| | | | |  | | By Lauren Gardner and Katherine Ellen Foley | | | | | | | 
An FDA decision on approval of a novel gene-editing therapy that can treat sickle cell disease is expected in December. | Eric Piermont/AFP via Getty Images | WHAT TO KNOW ABOUT CRISPR — The FDA is expected to decide by the end of next week whether to approve the first gene-editing therapy in the U.S., just weeks after the treatment for a rare genetic blood disorder received regulatory backing in the U.K. The therapy, known for now as exa-cel, uses a novel technology dubbed CRISPR to tweak genetic code in the stem cells of a person with sickle cell disease to produce healthy red blood cells. The process occurs after patients have their stem cells drawn; the edited, disease-free cells are then transplanted back into the patients. A panel of independent advisers to the American drug regulator signaled in October that there is sufficient data supporting its safety. The therapy’s promise has limitations. Its expected indication is for people with severe sickle cell disease, or roughly 10 to 20 percent of the disease’s patient population in the U.S. Plus, the treatment process is medically intensive and requires weeks of hospital care. Exa-cel is also expected to cost more than $1 million, a reflection of the one-time infusion of the treatment and the years of costly research and development. It’s unclear how public and private insurers would cover its cost (more on that below). Lauren and Katherine have more this morning on what you need to know ahead of the FDA’s decision on the breakthrough therapy. IT’S TUESDAY. WELCOME BACK TO PRESCRIPTION PULSE. The norovirus outbreak among Illinois students tied to $1 burritos reminds Lauren of a similar pre-Thanksgiving debacle back in her college days. Stay strong, kids. Send news, tips and pie recipes to Lauren Gardner (lgardner@politico.com or @Gardner_LM), David Lim (dlim@politico.com or @davidalim) and Katherine Ellen Foley (kfoley@politico.com or @katherineefoley). TODAY ON OUR PULSE CHECK PODCAST, host Ben Leonard talks with POLITICO health care reporter Robert King about the growing bipartisan concern — and scrutiny — on Capitol Hill over reports that Medicare Advantage plans are too often using the tool known as prior authorization to deny care.
| | | | | | | A message from Express Scripts by Evernorth: With drug prices on the rise, Express Scripts by Evernorth is redefining and reimagining what's possible for the pharmacy benefit. Prescriptions come with an easy-to-understand savings statement, detailing the cost of the drug, the price the consumer pays, and the value generated by Express Scripts. It’s all made simple with one clear fee. And if we don’t reach our client’s goals, they pay less – guaranteed. Simplicity, savings, and trust – that’s our pharmacy benefit. | | | | | | | 
Vertex Pharmaceuticals, which developed a new gene-editing technology to treat sickle cell disease with CRISPR Therapeutics, hasn't yet revealed the therapy's cost. | Charles Krupa/AP | THE MILLION-DOLLAR QUESTION — Vertex and CRISPR Therapeutics, makers of exa-cel, are discussing the cost of the therapy with U.K. authorities at its National Health Service, and the companies have declined to comment on how much the treatment may cost in the U.S. Two FDA-approved one-time gene therapies for blood diseases — which don’t directly edit a patient’s genetic coding — cost $2.8 million and $3.5 million, respectively. Regardless of the price, Medicaid and private insurers will likely struggle to cover the cost of exa-cel. “The insurers who pay upfront for the therapy may not recoup the benefits of improved patient health care down the road [for those who receive the treatment],” Beth Devine, a professor of health economics at the University of Washington, told Prescription Pulse. Katherine caught up with Devine on Monday to talk through the cost and value considerations for exa-cel. The conversation has been edited for length and clarity. How is the value of a treatment different from the cost? When we estimate value, we are comparing the cost of a drug with an alternative investment in a different therapy. We, as a society, want to pay for interventions that will provide the most value. Clinical improvement comes first … and then there are spillovers of an improved quality of life and productivity in life that really would raise society as a whole. A rising tide raises all boats. How do economists value a quality-of-life improvement? The U.S. has a couple of different willingness-to-pay thresholds. The thresholds are commonly $50,000 on the low end or $150,000 per quality-adjusted life year gained, which is one way of measuring how much a treatment improves or extends a person’s life. Our gene therapies that are now coming to market are on the higher end of those ranges — $150,000 to $170,000 per quality-adjusted life year gained. Does that value change when we consider systemic inequality? For example, most people with sickle cell disease are people of color (many of whom suffer from health care disparities), and an estimated 50,000 Medicaid beneficiaries have sickle cell disease. Those are the conversations that we are having now as a society. Is that willingness-to-pay threshold of $150,000 to $175,000 per quality-adjusted life years gained sufficient or should we be paying more? Should we be elevating the willingness-to-pay threshold because we have systemic inequalities or because we have rare diseases that occur in a fewer number of individuals than more common conditions? Those discussions are ongoing in the circles of health economics around the globe.
| | | | GET A BACKSTAGE PASS TO COP28 WITH GLOBAL PLAYBOOK: Get insider access to the conference that sets the tone of the global climate agenda with POLITICO's Global Playbook newsletter. Authored by Suzanne Lynch, Global Playbook delivers exclusive, daily insights and comprehensive coverage that will keep you informed about the most crucial climate summit of the year. Dive deep into the critical discussions and developments at COP28 from Nov. 30 to Dec. 12. SUBSCRIBE NOW. | | | | | | | | AI IN HEALTH CARE GETS HILL AIRING — The House Energy and Commerce Health Subcommittee will hold a hearing Wednesday on how artificial intelligence is changing health care. A committee memo on the hearing agenda noted AI’s implications for drug and device development. “This hearing will explore how AI is currently being used in medical devices, and in hospitals and what Congress should consider as AI in health care evolves,” it said. The memo said the FDA added 171 medical devices last month to its list of devices using AI or machine learning and drugmakers submitted more than 100 drug and biologic approval applications in 2021 with AI and machine learning elements. Witnesses will include representatives from Johns Hopkins University, UC San Diego Health and medtech giant Siemens Healthineers.
| | | | A message from Express Scripts by Evernorth:   | | | | | NOVO NORDISK DETAILS EXPANSION PLANS — ICYMI, Novo Nordisk will more than double the footprint of a production site in France, a move that will also grow its capacity to make GLP-1 treatments for diabetes and obesity, which have been in short supply worldwide for over a year. Construction has begun and “will gradually be finalized from 2026 to 2028,” the company said. Novo Nordisk announced an expansion of its Danish manufacturing facilities earlier this month to boost its “ability to meet future market demands,” including for GLP-1 drugs like Ozempic and Wegovy. But it’s unclear how much production capacity Novo Nordisk will add in the coming years once the upgrades are complete. A company spokesperson declined to comment beyond last week’s press release. “It’s really hard to say exactly what will happen,” said Erin Fox, a senior pharmacy director at University of Utah Health who tracks drug shortages. “But it’s good that they are expanding.”
| | | | JOIN WOMEN RULE ON 12/12: For centuries, women were left out of the rooms that shaped policy, built companies and led countries. Now, society needs the creativity and entrepreneurship of women more than ever. How can we make sure that women are given the space and opportunity to shape the world’s future for the better? Join POLITICO's Women Rule on Dec. 12 for Leading with Purpose: How Women Are Reinventing the World to explore this and more. REGISTER HERE. | | | | | | | | | FDA APPROVES DRUG FOR RARE TUMORS — On Monday, the FDA approved Ogsiveo, the first drug available for the treatment of desmoid tumors, a rare type of noncancerous growth.
| | OIG: LOW MISUSE OF PART D BUPRENORPHINE — A new report from HHS’ Office of the Inspector General found that in 2022, most Medicare Part D beneficiaries prescribed buprenorphine for opioid use disorder received recommended amounts, meaning there was likely little misuse of the drug. Those are similar findings to a 2021 report, which led OIG to conclude in the new review that there continues to be little risk of buprenorphine misuse among Part D enrollees.
| | Current and former University of Florida colleagues are voicing concerns about how Florida Surgeon General Joseph Ladapo so quickly secured a tenured position at the school, POLITICO’s Arek Sarkissian reports.
| | The FDA has added a Drug Supply Chain Security Act portal to its CDER NextGen Portal, allowing regulators and U.S. trading partners to communicate about agency requests for information during a product recall or investigations into illegitimate products.
| | | | A message from Express Scripts by Evernorth: With drug prices on the rise, Express Scripts by Evernorth is redefining and reimagining what's possible for the pharmacy benefit.
ClearCareRx ensures clients pay what Express Scripts pays pharmacies for member prescriptions, with 100% of rebates returned to payers, including all discounts.
Clients receive clear savings statements, outlining drug costs and Express Scripts’ value. They pay one simple fee, and if goals aren’t met, clients pay less – guaranteed.
Simplicity, savings, and trust – that’s our pharmacy benefit. | | | | | | | Follow us on Twitter | | | | Follow us | | | | |
