Some Blues Say They Won't Cover Aduhelm, as FDA's Acting Chief Calls for Investigation

Alzheimer's Coverage Denied

 health care professionals that a clinical trial has shown that Pepaxto (melphalan flufenamide) with dexamethasone increased the risk of death.

The trial, OCEAN, Study OP-103, compared Pepaxto with low-dose dexamethasone to Pomalyst (pomalidomide) with low-dose dexamethasone in patients with relapsed or refractory multiple myeloma following two to four lines of prior therapy and in patients who were resistant to (Revlimid) lenalidomide.

Regulatory officials have required the manufacturer, Oncopeptides, to suspend enrollment in this and other ongoing Pepaxto trials. Patients in the OCEAN will be able to continue if they sign a revised written informed consent.

 that patient safety is paramount and they have ongoing dialogue with the FDA and will provide updated information as soon as more information becomes available. They say this plan to submit complete data from the OCEAN-study to the International Myeloma Workshop meeting in Vienna, Austria, on September 8-11, 2021.

 accelerated approval to Pepaxto in February 2021. Oncopeptides was required to conduct the OCEAN trial as a post-approval requirement under the accelerated approval program.

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The agency has stopped enrollment in all ongoing trials.

FDA Safety Alert: Pepaxto is Associated with Increased Risk of Death

In a simulated evaluation, adding Farxiga (dapagliflozin) to guideline-directed medical therapy was projected to produce substantial clinical gains for patients with heart failure with reduced ejection fraction (HFrEF) with an acceptable increase in associated costs, according to a new 

At a willingness-to-pay threshold of $100,000 per quality-adjusted life year (QALY) gained, treatment with Farxiga, at an assumed annual cost of $4,192, would be a cost-effective therapy in patients with HFrEF.

Farxiga was projected to add 0.63 QALYs at an incremental lifetime cost of $42,800, for an incremental cost-effectiveness ratio of $68,300 per QALY gained. Findings were similar in people with or without diabetes. Investigators found adding Farxiga was cost-effective in 94% of 10,000 probabilistic simulations, and they said this suggests that the findings are robust across a wide range of estimates of key model parameters.

In this analysis, investigators simulated a cohort of patients at least 66 years of age and with 41.8% who had diabetes at baseline. Median survival in the guideline-directed medical therapy arm was 6.8 years, with a range of 3.5 to 11.3 years.

Investigators’ primary outcome was incremental cost-effectiveness ratio of adding Farxiga to guideline-directed medical therapy compared with guideline-directed medical therapy for the entire cohort and stratified by baseline diabetes status.

Farxiga, developed by AstraZeneca, is a sodium-glucose cotransporter 2 (SGLT2) inhibitor 

 to treat patients with type 2 diabetes, heart failure, and in 

The findings were similar in people with or without diabetes.

Analysis Finds Farxiga a Cost-effective Addition for Some Patients with Heart Failure

Since 2011, Medicare spending on oral anticoagulants has increased 16-fold to more than $7 billion annually, accounting for 5.9% of Part D spending in 2019, according to a 

Spending growth was driven by rising Part D enrollment, increased oral anticoagulant use, a shift from warfarin to direct oral anticoagulants (DOACs), and rising costs.

The 2019, the three most commonly used medications were Eliquis (apixaban) accounting for 41.4% of prescriptions and 59.5% of spending. Generic warfarin accounted for 29.6% of prescriptions; 1.6% of spending. Xarelto (rivaroxaban) accounted for 21.6% of prescriptions and 33.2% of spending.

Eliquis and Xarelto exhibited nearly identical cost growth, with average annual increases of 9.3% for Eliquis and 9.5% for Xarelto. For generic warfarin, annual estimated costs decreased by 27.6% over the study period.

Investigators found that competition among branded direct oral anticoagulants did not substantially curb annual spending increases, and they say this suggests a lack of price competition. The exception was Pradaxa (dabigatran), which faces a disadvantage because it requires multiple doses a day, investigators said the price of Praxada fell slightly from 2017.

Investigators used the Medicare Part D Prescription Drug Event file, which includes annual aggregate data for all Medicare Advantage and stand-alone Part D plans, to study oral anticoagulant use between 2011 and 2019.

They suggest that generic DOACs can help ensure competition prevents unsustainable Medicare drug spending. The first generic for Eliquis was 

 by the FDA in December 2019. But the product has not become available because of patent litigation with Eliquis’ developers Bristol-Myers Squibb and Pfizer.

There is no generic approved for Xarelto.

Competition among branded products did not substantially curb annual spending increases.

Study: Branded Direct Oral Anticoagulants Raised Medicare Part D Spending

Soliris (eculizumab) and efgartigimod improve function and quality of life for patients with myasthenia gravis, a rare neuromuscular disorder. But at Soliris' current price, its estimated cost-effectiveness is well above typical thresholds, and a substantial discount would be needed to meet commonly used thresholds, 

 to a draft report from the Institute for Clinical and Economic Review (ICER).

The cost-effectiveness of efgartigimod will depend on its eventual price, as it is not yet available, ICER officials said. But even at a projected price, this is also likely to be above cost-effectiveness thresholds.

Based on an annual cost of $470,200, the incremental cost/quality-adjusted life-year (QALY) and incremental cost/equal value of life years gained (evLYG) for Soliris were estimated to be $3,746,000. For efgartigimod, using a placeholder price of $286,100, the incremental cost/QALY and incremental cost/evLYG were estimated to be $1,426,000.

Soliris is a monoclonal antibody developed by Alexion Pharmaceuticals. (AstraZeneca

 its acquisition of Alexion on July 21, 2021.) The FDA 

 Soliris in October 2017 for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-AChR antibody positive.

Efgartigimod, developed by Argenx, is an immunoglobulin fragment that targets the neonatal Fc receptor. It is currently being reviewed by the FDA, and a decision is expected in December 2021.

ICER evaluated the cost-effectiveness of Soliris plus conventional therapy versus conventional therapy alone in patients with refractory anti-AChR antibody positive gMG, as well as efgartigimod plus conventional therapy versus conventional therapy alone in the patients with gMG.

Myasthenia gravis is a neuromuscular disorder primarily characterized by muscle weakness and muscle fatigue. It is caused by an antibody-mediated autoimmune response) in which the antibodies inappropriately attack certain proteins in muscles that receive nerve impulses. Autoimmune myasthenia gravis has a prevalence of about 14 to 40 per 100,000 people in the United States, 

 to the National Organization for Rare Disorders.

A substantial discount would be needed to meet cost-effectiveness thresholds.

ICER: Soliris and Efgartigimod Not Cost-effective

 a Complete Response Letter (CRL) for the Biologics License Application (BLA) for Incyte’s retifanlimab, an intravenous PD-1 inhibitor, for the treatment of adult patients with locally advanced or metastatic squamous cell anal cancer who have progressed on platinum-based chemotherapy.

 from the Oncology Drugs Advisory Committee meeting on June 24, 2021, which suggested additional data was needed to demonstrate clinical benefit. The committee voted 13-4 against approving retifanlimab.

“While we are not surprised with the FDA decision given the ODAC recommendation, we are disappointed. We remain committed to advancing science to find solutions for patients with unmet medical needs, and we will ensure close coordination with the FDA in order to address feedback and determine next steps for the review of retifanlimab,” Hervé Hoppenot, chief executive officer of Incyte, said in a statement.

The BLA for retifanlimab is based on data from the phase 2 POD1UM-202 trial evaluating retifanlimab in 94 previously treated patients. Incyte

 results from this trial in September 2020, which found retifanlimab monotherapy resulted in a objective response rate of 14% as determined by independent central review.

The FDA would like to see additional data on clinical benefit for patients with anal cancer.

Incyte Receives Complete Response Letter for Oncology Therapy

While patients starting treatment for high blood pressure can benefit equally from two different classes of medicine — angiotensin-converting enzyme (ACE) inhibitors and angiotensin receptor blockers (ARBs) — ARBs may be less likely to cause side effects, a new study reveals.

The extensive analysis, based on 3 million patients’ electronic health record and insurance claim databases in the United States, Germany, and South Korea, was 

While ACE inhibitors are prescribed more commonly for first time blood-pressure patients, ARBs work just as well and may cause fewer side effects, according to the American Heart Association.

“In professional guidelines, several classes of medications are equally recommended as first-line therapies. With so many medicines to choose from, we felt we could help provide some clarity and guidance to patients and health care professionals,” RuiJun Chen, M.D., lead author of the study and assistant professor in translational data science and informatics at Geisinger Medical Center in Danville, Pa., said in a 

"If a patient is starting hypertension therapy for the first time, our results point to starting with the ARB over the ACE inhibitor,” George Hripcsak, M.D., senior author of the study and professor and chair of biomedical informatics at Columbia University Vagelos College of Physicians and Surgeon, said in a 

The researchers analyzed health records for patients who began first-time blood pressure-lowering treatment with a single medicine between 1996 and 2018 and compared the occurrence of heart-related events and stroke among nearly 2.3 million patients treated with ACE inhibitors with about 674,000 patients treated with ARBs.

Compared with those taking ARBs, people taking ACE inhibitors were 3.3 times more likely to develop fluid accumulation and swelling of the deeper layers of the skin and mucous membranes, 32% more likely to develop a cough, 32% more likely to develop sudden inflammation of the pancreas, and 18% more likely to develop bleeding in the gastrointestinal tract.

While ARBs may have fewer side effects than ACE inhibitors among those just beginning treatment, “we unfortunately cannot extend these conclusions to people who are already taking ACE inhibitors or those who are taking multiple medications,” Chen said.


Angiotensin receptor blockers may be less likely to cause side effects than ACE inhibitors.


Study: Blood Pressure Meds Work Equally, But One Has Fewer Side Effects

Current drug shortages based on FDA data, prepared by Formulary Watch.

Formulary Watch: Drug Shortages, July 25, 2021

 late July 16 that the FDA had approved Vaxneuvance (Pneumococcal 15-valent Conjugate Vaccine), a vaccine that provides protection against invasive disease caused by several serotypes of 

. It is approved for adults 18 years of age and older, based on data from seven randomized clinical trials that showed immune responses elicited by Vaxneuvance were non-inferior to the currently available 13-valent pneumococcal conjugate vaccine (PCV13) for the 13 shared serotypes. Merck’s new vaccine is administered as a single dose via intramuscular injection. Common adverse reactions include pain at the injection site, fatigue, muscle pain, headache, redness and swelling at the injection site, and arthralgia.

Full approval for Keytruda in endometrial cancer. 

 full FDA approval for the combination of Keytruda (pembrolizumab) in combination with Eisai’s Lenvima (lenvatinib) for the treatment of patients with advanced endometrial carcinoma that is not microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR), who have disease progression following prior systemic therapy. Approval is based on results from the pivotal phase 3 KEYNOTE-775/Study 309 trial, which showed the combination significantly improved overall survival, reducing the risk of death by 32%, and progression-free survival, reducing the risk of disease progression or death by 40%, versus chemotherapy. Previously, the combination received accelerated approval.

Real-world evidence used in Prograf approval in lung transplants. 

 Astellas’ Prograf (tacrolimus), the first approval of an immunosuppressant drug to prevent organ rejection in adults and peditatric patients who receive lung transplant. Approval is based on an observational study using data are from the U.S. Scientific Registry of Transplant Recipients, which captures data on all transplants performed in the United States. The study analyzed outcomes based on discharge immunosuppression treatment regimens in recipients of a primary lung transplant between 1999 and 2017. In patients who received Prograf in combination with azathioprine, the one-year graft survival estimates from time of discharge were 90.8% for adults and 84.7% for pediatric patients. The safety profile for lung transplant patients treated was consistent with the safety profile in liver, kidney and heart transplant patients treated with Prograf.

Approval for AstraZeneca’s pediatric diabetes therapy Bydureon BCise.

 Bydureon BCise (exenatide extended-release), a once-weekly injectable suspension for the treatment of type 2 diabetes and to improve glycemic control in pediatric patients. The action was based on findings from a 24-week phase 3 trial with a 28-week open-label extension involving patients aged 10 to 17 years, which found those taking the study drug alongside a regimen of diet and exercise alone or diet, exercise and oral agents and/or insulin achieved a greater average reduction in A1C compared with placebo. Previously, Bydureon BCise was approved in the United States as a once-weekly single-dose autoinjector device for adults with type 2 diabetes.

 Wednesday that the FDA will not meet the goal date for a decision under the Prescription Drug User Fee Act (PDUFA) on the supplemental NDA for Xeljanz (tofacitinib) for the treatment of adults with active ankylosing spondylitis. The FDA had previously extended the PDUFA goal dates to early third quarter 2021. ​The FDA cited its ongoing review of Pfizer's post-marketing safety study, ORAL Surveillance, evaluating tofacitinib in rheumatoid arthritis patients, as a factor for the extensions. Xeljanz is approved in the United States for four indications: adults with moderately to severely active rheumatoid arthritis (RA) after methotrexate failure, adults with active psoriatic arthritis, adults with moderately to severely active ulcerative colitis after tumor necrosis factor inhibitor failure, and patients 2 years of age or older with active polyarticular course juvenile idiopathic arthritis.

First therapy for rare skin and muscle disease.

 to Octapharma USA for Octagam 10% [Immune Globulin Intravenous (Human)], the first intravenous immunoglobulin (IVIg) to be indicated for the treatment of adult dermatomyositis, a rare immune-mediated inflammatory disease. Approval is based on the results of ProDERM, a pivotal randomized clinical trial and the first study to evaluate the long-term efficacy and safety of intravenous immunoglobulin (IVIg) for adults with dermatomyositis. In the initial 16-week phase, 78.7% of patients receiving Octagam responded positively to treatment, compared with 43.8% receiving placebo. After switching to IVIg in the extension period, the placebo group attained a similar response rate at week 40. Dermatomyositis is a rare idiopathic autoimmune disorder of unknown cause that affects adults between age 40 and 60 years old, who suffer from skin rashes, chronic muscle inflammation and progressive muscle weakness. The estimated incidence of dermatomyositis is 9.63 cases per million people, 

 to the National Organization of Rare Disorders.

FDA approves therapy for severe itching for patients with liver disease.

 Albireo Pharma’s Bylvay (odevixibat), for treatment of pruritus, which causes a severe urge to scratch in patients aged three months and older who have progressive familial intrahepatic cholestasis (PFIC), a rare progressive liver disease that typically leads to liver failure. In many cases, PFIC leads to cirrhosis and liver failure within the first 10 years of life. Bylvay is a potent, non-systemic ileal bile acid transport inhibitor (IBATi), which does not require refrigeration and is easily administered as a once-daily capsule or sprinkled on food. The approval of was supported by from two global, phase 3 trials.

Keytruda gets a full approval in endometrial cancer; while Xeljanz will miss another PDUFDA date.

Merck Vaccine and Other FDA Updates

It takes nearly three years before first generics are covered on more than half of Medicare Part D formularies, according to a 

 by the Association for Accessible Medicines. In 2020, 72 first generics were approved. They typically enter the market at a discount between 40% to 60%.

On average, of the first generics launched in 2020, only 21% are covered by plan formularies in Medicare Part D plans. When first generics were covered, they were placed on non-generic tiers 79% of the time. In comparison, an average of 66% of commercial plans are covering first generics launched in 2020. Of those plans that cover first generics, 98% place them on generic tiers.

“There are structural elements of the Medicare drug program that unintentionally are causing plans to devalue or undervalue generics in the Medicare program compared to how they value them in commercial health care settings,” Craig Burton, vice president of policy at Association for Accessible Medicines, said in an interview with Formulary Watch®. “This means that seniors in Medicare are facing delayed access to lower costs drugs. As a result, it means they are paying more. It means their out-of-pocket costs are higher because the out-of-pocket cost is based on the list price. And when the only drug that is covered is the brand, their co-insurance is going to be higher.”

Burton noted the use of generics instead of branded products means lower costs overall and lower costs for seniors. He cited a 2019 report by the FDA, which found greater competition among generic drug makers is associated with lower generic prices.

Additionally, branded dispensing incurred an incremental annual cost of $1.67 billion to the Medicare program and $270 million to patients when compared with switching to generics, according to a 

This study examined data from January 2020 through October 2020. Among 169 million claims for 224 multisource drugs, 8.3 million claims (4.9%) were dispensed with a branded product. If all branded dispensing requested by prescribers had been substituted by the corresponding generics, the projected savings to the Medicare Part D program would have been $997 million (56.0%) and to Medicare patients $161 million (64.4%).

When Medicare D plans do provide coverage for first generics, they are often placed on non-generic tiers.

Medicare Part D Plans are Slower to Cover First Generics

 that the National Comprehensive Cancer Network (NCCN) added Rylaze (asparaginase erwinia chrysanthemi (recombinant)-rywn) to the Clinical Practice Guidelines in Oncology for acute lymphoblastic leukemia (ALL), for both pediatric and adult patients.

 Rylaze on July 21, just three weeks after the drug was approved by the FDA. The approval followed review of a biologics license application (BLA) under the FDA’s Real-Time Oncology Review program, and it was based on clinical data from a pivotal phase 2/3 trial conducted in collaboration with the Children’s Oncology Group.

“Asparaginase is a core component of chemotherapeutic regimens in ALL and lymphoblastic lymphoma; however, treatments derived from 

 are associated with the potential for hypersensitivity reactions, which can affect a substantial number of these patients,” Rob Iannone, M.D., executive vice president, research and development and chief medical officer at Jazz Pharmaceuticals, said in a statement. “Before the FDA approval of Rylaze, there was a significant need for an effective and reliable supply of asparaginase medicine that would allow patients the opportunity to start and complete their prescribed treatment program with confidence.”

The only other FDA-approved drug for such patients with allergic reactions has been under global shortage since 2016. Jazz Pharmaceuticals’ Erwinaze (asparaginase erwinia chrysanthemi) has 

 supply and manufacturing issues from the owner and manufacturer of the product, Porton Biopharma Limited. Jazz Pharmaceuticals’ agreement with Porton Biopharma ended in December 2020.

The June 30 approval meets an unmet need in this cancer.

NCCN Adds Jazz’s ALL Therapy Rylaze to Practice Guidelines

 the Clozapine Risk Evaluation and Mitigation Strategy (REMS). Starting November 15, 2021, pharmacies that dispense clozapine will now need to obtain a pre-dispense authorization that can be accessed on the Clozapine REMS website (

) or the Clozapine REMS Contact Center (888-586-0758). They will no longer be able to use the current system, which is called the “switch system,” for verification.

Dispensing pharmacies, as well as wholesalers and distributors, will also need to recertify or re-enroll in the program, which can be done on the website beginning August 16, 2021.

Health care professionals prescribing clozapine will need to re-enroll all patients that are currently prescribed clozapine into the new Clozapine REMS. Physicians can now submit Patient Status Forms monthly, but there is a new form that is being used to document the absolute neutrophil count (ANC) results, monitoring frequency, and appropriateness to continue treatment.

Clozapine, including generics and branded therapies (Clozaril, Fazaclo ODT, Versacloz), is an atypical antipsychotic medicine used to treat schizophrenia symptoms that are not controlled with standard antipsychotic drugs. The drug is also used in patients with recurrent suicidal behavior associated with schizophrenia or schizoaffective disorder.

Clozapine, however, is associated with neutropenia, which can lead to serious and fatal infections.

The Clozapine REMS program was originally approved in September 2015 to monitor patients taking any of the clozapine products. Before this, each manufacturer operated separate monitoring programs.

Pharmacies will need to obtain pre-dispense authorization for clozapine.

FDA Updates Clozapine REMS Program

 to safety labeling for Piqray (alpelisib), Novartis’ therapy for patients with advanced or metastatic breast cancer, saying there have been some fatal cases of ketoacidosis in the postmarket setting.

The Warnings and Precautions section of the label, under hyperglycemia, now reads: “Severe hyperglycemia, 

in some cases associated with hyperglycemic hyperosmolar non-ketotic syndrome (HHNKS)

 or ketoacidosis has occurred in patients treated with PIQRAY. 

Some fatal cases of ketoacidosis have occurred in the postmarketing setting 

…” (Additions/changes are in italics.)

HHNKS is a serious complication of type 2 diabetes resulting from high blood glucose levels, which can result in a coma or seizures. Ketoacidosis is a serious complication of diabetes that occurs when liver metabolizes fat for fuel, resulting in high levels of ketones, a byproduct of that process.  

 Piqray in 2019 for use in combination with Faslodex (fulvestrant) for postmenopausal women, and men, with HR+/HER2- PIK3CA-mutated advanced or metastatic breast cancer following progression on or after an endocrine-based regimen.

A Novartis spokesperson said the company continues to learn more about the therapy as additional patients are treated. In the postmarketing setting, it is a regulatory requirement to continue to collect real-world safety data.

This process showed increased severity of an already known adverse reaction ketoacidosis, which triggered the prescribing Information update, the spokesperson said in an email. “At Novartis, patient safety is our utmost priority. The updated guidance for additional safety monitoring is to help physicians appropriately manage potential serious side effects,” she said.

That guidance suggests that, in addition to monitoring HbA1C every three months, physicians monitor fasting glucose more frequently for the first few weeks of treatment in patients with high risk factors for hyperglycemia such as obesity, elevated fasting plasma glucose  and HbA1c levels, use of systemic corticosteroids, and those over the age of 75.

Piqray continues to demonstrate that it is an important treatment option to address the needs of about 40% of patients with HR+/HER2- advanced breast cancer whose tumors harbor a PIK3CA mutation, which is linked to a poorer prognosis in the metastatic setting, the spokesperson said.

Some fatal cases of ketoacidosis have occurred, and high-risk patients should be monitored when starting treatment.


Piqray’s Safety Label is Updated

 a Complete Response Letter (CRL) from the FDA for its NDA for tenapanor. The therapy is under review for the control of serum phosphorus in adult patients with chronic kidney disease (CKD) on dialysis. Regulators noted that data submitted indicate the treatment effect is small and of unclear clinical significance.

Additionally, the FDA noted that for the application to be approved, Ardelyx needs to conduct an additional well-controlled trial demonstrating clinical relevance on its effect on serum phosphorus. There were no safety, clinical pharmacology/biopharmaceutics, CMC or non-clinical issues identified in the CRL.

“We are saddened by this communication from the FDA and what it means for the patients and the physicians who treat them,” Mike Raab, president and chief executive officer of Ardelyx, said in a statement. “We continue to believe tenapanor represents an important, first-in-class treatment option for patients with elevated phosphorus. We do not agree with the FDA's subjective assessment on the clinical relevance of the treatment effect of tenapanor in our studies, which met all clinical endpoints agreed upon by the FDA. In our view, the serum phosphorus lowering data generated with tenapanor in all of our clinical studies is meaningful and clinically significant."

 an update indicating the FDA thought there were deficiencies in the NDA related to the size of the treatment effect and its clinical relevance.

In April, the FDA extended the PDUFA review period for tenapanor to July 29, 2021. The agency had requested the company to submit additional analyses in light of tenapanor’s novel mechanism of action. Ardelyx has submitted its NDA in June 2020.

The FDA is requesting an additional study to demonstrate tenapanor lowers serum phosphorus. 

Ardelyx Receives Complete Response Letter for Kidney Disease Therapy

There has been a rapid increase in the costs of oncology drugs, but often they are not priced based on the value they provide, according to Andrew Hertler, M.D., chief medical officer of New Century Health, a specialty care management company focused on oncology and cardiology in Brea, Calif.

The value of new oncology therapies comes from increasing survival and improving the quality of life for patients, but newer therapies only provide incremental benefit that comes with a high price tag. “In 2020 alone, the average prices of new drugs coming to market [were] between $100,000 and $200,000 a year, with some drugs costing as much as $400,000 for an individual dose,” he said in an interview with 

Competition from newly approved therapies in the same class hasn’t worked to lower the cost. “Prices go up about 10% a year at pricing that is determined by what the market will bear. Patients really don’t have an option, because they want to live. We don't really have a shock on the price,” Hertler said.

An example, he said, is the checkpoint inhibitor class. Merck’s Keytruda (pembrolizumab), the first checkpoint inhibitor that targets the PD-1/PD-L1 pathway, was seen as a breakthrough when it was approved in 2014 for advanced melanoma. Since that time, it has received additional approvals and is now approved for 30 indications, with the most 

 being for the treatment of patients with high-risk early-stage triple-negative breast cancer (TNBC) in combination with chemotherapy before surgery and then continued as a single agent after surgery.

Today, Keytruda has a wholesale acquisition

 of $10,067 when given every three weeks, compared with a monthly 

 of $8,725 when it was approved. Since then, several other PD-1 checkpoint inhibitors have become available, including Bristol Myers Squibb’s Opdivo (nivolumab), Genentech’s Tecentriq (atezolizumab), and Regeneron’s Libtayo (cemiplimab).

The newest innovations, the chimeric antigen receptor (CAR) T-cell therapies, have an even higher price tag, especially when the total cost of patient care is considered. In fact, the total cost of administering CAR T-cell therapies averaged more than $700,000, and in some cases exceeded $1 million, even though the wholesale acquisition cost of the treatment is $373,000, according to data from a recent real-world data 

Over two-and-a-half years, Prime investigators looked at 74 members of Blue Cross and Blue Shield plans who received Novartis’ Kymriah (tisagenlecleucel) or Kite Pharma’s Yescarta (axicabtagene ciloleucel for the treatment of B-cell lymphoma.

The cost for CAR-T medication alone was $527,000 on average, which was 74% of the total cost during the initial treatment period. However, 12% of patients with post–CAR-T events incurred more than $1 million for their total cost of care, including drug and other costs, such as infusion center visits or transplants.

A 2018 Institute for Clinical and Economic Review report estimated the cost-effectiveness of the two CAR-T therapies in B-cell cancers and suggested the therapies are priced in alignment with their clinical value, despite their high price. According to the report, the cost-effectiveness of both CAR-T therapies fell near or within commonly cited thresholds of $100,000 to $150,000 per quality-adjusted life years (QALY). Because ICER’s study only estimated total treatment cost, Prime’s real-world evidence is another barometer to consider when evaluating whether CAR-T’s pricing is in line with value.

“Realistically, there has to be a consensus solution for these high costs,” Hertler said. “What we can do as physicians is to follow the clinical pathways for any clinical situation for what is the highest value drug. To me, if a drug just isn't that good, we shouldn’t use it. We routinely see approved drugs with 1.4 months to 1.6 months improvement in progression free survival. For marginal drugs, we don’t have to put them on our pathways.”

Drugs that provide only incremental benefit may not be included on clinical pathways.


Specialty Pharmacies Grapple with Assessing Costs and Value of Oncology Therapies

 the first biosimilar insulin to improve glycemic control in adults and pediatric patients with type 1 diabetes and in adults with type 2 diabetes.

Developed by Mylan Pharmaceuticals, now Viatris, in partnership with Biocon Biologics, Semglee (insulin glargine-yfgn) is interchangeable with its reference product, Sanofi’s Lantus (insulin glargine), a long-acting insulin analog. Semglee is the first interchangeable biosimilar product approved in the United States for the treatment of diabetes.

Semglee, offered in 10 mL vials and 3 mL prefilled pens, is administered subcutaneously once daily. It is not recommended for treating diabetic ketoacidosis. In a 

, Viatris officials said commercial preparations for launch are under way.

FDA officials noted that biosimilars typically have launched with initial list prices 15% to 35% lower than comparative list prices of the reference products.

Viatris and Biocon Biologic's insulin glargine has received regulatory approval in more than 60 countries around the world and was the third product approved by the FDA through the Viatris-Biocon Biologics collaboration. The company is eligible to have exclusivity for 12 months before the FDA can approve another biosimilar interchangeable to Lantus.

 in November 2020 after the merger of Mylan and Pfizer’s Upjohn business and has U.S. headquarters in Pittsburgh, Pa.

The approval allows for substitution at the pharmacy counter for Lantus, its reference product.

FDA Updates Clozapine REMS Program

Piqray’s Safety Label is Updated

Ardelyx Receives Complete Response Letter for Kidney Disease Therapy

Specialty Pharmacies Grapple with Assessing Costs and Value of Oncology Therapies

FDA Approves First Biosimilar Insulin