are pretty dismayed at the outcome,” Kasmer said, acknowledging the company would have to move quickly in filing a legal challenge.

Pharma Worldwide

EBOLA MEDS SHOW PROMISE IN DRC TRIAL — U.S. and WHO authorities said Thursday that a clinical trial of four possible Ebola treatments in the Democratic Republic of the Congo had been narrowed to two — Regeneron’s REGN-EB3 and a monoclonal antibody called mAb114. Their 90 percent cure rate justified offering those two drugs to all patients.

…The remaining patients on Gilead’s remdesivir and Mapp Biopharmaceutical’s ZMapp — one of the first experimental treatments launched during the 2014 West African outbreak — will have the option of switching over to the other two products, the National Institutes of Health, the trial’s co-sponsor, said.

The Congo outbreak began August 2018 and has claimed at least 1,800 lives, making it the second largest Ebola epidemic in history. WHO recently declared it a global emergency because of the risk for transmission to nearby countries and the violence that has stalled some outreach and treatment efforts.

EU REBUFFS AMERICAN CALLS FOR DELAY ON MED DEVICE CHANGES — The European Commission shows no signs of wavering on its May 26, 2020, deadline for medical devices to adhere to stringent new requirements, despite U.S. calls for a delay, our POLITICO EU colleague Sarah Wheaton reports. A spokesperson said the region-wide regulatory agency is on track for implementing its new system, including tightened standards and new “notified bodies"--which assess products--after a series of subpar device scandals like leaking silicone breast implants.

The United States, backed by Canada and South Korea, has called on the EU to delay enforcement for three years. The U.S. government called the process “critically behind schedule,” at the World Trade Organization meeting last month, and noted that American manufacturers make up about a fifth of the region’s $135 billion devices market. Device makers are worried about the slow process of certifying the notified bodies that must re-evaluate roughly half a million devices on the EU market.

Quick Hits

IMMUNE SERA SHORTAGES CAUSE PROVIDER RATIONING — A shortage of immune globulin in the United States is causing health providers to ration treatments, giving priority to patients who need it to stay alive, The Wall Street Journal’s Peter Loftus reports. The material helps people with weakened immune systems fight a wide range of infections. Paul Biddinger, director of the Massachusetts General Hospital’s center for disaster medicine, told WSJ that infusions have been canceled due to insufficient supplies, putting patients at a bigger risk of infection. The news comes as EpiPen and cancer medication shortages also have peaked.

ICER CHANGING GENE THERAPY EVALUATION PROCESS — The Institute for Clinical and Economic Review said it needs to incorporate new factors into its cost-effectiveness framework to account for the “single and short-term transformative” cell and gene therapies coming through the pipeline. Those changes include standardizing methods to tackle uncertainty and establishing new effectiveness thresholds, S&P Global’s Michael Gibney reports.

FDA WARNING TO NOVARTIS A MESSAGE TO GENE THERAPY WORLD — FDA’s warning letter last week to Zolgensma maker Novartis over a data error in its regulatory filing was an unambiguous signal to others in the fast-growing world of gene therapies, writes BioPharma Dive’s Ned Pagliarulo.”This is a rapidly evolving field where there are going to be accelerated approvals. The quality of the data is critical,” said former FDA chief Robert Califf in an interview with BioPharma Dive.

Pharma Moves

GlaxoSmithKline’s President of U.S. Pharmaceuticals Jack Bailey is stepping down at the end of the year. Maya Martinez-Davis, who currently serves as the Latin America regional president at EMD Serono, will replace Bailey.

Heyward Donigan will become Rite Aid Corp.’s new CEO, The Wall Street Journal reports. Donigan previously served as Sapphire Digital’s CEO.

Document Drawer

FDA announced the agency will be holding a meeting Nov. 7 for public comment on drug development programs.

ICER said it plans to do research on the clinical effectiveness and value of crizanlizumab and voxeloto, drugs used for the treatment of sickle cell disease.

CDC announced it is looking for nominees for the membership of the CDC/HRSA Advisory Committee on HIV, Viral Hepatitis and STD Prevention and Treatment.

A recently released GAO report assesses U.S. federal agency efforts in global tobacco control.

Biotech Amgen won its patent case for etanercept, a drug that treats autoimmune diseases.

CMS Administrator Seema Verma wrote a blog post on the importance of accessible antimicrobial drugs for seniors.