Last week the FDA approved a new therapy, BESREMi (ropeginterferon alfa-2b-njft) from PharmaEssentia, for the treatment of adults with polycythemia vera (PV). The drug was granted an orphan designation. BESREMi is administered subcutaneously.

Polycythemia Vera (PV) is a cancer that originates from a disease-initiating stem cell in the bone marrow leading to a chronic increase of red blood cells, white blood cells, and platelets. The cancer then frequently results in cardiovascular complications as well as secondary myelofibrosis or leukemia.

BESREMi is a long-acting interferon and is administered once every two weeks until hematologic targets are realized. Patients with a complete hematologic response can then be treated every four weeks.

Analysts expect the annual cost per patient per year (PPPY) to be in the $40,000 range. Details related to channel access were not released. As an orphan drug, BESREMi will very likely be released through limited distribution.

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U.S. FDA Approves BESREMi (ropeginterferon alfa-2b-njft) as the Only Interferon for Adults With Polycythemia Vera

November 12, 2021 — BURLINGTON, Mass.–(BUSINESS WIRE)–PharmaEssentia Corporation (TPEx: 6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, today announced that the U.S. Food and Drug Administration (FDA) has approved BESREMi for the treatment of adults with polycythemia vera (PV).

“With the availability of an FDA-approved, next-generation interferon for this indication, it’s time that we focus on preserving the long-term health of patients with polycythemia vera.”

BESREMi is an innovative monopegylated, long-acting interferon, which exhibits its cellular effects in polycythemia vera in the bone marrow. BESREMi was approved with a boxed warning for risk of serious disorders including aggravation of neuropsychiatric, autoimmune, ischemic and infections disorders. PharmaEssentia is preparing to make BESREMi available in the coming weeks in the U.S.

“The FDA approval of BESREMi for people with polycythemia vera represents the next step in advancing patient care as it provides a critical addition to managing not only symptom burden and near-term complications, but also treating the cancer early, which may help reduce the risk of disease progression over time,” said Srdan Verstovsek, M.D., Ph.D., Director of the Hanns A. Pielenz Clinical Research Center for Myeloproliferative Neoplasms, Department of Leukemia at the University of Texas MD Anderson Cancer Center. “With the availability of an FDA-approved, next-generation interferon for this indication, it’s time that we focus on preserving the long-term health of patients with polycythemia vera.”

“The reality of living with a rare and chronic cancer like polycythemia vera is that it is often under recognized and the limited treatments available cannot properly address the disease beyond the symptoms. Our community welcomes the FDA approval of a new treatment that has the potential to deliver what has been unavailable for so many patients hoping for a better outlook,” said Ann Brazeau, CEO of MPN Advocacy and Education International.

The U.S. FDA approval was based on safety from the PEGINVERA and PROUD/ CONTINUATION-PV studies and efficacy data from the PEGINVERA clinical study program. The study showed that after 7.5 years of treatment with BESREMi, 61% of patients with PV experienced a complete hematological response (defined as hematocrit <45% without phlebotomy for at least 2 months since last phlebotomy, platelets ≤ 400 x 109/L, leukocytes ≤10 x 109/L, normal spleen size (longitudinal diameter ≤12 cm for females and ≤ 13 cm for males). Importantly, 80% of patients achieved a hematological response (based on objective laboratory parameters only, with the exclusion of normal spleen size and thrombosis). These parameters are the most commonly used metrics to make therapeutic decisions.1 In the pooled safety population of patients treated with BESREMi, the most common adverse reactions (incidence >40%) were influenza-like illness, arthralgia, fatigue, pruritis, nasopharyngitis, and musculoskeletal pain. Serious adverse reactions (incidence > 4%) were urinary tract infection, transient ischemic attack and depression.1

“We are incredibly proud to deliver on our goal of bringing treatments like BESREMi to the polycythemia vera community where there is clear unmet need for more effective, tolerable and durable treatments to preserve patients’ health and well-being,” said Ko-Chung Lin, Ph.D., Co-Founder and Chief Executive Officer for PharmaEssentia and inventor of ropeginterferon alfa-2b-njft. “As we begin working closely with the community to integrate this important treatment into clinical practice, we also continue to expand our scientific efforts to unlock the full potential of our pioneering molecule.”

The FDA just approved a new ORAL therapy, Scemblix (asciminib) from Novartis, for patients with Philadelphia chromosome-positive chronic myeloid leukemia previously treated with two or more tyrosine kinase inhibitors (TKIs). Studies show that around 55% of CML patients are intolerant to TKIs and 70% receiving TKIs as a second-line treatment fail to achieve a significant response. Patients who acquire T314I mutations also tend to be resistant to existing TKI therapies. The drug also received full approval for adult patients with Ph+ CML in CP with the T315I mutation.

Chronic myeloid leukemia (CML) is the most common myeloproliferative disorder accounting for ~20% of all leukemia cases. Its annual incidence has been estimated at between 1 and 1.5 cases per 100,000 and its prevalence at around 1 in 17,000. The Philadelphia chromosome is seen in more than 90% of patients with CML.

Pricing for Scemblix was not released. Channel access was not announced. However, given the small patient base for its indication it would be expected that Scemblix will launch via limited distribution.

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FDA approves Scemblix (asciminib) for Philadelphia chromosome-positive chronic myeloid leukemia

On October 29, 2021, the Food and Drug Administration granted accelerated approval to asciminib (Scemblix, Novartis AG) for patients with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP), previously treated with two or more tyrosine kinase inhibitors (TKIs), and approved asciminib for adult patients with Ph+ CML in CP with the T315I mutation.

ASCEMBL (NCT03106779), a multi-center, randomized, active-controlled, open-label clinical trial, is evaluating asciminib in patients with Ph+ CML in CP, previously treated with two or more TKIs. A total of 233 patients were randomized (2:1) and stratified according to major cytogenetic response (MCyR) status to receive either asciminib 40 mg twice daily or bosutinib 500 mg once daily. Patients continued treatment until unacceptable toxicity or treatment failure occurred. The main efficacy outcome measure was major molecular response (MMR) at 24 weeks. The MMR rate was 25% (95% CI: 19, 33) in patients treated with asciminib compared with 13% (95% CI: 6.5, 23; p=0.029) in those receiving bosutinib. With a median duration of follow-up of 20 months, the median duration of MMR has not yet been reached.

CABL001X2101 (NCT02081378), a multi-center, open-label clinical trial, is evaluating asciminib in patients with Ph+ CML in CP with the T315I mutation. Efficacy was based on 45 patients with the T315I mutation who received asciminib 200 mg twice daily. Patients continued treatment until unacceptable toxicity or treatment failure occurred. The main efficacy outcome measure was MMR. MMR was achieved by 24 weeks in 42% (19/45, 95% CI: 28% to 58%) of the patients. MMR was achieved by 96 weeks in 49% (22/45, 95% CI: 34% to 64%) of the patients. The median duration of treatment was 108 weeks (range, 2 to 215 weeks).

The most common adverse reactions (≥20%) are upper respiratory tract infections, musculoskeletal pain, fatigue, nausea, rash, and diarrhea. The most common laboratory abnormalities are decreased platelet counts, increased triglycerides, decreased neutrophil counts and hemoglobin, and increased creatine kinase, alanine aminotransferase, lipase, and amylase.

The recommended asciminib dose in patients with Ph+ CML in CP, previously treated with two or more TKIs, is 80 mg taken orally once daily at approximately the same time each day or 40 mg twice daily at approximately 12-hour intervals. The recommended asciminib dose in patients with Ph+ CML in CP with the T315I mutation is 200 mg taken orally twice daily at approximately 12-hour intervals.

This review used the Real-Time Oncology Review (RTOR) pilot program, which streamlined data submission prior to the filing of the entire clinical application, and the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment. The FDA approved this application 4 months ahead of the FDA goal date.

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

We missed one…….

At the end of August the FDA approved a new therapy, Skytrofa from Ascendis Pharma (lonapegsomatropin-tcgd), indicated for pediatric patients that have short stature due to inadequate secretion of endogenous growth hormone. Skytrofa is the first weekly subcutaneous injectable growth hormone for children with growth hormone deficiency (GHD).

According to the American Academy of Pediatrics, growth hormone deficiency among humans is a rare condition and affects less than one in 3,000. In 2020 there were 74 million kids under the age of 18 in the US. So, we can estimate that there are as many as 25,000 children eligible for hGH.

Skytrofa joins a growing number (pun intended) of competing growth hormone products.
– Accretropin
– Humatrope
– Norditropin
– Norditropin
– Nutropin
– Nutropin AQ
– Nutropin Depot
– Omnitrope
– Saizen
– Serostim
– Skytrofa
– Tev-Tropin
– Zomacton
– Zorbtive

Analysts indicate that the hGH market globally surged in 2019-2020 and is expected to realize a robust 5% compound annual growth rate for the foreseeable future.

Skytrofa is dosed based on weight. It is supplied in nine quantities from 3mg through 13.3mg in prefilled cartridges. Most noteworthy is that this therapy is also supplied with an electric auto-injector (the instructions for use resemble something from IKEA). Each cartridge includes a chamber for the dry powder and a sterile water chamber for mixing with the powder….. inside the electric injector. Safety instructions state not to use near a microwave oven…. now that is something you don’t see in prescribing information every day. The electric injector is replaced after every six injections.

Skytrofa is supplied in a 4-dose carton for once weekly administration. A carton of Skytrofa with a GoodRx discount costs $2,500 – $2,700 depending on pharmacy. Finally a new therapy that is not in limited distribution.

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FDA Approves Weekly Therapy for Pediatric Patients with Growth Hormone Deficiency

FDA has approved Skytrofa (lonapegsomatropin-tcgd) injection to treat pediatric patients age one year and older who weigh at least 11.5 kg (25.4 pounds) and have short stature due to inadequate secretion of endogenous growth hormone. Skytrofa is a human growth hormone (hGH) approved for pediatric patients to take by under-the-skin injection weekly. Other available FDA-approved hGH formulations for pediatric patients with growth hormone deficiency must be administered daily.

Growth hormone deficiency is a disorder characterized by inadequate growth hormone production from the anterior pituitary gland, a small gland located at the base of the brain that produces several hormones. Children with this disorder can receive growth hormone to stimulate growth.

Skytrofa was studied in a 52-week trial that enrolled 161 pre-pubescent research participants (average age was 8.5 years) with growth hormone deficiency who had received no previous growth treatment. Participants were randomly assigned to receive either weekly injection of Skytrofa or daily injection of somatropin, an FDA-approved human growth hormone. Efficacy was determined by the increase in growth rate after 52 weeks of treatment. Weekly Skytrofa treatment for 52 weeks resulted in a growth rate increase of 11.2 cm (4.4 inches) per year and was not inferior to daily somatropin treatment.

The most common side effects of Skytrofa include viral infection, pyrexia (fever), cough, nausea and vomiting, hemorrhage (blood loss), abdominal (stomach) pain, arthralgia (joint pain), arthritis, and diarrhea. Patients with acute critical illness, allergies to somatropin or any Skytrofa excipients, active malignancy, certain types of diabetic retinopathy (eye-related diabetes complications), and Prader-Willi syndrome with other risk factors should not take Skytrofa. Children with closed epiphyses (end part of a long bone) should also not take the drug.

Patients taking Skytrofa are also at increased risk of neoplasms (abnormal tissue growth), intracranial hypertension (buildup of pressure around the brain), fluid retention, hypoadrenalism (underactive adrenal gland), hypothyroidism (underactive thyroid), slipped capital femoral epiphysis (a pediatric hip condition), progression of preexisting scoliosis, and pancreatitis (inflammation of the pancreas).

Skytrofa may interact with glucocorticoid (a type of steroid) treatment, oral estrogen, insulin, and other antihyperglycemic (blood sugar-reducing) agents.

Most of us that work in the specialty pharmacy segment are very familiar with the concept of a HUB. At its rudimentary level a HUB acts as a clearinghouse for a drug to expedite getting the therapy from the prescriber to the patient as efficiently as possible. As is often the case, the therapy is in limited distribution, so the HUB directs traffic to put the prescription on a track to a designated pharmacy. That’s about where the knowledge level starts to fall off for many.

The article below is a good read for new SP staff to get a thorough introduction to the HUB concept. But, it is also a good read for more experienced staff to fill in any blanks so they can give a fulsome explanation of what a HUB does and the benefits and complexities associated with the model.

A couple points that the article did not include—–
– First, many HUBs include multiple participating specialty pharmacies. An underlying reason is that not all specialty pharmacies have payer contracts for reimbursement. By stitching together a network that expands payer reach fewer roadblocks to patient authorization will be encountered.
– Secondly, the importance of data is understated. Manufacturers have increasingly relied on specialty pharmacies to report on ever more granular details including patient status, reasons for termination of therapy, occurrence of ER admissions, patient health progress, etc., etc. These additional data items are usually tailored to each specific therapy. Such services also provide an important revenue source for the specialty pharmacy.

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The Value of a Hub in a Limited Distribution Specialty Pharmacy Network

Although specialty pharmacies may be able to offer some of the services that a hub provider does, a hub can standardize patient and provider support across the network.

“What is a hub?”
“What does a hub do?”
“Why is a hub needed?”
“Why would a manufacturer select a hub as part of their network?”

I find myself answering these questions often. For a non-industry native, I find it hard to provide a succinct answer. Fundamentally, a hub provider serves as an extension to the drug manufacturer and administers their branded support programs.

In many instances, a hub proves to be extremely valuable in a network because it can provide unique program services. These services aim to enhance coordination of care, patient access, financial support, and patient and prescriber engagement. Services include, but are not limited to:
– case management
– reimbursement and co-pay support
– specialty pharmacy triage
– network coordination and reconciliation of referrals
– field team support
– nurse navigator and psychosocial support
– free drug qualification and non-commercial pharmacy dispensing
– patient clinical support
– health care provider education
– execution of Risk Evaluation and Mitigation Strategy (REMS) program
– data reporting, analytics, and digital solutions

To get a better understanding of these services and why a hub is poised to provide these services, an understanding of the limited distribution network is warranted.

Understanding the Limited Distribution Network
Beginning at a few years prior to the anticipated Prescription Drug User Fee Act (PDUFA) date and launch of a product, a biopharmaceutical company must define its distribution strategy. This includes designing the network of specialty pharmacies and whether to include a hub service provider, data aggregator, and other vendors.

The number of specialty pharmacies to include in the network heavily depends on the size of the patient population anticipated to be treated with the therapy being commercialized. The larger the patient population, the more pharmacies are likely included in the network.

Depending on the disease state, product considerations and the anticipated size of the patient population, certain specialty pharmacies are considered. For example, if a biopharmaceutical manufacturer is launching an oncology product, they should consider oncology-focused specialty pharmacies.

Similarly, if a product is ultra-rare, then a rare disease-focused specialty pharmacy is the obvious choice to pursue.

Payer and reimbursement factors also play a role in the number and type of specialty pharmacies to include in the network. A biopharmaceutical company must ensure that the network they select enjoys broad coverage of the major payers to guarantee product reimbursement. This can be accomplished by selecting all pharmacy benefit manager (PBM)-owned pharmacies.

Alternatively, a manufacturer can select all independently owned pharmacies without providing access to the product to the PBM-owned pharmacies, which prevents payers from mandating product be filled at their pharmacies.

Once the commercial specialty pharmacy strategy is understood, a decision is made on whether to include a hub service provider in the network. If the patient population is small and the manufacturer decides on an exclusive model in which a single specialty pharmacy has access to the product, then including a hub may not be necessary.

However, as the number of pharmacies grow, positioning a hub to “quarterback” the network becomes the right choice to make.

“So why do I need a hub if specialty pharmacies can do it?”

The Value a Hub Service Provider
A hub can play an essential role in coordinating the specialty pharmacy limited distribution network. Although specialty pharmacies may be able to offer some of the services that a hub provider does, a hub can standardize patient and provider support across the network and coordinate referrals to ensure patients end up at their most appropriate destination.

Referral Reconciliation: Depending on whether the hub model is mandatory or not, referrals can end up at multiple places within the network. The right hub strategy uses a proactive approach to reconcile referrals across the network and ensure no patients are left behind. If referrals are in a stalemate or are being serviced by multiple pharmacies simultaneously, causing unnecessary payer blocks, the hub can step in to assist. This ensures only one pharmacy is moving the patient to therapy or preventing a lapse in therapy.

Sophisticated Network Triage: Sending hub referrals to the appropriate pharmacy is an important role only a hub can play. If the right hub strategy is chosen, it can set up automated triage rules based on payer mandates, physician preference, round robin, and other rules to guarantee referrals make it to the pharmacy that has the highest likelihood of getting a paid claim. If done right, a hub can have a tremendously positive effect on time to therapy by minimizing network transfers.

Enhanced Data Visibility: A hub plays an important role in increasing Health Insurance Portability and Accountability Act (HIPAA) authorization and consent rates into additional programs. Referrals can come into the hub with patient signatures. If that is not the case, the hub can follow up during a welcome call to obtain consent, ideally digitally via e-consent. With increased HIPAA authorization rates comes increased data visibility to the manufacturer. Additionally, if the right hub strategy is chosen, it can set up data agreements across the network to enhance data visibility, allowing it to play an active role in referral reconciliation.

Customizability: Hub providers have a core set of services they offer, but also have the ability to highly customize when necessary. This involves customizing the process flow based on patient and provider needs, the patient engagement journey, and data reporting. The right hub strategy is nimble, able to make process changes on the fly, and captures any data element required for reporting.

Other Important Services a Hub Provides
Case Management and Field Team Support: A hub can employ dedicated case managers that serve as a main point of contact to patients and prescribers. They can assist patients in navigating their journey to treatment and beyond. They can serve as a patient advocate. They can also assist health care providers with prescribing the medication and send field-based team members to properly support prescribers.

Reimbursement and Co-pay Support: Depending on the model, the hub can assist with drug reimbursement and co-pay support. In a mandatory hub model, the hub performs detailed benefits investigation, supports prescribers through prior authorizations and appeals, and triages clean claims to pharmacies for dispensing. After launch, a hub can quickly learn payer requirements, stay up to date with formulary updates, and work with the manufacturer’s market access team to ensure optimization of payer coverage. For commercially insured patients, the hub can enroll them in a co-pay program and communicate the co-pay identification to the pharmacy to use at adjudication, minimizing the patient’s out-of-pocket burden.

Nurse Navigator and Psychosocial Support: A hub can enhance patient engagement through nursing and psychosocial support. Nurse navigators can serve as a patient resource through disease state education. They can conduct needs assessments to understand the patient’s knowledge and familiarity with their diagnosis. They can assess the patient’s support system and guide them through their journey. They set the expectation for next steps regarding coverage determination and specialty pharmacy triage. Based on findings from needs assessments, nurse navigators schedule follow ups to discuss appropriate topics that will be beneficial to the patient.

Free Drug Qualification and Non-Commercial Pharmacy Dispensing: A hub can administer free drug programs, such as patient assistance programs, bridge, quick start, dose exchange, voucher, and other forms of “free” drug. This includes qualifying patients based on business rules and triage to a non-commercial pharmacy for dispensing. Ideally, the hub owns a non-commercial pharmacy licensed in 50 states. Having an in-house non-commercial pharmacy prevents roadblocks associated with an outsourced pharmacy that may not be able to accommodate specific program nuances.

Patient Clinical Support: If the hub leverages nurse navigators for disease and psychosocial support, the non-commercial pharmacy can leverage pharmacist clinical support for patients on free drug. Clinical pharmacists can educate the patient about the medication, side effect profile and potential drug-drug interactions. They can set the expectation of the benefit of taking the medication, what to do if doses are missed, how to store the medication and address any questions. If the medication requires a titration, weight-based dosing or has special dosing considerations, the pharmacy can support patients and prescribers through proper administration and dose optimization.

Health Care Provider Education: This includes educating prescribers on sending complete enrollments, guiding them through the reimbursement process and supporting them with medication prescribing and dose adjustments. The hub can provide a dedicated, single point-of-contact to prescribers for protocol support.

REMS Program Execution: If a REMS program is required by the FDA, the hub can customize its process flow to fulfill REMS requirements. This can include medication guide requirements, guiding prescribers to enroll in a program and provide the necessary attentions, enrolling patients and counseling them on potential risks, verifying these enrollments prior to shipments, adverse events reporting, and so forth.

Data Reporting, Analytics, and Digital Solutions: In addition to the services discussed above, hubs communicate activities to the manufacturer through reporting and analytics. Standard reports include status, dispense, and inventory files. Hubs have experience working with data aggregators, have the ability provide custom reports, and have the capability to provide real time insights. Other digital solutions include texting and emailing capabilities, e-consent, electronic prescribing, etc.

Selecting the “Right Hub”
Similar to selecting specialty pharmacies, it is preferred that the hub is focused in the treatment area, such as rare disease for an orphan drug or oncology-focused for a cancer medication. In addition to that, the right hub is dedicated, proactive, attentive to detail, and plays an active end-to-end role in the network.

The right hub is nimble, quickly adaptable, and delivers on promises. Similarly, the right hub has an in-house non-commercial pharmacy that provides a consistent experience with the commercial specialty pharmacies in the network and integrates seamlessly with the patient service provider.

In conclusion, the right hub service provider acts as an internal, external hub to the manufacturer, executing their brand support program flawlessly and setting up the limited distribution network to succeed.

Rami Chammas, PharmD, MPBA
Pharmacy Times

Announcements for newly approved specialty drugs often state that the product will be available through specialty pharmacy in limited distribution. However, the press releases rarely specify the specialty pharmacy(ies) selected as the designated partner(s).

Here are a few LD deals that have been recently publicly confirmed subsequent to the approvals.

Orsini Specialty Pharmacy
Sanofi has selected Orsini as a limited distribution partner for Nexviazyme (avalglucosidase alfa-ngpt). Nexviazyme, an enzyme replacement therapy (ERT), is used for treating patients one year of age and older with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency).

AllianceRx Walgreens Prime.
Arcalyst (rilonacept) is the first and only FDA-approved product for the treatment of recurrent pericarditis and reduction in risk of recurrence. Pericarditis is a painful and debilitating autoinflammatory cardiovascular disease associated with swelling of the sacklike membrane surrounding the heart. Arcalyst is also approved by the FDA for the treatment of Cryopyrin-Associated Periodic Syndromes (CAPS), a group of rare hereditary autoinflammatory disorders, including Familial Cold Autoinflammatory Syndrome and Muckle-Wells Syndrome, and for the maintenance of remission of Deficiency of Interleukin-1 Receptor Antagonist (DIRA), an autoinflammatory disease affecting fewer than 50 people worldwide. Kiniksa Pharmaceuticals manufactures Arcalyst, which is also a registered trademark of Regeneron Pharmaceuticals, Inc.

Gavreto (pralsetinib) is a once-daily oral prescription medicine used to treat certain cancers caused by abnormal RET-positive (rearranged during transfection) genes in non-small-cell lung cancer that has spread (metastatic) or advanced thyroid cancer that may have spread. Blueprint Medicines manufactures Gavreto.

Maxor Specialty Pharmacy
Elixir has selected Maxor as its exclusive Cystic Fibrosis (CF) provider in its specialty pharmacy network. Maxor Specialty Pharmacy will provide Elixir CF patients with case management, education, such as how to clean and maintain their equipment, and fulfill their prescriptions. Maxor is one of four pharmacies in the United States with access to all CF drugs, including Trikafta.

The FDA approved a new therapy, Susvimo (ranibizumab injection) from Genentech, indicated for intravitreal use via ocular implant for wet age-related macular degeneration (AMD) with at least two prior anti-vascular endothelial growth factor (VEGF) injections. The implant is surgically inserted into the eye during a one-time, outpatient procedure and can be refilled every six months.

Wet AMD impacts approximately 1.1 million people in the United States and is a leading cause of blindness in people aged 60 and older. Age-related macular degeneration (AMD) is a condition that affects the macula, the part of the eye that provides sharp, central vision. AMD is an advanced form of the disease that can cause rapid and severe vision loss. Approximately 11 million people in the United States have some form of AMD.

Genentech developed the first anti-VEGF medicine, Lucentis (ranibizumab injection), approved for wet AMD by the FDA in 2006. Susvimo, however, is different from the ranibizumab intravitreal injection.

Pricing for Susvimo was not released. By way of reference, Lucentis costs approximately $2,000 / month and is administered monthly….. so upwards of $24,000 annually. Analysts believe that Susvimo will generate more than $1 billion in 2022 sales. The 2020 AMD market in the US generated more than $35 billion in sales.
Distribution details on were not released. However, virtually all other therapies for AMD and other high-cost Ophthalmology injectable therapies are currently in limited distribution, direct-to-provider, via specialty pharmacies.
Susvimo will be available in the United States later this quarter.

CLICK HERE to access the full Genentech press release.

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FDA Approves Genentech’s Susvimo, a First-of-Its-Kind Therapeutic Approach for Wet Age-Related Macular Degeneration (AMD)

Susvimo, previously called Port Delivery System with ranibizumab, is the first wet AMD treatment in 15 years to provide an alternative to standard-of-care eye injections needed as often as once a month
By continuously delivering medicine into the eye through a refillable implant, Susvimo may help people with wet AMD maintain their vision with as few as two treatments per year

October 22, 2021 — Genentech, a member of the Roche Group, today announced that the U.S. Food and Drug Administration (FDA) has approved Susvimo (ranibizumab injection) 100 mg/mL for intravitreal use via ocular implant for the treatment of people with wet, or neovascular, age-related macular degeneration (AMD) who have previously responded to at least two anti-vascular endothelial growth factor (VEGF) injections. Wet AMD is a potentially blinding condition that requires treatment with eye injections as often as once a month. Susvimo, previously called Port Delivery System with ranibizumab, is the first and only FDA-approved treatment for wet AMD that offers as few as two treatments per year.

“Susvimo represents a major advancement in the treatment of retinal disease and is an important new option for patients with wet AMD,” said Carl Regillo, M.D., Chief of Retina Service at Wills Eye Hospital in Philadelphia and an Archway study investigator. “With Susvimo, my patients now have an option that can help them maintain their vision as well as anti-VEGF injections, but on a more manageable twice-yearly treatment schedule.”

Susvimo delivers ranibizumab continuously, offering people living with wet AMD an alternative to anti-VEGF eye injections needed as often as once a month

The approval is based on positive results from the Phase III Archway study primary analysis, which showed wet AMD patients treated with Susvimo achieved and maintained vision gains equivalent to monthly ranibizumab injections – +0.2 and +0.5 eye chart letters from baseline, respectively – at weeks 36 and 40 of treatment. In addition, only 1.6% of Susvimo patients received supplemental ranibizumab treatment before their first refill, and more than 98% could go six months before their first refill.

Genentech is committed to helping people access the medicines they are prescribed and will be offering comprehensive services for people prescribed Susvimo to help minimize barriers to access and reimbursement. Patients can call 833-EYE-GENE for more information. For people who qualify, Genentech plans to offer patient assistance programs through Genentech Access Solutions. More information is also available at (866) 4ACCESS/(866) 422-2377 or http://www.Genentech-Access.com.

Genentech’s late-stage ophthalmology portfolio also includes faricimab, a bispecific antibody under FDA and EMA review for the treatment of wet AMD and DME. The FDA is additionally reviewing faricimab for the treatment of diabetic retinopathy.

Press release continues…………

A little over two years ago we reported on the approval of yet another biosimilar to Humira (yawn), Cyltezo (adalimumab-adbm) a sub-q therapy from Boehringer Ingelheim. It was noteworthy at the time, but didn’t create any ripples in the pharmacy world….. till now.

This week the FDA approved Cyltezo….. again.
But the big news is that now the therapy has earned a precious gold star designation as an interchangeable biosimilar to AbbVie’s Humira. This is only the second interchangeable biosimilar to date.

Cyltezo still won’t be available until July 1, 2023 as a result of settlement with AbbVie that allows marketing in the United States. Pharmacists will then be able to routinely substitute Cyltezo for AbbVie’s mega-blockbuster without a prescriber’s OK. Payers and PBMs will also have broader latitude to regigger their formularies to steer selection to the lower cost interchangeable(s?).

The remaining question is….. “So, how much will it cost?”
Analysts suggest that the discounts (vs. Humira) of up to 50% as seen in Europe may be the upper limit in the US. Even a 30+% discount would deliver huge $$ savings in the US.

Cyltezo has the leg up on a crowded field of Humira competitors with its interchangeable title. Current frontrunners include Amgen’s Amjevita (adalimumab-atto) (also likely to launch in January 2023), Sandoz’s Hyrimoz (adalimumab-adaz), Pfizer’s Abrilada (adalimumab-afzb) and Samsung’s Hadlima (adalimumab-bwwd). Others are pending FDA approval and/or are seeking the interchangeable designation crown as well.

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FDA Approves Cyltezo, the First Interchangeable Biosimilar to Humira

Second Interchangeable Biosimilar Product Approved by Agency

The U.S. Food and Drug Administration approved the first interchangeable biosimilar product to treat certain inflammatory diseases. Cyltezo (adalimumab-adbm), originally approved in August 2017, is both biosimilar to, and interchangeable with (may be substituted for), its reference product Humira (adalimumab) for Cyltezo’s approved uses. Cyltezo is the second interchangeable biosimilar product approved by the agency and the first interchangeable monoclonal antibody. Once on the market, approved biosimilar and interchangeable biosimilar products can play a role in facilitating access to treatments for many serious health conditions.

“The biosimilar and interchangeable approval pathway was created to help increase access to treatment options for patients with serious medical conditions,” said Acting FDA Commissioner Janet Woodcock, M.D. “We continue to be steadfast in our commitment to provide patients with alternative high-quality, affordable medications that are proven to be safe and effective.”

Cyltezo is approved for the following indications in adult patients:
– moderately to severely active rheumatoid arthritis;
– active psoriatic arthritis;
– active ankylosing spondylitis (an arthritis that affects the spine);
– moderately to severely active Crohn’s disease;
– moderately to severely active ulcerative colitis; and
– moderate to severe chronic plaque psoriasis
– moderately to severely active polyarticular juvenile idiopathic arthritis in patients two years of age and older
– pediatric patients six years of age or older with Crohn’s disease.

Biological products, generally derived from a living organism, include medications for treating many serious illnesses and chronic health conditions. A biosimilar is a biological product that is highly similar to, and has no clinically meaningful differences from, a biological product already approved by the FDA (also called the reference product). Patients can expect the same safety and effectiveness from the biosimilar as they can from the reference product. Interchangeable biosimilar products can be expected to produce the same clinical result as the reference product in any given patient and, for biological products administered more than once to an individual, the risk in terms of safety or diminished efficacy of switching between the two products is not greater than the risk of using the reference product without such switching.

Cyltezo, offered in a single-dose, pre-filled glass syringe (40 mg/0.8 mL, 20 mg/0.4 mL), is administered subcutaneously (under the skin) under the guidance of a physician. The most serious known side effects with Cyltezo are infections and malignancies (cancers). The most common expected adverse reactions are upper respiratory and sinus infections, injection site reactions, headache and rash.

Like Humira, the labeling for Cyltezo contains a boxed warning to alert health care professionals and patients about an increased risk of serious infections that may lead to hospitalization or death. The boxed warning also notes that lymphoma and other malignancies, some fatal, have been reported in children and adolescent patients treated with tumor necrosis factor blockers, including adalimumab products. The drug must be dispensed with a patient Medication Guide that describes important information about its uses and risks.

The FDA granted approval of Cyltezo to Boehringer Ingelheim on October 15, 2021.

Last week we published a review of the 2021 Medication Access Report which detailed a variety of access barriers including COVID-19, logistics, financial burdens, and more. We suggested that understanding the scope of access challenges would be good knowledge for anyone working in the specialty pharmacy segment, but especially those that work in marketing, communications and web messaging. Today we turn up the heat.

The results of a recent research survey, sponsored by AllianceRx Walgreens Prime, offer deep dive insights into the social determinants of health and, specifically, how these determinants impact patient access to health.

Here are the key areas covered in the survey…..
When we say it is a deep dive we mean it – especially for a specialty pharmacy.
Safe housing, transportation, and neighborhoods
Racism, discrimination, and violence
Education access, job opportunities, and income
Access to nutritious foods and physical activity opportunities
Polluted air and water
Language and literacy skills

The findings are highly insightful and are appropriate for leadership in specialty pharmacies as well as those involved in communications to better know their customers.

CLICK HERE to access the survey findings

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How Specialty Pharmacies Address Social Determinants of Health
Specialty pharmacies are uniquely positioned to identify social determinants of health that could impact medication adherence and health outcomes.

Specialty pharmacies play an integral role in ensuring patients receive, understand, and adhere to medications prescribed by their providers. This role also includes coordinating the numerous aspects of patient care and disease management.

“Specialty pharmacies are responsible for assisting patients through their treatment journey, which in many cases may be a lifelong journey with many complexities and challenges,” says AllianceRx Walgreens Prime Vice President of Clinical and Professional Services Rick Miller, MS Pharm, MBA, BS Pharm, CSP.

Many specialty patients present with autoimmune disorders, cancer, bleeding and blood disorders, HIV/AIDS, multiple sclerosis (MS), along with other chronic and rare conditions. As a result of working so closely with patients, specialty pharmacies are uniquely positioned to spot obstacles likely to impact the patient’s ability to begin and stay on their medications — including those obstacles that are commonly known as the social determinants of health.

The US Department of Health and Human Services defines social determinants of health (SDOH) as the conditions in the environments where people are born, live, learn, work, play, worship, and age that affect a wide range of health, functioning, and quality-of-life outcomes and risks.

These social determinants of health have a major impact on patient care, with access to and the quality of medical care impacting approximately 20 percent of a patient’s health and well-being, SDOH factors impact the remaining 80 percent of health outcomes.

Economic stability is a leading social determinant of health with implications for patient outcomes when patients cannot afford necessary care, including medications. Individuals facing financial hardship are in a precarious situation and often opt to avoid care, which can have severe implications for those with complex and chronic medical conditions requiring specialty medications.

A growing body of literature indicates that cancer patients are more likely to not receive necessary medical care due to financial hardship and report changes in prescription drug use due to financial reasons. What’s more, estimates show that medication non-adherence costs the healthcare industry $300 billion, one-third of total waste, with $100 billion alone tied to avoidable hospitalizations.

High costs are a defining feature of specialty medications, though definitions vary. Whereas the Centers for Medicare & Medicaid Services indicated costs of more than $670 per month back in 2017, the Congressional Budget Office stated an annual cost of $6,000 as recently as 2015.

With cost being a significant factor in medication adherence, specialty pharmacies on the frontlines have the power to identify and address economic stability among the patients they serve, improving health outcomes and reducing costs by avoiding hospitalizations and emergency care services were a patient’s condition to deteriorate.

Specialty pharmacies assist with implementing the care plan developed in collaboration between the physician, patient, and/or patient’s caregiver and ensuring the medications are properly dispensed and taken.

Earlier this year, AllianceRx Walgreens Prime and Highmark launched a pilot to identify the impact of SDOH on patients with MS in terms of quality of life and health outcomes. Trained nurses at the specialty pharmacy contact patients who have agreed to participate in the pilot by phone to administer a 13-question SDOH survey. The results of which are reviewed by the health plan to determine the support necessary for individuals facing these financial, behavioral, or environmental challenges.

In addition to the SDOH pilot, AllianceRx Walgreens Prime’s staff focuses on identifying potential obstacles to patient care.

“At AllianceRx Walgreens Prime, many of the cues will be verbal in nature due to the high prevalence of the way in which we telephonically interact with patients,” Miller explains. “We work with our staff to properly train them in active listening. You need to listen to the patient, listen to their responses. For example, focusing on any hesitation, any language barriers, or any confusion.”

A combination of staff and services allows the specialty pharmacy to holistically meet the needs of patients head-on, including pharmacists, nurses, patient care coordinators, and financial experts. The ability to provide wrap-around support services puts the specialty pharmacy in a position to work with charitable foundations and pharmaceutical companies as well as health plans and providers to coordinate appropriate care for each patient.

But the coordination of services is only as effective as the specialty pharmacy staff’s ability to recognize that a problem exists. This is especially true for patients with MS who often experience depression and fatigue during therapy. Nurses at the specialty pharmacy are equipped to screen for these factors and report the results to the appropriate specialist.

Responsible for the initiation of therapy and each subsequent refill, the specialty pharmacy has numerous opportunities to engage with patients and screen them for potential obstacles for optimal care and outcomes. In truth, providing holistic care to patients has been a staple of successful specialty pharmacies, given their central role in medication adherence.

There are two ways the specialty pharmacy helps ensure medication adherence among its patient population. First, they can empower patients by educating them about their condition, therapy, medication, expected clinical outcomes, and potential adverse events. Second, specialty pharmacies can reach out to patients “off-cycle” to identify potential issues between refills that could negatively impact adherence.

In short, it all comes down to working closely with patients and listening to their needs.

“We collaborate with the patient. We let them also know that they’re not traveling through this journey alone and that we’re here to help them 24/7,” Miller concludes.

While the focus on social determinants of health has grown considerably before and throughout the pandemic, specialty pharmacists have remained well suited to address and overcome these obstacles to improve the lives of patients they serve.

as published in Health Payer Intelligence

We’ve written frequently on two of the biggest trends in channel access, the shift away from buy-and-bill to other, less costly, sites of service as well as payer policies that have shifted reimbursement away from physician / hospital purchased drugs to specialty pharmacies. An article we want to review today is from Drug Channels. It is a great read and includes some fresh data to document these trends.

That article, an excerpt from a larger market report available for purchase from the company, first refreshes our understanding of the types of patient access now being employed. By now we are all familiar with White Bagging and Brown Bagging (if not, read the article). A term that is beginning to catch on is Clear Bagging in which the hospital-owned specialty pharmacy does the fulfillment.

What the data shows is that there has been a significant increase in White Bagging over the past two years with a 20% increase at physician practices and a 25% increase at hospital outpatient departments. Brown Bagging has virtually disappeared in these settings. Strangely, buy-and-bill at Home Infusion companies actually increased by a whopping 41% in the same time period and Brown Bagging more than doubled. Go figure! The data is based on a very large ‘n’ of payers nationally. 

There are several points for further consideration. 

• First, the list of impacted drugs goes beyond Oncology….. and is growing.  
• Secondly, a number of specialty pharmacies have been designated by manufacturers as limited distribution (LD) partners (often exclusive) for certain drugs….. even bypassing traditional wholesalers. Since virtually all of these SPs are now also licensed distributors they can sell direct for professional use as a buy-and-bill drug….. under the medical benefit. (Not popular with hospitals and many providers.) 
• Next, Payer and PBM policies have ramped in the past year+ to push these transactions over to the pharmacy benefit (even less popular) through rewriting the patient benefit plan…. including introducing patient OOP if billed as a medical benefit. 
• Concurrently, PBMs and Payers have been able to co-opt these dynamics to further their cost management efforts on other, non-LD drugs. The data does not separately break out these LD drugs which further muddies up the total White Bagging picture.  
• And finally, we can more easily understand why hospitals are increasingly desperate to redirect fills internally to owned specialty pharmacies to recapture lost buy-and-bill revenue.

White Bagging Update: PBMs’ Specialty Pharmacies Keep Gaining on Buy-and-Bill Oncology Channels Drug Channels, October 2021

We all know that the past year has been nothing short of a massive monkey wrench thrown into the US healthcare machinery and the impacts are only now being assessed. Specialty pharmacies have been acutely attuned to patient psycho-social needs for a long time and need to adjust their patient facing programs according to changing times. Some fresh guidance might be timely.

A new study was recently released that you may not have seen. For many readers it may be outside your bailiwick, but the info is something that your marketing, media, and web messaging colleagues might want to review to keep pace with these challenging times.

The link below will connect to the 2021 Medication Access Report published by CoverMyMeds, part of McKesson’s Prescription Technology Solutions. It uses industry research, patient interviews and new survey data from patients, pharmacists and providers to investigate medication access barriers and how we can overcome them as an industry, especially in the wake of the COVID-19 pandemic.

CLICK HERE to read the report

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The 2021 Medication Access Report emphasizes medication access barriers further impacted by the COVID-19 pandemic and technology that can make a difference

• 36% of Patients Sacrifice Medications to Afford Basic Needs

COLUMBUS, Ohio, Jan. 26, 2021 /PRNewswire/ — The impact from the COVID-19 pandemic caused tens of millions of Americans to lose their jobs, health insurance and, for some, access to their medications. New research released today by CoverMyMeds found that 65 percent of patients were financially impacted by the COVID-19 pandemic and 36 percent sacrificed their treatments or medications to pay for bills and basic needs. Others have done the inverse: 43 percent have had to forgo paying for essential items and bills to afford needed medications,2 while 41 percent have skipped or modified medication doses to stretch out prescriptions.

“I don’t know how people can afford to be sick and still live their lives,” said Patricia F., a patient featured in the 2021 Medication Access Report who shared her experience managing multiple sclerosis. “I take it one day at a time and hope I’m not going to find out when it’s too late that I really should have been on medication.”

The 2021 Medication Access Report also highlights technology designed to help inform medication conversations with patients, support prescribing decisions, and empower patients and caregivers to become part of their own care team, including:

Prescription Price Transparency
Sixty-two percent of patients ask their provider about medication price and affordability options.2 Equipping providers and care team members with tools that show prescription cost, remaining deductible, formulary alternatives, cash price and cost savings programs at the point of prescribing can help bolster affordability conversations with patients.
When a prescription cost more than expected, 43 percent of patients in 2020 said they checked a pharmacy comparison app to find a better price, up from 28 percent in 2019.

Tech-Enabled Patient Support Services
Specialty therapies are often the hardest to access for patients, usually due to process complexity and affordability challenges. Nearly half of providers surveyed indicated they changed how they approach specialty patient appointments due to COVID-19 while nearly one in five providers saw a decrease in patient therapy compliance.
Through continued development of electronic patient support services, available at the point of prescribing for prescribers and their staff, more patients can experience end-to-end support to access specialty medications. In some cases, such programs have contributed to a 34 percent reduction in time to therapy.

Electronic Prior Authorization
Due to manual prior authorization management, over one third of providers reported having less time for face-to-face interaction with patients—during which meaningful conversations about affordability, access or social circumstances could occur.

Adoption of a robust electronic prior authorization solution can help reduce manual processes and improve patient outcomes. In fact, 62 percent of prior authorization requests submitted electronically receive a determination within two hours, and 43 percent receive a determination automatically.

“Patients need reliable paths to medication access, and they shouldn’t have to choose between basic needs to access and afford their medications,” said Miranda Gill MSN, RN, NEA-BC, Senior Director, Provider Services and Operations at CoverMyMeds. “Encouraging patients to proactively participate in their care plan has shown to help improve outcomes. By utilizing reliable technology to view cost, quality and convenience options, the patient and provider can collaborate to make the best treatment decision for the patient.”