A ‘rare’ chance to revisit drug pricing?

Delivered every Tuesday and Friday by 12 p.m., Prescription Pulse examines the latest pharmaceutical news and policy.

Feb 23, 2024 View in browser   By David Lim and Lauren Gardner Driving The Day Rare disease advocates will be in Washington next week to urge Congress and the Biden administration to make adjustments to the IRA that would help shield orphan drugs from Medicare price negotiations. | Kirsty Wigglesworth-Pool / Getty Images AN OPENING TO RAISE IRA WORRIES — Hundreds of rare disease advocates will descend on Washington next week to lobby Congress and the executive branch to speed potentially life-changing therapies to market — and some may seek tweaks to President Joe Biden’s biggest legislative win to better shield those drugs from Medicare price negotiations. Patient advocates — and the pharma companies behind potential treatments — are pushing for lawmakers to amend the Inflation Reduction Act to soften its impact on orphan drugs — the medicines geared toward rare conditions that are harder for drug companies to profit from because of their small patient populations. Under the landmark law, products designated by the FDA as a rare disease drug and approved for the treatment of one specific disease or condition are exempted from Medicare price negotiations. But the agency can designate the drug for a different rare disease — a move that typically occurs early in the regulatory process and might not lead to additional approvals for other specific conditions. If that happens, the drug is no longer exempt, which advocates worry could discourage biotechs from studying potential treatments for uncommon illnesses. “Having a designation does not enable you to market the product,” said Heidi Ross, vice president of policy and regulatory affairs at the nonprofit National Organization for Rare Disorders. But getting Democrats to sign onto any effort to amend the law, touted as a major Biden administration achievement, will be a tough sell, former congressional aides said in December at a rare disease forum. “It has to be bipartisan; it has to be incremental,” Corey Malmgren, a former Democratic health counsel who now lobbies for drug companies, said at the event. “We have to socialize it.” Other asks: Some groups are focusing on other priorities, such as pushing Congress to reauthorize the FDA’s rare pediatric disease priority review voucher program, which expires Sept. 30. The initiative lets companies that win approval for such drugs redeem vouchers for expedited agency review of a subsequent product application. The week culminates with an all-day conference on March 1 at the FDA. Congress weighs in: The House Energy and Commerce Health Subcommittee has scheduled a hearing for the day before on 18 rare disease-focused bills, including a bipartisan bill to broaden the orphan drug exclusion from Medicare negotiations. IT’S FRIDAY. WELCOME BACK TO PRESCRIPTION PULSE. Your host is back from the great Midwestern city of Indianapolis. Send news and tips to Lauren Gardner (lgardner@politico.com or @Gardner_LM) or David Lim (dlim@politico.com or @davidalim). Around the Agencies The FDA says an EPA regulation for ethylene oxide that's due next week could affect the supply chain for medical devices. | Pablo Martinez Monsivais/AP Photo DEVICE INDUSTRY GIRDS FOR FINAL EtO REGS — A looming EPA regulation aimed at stanching the industrial release of a carcinogenic gas is a key part of the agency’s environmental justice work. But the FDA and the medical device industry continue to raise concerns that the rule could disrupt the supply chain for essential medical products for surgeries and other procedures. Under a court-ordered settlement, the ethylene oxide regulation is due out March 1. But environmental and industry advocates say the prospect of medical supply-chain disruptions is raising major concerns among White House policy staffers, POLITICO’s Annie Snider and David report. Both environmental advocates and medical device suppliers have been meeting with White House staffers in recent weeks to discuss the regulation’s potential impact and rallying their supporters on Capitol Hill. The White House did not respond to a request for comment. Medical device industry reps have also met with officials at the Defense Department and the National Institutes of Health to raise the alarm on potential supply-chain impacts if the rule’s provisions remain unchanged. The agencies: An EPA spokesperson declined to comment on potential changes to the regulation, insisting the agency is “working to finalize a rule that will address pollution concerns associated with [ethylene oxide], ensuring facilities can meet emissions requirements while also ensuring supply chains for sterilized medical supplies are not disrupted.” A spokesperson for HHS, which houses the FDA, said the department is working with the industry, the EPA and other federal agencies to “help answer questions related to medical device sterilization and provide insight on potential impacts to the medical device supply chain to further inform their decisionmaking.” The industry’s perspective: In a Feb. 14 meeting with the Office of Management and Budget, sterilizer company Sterigenics argued that the costs to upgrade facilities “far exceed EPA estimates.” It also said the regulation could reduce domestic sterilization capacity — and that the U.S. “cannot count on foreign capacity in times of crisis.”   CONGRESS OVERDRIVE: Since day one, POLITICO has been laser-focused on Capitol Hill, serving up the juiciest Congress coverage. Now, we’re upping our game to ensure you’re up to speed and in the know on every tasty morsel and newsy nugget from inside the Capitol Dome, around the clock. Wake up, read Playbook AM, get up to speed at midday with our Playbook PM halftime report, and fuel your nightly conversations with Inside Congress in the evening. Plus, never miss a beat with buzzy, real-time updates throughout the day via our Inside Congress Live feature. Learn more and subscribe here.     Eye on the FDA FDA: NO SMARTWATCHES TO MONITOR GLUCOSE — The FDA cautioned consumers Wednesday that smartwatches and smart rings that claim to test blood sugar levels without puncturing the skin have not been approved by the agency and should not be purchased for that purpose. “Sellers of these smartwatches and smart rings claim their devices measure blood glucose levels without requiring people to prick their finger or pierce the skin,” said the safety communication. “These smartwatches and smart rings do not directly test blood glucose levels.” The FDA said using the devices “could result in inaccurate measurements of blood glucose levels” and lead to people with diabetes taking an incorrect dose of insulin. Errors in low blood sugar readings can be a consequential mistake that can result in mental confusion, coma or death, according to the agency. In the Courts BIG IRA COURT DATE — Mark your calendars: A New Jersey federal district court judge will hear oral arguments on March 7 in four drugmakers’ challenges to the IRA’s drug price negotiation provisions. Biden-appointed Judge Zahid N. Quraishi will hear arguments by Bristol Myers Squibb, Janssen, Novartis and Novo Nordisk in the U.S. District Court for the District of New Jersey. It marks the second court date for drugmakers to make their case against the law; AstraZeneca appeared in court in Delaware last month. Pharma Moves Neurocrine Biosciences joined the PhRMA. Daniel O’Day, CEO of Gilead Sciences, is now the chair of PhRMA’s board, succeeding Novartis CEO Vas Narasimhan. Albert Bourla, CEO of Pfizer, is now board chair-elect and Paul Hudson, CEO of Sanofi, will be named treasurer. Astellas Pharma has hired Michael Petroutsas to be head of its U.S. commercial operations. Petroutsas previously was senior vice president for GSK’s U.S. Specialty and Oncology Business. WHAT WE'RE READING Moderna’s fourth-quarter revenue slumped year over year because fewer people are getting its Covid-19 vaccine, The Wall Street Journal’s David Wainer reports. A cyberattack at health tech company Change Healthcare impacted pharmacy orders nationwide Thursday, Fox Business’ Timothy Nerozzi reports. Document Drawer FDA published final guidance for drugmakers on Thursday, with advice for assessing Covid-19 symptoms in adults and adolescents participating in clinical trials for drugs and biologics that seek to prevent or treat the disease.   YOUR TICKET INSIDE THE GOLDEN STATE POLITICAL ARENA: California Playbook delivers the latest intel, buzzy scoops and exclusive coverage from Sacramento and Los Angeles to Silicon Valley and across the state. Don't miss out on the daily must-read for political aficionados and professionals with an outsized interest in California politics, policy and power. Subscribe today.       Follow us on Twitter David Lim @davidalim Lauren Gardner @Gardner_LM   Follow us

To change your alert settings, please log in at https://login.politico.com/?redirect=https%3A%2F%2Fwww.politico.com/settings This email was sent to rouf@idiot.cloudns.cc by: POLITICO, LLC 1000 Wilson Blvd. Arlington, VA, 22209, USA Unsubscribe | Privacy Policy | Terms of Service