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Monday, January 13, 2020

Drugmakers Test New Ways to Pay for Six-Figure Treatments

Drugmakers are experimenting with new ways to get paid for their most-expensive medicines, as resistance to escalating prices builds and improvements are made in collecting and analyzing patient data.
Now that six-figure price tags are common, drug companies are finding creative ways to get reimbursed, from installment plans to subscriptions to more complex value-based contracts that tie payment to when a drug helps a patient. For years, pharmaceutical companies would typically set a price for a drug and then get paid per pill sold at that price, less any negotiated rebates.
Alnylam Pharmaceuticals Inc. now will charge full value for a nearly $600,000 new rare-disease drug only if a patient gets a benefit akin to what was seen in clinical testing, and it will make the drug cheaper for insurers if they cover more patients than expected. Sanofi SA is offering $99-a-month subscriptions for insulin. Novartis AG — which sells a gene therapy at $2.1 million, the most expensive drug in the world — is giving insurers the opportunity to pay over five years.
The drug-reimbursement innovation comes as cries for relief mount. Congress is considering plans to lower drug costs, while the Trump administration has proposed importing drugs from Canada. Earlier this month, drugmakers raised prices of hundreds of prescription medicines, The Wall Street Journal reported.
Meanwhile, health plans are controlling costs by restricting prescriptions for certain high-price medicines to a narrow set of patients.
Drugmakers “understand that if they come to the market with superhigh-cost drugs and aren’t willing to share the risk then they are going to face pushback and access challenges,” said Michael Sherman, chief medical officer of insurer Harvard Pilgrim Health Care.
It remains to be seen, however, how widely the innovative reimbursement programs will be adopted. Many previous experiments with installment-plan payments, for instance, were directed at drugs for rare diseases, not at more widely used treatments.
Dr. Sherman and other health-insurance officials worry the new efforts might give drugmakers cover to keep raising prices, limiting the overall impact on costs.
Insurer Cigna Corp.’s most popular version of value-based contracts refunds two-thirds of the cost to employers if a patient ends up taking a different anti-inflammatory therapy within the first 90 days — which happens in 25% of patients, said Steve Miller, Cigna’s chief clinical officer.
Value-based contracting is “a great lever to pull, but it’s just one more tool in our toolbox,” he said. “It’s definitely not going to revolutionize the system to make it more affordable.”
A big factor driving drug companies to explore new payment mechanisms, industry officials say, is rising employer, patient and political pressure to control health spending, with some prescription drugs costing hundreds of thousands of dollars a year.
Caught in the middle are health plans: They are trying to keep a lid on drug spending for employers while not inciting members by denying coverage.
“For payers, there really is a challenge in wanting to pay for these things,” said Walid Gellad, a drug-policy researcher at the University of Pittsburgh School of Medicine. “So if you can somehow make it easier, it’s going to be better for the payer and for the manufacturers.”
In November, Alnylam said it would calibrate the $575,000-a-year price of newly-approved Givlaari, depending on how patients do on the drug and how many take it. Givlaari treats acute hepatic porphyria, an inherited liver condition in an estimated 3,000 patients in the U.S. and Europe that often requires hospitalization.
Public and private health insurers that agree to participate in the program will pay full value only if patients show a benefit similar to clinical trials, Alnylam Chief Executive John Maraganore said. The company also will charge less if more patients than expected take the therapy.
The concessions may help Alnylam secure reimbursement from health plans that otherwise might recoil at such a high price tag, while maximizing prescriptions, Dr. Maraganore said. “We can work together without creating misaligned incentives around the cost of a new medicine.” he said.
Sanofi in June expanded its $99-a-month subscription program for insulins Admelog, Apidra, Lantus and Toujeo. Without the program, uninsured patients taking Lantus might face an annual bill of more than $4,000.
Sanofi said the program was used more than 52,000 times in 2019.
Novartis launched its gene therapy Zolgensma with an option for insurers to pay over five years in equal annual installments. Zolgensma treats an inherited disease called spinal muscular atrophy.
Gene therapies treating just 11 conditions are projected to cost $45 billion over the next five years and are “perfect candidates” for value-based contracts, according to a research report by CVS Health shared with The Wall Street Journal and that will be published soon.
Such contracts, previously used sparingly in part because of problems assessing how a patient fared on a drug, are becoming more popular, industry officials say.
Digitized medical records, combined with technology to analyze data and see how a patient does on a drug, is making it easier for health plans and drugmakers to agree on metrics for pegging payments.
Eli Lilly & Co. signed 15 such agreements with eight payers last year, according to Frank Cunningham, who leads Lilly’s managed health-care services. He said some agreements are linked to whether medicines successfully lead to patients showing up for work.
Amgen Inc. negotiated value-based contracts for migraine drug Aimovig that reward the drugmaker for factors like reducing emergency room visits, said Kave Niksefat, Amgen’s vice president and head of value & access.
“Tracking multiple metrics across multiple different health-care systems is something that is hopefully the blueprint for the next wave of value-based contracts,” he said.

Accelerate Diagnostics sees FY 2019 revenue as high as $9.3M

Accelerate Diagnostics (NASDAQ:AXDXreports preliminary financial results for Q4 and FY 2019.
Commercially contracted instruments were 137 in Q4 and 304 for FY 2019.
Total revenue for Q4 and FY 2019 is expected to be ~$3.5M and $9.3M, respectively.
FY 2019 Gross margin is expected to be roughly 50%.
Net cash used is expected to be ~$58M for FY 2019.
Full audited financial results for Q4 and FY 2019 will be filed in late February.
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AcelRx +4% premarket on corporate update

AcelRx Pharmaceuticals (NASDAQ:ACRXprovides an update on its business and DSUVIA launch metrics.
166 healthcare facilities are REMS-certified and 148 formulary approvals have been achieved through December 31, 2019, exceeding year-end goals of 125 for each metric.
Year-end 2020 goals of 465 REMS-certified facilities and 465 formulary approvals.
FY 2019 total revenues of $2.3M.
Cash and short-term investments of $66.1M as of December 31, 2019.
Department of Defense Milestone C meeting for DSUVIA is expected in Q2 2020.
Commencement of investigator-initiated studies of DSUVIA in post-operative pain management and Enhanced Recovery protocols is anticipated beginning in H1 2020.
Shares are up 4% premarket.
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Inogen expects Q4 revenue drop, looks to improvement in 2020

Inogen (NASDAQ:INGN-18.2% pre-market after saying it expects Q4 revenues of $78.4M-$79.4M based on preliminary data, down 8.2%-9.4% from the year-ago quarter, citing softer than expected demand primarily in its direct-to-consumer and international channels.
For FY 2019, INGN expects revenues of $361M-$362M, up 0.9%-1.2% from 2018.
INGN updates FY 2020 revenue guidance to $385M-$400M, which would represent 6.4%-10.5% Y/Y growth vs. the 2019 preliminary revenue mid-point of $361.9M, with direct-to-consumer sales expected to be its fastest growing channel.
The company also expects manufacturing challenges to continue in Q1, where certain component part shortages may delay shipments.

Bluebird bio launches Zynteglo for thalassemia in Germany

Bluebird bio (NASDAQ:BLUEannounces the launch in Germany of ZYNTEGLO (autolog
https://seekingalpha.com/news/3531175-bluebird-bio-launches-zynteglo-for-tdt-in-germanyous CD34+ cells encoding βA-T87Q-globin gene), a one-time gene therapy for patients 12 years and older with transfusion-dependent β-thalassemia (TDT) who do not have a β0genotype, for whom hematopoietic stem cell transplantation is appropriate but a human leukocyte antigen-matched related HSC donor is not available. This is the first time ZYNTEGLO is commercially available.
Bluebird has also collaborated with University Hospital of Heidelberg as the first qualified treatment center in Germany.
In addition, bluebird has entered into value-based payment agreements with multiple statutory health insurances in Germany to help ensure patients and their healthcare providers have access to ZYNTEGLO.
Bluebird has initiated the rolling BLA submission for U.S. FDA approval. The company is planning to complete the BLA submission in H1 2020.

Ultragenyx files U.S. application for expanded use of Crysvita

Ultragenyx (NASDAQ:RARE) and collaboration partner Kyowa Kirin (OTCPK:KYKOFannounce the filing of a supplemental marketing application in the U.S. seeking approval to use Crysvita (burosumab) to treat FGF23-related hypophosphatemia associated with phosphaturic mesenchymal tumors (tumor-induced osteomalacia) that cannot be curatively resected or localized.
The companies are co-commercizing burosumab in the U.S. under a 2013 agreement.
The agency approved the FGF23-blocking antibody in April 2018 for X-linked hypophosphatemia.

Blueprint Medicines lays out growth plans

Blueprint Medicines Corporation (NASDAQ:BPMC) formally sets out its corporate goals for 2020.
“As we complete our evolution into a fully-integrated biopharmaceutical company this year, we will also aim to bring a second product to market, expand across multiple indications and extend our global commercial footprint with our first anticipated regulatory approval in Europe.”
The company says its future growth will also be fueled by an expanded strategic focus on systemic mastocytosis and related mast cell disorders, which represent a large population of underserved patients with significant medical needs.
In particular, Blueprint expects to report top-line data from the Phase 3 VOYAGER trial of avapritinib in third-line GIST in Q2 and gain regulatory approval and launch avapritinib in fourth-line GIST in the U.S. During Q3, the company aims to gain regulatory approval and launch avapritinib in PDGFRA D842V GIST in Europe.
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Source: Press Release