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CVS Announces It Will Launch Clinical Trials Division

Here’s something that you don’t see every day….. CVS has announced that it is opening a new division to provide Clinical Trial Services. Yep, the whole magilla, including patient recruitment.

So, are clinical trial services a good fit for CVS? In short, yes.

With the number of new therapies growing exponentially the need for effective clinical trial management partners are top of mind with manufacturers. There are many CRO / Clinical Trial companies, but demand is now exceeding supply. Also, the track record for established companies is less than stellar. Participant recruitment and retention prevent many trials from finishing successfully. Upwards of 80% of studies don’t meet participant enrollment deadlines, and around 30% of participants drop out before a study is completed due to reasons ranging from inconvenient site location, complex participation requirements, and trial duration.

CVS has some limited experience with clinical trials and offers some good results to showcase. One example includes CVS’ infusion subsidiary, Coram. That particular trial achieved a patient retention rate close to 90% – well over the 70% average rate – due to Coram’s integrated position as the home care / infusion & enteral nutrition provider for the trial participants.

CVS can fast-track recruitment by tapping into its massive customer base as pharmacy benefit manager, its national retail pharmacy network, and its insurance business to find eligible patients. The cherry on the top is that 75% of people in the US live within 3 miles of a CVS store, which boosts accessibility….. about 40% of patients say that distance from trial sites and schedule conflicts are top reasons for dropping out.

But….. the there is one huge reason to use clinical trials as a way to get in on the ground level with manufactures….. CVS can increase its chances of being selected as the HUB provider, and/or the exclusive or a select specialty pharmacy if the product launches through limited distribution.
A good example of having one’s cake and eating it too!

CVS Health Introduces Clinical Trial Services

The new business is uniquely positioned to continue delivering research solutions for COVID-19 and beyond with community presence, data capabilities and health care expertise

WOONSOCKET, R.I., May 20, 2021 /PRNewswire/ — Clinical trials are critical in evaluating the safety and efficacy of investigational new drugs and devices and making them available to those who need them. Despite their importance, less than 4 percent of the U.S. population participates in clinical studies. In addition, 80 percent of studies don’t meet participant enrollment deadlines, and an average of 30 percent of participants drop out before a study is completed.1 CVS Health (NYSE: CVS) today announced its new Clinical Trial Services business that brings together innovation and experience to help solve these challenges, driving greater access to clinical trials across the communities it serves and creating a more efficient, convenient experience to improve participant retention and research effectiveness.

CVS Health
“Traditionally low patient enrollment, diversity and engagement coupled with inconvenient trial sites, challenging study participation requirements, including the length of participation, show the need to improve the current model – particularly in response to the COVID-19 pandemic,” said Troyen A. Brennan, M.D., MPH, Executive Vice President and Chief Medical Officer of CVS Health. “Combining clinical trial expertise from across the CVS Health enterprise with our growing connection to the communities we serve, will help create a new clinical trial experience that works better for participants, health care providers, clinical research organizations and study sponsors.”

CVS Health Clinical Trial Services is working with key stakeholders in the biopharmaceutical industry and across the clinical trial ecosystem to design and deliver innovative approaches to research and real-world evidence generation. The new business will initially focus on scaling three core capabilities:

Precision patient recruitment: Leveraging analytics, national reach, and local community connections to engage individuals by helping them learn about clinical trial opportunities that may be appropriate for them
Clinical trial delivery: Innovative, decentralized options for the delivery of Phase III/IV clinical trials and real-world evidence studies at CVS locations, at home or virtually
Real-world evidence generation and studies: Retrospective and prospective studies that measure the impact of novel devices and therapeutics in real-world settings
CVS Health collaborated with the pharmaceutical industry to help facilitate clinical trials for investigational COVID-19 vaccines and treatments. Using a specially designed digital model and screening protocols, CVS Health engaged more than 300,000 volunteers who met the study inclusion criteria for COVID-19 vaccine trial consideration and helped them connect to studies close to where they live.

The Clinical Trial Services business will continue to use CVS Health’s expertise in both health care services and clinical research, including more than 200 in-home clinical trials, real-world evidence studies, and support for publication of peer-reviewed articles.


FDA Approves New Specialty IV Tx for NSCLC – Rybrevant

The FDA recently approved a new Specialty Infusion therapy, Rybrevant (amivantamab-vmjw) from Janssen Pharmaceutical, as the first treatment for adult patients with non-small cell lung cancer (NSCLC) with specific genetic mutations.

Infused specialty therapies were once relegated only to the medical benefit and traditional buy-and-bill. However, Rybrevant’s approval continues the growing trend that has created an integral role for specialty pharmacies supporting distribution under the medical benefit or direct-to-office using PBM billing (payer specific).

As with many of these next generation therapies, Rybrevant was approved in combination with a companion diagnostic test to identify targeted EGFR exon 20 mutations. This group represents only 2 or 3 percent of NSCLC patients.

Pricing for Rybrevant has not yet been confirmed by Janssen. Additionally, Janssen has not announced its plans for distribution and pharmacy access once the product launches. Analysts have indicated that its expected cost, the very small patient population, and need for genetic testing will push this therapy into limited distribution.

FDA Approves First Targeted Therapy for Subset of Non-Small Cell Lung Cancer

May 21, 2021 — Today, the U.S. Food and Drug Administration approved Rybrevant (amivantamab-vmjw) as the first treatment for adult patients with non-small cell lung cancer whose tumors have specific types of genetic mutations: epidermal growth factor receptor (EGFR) exon 20 insertion mutations.

The FDA also approved the Guardant360 CDx (Guardant Health Inc.) as a companion diagnostic for Rybrevant today.

“Advances in precision oncology continue to facilitate drug development, allowing diseases like lung cancer to be subset into biomarker-defined populations appropriate for targeted therapies,” said Julia Beaver, M.D., chief of medical oncology in the FDA’s Oncology Center of Excellence and acting deputy director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research.

Lung cancer is the most common cancer type and the leading cause of cancer-related deaths worldwide, with non-small cell lung cancer accounting for 80% to 85% of all lung cancers, according to the American Cancer Society. Approximately 2% to 3% of patients with non-small cell lung cancer will have EGFR exon 20 insertion mutations, which are a group of mutations on a protein that causes rapid cell growth, and consequently, helps cancer spread. EGFR exon 20 insertion mutations are the third most common type of EGFR mutation.

Researchers evaluated Rybrevant’s efficacy in a study of 81 patients with non-small cell lung cancer and EGFR exon 20 insertion mutations whose disease had progressed on or after platinum-based chemotherapy. The main outcome measured was overall response rate (proportion of patients whose tumor is destroyed or reduced by a drug). In the trial population in which all patients received Rybrevant, the overall response rate was 40%. The median duration of response was 11.1 months, with 63% of patients having a duration of response of 6 months or more.

The most common side effects of Rybrevant include rash, infusion-related reactions, skin infections around the fingernails or toenails, muscle and joint pain, shortness of breath, nausea, fatigue, swelling in the lower legs or hands or face, sores in the mouth, cough, constipation, vomiting and changes in certain blood tests. Rybrevant should be withheld if patients develop symptoms of interstitial lung disease and permanently discontinued if interstitial lung disease is confirmed. Patients taking Rybrevant should limit sun exposure during and for two months after treatment. Rybrevant may cause problems with vision. Rybrevant can also cause fetal harm when administered to a pregnant woman; therefore, the pregnancy status of females of reproductive potential should be confirmed before treatment is started.

Rybrevant received Priority Review and Breakthrough Therapy designation for this indication. Priority Review directs overall attention and resources to the evaluation of applications for drugs that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis or prevention of serious conditions when compared to standard applications. Breakthrough Therapy designation is designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).

The FDA granted approval of Rybrevant to Janssen Pharmaceutical Companies of Johnson & Johnson.


Specialty Pharmacies Should Study the Magellan Medical Pharmacy Trend Report

We recently expressed our disappointment in the meager annual pharmacy trend reports now being offered by the leading PBMs. They once were the most insightful benchmark documents to help specialty pharmacies gauge the specialty pharmacy market.

However, there is one bright light on the trend report horizon, the Magellan Medical Pharmacy Trend Report. The new, 2020 report was recently released, and it is chock-a’-block full of great insights into what is happening in the most obscure segment of pharmacy, medical pharmacy spend.

The 2020 report has gone heavy into charts and graphs, which is great since many of the issues that fall under medical trend are more diffuse than under pharmacy trend. Some of the issues are easier to understand when they are color coded.

Specialty pharmacy has been a core element in medical pharmacy trend for years….. and is rapidly increasing. The fastest growing specialty pharmacies have all jumped into drug fulfillment under the medical benefit…. including buy-and-bill, direct distribution. Any specialty pharmacy interested in playing in that lucrative sandbox would be advised to download this report and use the insights as a road map to develop a strategy.


Magellan Rx Management’s Medical Pharmacy Trend Report Unlocks the Latest Trends and Emerging Strategies to Manage Rising Medical Benefit Specialty Drug Spend

The 11th edition of this unique industry resource features surprising changes to the top commercial drug list

May 20, 2021, PHOENIX–(BUSINESS WIRE)–Magellan Rx Management, a division of Magellan Health, Inc. (NASDAQ: MGLN), released its eleventh annual Medical Pharmacy Trend Report, featuring a comprehensive view of provider-administered medical benefit drug trends—one of the largest drivers of total specialty drug spend.

“At Magellan Rx, we have a deep understanding of trends based on intricate knowledge and detailed analysis of medical benefit drug claims as well as current management approaches and innovative solutions that are being deployed to combat rising costs while maintaining quality care for patients.”

Through 11 years of market evolution and claims data trends, the Report highlights how total medical benefit costs and per-member-per-month (PMPM) trends have continued to rise. Since the first edition of the Report, commercial PMPM has nearly doubled. Market dynamics have evolved significantly, including the introduction and growth of biosimilars, and the science has advanced faster than ever with new mechanisms of action, novel therapies for orphan diseases, targeted oncology therapeutics, and the introduction of gene therapy. These innovations have helped countless patients, while also presenting new complexities for managed care organizations and clinicians. These challenges are why it is critical for payers to be increasingly focused on managing drug spend on the medical benefit.

“These trends continue to be a challenge for all stakeholders involved in the care of patients with complex specialty conditions, making it vital for them to stay current and informed for better decision-making,” said Kristen Reimers, RPh, senior vice president, specialty clinical solutions, Magellan Rx Management. “At Magellan Rx, we have a deep understanding of trends based on intricate knowledge and detailed analysis of medical benefit drug claims as well as current management approaches and innovative solutions that are being deployed to combat rising costs while maintaining quality care for patients.”

Key Findings and Trends in the 2020 Report
The top five commercial drugs are now: Remicade, Neulasta, Ocrevus, Rituxan, and Herceptin. This is a historic shift—after a decade of Remicade, Neulasta, Rituxan, Herceptin, and Avastin being the top-five drugs, Avastin has been replaced by Ocrevus (No. 3).
Biosimilars Renflexis and Inflectra in the Biologic Drugs for Autoimmune Disorders (BDAIDs) category are making an impact with a 4-6 percentage point increase in market share for commercial and Medicare and a substantial 24 percentage point increase in Medicaid. The average annual cost per patient for these agents were 22-28 less than the reference product, Remicade.
Colony stimulating factors (CSFs) in Medicare and Medicaid experienced a decline in spend, potentially attributed to a decrease in the use of cytotoxic chemotherapy. This reduction in the use of cytotoxic agents drove higher oncology spend as use of immunotherapy and monoclonal antibody (MoAb) drugs increased.
Driven by the decrease in CSF cost and spend, oncology support spend is forecasted to decrease by 32% over the next five years—the only category profiled with a steady reduction in cost.
The oncology pipeline had more than 700 drugs in clinical trials in 2019 and is forecasted to increase 105% in PMPM spend from $52 in 2019 to $106 in 2024.

This Report is just one of the ways Magellan Rx keeps the market updated on the latest trends for medical benefit drugs. To learn about Magellan Rx’s 16+ years of experience and commitment to developing innovative clinical programs that address these trends and help payers stay ahead of the curve to improve overall cost and quality, click here to read about the company’s total specialty drug management approach and medical pharmacy solutions.


FDA Approves Sub-q Tx for PNH – Empaveli

Last week the FDA approved a new sub-q therapy, Empaveli (pegcetacoplan) from Apellis Pharmaceuticals, for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) who are treatment naïve and those who are switching from Alexion’s two marketed PNH C5 inhibitor therapies, Soliris (eculizumab) and Ultomiris (ravulizumab).

PNH is an acquired disorder that leads to the rupture or destruction of red blood cells called hemolysis. Patients with the disease experience severe anemia, profound fatigue, shortness of breath, intermittent episodes of dark colored urine, kidney disease, or recurrent pain. Chronic hemolysis can be devastating, with the potential to damage vital organs and cause premature death.

Empaveli is administered by subcutaneous infusion twice weekly via a commercially available pump. By comparison, both Soliris and Ultomiris are administered by infusion.

PHN is an ultra-rare disorder with only about 300 patients in the US.
Apellis set Empaveli’s initial price at $458,000, which the company says is on par with Ultomiris and lower than the price tag on Soliris.

Apellis has announced that Empaveli will be available only through physicians and pharmacies that are registered/approved through their REMS management program. Their web site references PantherRx Rare as the program lead pharmacy.

FDA approves new treatment for adults with serious rare blood disease

FDA has approved Empaveli (pegcetacoplan) injection to treat adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare, life-threatening blood disease. Empaveli is the first PNH treatment that binds to compliment protein C3.

PNH is characterized by red blood cell destruction, anemia (red blood cells unable to carry enough oxygen to tissues), blood clots, and impaired bone marrow function (not making enough blood cells). The disease affects 1-1.5 people per million. Individuals are typically diagnosed around ages 35 to 40. PNH can be serious, with median survival of 10 years after diagnosis. However, some patients live for decades with only minor symptoms.

PNH is caused by gene mutations that affect red blood cells. Red blood cells in people with these mutations are defective and can be destroyed by the immune system, which causes anemia.

The effectiveness of Empaveli was evaluated in a study enrolling 80 patients with PNH and anemia who had been taking eculizumab, a treatment previously approved for PNH. Patients first completed a four-week period during which they received Empaveli 1,080 mg twice weekly in addition to eculizumab at their previous dose. After the first four weeks, patients were randomly assigned to receive either Empaveli or their current dose of eculizumab for 16 weeks.

After 16 weeks, the severity of anemia was compared in the two treatment groups on the basis of hemoglobin concentration (a laboratory measure of anemia). In both treatment groups, the average hemoglobin was 8.7 g/dL at baseline, indicating severe anemia. (Normal hemoglobin values in adult men are 14 g/dL or above; normal values in adult women are 12 g/dL or above.) During the 16 weeks of treatment, patients in the Empaveli group had an average increase in their hemoglobin of 2.4 g/dL. Meanwhile, patients in the eculizumab group had an average decrease in their hemoglobin of 1.5 g/dL.

Empaveli is available only through a restricted program under a risk evaluation and mitigation strategy. Meningococcal (a type of bacteria) infections can occur in patients taking Empaveli and can become life-threatening or fatal if not treated early. Empaveli may also predispose individuals to serious infections, especially infections caused by encapsulated bacteria. Patients should be monitored for infusion-related reactions. Empaveli can interfere with certain laboratory tests. The most common side effects are injection site reactions, infections, diarrhea, abdominal pain, respiratory tract infection, viral infection, and fatigue.


Hospital Specialty Pharmacy Network Nails Another LDD

We are always on the lookout for updates on health systems that own or operate their own specialty pharmacies as well as third-party organizations that have formed networks to proliferate specialty pharmacy in the hospital world. Today’s article offers an update on one of these networks, Acentrus Specialty.

We started to cover Acentrus in 2017. Since then they have grown significantly. According to them, Acentrus Specialty represents the “largest integrated care network for health system specialty pharmacy in the United States”. The network now tips in with 115 health systems consists (mainly academic medical centers and integrated delivery networks) serving more than 4.6 million inpatient admissions annually, treated by nearly 200,000 physicians. That translates into a mountain of specialty prescriptions.

Acentrus’ service menu includes channel partner access (e.g., contract pharmacies or backup partners, access to limited distribution drugs via their connections with manufacturers, consulting services, Boards of Pharmacy licensing, payer contracts, and accreditation in partnership with Center for Pharmacy Practice Accreditation (CPPA) launched in mid-2018.

The article underscores that these hospitals are strategically gaining access to limited distribution therapies. In fact, we first tripped access Acentrus in late 2017 when they announced that they had gained access to five big LD therapies, Ibrance, Bosulif, Sutent, Inlyta and Xalkori.

Acentrus Specialty Contracts with Chiasma to Bring Mycapssa (octreotide) to Health Systems and Hospitals in the Acentrus Network

May 13, 2021 — IRVING, Texas–(BUSINESS WIRE)– Acentrus Specialty, a national network of health systems and hospitals committed to providing specialty pharmacy care for their patients, has finalized an agreement with Chiasma, Inc. to provide qualified Acentrus clients access to Mycapssa (octreotide), delayed-release oral capsules for long-term maintenance treatment in acromegaly patients who have responded to and tolerated treatment with octreotide or lanreotide.

“We are pleased to provide our network hospitals and their patients access to the first and only FDA-approved oral somatostatin analog (SSA) treatment for acromegaly,” said Acentrus Specialty Vice President George Zula. “Limited distribution drugs like Mycapssa are highly sought after and Acentrus works with drug companies to open the door to these medications for specialty pharmacies operated by our network hospitals. This means that patients may begin treatment sooner and with the significant advantage of integrated care from pharmacy professionals they know and trust.”

“Chiasma is very pleased to enter into this agreement with Acentrus to significantly expand the access and distribution of Mycapssa to qualified hospital specialty pharmacies that want to offer our oral treatment for appropriate acromegaly patients within their institutions and are committed to providing dedicated patient services consistent with those offered through our own Chiasma Access and Patient Support program,” said Chiasma Vice President Market Access Dan Thornton. “We have entered into this partnership agreement with Acentrus to continue to help address the significant unmet needs of patients living with acromegaly by expanding patient access to oral Mycapssa therapy.”

Participation in Acentrus is open to hospital organizations of all sizes, types and affiliations. Clients, which range from academic medical centers to community-based hospitals, share one goal: to provide their patients with high-quality care in a setting where clinical and pharmacy services are integrated.

There is no cost for an organization to join the Acentrus network, which provides 115 health systems and hospitals with the following benefits:

Access to difficult-to-obtain specialty medications
Data management
Collaboration with health system peers dedicated to sharing best practices
Portfolio of other services to help hospital-based specialty pharmacies meet the needs of their patients, including backup and contract pharmacy as well as expertise in licensing and accreditation

About Acentrus Specialty
Acentrus Specialty is the integrated care network and health system solution for specialty pharmacy, addressing the largest and fastest growing area of health care expenditures by empowering members to deliver exceptional, cost-effective, locally integrated care. This is accomplished through a portfolio of services and specialty pharmacy products, as well as member sharing of clinical and operational best practices. The network of health systems consists mainly of academic medical centers and integrated delivery networks, serving more than 4.6 million inpatient admissions annually, treated by nearly 200,000 physicians. Follow Acentrus on LinkedIn or on the web at


How a $250,000 Tx can End Up Costing $2+ Million

Today we now better understand how UBER costly drugs, such as the big-ticket CAR-T therapies, are billed and paid for in our highly fractional health market.

AIS Health just published a very informative article in the May issue of Radar on Specialty Pharmacy titled Study Finds Wide Variation in Payers’ CAR-T Drug Costs. The report was produced by Prime Therapeutics.

The report examined integrated pharmacy and medical claims data with a CAR-T drug claim line of more than $250,000. Patients also had to be continuously enrolled for an 86-day CAR-T episode: 30 days prior to and 56 days following administration (such a protocol adds a ton of $$$$ beyond drug cost).

What was most stunning was the variance in drug cost. The CAR-T therapies are available at only a handful of leading medical centers in the US. National payers may have an existing contract with these hospitals as noted in the article, but many regional payers likely do not. As such, these hospitals are ‘out-of-network’ and these hospitals will submit claims for CAR-T therapies based on the hospitals’ arbitrary ‘Usual & Customary’ rate….. a rate that appears to range from about 150% of WAC to about 10 X WAC. (It is unclear whether a discount is applied to the billed charges, which is common practice.) Further, these drugs are billed under the medical benefit which gives hospitals significantly greater wiggle room to squeeze payers for big $$s

Here’s the bottom line—-
– The median CAR-T drug claim was $411,711 and ranged from $275,244 (essentially WAC) to $2,101,934. The mean CAR-T drug cost was $527,547
– The median cost for the total episode was $610,999, ranging from $358,980 to $2,235,658.
– Twelve percent of the episodes totaled more than $1 million.

Payers are at the mercy of these out-of-network hospitals and have no alternatives. Given the actual billed costs it is no surprise that payers are looking for any way to bring down the cost burden for these new drugs. We anticipate that payers will start to press hard for outcomes-based deals with manufacturers.

Study Finds Wide Variation in Payers’ CAR-T Drug Costs

Chimeric antigen receptor T cell (CAR-T) therapies have been available in the U.S. since August 2017. While the list prices for the one-time treatments are known, a recent study from Prime Therapeutics LLC examined their total cost of care and clinical events following administration and found that payer costs for the therapies varied widely. Payers could use this information to help forecast costs for these drugs and strike value-based deals, says one author of the study, which was presented at the Academy of Managed Care Pharmacy’s AMCP 2021 virtual conference in April.

Study Examined First Two CAR-Ts
Researchers examined the first two CAR-Ts on the U.S. market. Yescarta (axicabtagene ciloleucel) from Kite Pharma, Inc., a Gilead Sciences, Inc. company, was approved Oct. 18, 2017, for the treatment of adults with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy (RSP 11/17, p. 8). The drug — which gained FDA approval for relapsed or refractory follicular lymphoma after at least two forms of treatment on March 6 (RSP 4/21, p. 8) — is priced at $373,000.

On May 1, 2018, the FDA approved Kymriah (tisagenlecleucel) from Novartis Pharmaceuticals Corp. — first approved on Aug. 30, 2017 (RSP 9/17, p. 4) for the treatment of people up to 25 years old with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or has relapsed at least twice — for the same large B-cell lymphoma indication (RSP 5/18, p. 8) and is priced the same for this use as Yescarta.

The process of administering CAR-Ts is complex and involves extracting a person’s T-cells, shipping them to a facility where they are genetically reprogrammed and infusing them into the patient. Patients must stay near their infusion facility for a period of time afterwards to be monitored for any adverse reactions such as cytokine release syndrome and neurological toxicities.

Researchers examined integrated pharmacy and medical claims data among an average of 15 million commercially insured members from January 2018 to June 2020 with a CAR-T drug claim line allowed cost of more than $250,000. Members had to be at least 18 years old and have a lymphoma diagnosis but not a leukemia or ALL diagnosis. They also had to be continuously enrolled through an 86-day CAR-T episode: 30 days prior to and 56 days following administration.

Members’ non-financial outcomes were followed from the CAR-T episode date through Oct. 31, 2020. Members were separated into three groups:
Experienced any claims-identified clinical event,
Disenrolled without experiencing an event or
Remained enrolled and did not experience an event by Oct. 31, 2020.

Researchers identified 74 members who met the study criteria. More than half — 59% — were male and ranged from 18 to 76 years old, with an average age of 55. The outcome events assessment follow-up period averaged 288 days after the CAR-T episode, ranging from 26 to 990 days.

The mean total cost of care for the 86-day episode was $711,884; the mean CAR-T drug cost was $527,547, while the mean non-CAR-T drug cost was $184,337. The median CAR-T drug claim was $411,711 and ranged from $275,244 to $2,101,934. The median cost for the total episode was $610,999, ranging from $358,980 to $2,235,658. Twelve percent of the episodes totaled more than $1 million.

Among the outcome events following the CAR-T episode, 29 of the 74 members experienced one, 21 disenrolled or ceased all claim activity without experiencing an event, and 24 remained enrolled and did not experience an event. Of the members experiencing a clinical event, 22 received chemotherapy, four had a bone marrow transplant, and 13 had an identified death or hospice. Nine members had more than one type of event.

“There has been little real-world data reported about the clinical and financial aspects of CAR-T therapy for adults with lymphoma,” points out Joseph Leach, M.D., senior vice president and chief medical officer at Prime and a study co-author. “We went into this analysis with the goal of using the integrated medical and pharmacy claims data of over 15 million commercially insured lives to identify potential avoidable future costs and begin to assess real-world CAR-T therapy outcomes. Though we knew the CAR-T drug list price, we still expected variance, and it did vary.

Non-Drug Costs Previously Were Unknown
“Conversely, the non-drug costs, as they relate to what we defined as the 56-day ‘CAR-T episode,’ were unknown and therefore [this is] a noteworthy finding,” he continues. “For example, we knew that stem cell transplantation was costly but needed to perform this analysis to understand what CAR-T drug plus all other costs would sum to, providing a potential expected financial range cost. Our clinical outcomes findings appear similar to those in the clinical trial findings.”

Asked if payers could take steps to bring down some of the non-CAR-T costs, Leach replies, “The wide variation in payer CAR-T drug cost sticks out as an area for opportunity. The CAR-T drug wholesale acquisition cost (WAC) is consistent across all cases, but the total insurer payment showed substantial difference that has optimization potential. It’s difficult to say the same thing for the non-drug costs knowing that patients may vary in their response to therapy, side effect management costs — for example, cytokine release syndrome — or hospital stay length.”

As far as the CAR-T drug costs being higher than the WACs, Leach tells AIS Health, a division of MMIT, that those costs were paid “under the medical benefit through contracts or agreements held between the payer and hospital. It was anticipated that the real-world CAR-T drug cost would be higher than WAC, based on past drug pricing through the medical benefit observations, as drugs paid through the medical benefit are frequently priced higher than WAC.”

Data Can Help Insurers With Forecasting
Leach maintains that “having an understanding of the CAR-T therapy median and range of drug and total costs may be useful as insurers forecast their own future costs for these or similar drugs. Payers may use these findings in contract conversations with providers, as well as value-based purchasing agreement conversations with drug manufacturers. This information provides real-world, integrated medical and pharmacy insurance benefits, fact-based data to negotiate pharmaceutical manufacturer–insurance payer value-based purchasing agreements ensuring the CAR-T therapy price to value is fair. It should be noted that value-based purchasing agreement reporting fulfillment will require comprehensive outcomes data and sophisticated analytic insurer/PBM reporting capabilities.”

Earlier this year on Feb. 5, the FDA approved a third CAR-T for relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy: Bristol Myers Squibb’s Breyanzi (lisocabtagene maraleucel) (RSP 3/21, p. 8). With the approval, Leach says it’s possible that payers may begin preferring one of the drugs, “especially if a value-based purchasing agreement with either manufacturer, provider or both results in fair pricing and cost-variation control.”

That said, “although there are differences in the design of the available products, lack of comparative clinical trial data will make choosing a preferred CAR-T based on clinical efficacy challenging. Additional real-world studies such as this one may help inform differences in efficacy and toxicity. Value-based purchasing agreements based on meaningful endpoints such as duration of response and total cost may also be important factors in choosing a preferred product.”


All Specialty Pharmacy Staff Should Be Data Savvy

Over the years I’ve been able to visit dozens of specialty pharmacies working with pharmacy leadership to help develop new programs. Each of these new programs required staff to be trained and, to no surprise, many recent hires simply did not understand the specialty pharmacy model with all its moving parts.

One area that specialty pharmacies don’t spend a lot of time on in training is DATA. Why should staff care about data if they aren’t involved in crunching the numbers? The answer is simple. Garbage in…. Garbage out. Staff need to understand the value of data and how data is integral to the long-term value proposition of the SP model.

The article below is all about data. The article explains data, data capture (process), and reporting. It then pivots to address challenges related to specialty data and where and why data capture is increasingly important. It is a very good primer for any recent hire in specialty pharmacy to begin to understand the value of data to the business.

Data Capture and Reporting in Specialty Pharmacy

May 6, 2021 — Data can not only provide visibility into the process, day-to-day operations, and the patient journey, but can also help specialty pharmacies meet contractual obligations and provide a closer look at performance and improvement opportunities.

Specialty pharmacy is uniquely positioned to capture and deliver data to drive value in the care of patients who need specialty medications. Data can not only provide visibility into the process, day-to-day operations, and the patient journey, but can also help specialty pharmacies meet contractual obligations and provide a closer look at performance and improvement opportunities.

Data in specialty pharmacy provides visibility into prescribers, patients, and payers. A specialty pharmacy can identify which kind of health care providers are prescribing certain medications and can provide insights into their prescribing habits.

Trends in prescribing habits that may delay time to fill can be identified and interventions can be made to address these trends. Examples include poor documentation and lack of guidelines support when prescribing.

Data can also provide visibility into the regional distribution of patients and can offer insights into the social determinants of health that may be impacting patient outcomes. Further, data can demonstrate patterns that can help specialty pharmacies better treat their patients.

Although each patient is unique, there are patterns that can be identified as data are collected on many patients, which can help improve the treatment approach.

Lastly, data can provide visibility into payer activity, their coverage patterns, benefit structures, and their reimbursement rates and out-of-pocket obligations. This helps specialty pharmacies identify opportunities to obtain secondary coverage for patients or help them identify when payer policies are not representative of treatment guidelines.

Data Capture
Data are captured through documentation platforms that capture standardized claims data and through patient management systems that can capture additional meaningful information about the patient. Capturing data at any point within the patient experience can offer value, from referral to discharge.

At the time of referral, patient and prescriber demographic information is captured. When the specialty pharmacy initiates the benefit investigation and verification process, plan and policy details are captured.

Once claims are submitted, data regarding the denied or paid claim is retuned and captured. This includes information such as restrictions or quantity limits, co-pay, and deductible amounts.

Once this granular information is captured, it allows for the calculation of broader metrics such as time to clean claim, time to fill, and incremental time to process. These metrics provide a closer look into the performance of pharmacy operations and allow for diagnostic analytics to identify areas of improvements within the process to cut down on those metrics.

When claims are denied, prior authorization and appeal information is documented. This sheds light on what it takes to get a paid claim across payers, whether a letter of medical necessity is required or if a peer-to-peer would suffice. This information is of great benefit because it can provide guidance on how to handle the next patient’s reimbursement support.

From a patient perspective, data regarding their self-reported outcomes is collected during periodic shipment set up calls and other support outreach that the specialty pharmacy is conducting. Financial information can also be captured that allows the pharmacy to look for financial support options to ensure patient access to their much-needed medication.

Furthermore, specialty pharmacies are typically contractually obligated to document and report patient, caregiver, or physician reported adverse events. From there, specialty pharmacies learn about the patient through their fill history data and can calculate adherence measures, such as their medication possession ratio or proportion of days covered.

In addition to specialty pharmacies collecting data, other entities could do so as well depending on the makeup of the distribution network. Specialty hubs are involved in certain networks and can receive enrollments directly from prescribers. Hubs, in turn, can collect data about the patient, prescriber, and payer depending on the level of service they provide. Hubs can also collect metrics regarding hub utilization and which services patients and prescribers are taking advantage of the most.

A lot of data are available for collection for any entity servicing specialty patients, whether the pharmacy or the hub. In addition to managing incoming data, there is an expectation of data to be reported externally.

Data can be transmitted to a payer, data aggregator, or directly to a manufacturer. Typically, data are encrypted to ensure security during transmission from a pharmacy to a third party. Files can be transmitted as an excel, comma-separated values, or pipe-delimited flat file.

Mechanisms to transmit these files include placement on a secure file transfer protocol site to ensure encryption through transmit. It can go through an electronic data interchange, which are industry standard file formats. Alternatively, it can be displayed and accessed through a portal.

When reporting data, specialty pharmacies are responsible for managing the de-identification of protected health information in compliance with the Health Insurance Portability and Accountability Act (HIPAA). Section 164.514(a) of the HIPAA Privacy Rule provides the standard for de-identification of protected health information. Under this standard, health information is not individually identifiable if it does not identify an individual and if the covered entity has no reasonable basis to believe it can be used to identify an individual.1

De-identification methods are the expert determination and the safe harbor method. The expert determination method uses statistical principles to ensure that there is a very small risk of patients being individually identifiable.

This method looks at separate populations individually and tends to be costly. The safe harbor method is more commonly used because it removes 18 major identifiers relating to patient demographics, vehicle, device, biometric, account number, and other identifiers.

Specialty pharmacies and hubs are typically asked to report patient level information through a status report. Status reports provide visibility to the drug manufacturer on each patient’s journey within the specialty pharmacy network.

As these providers capture data from referral to discharge, these data can be translated into statuses that describe where the patient resides in their treatment journey. Statuses pinpoint whether a patient is pending benefit verification, has been referred to a pharmacy for dispense, has received a shipment, or has experienced a discontinuation in treatment.

Supplementing the statuses are additional information about the patient, prescriber, and payer that specialty pharmacy providers can communicate via reporting. Other types of reports that specialty pharmacies and hubs can be responsible for include dispense and inventory reports, among others.

Specialty pharmacy networks can include third party data aggregators that collect data reported from all network constituents. These organizations are designed to pull data together from multiple parties and package them for the end user.

They come into play when there are multiple pharmacies in a network or when extensive reporting is required. They clean up the data, aggregate it, and manages its quality prior to passing on to the drug manufacturer.

Beyond individual networks that data aggregators service, they can collect data at the industry level and offer it to drug companies as a way of providing industry insights. In addition to data reporting within the specialty network, third party payers that specialty pharmacies contract with may require data reporting on a quarterly or yearly basis.

Payers can ask for certain data to be submitted to demonstrate the value that the specialty pharmacy is providing to the patients that the payer covers. Specialty pharmacies end up reporting to multiple payers as they expand their contracts.

Since there is not a standardized reporting approach to payers, pharmacies end up having to create different reports to different payers that meet contractual obligations to ensure proper reimbursement. Similarly, specialty pharmacies that are accredited must report to accreditors to demonstrate that they are meeting required metrics.

Many challenges arise when it comes to data capture and reporting in specialty pharmacy. The biggest challenge is the lack of standardization. Since there is no reporting standard between entities, it makes it difficult to align data.

Therefore, specialty pharmacies need to act upon, capture, and report the necessary fields. Also, data reporting transmission techniques could differ between entities, so specialty pharmacies need all appropriate systems to transmit data appropriately.

This includes de-identification as well as the means and frequency of transmission. Another challenge is identifying the appropriate use cases for the data, especially when baseline data is not available or has not been captured. Lastly, data availability may be limited with smaller populations, particularly in the rare disease space.

Perhaps the most important question specialty providers can ask themselves is, “how can we use the data to get better?”

Operationally, providers need to know what questions to ask and to differentiate between operational inefficiencies versus complexities in the disease state, such as the difficulty of getting the medication approved by payers. Additionally, specialty pharmacies should measure patient satisfaction and use it as a way of highlighting their strengths and identifying areas for improvement.

The Future
Looking ahead, real time data can be a great way of getting a view into the patient at any point in time. Currently, data reports are typically scheduled for daily, weekly, monthly, or quarterly delivery.

A better way of sharing data would be to display it in real time through a portal or a dashboard. This would require integration, so specialty providers are capturing data within the workflow instead of it being an afterthought.

Another opportunity that the larger specialty providers can get a head start on compared to their competition is the use of advanced analytics. Currently, data analytics in specialty pharmacy is centered around descriptive and diagnostic analytics.

It is a hindsight approach that presents past data to try to make sense of what happened. This is typically communicated in a quarterly business review fashion.

A great opportunity for specialty pharmacies is to shift gears into foresight-type analytics through predictive and prescriptive analytics. This allows providers with large data sets for populations they treat to predict the likelihood of certain events happening to their patients.

This allows them to implement interventions to prevent those events, which can lead to reduced adverse events, increased retention, and enhanced patient care.

About the Author
Rami Chammas is the Director of Pharmacy Services at RareMed Solutions, the nation’s first rare disease focused specialty pharmacy HUB.

Rami Chammas, PharmD, MPBA Candidate
Pharmacy Times


When Will Amazon Pose a Threat to Specialty Pharmacy?

How time flies!
About three years ago Amazon jumped into the prescription business throwing a new monkey wrench in the segment. So, what’s happened in that time. If you are primarily concerned about specialty pharmacy, not a lot.

Following the acquisition of Pill Pack, Amazon has been slowly and methodically building out its mail service dispensing program. They are now licensed in 45 states (not yet licensed in Hawaii, Illinois, Kentucky, Louisiana, or Minnesota.) But, they tout that they are now contracted with virtually all payers. As any large /national specialty pharmacy knows, access is critical to growing the business…. and Amazon likes nothing better than growth.

They are laser focused on mailing maintenance meds and aren’t shy to tell prospective customers that the ‘one and done’ meds should go to local retail. A GoodRx like option is also now seamlessly linked into the patient’s online pharmacy profile offering the option to select the insured/out-of-pocket price or a cash price. They promote the fact that the cash price can often be much less expensive than the insured copay or coinsurance.

Amazon and Specialty Pharmacy
Is Amazon poised to challenge the specialty pharmacy segment?
Surprisingly, it is not evident at this point.

Their web site states that they do not fill specialty pharmacy medications, compounds, and, curiously, REMS medications . Earlier we touched on contracts and licensure as key factors limiting growth. National payers have shown that they prefer to work with specialty pharmacies with broad access to specialty meds across all the major therapeutic categories (or conversely just rare or Oncology meds) and sophisticated patient medication management platforms. How long will it take for Amazon to build out those competencies? The answer is years if they build them in-house…. or, very quickly if they can find an acquisition to fast-track the effort (but who is left to acquire??).

So, is Amazon still committed to health care for the long haul? So it seems. The article below details their expansion into Telehealth services called Amazon Care. Telehealth took off in 2020 with the pandemic (what timing, huh?) and could be a very effective way for Amazon to establish their creds in a new way with payers. The article also suggests that there may be real synergies between the Telehealth business and mail pharmacy…… Just ‘sayin.

Reality check: Amazon Care may not be that big of a deal

At the moment, the effort is a more direct threat to employer-facing point solutions, instead of the more comprehensive offerings peddled by entrenched vendors.

March 23, 2021 — Amazon’s expansion of its virtual care pilot, Amazon Care, to employees and third-party employers nationwide is sparking fears the e-commerce giant could represent a significant threat to telehealth vendors after a year of unrestrained growth in the sector.

But though the idea of Amazon offering virtual care nationwide is unsettling for incumbents, Amazon Care’s current offerings and structure aren’t that disruptive, experts say, and shouldn’t noticeably affect market giants like Teladoc Health, Amwell or Doctor on Demand — at least in the near term.

“I don’t necessarily think that we see the big Teladoc and Amwells of the world shaking in their boots,” Arielle Trzcinski, principal analyst at Forrester, said. “They have a pretty strong hold on the market.”

Amazon started piloting Amazon Care 18 months ago as an on-demand preventative, urgent and wellness service for employees. Then last week, the Seattle-based behemoth announced plans to offer the program’s virtual care benefit to employers across the U.S. beginning this summer, and would offer its in-home primary care service to other Washington state-based companies and the cities of Washington, D.C. and Baltimore in subsequent months.

Analysts say the scale Amazon can bring to bear and the implications of its entrance for pricing, marketing and capital access in the telemedicine sector are indeed concerning, but the virtual care space is big enough to support multiple winners.

Despite surging demand caused by COVID-19, it’s still a massively underpenetrated market. Teladoc, the largest virtual care provider in the U.S., expects to provide between 12 million and 13 million visits in 2021, but the country sees about 850 million outpatient visits annually, Cowen analysts said in a research note on Amazon Care’s expansion.

That wiggle room bodes well for existing players, which also enjoy a significant first-mover advantage over Amazon.

Teladoc, for example, has had 19 years to build out its suite of telemedical services and capture market share. The New York-based company has thousands of clients amid major health systems, payers and employers, including over 40% of the Fortune 500.

Teladoc had been ramping up its offerings to become a one-stop virtual shop even before the pandemic, but exponentially accelerated growth plans in 2020 amid mounting consumer demand. With its $18.5 billion acquisition of chronic care manager Livongo last year, the vendor has expanded into previously niche and specialty areas like behavioral health and chronic care, and is testing other adjacencies like virtual-first primary care.

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It’s likely Teladoc will maintain the competitive advantage moving forward, according to SVB Leerink analyst Stephanie Davis. Though its stock took an initial hit late last week following the news, shares in Teladoc have ticked back up since as Wall Street digests the peripheral near-term implications of Amazon Care’s nationwide rollout.

“They’re not going to be knocked out of place,” Davis said.

At the moment, Amazon Care is a more direct threat to employer-facing virtual point solutions, instead of the more comprehensive offerings peddled by larger, more entrenched vendors, analysts say.

Such small companies like Omada, Cleo, Hinge Health and Kaia that focus on select conditions like diabetes, muscluoskeletal, hypertension or behavioral health generally have a few important employer clients and compete on price. Going toe-to-toe against Amazon, which brought in $386 billion in revenue last year, is likely a daunting prospect for the crop of digital health startups.

“The employer space is the closest thing to the wild, Wild West in the health sector,” Davis said. Employers are more willing to shop around or bet on unestablished players than health insurers or providers, which are more directly culpable for quality of care.

Amazon says it’s in discussions with numerous employers about Amazon Care, but isn’t sharing specifics. It’s likely Amazon will target smaller employers that need to save money as a starting point for sales efforts for Amazon Care, or mid-sized or large companies with concentrated workforces, Michael Abrams, managing partner at Numerof & Associates, said.

Amazon isn’t currently disclosing pricing for its virtual care platform, but will likely argue they can provide cost savings to employers that feel their current vendors aren’t delivering.

Looking strictly at the virtual visit component of Amazon Care, there’s very little distinguishing it from other large vendors. Most telehealth platforms are available right now at little to no cost and offer short wait times, though Amazon says it provides free access to a medical professional in 60 seconds or less — and will eventually link telehealth with in-home care across the U.S.

It’s also angled more as a virtual-first primary care option, treating patients through groups of providers matched with each patient based on their needs.

There’s an appetite for this on the consumer side, analysts say. Research has shown people like connecting with their own doctors, and telemedicine vendors are banking on their ability to build a sense of familiarity with virtual providers without sacrificing the convenience of an in-home visit.

Teladoc does something similar, allowing patients to connect with a preferred provider — and can match patients with chronic conditions to coaches and other support, including connected devices, thanks to the Livongo buy.

However, Amazon Care does offer some services that have been overlooked by larger vendors, including joint care. Existing joint conditions have likely been exacerbated by non-ergonomic work from home settings, Trzcinski said.

That could be a major plus for Amazon, as employers are starting to face the risk of potential short- and long-term disability claims bubbling up over the next year or so due to work from home, and may be willing to pay a premium to head those off.

Pharmacy is another key differentiator, experts say. Amazon Pharmacy, which allows consumers to complete a transaction on their desktop or smartphone through the Amazon app and get medications delivered to the home, is an option to fill scripts on Amazon Care.

That could greatly simplify the process for existing Amazon customers, linking prescription fulfillment and making it easier to order and receive all kinds of over the counter and durable medical equipment products at the same time.

With its Livongo tie-up, Teladoc checked the box on chronic care management, but pharmacy overall is “a piece I don’t see filled yet,” Trzcinski said, noting how successfully Amazon threatens existing telehealth vendors is a question of which is most effective —​ and most affordable. “There’s a couple of areas I think Amazon is positioned well. That doesn’t mean Teladoc or Amwell couldn’t step in and close those gaps if they wanted to.”

And familiarity with the brand is, of course, a significant competitive advantage. In the 25 years since its creation, Amazon has grown to capture almost 40% of the U.S. e-commerce market, according to eMarketer. As many as 82% of American households may have a Prime subscription according to one estimate, and that convenience — the linchpin of its brand — could smooth Amazon Care’s path in the opaque healthcare sector.

But despite these differentiators, Amazon Care has a ways to go before it’s ready to compete with the likes of Teladoc, experts say. And Amazon has been burned in healthcare before, disbanding its joint venture with J.P. Morgan and Berkshire Hathaway to lower medical costs, called Haven, earlier this year after failing to move the needle on prices.

Amazon Care could run into similar roadblocks.

“This is Amazon’s first foray into direct care delivery, and the complexities of the business more than rival those of mail order merchandizing,” Abrams said. “Amazon has a steep hill to climb.”


No Fireworks as Specialty Pharmacy Breaks the 50% Milestone

Were there fireworks to mark the event?
Was there a parade to commemorate the long-awaited day?
In short, NO!

Annual Drug Trend Reports from Express Scripts (Evernorth) and CVS were recently released as they have been for many years. As in prior years we attempt to point out the important details that are pertinent to the specialty pharmacy industry. After all, these two giants represent upwards of 70% of all specialty pharmacy spend.

So, what big event are we talking about?
After hearing for a decade that specialty pharmacy spend would soon top 50% of all pharmacy spend it has finally happened, as confirmed by both PBMs. ESI set it at 51% while CVS suggests 52%. So much for a long-awaited benchmark to arrive.

You can read the trend reports by clicking below. Don’t set aside much time, however. There is even less detail in the 2020 reports than in the already lean 2019 report.

Unit cost trend was up by less than 1%. However, 2020 specialty pharmacy utilization spiked about 300% at 3+%. Average SP utilization trend for 2017-2019 averaged a very low 1% annually. Neither report offers much explanation of the drivers behind these trend numbers.

There is little explanation as to why specialty pharmacy utilization spiked in 2020 in spite of the pandemic and numerous analyst assessments that specialty utilization would be negatively impacted especially with expected drop in new diagnoses (fewer doctor office visits). CVS offers some comment on the swing in utilization in 2020 as noted below….. but not much in the specialty pharmacy category.

Lastly, neither organization offered much detail of their specialty pharmacy management programs to drive down utilization through limited networks, tighter formularies, and cost shifting to patients (e.g., copay accumulators, etc.)

2020 Drug Trend Reports

ESI / Evernorth
For the first time, spending on specialty drugs accounted for more than half of total drug spend (51%). This was largely due to the introduction of costly treatments for inflammatory conditions and cancer with no real competition. Evernorth moved to mitigate specialty costs in 2020 and beyond with:

The industry’s first solution for affording incredibly expensive, potentially curative gene therapies
New, comprehensive and cost-effective solution to make fertility treatment more accessible
Strategic formulary management, including plan-level customization by therapeutic category
Value-based care solutions for inflammatory conditions, cancer, HIV, multiple sclerosis, complex neurological conditions and more

Specialty treatments continue to grow as a portion of pharmacy spend. They now account for more than half of pharmacy spend — 52 percent in 2020 — and are concentrated in relatively few therapeutic categories. Specialty trend has continued to remain high over recent years, but that spend is now largely driven by members new to treatment. Utilization is now the main driver of specialty drug trend as we have successfully implemented approaches to mitigate manufacturer driven price inflation.

CVS Caremark’s overall drug spend trend was 2.9% in 2020 and about one-third of its clients had a negative trend, the PBM announced today. Utilization increased just 1.7% and price growth was 1.2%, according to its 2020 Drug Trend Report.

The report from the PBM unit of CVS Health includes a graph that shows the steep drop in healthcare utilization in April 2020 as the COVID-19 pandemic took off and lockdowns ensued had a similar effect on patients starting new drugs. The number of CVS Caremark prescriptions for a new drug for depression, diabetes, dyslipidemia (high cholesterol) and heart failure fell precipitously in April, the graph shows. By September, new starts for depression, diabetes, and dyslipidemia had recovered somewhat so they were roughly 10% higher than new starts in 2019. But for heart failure, the new starts continued to lag behind 2019 and were about 10% lower in September 2020 than in September 2019.

The drug trend report also updates the long-term trend in the growth of the specialty drug spend as a share of the overall pharmacy spend. In 2020, specialty drugs accounted for 52% of the CVS Caremark drug spend, according to this report. Five therapeutic area — autoimmune, oncology, cystic fibrosis, atopic dermatitis, HIV — accounted for 90% of that spend.

The report acknowledges the specialty trend but touts CVS Caremark’s ability to manage it. “Ensuring appropriate utilization, avoiding waste and maximizing cost reduction programs helped us keep specialty trend in check for clients who used our services,” the report says, citing figures that show that more than 40% of clients had single-digit specialty trend and 18% had a negative trend

Click here to access the ESI / Evernorth Trend Report
Click here to access the CVS Trend Report


FDA Approves New Specialty Infused Tx for DLBCL – Zynlonta

The FDA approved a new infused specialty therapy last week, Zynlonta (loncastuximab tesirine-lpyl) from ADC Therapeutics, for the treatment of adult patients with relapsed or refractory large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy.

It is estimated that approximately 77,240 new cases of NHL will be diagnosed, and 19,940 patients will die from NHL in 2020, despite currently available treatment. NHL is the seventh most common cancer in the United States, accounting for 4.3% of all cancers, and the eighth leading cause of cancer deaths, accounting for 3.3% of cancer-related deaths. Lymphomas are a heterogeneous group of malignancies with diverse biology, clinical behavior, and prognosis.

According to an investor presentation, ADC expects to launch Zynlonta this week at a wholesale price of $23,500 per 10 mg vial, before any discounts or rebates. This therapy is weight based and the dosing schedule for a 150-pound person would require 10 vials should the schedule last one full year. The annualized cost of therapy for that patient would tip in at $235,000.

Onco360, has been selected by ADC Therapeutics to be the exclusive specialty pharmacy partner for Zynlonta. ADC did not disclose details related to buy-and-bill vs. PBM adjudication under either the medical vs. pharmacy benefit.

FDA grants accelerated approval to loncastuximab tesirine-lpyl for large B-cell lymphoma

On April 23, 2021, the Food and Drug Administration granted accelerated approval to loncastuximab tesirine-lpyl (Zynlonta, ADC Therapeutics SA), a CD19-directed antibody and alkylating agent conjugate, for adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, DLBCL arising from low grade lymphoma, and high-grade B-cell lymphoma.

Approval was based on LOTIS-2 (NCT03589469), an open-label, single-arm trial in 145 adult patients with relapsed or refractory DLBCL or high-grade B-cell lymphoma after at least two prior systemic regimens. Patients received loncastuximab tesirine-lpyl 0.15 mg/kg every 3 weeks for 2 cycles, then 0.075 mg/kg every 3 weeks for subsequent cycles. Patients received treatment until progressive disease or unacceptable toxicity.

The main efficacy outcome measure was overall response rate (ORR), as assessed by an independent review committee using Lugano 2014 criteria. The ORR was 48.3% (95% CI: 39.9, 56.7) with a complete response rate of 24.1% (95% CI: 17.4, 31.9). After a median follow-up of 7.3 months, median response duration was 10.3 months (95% CI: 6.9, NE). Of the 70 patients who achieved objective responses, 36% were censored for response duration prior to 3 months.

Most common (≥20%) adverse reactions in patients receiving loncastuximab tesirine-lpyl, including laboratory abnormalities, are thrombocytopenia, increased gamma-glutamyltransferase, neutropenia, anemia, hyperglycemia, transaminase elevation, fatigue, hypoalbuminemia, rash, edema, nausea, and musculoskeletal pain.

The prescribing information provides warnings and precautions for adverse reactions including edema and effusions, myelosuppression, infections, and cutaneous reactions.

The recommended loncastuximab tesirine-lpyl dosage is 0.15 mg/kg every 3 weeks for 2 cycles, then 0.075 mg/kg every 3 weeks for subsequent cycles, by intravenous infusion over 30 minutes on day 1 of each cycle (every 3 weeks). Patients should be premedicated with dexamethasone 4 mg orally or intravenously twice daily for 3 days beginning the day before loncastuximab tesirine-lpyl.

View full prescribing information for Zynlonta.

This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment.

This application was granted priority review and orphan drug designation. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.

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