Since the last FOMC statement (on May 1st), stocks and bonds have outperformed (with the former at record highs), gold is flat while the dollar is down modestly. Amid all that, oil is down significantly...
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Wednesday, June 12, 2024
Who’s Paying for Million-Dollar Gene Therapies?
When Orchard Therapeutics announced the $4.25 million price tag in March for its newly approved rare disease gene therapy Lenmeldy, it became the most expensive on the market. But since 2019, the FDA has approved five others that cost more than $2 million. Some insurers, even when legally required, are not having it when it comes to paying out the cost of a treatment.
A November 2023 ProPublica investigation found that insurance provider Priority Health quietly nixed coverage of gene therapies as a cancer treatment, even though under Michigan state law the payer was required to cover clinically effective cancer drugs. The gene therapy in question, CAR-T,
Globally, many healthcare systems are choosing not to cover gene therapies at all. Even though every health regulatory body reviews the same scientific and trial information, Canada and countries across Europe have completely different gene therapy coverage models than the U.S., according to a December 2021 study in Health Policy.
“The primary underlying reasons for access restrictions in all regions appear to be scientific uncertainty at the time of approval, limited evidence, and the high cost of these therapies,” the study authors wrote.
But even with the high price tag and what some call questionable evidence that a treatment will provide a lifetime benefit, private American payers are already shelling out for gene therapy treatments, said Graig Suvannavejh, the managing director of biopharma and biotech at Mizuho Securities. However, debate remains over what conditions warrant six-figure prices and how payers can recoup that money if a gene therapy fails.
“This will continue to be an evolving dynamic over the next 10 to 20 years,” Suvannavejh said.
At this juncture, both public and private payers can pay for gene therapies either upfront or in annual installments over a handful of years. Both types of agreements can, and largely do, build in a caveat that if a patient does not see a significant benefit over time, the payer can receive a rebate of part of the gene therapy's cost. These are known as outcome- or value-based agreements.
“Gene therapy [means] that you’re given it one time [and] you’re stuck with it, good and bad,” Suvannavejh told Biospace. “The issue then is if it is meant to be given as a one-time treatment, are you actually getting the benefits for the duration of your life?”
Suvannavejh said that other factors, such as existing therapeutic options beyond gene therapy and the disease’s fatality rate, are part of the coverage discussion. If a condition has a high fatality rate and few other options, there may be greater willingness to agree to a high price tag. Right now, he added, gene therapy payments tend to be upfront in a lump sum.
Bob Lojewski, senior vice president and general manager of CSL Behring North America, said that private payers have been largely receptive to the $3.5 million price tag of the company’s HEMGENIX, a gene therapy treatment for hemophilia B.
Currently, payers are covering “the vast majority of the U.S. population” who may need HEMGENIX, he said. “To date, every patient referred for HEMGENIX has been covered through insurance, and we expect access for eligible people interested in this one-time treatment will continue,” he said.
Lojewski added that the CSL team has seen an “accelerating rate” of patients referred for treatment in the past six months, potentially due to the agreements in place to cover the therapy. He did not say whether insurers were paying by lump sum or in installments.
Internal CSL figures estimate that between annual bleed rates and required therapies to address hemophilia B, a patient with the disease can cost an insurer $20 million over the course of their life. That puts the expenditure of $3.5 million for a gene therapy to reverse or cure the condition into perspective. When that is extrapolated out, HEMGENIX could save the U.S. healthcare ecosystem tens of billions of dollars over the next 20 years, Lojewski said.
However, saving all of that money in the long run comes with tens of millions of dollars in upfront costs to private companies or the federal government, while it may take decades to realize the cost savings from that investment.
According to the Alliance for Regenerative Medicine, an analysis by NEWDIGS at Tufts University estimates that gene therapy expenditure in the U.S. will reach
Public Payers Negotiate Costs for Gene Therapies
Medicare and Medicaid both cover many patients affected by conditions addressed by new gene therapies. Medicaid covers two-thirds of patients with sickle cell disease, which bluebird bio’s Lyfgenia and Vertex Pharmaceuticals’ and CRISPR Therapeutics' Casgevy, listed at $3.1 and $2.2 million respectively, are designed to treat. Furthermore, Medicaid provides healthcare coverage for over half of children with spinal muscular atrophy, some cases of which can be treated with Novartis’ $2.1 million Zolgensma treatment.
But public payers may be negotiating discounts on those list prices down year over year, while it’s unclear whether private payers are negotiating regularly for treatments to be significantly less than their list prices.
Take Zolgensma. With a $2.1 million listing price, public insurers paid $1.89 million
Like private insurers, public payers are setting up rebate programs in case these therapies do not deliver on their promises, although this strategy isn’t commonly used just yet.
“Supplemental rebates only accounted for 9% of all Medicaid drug prices in fiscal year 2022,”
https://www.biospace.com/article/who-s-paying-for-million-dollar-gene-therapies-/
Pfizer, Flagship Pioneering to Target Obesity Treatments Under 2023 Partnership
ProFound Therapeutics, a Flagship Pioneering-founded company, announced Wednesday that it will conduct research on potential first-in-class therapies for the treatment of obesity with Pfizer as part of a potential $7 billion collaboration with Flagship unveiled last year.
According to Flagship, its in-house drug discovery and development unit Pioneering Medicines will lead the partnership with Pfizer. ProFound will use its proprietary platform alongside Pioneering Medicine to find new proteins and evaluate them for treating obesity. ProFound’s platform uses protein detection technologies to find and validate proteins and determine their therapeutic potential.
Per the latest agreement, Pfizer will have the option to advance selected research programs under the terms of the strategic partnership announced in 2023 between Pfizer and Flagship, which will allow the pharma to add up to 10 new medicines to its pipeline. Both companies contributed an upfront investment of $50 million, while Flagship and its respective entities can receive up to $700 million in milestones and royalties for each program on the market, giving the deal a total potential value of $7 billion.
“Since launching this unique alliance between Flagship and Pfizer we have been working together to rapidly build out a portfolio of exploration programs,” Paul Biondi, Flagship executive partner and president of Pioneering Medicines said in a statement. “This agreement will enable ProFound to explore how its pioneering platform can tackle unmet needs in obesity.”
The original deal between Pfizer and Flagship was also meant to discover candidates in several areas, such as rare diseases, oncology, vaccines, inflammation and immunology. However, according to Charlotte Allerton, Pfizer’s head of discovery and early development, given that obesity will impact 1.9 billion people globally by 2035, it is “critical to continue breakthrough discovery research” that can find new therapies.
“This agreement, the first under our broader strategic partnership with Flagship, is designed to push the boundaries of science to potentially unlock new protein therapeutics for obesity leveraging ProFound’s proprietary discovery platform,” Allerton said in a statement.
Flagship is also working with another pharma in the obesity space. In May, Flagship and Metaphore Biotechnologies inked a research collaboration with Novo Nordisk to find new therapies for obesity management. The terms of the agreement will see Novo paying up to $600 million in upfront, development and commercial milestone payments and royalties on net sales. Novo is also reimbursing R&D costs and participating in a future financing round for Metaphore.
https://www.biospace.com/article/pfizer-flagship-pioneering-to-target-obesity-treatments/
Teva Loses Inhaler Patent Case Against Amneal, Must Delete or Amend Orange Book Listing
A New Jersey federal court on Monday ruled against Teva Pharmaceuticals, finding that five key patents for its inhaler product ProAir HFA (albuterol sulfate) are “improperly listed” on the FDA’s Orange Book.
District Judge Stanley Chesler agreed with the plaintiffs—Amneal Pharmaceuticals, with the backing of the FTC—that Teva’s patents only apply to the inhaler device of ProAir HFA and not to its albuterol sulfate drug formulation. The judge ruled that Teva’s inhaler patents do not claim a “finished dosage form” of albuterol, allowing Amneal to develop a generic version of the drug.
“It is undisputed that no claim in any of the Inhaler Patents discloses albuterol sulfate,” Chesler wrote in his decision, adding that Teva must either “correct or delete” its patent listings in the FDA’s Orange Book to reflect the court’s judgement.
Amneal in July 2023 submitted an Abbreviated New Drug Application (ANDA) for a generic version of ProAir HFA, though its filing specifically focused on inhalable albuterol sulfate. Teva in 2023 sued Amneal, claiming that the ANDA infringed on its patent protection for its drug-device combination product. Amneal filed a countersuit soon after claiming that Teva’s patents should not have been listed on the Orange Book in the first place.
Teva’s lawsuit focuses on five key patents, dubbed the ‘712, ‘289, ‘587, ‘808 and ‘889 patents. Together, these patents protect aspects of Teva’s inhaler device and its metered dose system. However, in its suit, the pharma sought to expand this coverage to also include the albuterol formulation that the inhaler delivers.
In his opinion, Chesler agreed with Teva that the term “drug” has a “broad scope” and could include devices or other components “intended for use for the treatment of disease.” However, the Orange Book’s listing statute modifies this definition with the restrictive phrase “for which the applicant submitted the application,” which Chesler argues nullifies Teva’s argument.
“The fact that the statutory definition of ‘drug’ expressly includes devices for treating disease, and their components, does not nullify the restrictive action of the modifying phrase, ‘for which the applicant submitted the application,’” Chesler wrote.
Monday’s ruling comes as the FTC is ramping up its scrutiny of “junk” patents listed on the FDA’s Orange Book. The antitrust watchdog launched its initial salvo in November 2023, challenging more than 100 patents by companies including AbbVie, GSK and Teva. The FTC’s opening campaign included a wide range of pharmaceutical products, including autoinjectors and inhalers.
In April 2024, the FTC sent a warning letter to Novo Nordisk calling into question the patent of its blockbuster diabetes drug Ozempic (semaglutide). The federal agency also wrote to GSK to raise concerns about its asthma inhaler Trelegy Ellipta (fluticasone, umeclidinium, vilanterol).
"Pelosi SHOULD Take Responsibility!" J6 Capitol Police Chief Says Her Staff Blocked Added Security
Former Capitol Police Chief Steven Sund responded to a viral video of former Speaker Nancy Pelosi (D-CA) admitting that she was responsible for the lack of preparedness on Jan. 6, 2021.
"Pelosi should take responsibility!" Sund posted on X, adding "She put herself in the security decision process and her Sergeant at Arms denied my requests for support before and during the Jan. 6 chaos. She undermined my law enforcement capabilities."
Sund, who was in charge of the Capitol Police during Jan. 6, then asked "Why did they change the law (2US1970) that tied my hands?"
On Monday, the House Oversight Committee posted footage of Pelosi admitting "I take responsibility" for the lack of security on Jan. 6.
The video shows Pelosi in an exchange with Chief of Staff Terri McCullough on the evacuation. Pelosi states:
“We have responsibility, Terri. We did not have any accountability for what was going on there. And we should have. This is ridiculous.
You’re going to ask me in the middle of the thing when they’ve already breached…that, should we call the Capitol Police? I mean the National Guard?
Why weren’t the National Guard there to begin with?
…
They clearly didn’t know, and I take responsibility for not having them just prepared for more.”
In February of last year, Sund told journalist Tucker Carlson that Jan. 6 was a "setup" - noting that Pelosi's staff refused to authorize the deployment of the National Guard at the Capitol despite his pleas, and that federal agencies withheld information and warning signs of potential dangers prior to the riot.
"It doesn’t seem like people really want to get to the bottom of it," said Sund, adding "It really doesn’t. And it just gets worse. It gets worse from there."
Sund got approval to bring in the National Guard at 2:09 p.m. Before his approval, he alleged that he begged several generals, including General Michael Flynn, to bring the National Guard. The officials told Sund they did “not like the optics of the National Guard” as he allegedly begged for their assistance to intervene in the violence. -Daily Caller
"This sounds like a set up to me," Carlson said, adding "I'm sorry, it does."
To which Sund replied:
"It gets better. So I beg and beg and he goes ‘well, I’m gonna walk down the hall and we’ll talk to the Secretary of Defense or whoever he’s gonna talk to. Right then I get a notification, oh, I’m still on the call, we have the shooting of Ashli Babbitt. And I said we have shots firing, I still remember yelling over the phone. We have shots firing on the U.S. Capitol, is that urgent enough for you now?"
According to Sund, the National Guard didn't show up until 6 p.m., hours after the fatal shooting of Babbitt. He also claimed that the Pentagon deployed resources to the homes of generals, but not the Capitol.
Watch:
Sanders Seeks Subpoena for Novo Nordisk President Regarding Ozempic, Wegovy Pricing
Sen. Bernie Sanders (I-Vt.) on Tuesday moved to subpoena Novo Nordisk President Doug Langa, forcing him to testify before the Senate’s Health, Education, Labor and Pensions Committee regarding the pricing of the pharma’s top-selling semaglutide brands Wegovy and Ozempic.
Sanders, chair of the committee, wants to know why Novo charges up to 10 to 15 times more for Ozempic and Wegovy in the U.S. compared with other developed countries. The committee is set to vote on the subpoena on June 18 and a hearing is scheduled for July 10.
“It is not acceptable to me or the American people that we continue to be ripped off by giant pharmaceutical companies who make huge profits every year while charging us outrageous prices,” Sanders said in a statement, adding that the committee has “time and time again” reached out to Novo to request its voluntary attendance at a hearing.
“Unfortunately, despite all of our efforts, they have repeatedly denied our requests,” Sanders said.
Tuesday’s news comes after the senate committee in April 2024 launched a probe into the pricing of Ozempic and Wegovy. At the time, Sanders highlighted the differences in cost of these treatments among countries. In the U.S., Ozempic goes for $969 per month, while the drug is available for $155 in Canada and $59 in Germany. Wegovy, which costs $1,349 a month in the U.S., can be bought for $92 in the U.K.
Sanders reiterated these figures in his statement on Tuesday, adding that at their current prices, Wegovy and Ozempic could have far-reaching implications for the U.S. healthcare system.
The senator contends that Wegovy and other anti-obesity medications would cost Medicare and Medicaid $166 billion per year if half of its beneficiaries with obesity took these treatments. This sum rivals “what the two federal health programs spent on all retail prescription drugs in 2022,” according to Sanders.
In March 2024, a study was published in the Journal of the American Medical Association which found that injectable formulations of semaglutide, such as Wegovy and Ozempic, could be profitably manufactured for less than $5 per month, much lower than the treatments’ current list prices.
Generic versions of these treatments, as well as a more robust competitive landscape, could further push GLP-1 prices down to as low as $0.75 per month, according to the study.
Merck Eyes Next-Generation Obesity Drugs That Offer Additional Benefits
To break into the lucrative weight loss market, Merck will focus on the development of next-generation anti-obesity medications that offer additional cardiometabolic benefits beyond simple body weight reduction, company executives said on Tuesday.
During the 45th Annual Goldman Sachs Healthcare Conference, Merck CEO Robert Davis said that the pharma is focusing its R&D efforts more on small molecule oral drugs rather than injectables. “We’re very much focused on the second- and third-generation opportunities,” Davis said, adding that it is looking to advance GLP-1-targeting candidates that target other conditions on top of eliciting weight loss.
“I think everything is couched in terms of obesity first, and then we think about some of these outcomes kind of supporting the reimbursement within the broader obesity space,” Davis said.
Merck’s entry to the GLP-1 market is efinopegdutide, which is an investigational peptide therapeutic that also activates the glucagon receptor. While activating both the GLP-1 and glucagon pathways is a well-known mechanism of action for its weight loss and anti-diabetic effects, Merck is instead trialing efinopegdutide primarily as a treatment for metabolic dysfunction-associated steatohepatitis (MASH).
The exact mechanism of efinopegdutide for treating MASH is not yet completely understood, but it might reduce liver damage, steatosis and inflammation.
Merck exclusively licensed efinopegdutide from Hanmi Pharmaceutical in 2020 for $10 million upfront and the promise of up to $860 million in milestones. In June 2023, the FDA granted the GLP-1/glucagon receptor dual agonist its Fast Track designation for MASH.
In addition to MASH, Merck is also developing efinopegdutide for non-alcoholic fatty liver disease (NAFLD), for which it is running a mid-stage study testing the candidate against Novo Nordisk’s semaglutide. Results from the trial showed that efinopegdutide achieved a significantly greater reduction in liver fat content, while eliciting similar weight loss.
Merck’s strategy of focusing on next-generation oral GLP-1 therapies with additional clinical benefits is meant to help it break into the lucrative obesity market, which many analysts expect to reach $150 billion in value by the early 2030s.
Novo and Eli Lilly are the two early leaders in the obesity space—and are anticipated to control around 80% of the market—but, like Merck, several other biopharma players are vying for a spot in the GLP-1 game. Boehringer Ingelheim is advancing survodutide, which also targets the GLP-1 and glucagon receptors. The candidate is in five Phase III trials, which the pharma launched in 2023.
Boston-based startup Syntis Bio debuted earlier this week to advance alternative obesity treatments that do not operate via the GLP-1 pathway. Its lead program, SYNT-101, blocks the absorption of nutrients in the duodenum and mimics the effects of gastric bypass surgery.
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