Last week the FDA approved a new, INFUSED enzyme replacement therapy, Nexviazyme (avalglucosidase alfa-ngpt) from Genzyme, indicated to treat patients 1 year of age and older with late-onset Pompe disease.

Pompe disease is a progressive, multisystemic, debilitating, and often fatal neuromuscular disorder. Enzyme replacement therapy (ERT) is the only approved treatment for all patients with Pompe disease. The first enzyme treatment, Lumizyme (also from Genzyme), was approved in 2006.

There are three types of Pompe disease:

• Classic infantile onset appears within a few months of birth.
• Non-classic infantile onset appears at about 1 year of age.
• Late-onset appears later in a child’s life, or even into the teen years or adulthood.

Pompe disease is estimated to affect 1 in 40,000 in the U.S. with a genetic carrier frequency estimated at 1 in 100. In Pompe disease, mutations in the GAA gene reduce or completely eliminate an essential enzyme needed to prevent excessive lysosomal glycogen accumulation throughout the body. Patients with Pompe disease experience heart enlargement, generalized skeletal muscle weakness, and a life expectancy of less than 2 years if untreated (classic infantile and non-Classic infantile onset).

Genzyme has announced pricing for Nexviazyme similar to that of its sister drug Lumizyme. An annual course of therapy for a pediatric patient will run $330,000 and $758,000 per year for an adult patient. Details on access were not announced but it is almost certain that Nexviazyme, as with Lumizyme, will launch through specialty pharmacy distribution – direct to provider.

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FDA Approves New Treatment for Pompe Disease

August 06, 2021 — Today, the U.S. Food and Drug Administration approved Nexviazyme (avalglucosidase alfa-ngpt) for intravenous infusion to treat patients 1 year of age and older with late-onset Pompe disease.

Patients with Pompe disease have an enzyme deficiency that leads to the accumulation of a complex sugar, called glycogen, in skeletal and heart muscles, which cause muscle weakness and premature death from respiratory or heart failure. Normally, glycogen—the stored form of glucose—breaks down to release glucose into the bloodstream to be used as fuel for the cells.

“Pompe disease is a rare genetic disease that causes premature death and has a debilitating effect on people’s lives,” said Janet Maynard, M.D., deputy director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research. “Today’s approval brings patients with Pompe disease another enzyme replacement therapy option for this rare disease. The FDA will continue to work with stakeholders to advance the development of additional new, effective and safe therapies for rare diseases, including Pompe disease.”

Nexviazyme, an enzyme replacement therapy, is an intravenous medication that helps reduce glycogen accumulation. The effectiveness of Nexviazyme for the treatment of Pompe disease was demonstrated in a study of 100 patients who were randomized to take Nexviazyme or another FDA-approved enzyme replacement therapy for Pompe disease. Treatment with Nexviazyme improved lung function similar to the improvement seen with the other therapy.

The most common side effects included headache, fatigue, diarrhea, nausea, joint pain (arthralgia), dizziness, muscle pain (myalgia), itching (pruritus), vomiting, difficulty breathing (dyspnea), skin redness (erythema), feeling of “pins and needles” (paresthesia) and skin welts (urticaria). Serious reactions included hypersensitivity reactions like anaphylaxis and infusion-associated reactions, including respiratory distress, chills and raised body temperature (pyrexia). Patients susceptible to fluid volume overload or with compromised cardiac or respiratory function may be at risk for serious acute cardiorespiratory failure.

The FDA granted this application Fast Track, Priority Review and Breakthrough Therapy designations. Nexviazyme also received an orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. The FDA granted the approval of Nexviazyme to Genzyme Corporation.

It was way back in March of 2015 when the first biosimilar was approved. There was much consternation over the impact that biosimilars would have on the ‘land of plenty’ world of brand name drugs. As we all know, those concerns never realized as the biggest impediment to triggering a shift to the ‘copycat drugs’ was the lack of interchangeability.

The interchangeability ceiling was finally broken last week with the approval of the first interchangeable biosimilar insulin product, Semglee (insulin glargine-yfgn) from Mylan, indicated to improve glycemic control in adults and pediatric patients with Type 1 diabetes mellitus and in adults with Type 2 diabetes mellitus. Semglee is both biosimilar to and interchangeable with its reference product, Lantus (insulin glargine), a long-acting insulin analog.

It is ironic that the first interchangeable biosimilar is for a non-specialty therapy when much of the original concern over biosimilars related to specialty biologics. As we know, the biologics that have been approved and are commercially available are for therapies not generally dispensed by specialty pharmacies. The remainder are NOT available commercially as they have have been sidelined due to massive legal challenges usually related to patent protections.

Patients have long awaited relief from brand prices and have been disappointed. The approval of Semglee finally delivers measurable relief. The GoodRx price for Semglee with a coupon is only $104 compared to the reference product, Lantus, at more than $400.

Side notes
First, now that we have proof of interchangeability will the FDA be more inclined to grant that designation to other biosimilars dispensed by specialty pharmacies. Probably. However, having an interchangeable designation won’t remove the legal roadblocks can keep FDA approved biosimilars off the commercial market for years.

Second, Walmart announced that it will start selling its own private brand of analog insulin at prices up to a 75% discount to competing products. The private-brand insulin will cost $73 per vial and $86 per FlexPen, prices that are 58% to 75% lower than other insulin products. Details as to source for the ‘private brand’ were not released.

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FDA Approves First Interchangeable Biosimilar Insulin Product for Treatment of Diabetes

July 28, 2021 — Today, the U.S. Food and Drug Administration approved the first interchangeable biosimilar insulin product, indicated to improve glycemic control in adults and pediatric patients with Type 1 diabetes mellitus and in adults with Type 2 diabetes mellitus. Semglee (insulin glargine-yfgn) is both biosimilar to, and interchangeable with (can be substituted for), its reference product Lantus (insulin glargine), a long-acting insulin analog. Semglee (insulin glargine-yfgn) is the first interchangeable biosimilar product approved in the U.S. for the treatment of diabetes. Approval of these insulin products can provide patients with additional safe, high-quality and potentially cost-effective options for treating diabetes.

“This is a momentous day for people who rely daily on insulin for treatment of diabetes, as biosimilar and interchangeable biosimilar products have the potential to greatly reduce health care costs,” said Acting FDA Commissioner Janet Woodcock, M.D. “Today’s approval of the first interchangeable biosimilar product furthers FDA’s longstanding commitment to support a competitive marketplace for biological products and ultimately empowers patients by helping to increase access to safe, effective and high-quality medications at potentially lower cost.”

Biological products include medications for treating many serious illnesses and chronic health conditions, including diabetes. 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). This means you can expect the same safety and effectiveness from the biosimilar as you would the reference product.

An interchangeable biosimilar product may be substituted for the reference product without the intervention of the prescriber. The substitution may occur at the pharmacy, a practice commonly called “pharmacy-level substitution”—much like how generic drugs are substituted for brand name drugs, subject to state pharmacy laws, which vary by state. Biosimilar and interchangeable biosimilar products have the potential to reduce health care costs, similar to how generic drugs have reduced costs. Biosimilars marketed in the U.S. typically have launched with initial list prices 15% to 35% lower than comparative list prices of the reference products.

More than 34 million people in the U.S. today have been diagnosed with diabetes, which is a chronic (long-lasting) health condition that affects how the body stores and uses sugars and other nutrients for energy. Most food is broken down into sugar (also called glucose) and released into the bloodstream. When blood sugar levels increase, it signals the pancreas to release insulin, which acts like a key to allow blood sugar to enter the body’s cells for use as energy. With diabetes, the body doesn’t make enough insulin to keep sugar levels regulated in the normal range.

“Access to affordable insulin is critical and long-acting insulin products, like insulin glargine, play an important role in the treatment of Types 1 and 2 diabetes mellitus,” said Peter Stein, M.D., director of the Office of New Drugs in the FDA’s Center for Drug Evaluation and Research. “The FDA’s high standards for approval mean health care professionals and patients can be confident in the safety and effectiveness of an interchangeable biosimilar product, just as they would for the reference product.”

All biological products are approved only after they meet the FDA’s rigorous approval standards. The approval of Semglee (insulin glargine-yfgn) as biosimilar to, and interchangeable with Lantus (insulin glargine), is based on evidence that showed the products are highly similar and that there are no clinically meaningful differences between Semglee (insulin glargine-yfgn) and Lantus (insulin glargine) in terms of safety, purity and potency (safety and effectiveness). It also showed that Semglee (insulin glargine-yfgn) can be expected to produce the same clinical result as Lantus (insulin glargine) in any given patient and that the risks in terms of safety or diminished efficacy of switching between Semglee (insulin glargine-yfgn) and Lantus (insulin glargine) is not greater than the risk of using Lantus (insulin glargine) without such switching.

Semglee (insulin glargine-yfgn), offered in 10 mL vials and 3 mL prefilled pens, is administered subcutaneously once daily. Dosing of Semglee (insulin glargine-yfgn), like Lantus, should be individualized based on the patient’s needs and should not be used during episodes of hypoglycemia (low blood sugar) or in patients with hypersensitivity to insulin glargine products. Also, like Lantus, Semglee (insulin glargine-yfgn) is not recommended for treating diabetic ketoacidosis. Semglee (insulin glargine-yfgn) may cause serious side effects, including hypoglycemia (low blood sugar), severe allergic reactions, hypokalemia (low potassium in blood) and heart failure. The most common side effects associated with insulin glargine products other than hypoglycemia include edema (fluid retention), lipodystrophy (pitting at the injection site), weight gain and allergic reactions, such as injection site reactions, rash, redness, pain and severe itching.

The FDA released new materials for health care providers to enhance understanding about biosimilar and interchangeable biosimilar products, including a fact sheet about interchangeable biosimilar products.

The FDA granted approval of Semglee (insulin glargine-yfgn) to Mylan Pharmaceuticals Inc.

The FDA recently approved a new therapy, Saphnelo (anifrolumab-fnia) from AstraZeneca, as a treatment for adults with systemic lupus erythematosus (SLE) who are receiving standard therapy. It is only the third FDA approval of a lupus therapy since 2011. Saphnelo is a first-in-class, type I interferon inhibitor administered by intravenous (IV) infusion.

SLE is the most common form of lupus affecting up to 300,000 people in the US. SLE disproportionately affects women of African-American, Hispanic or Asian lineage. It is a complex autoimmune condition that can affect any organ, and people often experience debilitating symptoms, long-term organ damage and poor health-related quality of life. There is no cure for SLE.

Saphnelo offers a HCP-administered option with a novel mechanism of action. Both Saphnelo and Benlysta are administered IV every 4 weeks in adults. Benlysta IV is also approved for SLE in pediatrics ≥ 5 years of age and a SC formulation is also approved for use in adults. Several other products with varying mechanisms of action are also in phase 3 trials for SLE. A SC formulation of Saphnelo for SLE is in development.

At this time pricing for Saphnelo has not been confirmed by AZ. As a point of reference the two leading therapies for Lupus, Benlysta (SLE, Infused-SC) and Lupkynis (LN, Oral) , are available with a GoodRx coupon as follows: Benlysta for +-$4,000 monthly and Lupkynis at +-$12,000 monthly. Benlysta IV is available through limited distribution, direct-to-office. As such, it is expected that Saphnelo will also be distributed, direct-to-office, via a traditional specialty distributor &/or a SP Distributor.

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Lupus Foundation of America Celebrates FDA Approval of Saphnelo as a New Treatment for Lupus

This type I interferon receptor antagonist demonstrated early and sustained benefits for people with moderate to severe systemic lupus erythematosus.

The U.S. Food and Drug Administration (FDA) has approved Saphnelo (anifrolumab-fnia) as a treatment for adults with systemic lupus erythematosus (SLE) who are receiving standard therapy.

“After having only one therapy approved for lupus during the past 60 years, it is a cause for celebration to have two new treatment options approved in 2021 alone for this life-threatening autoimmune disease that affects an estimated 1.5 million Americans,” said Stevan W. Gibson, president and CEO, Lupus Foundation of America. “The pipeline of potential new treatments for lupus remains vibrant, and the Lupus Foundation of America continues its work to bring down barriers to lupus drug development and ensure that people with lupus will have access to the medications they need to improve their quality of life.”

Dr. George Tsokos, a member of the Lupus Foundation of America Medical-Scientific Advisory Council, Professor of Medicine, Harvard Medical School, Chief, Division of Rheumatology and Clinical Immunology, Beth Israel Deaconess Medical Center, noted that Saphnelo is the first lupus therapy designed to inhibit type I interferons. “We have known since the 1970’s that interferons were involved with lupus. With the approval of Saphnelo, we now have one more drug that allows us to translate valuable research knowledge into clinical practice with multiple benefits for our patients with lupus.”

Saphnelo inhibits a key protein in the immune system called the IFNAR receptor that acts as a transmitter, amplifying signals from tiny messengers called type I interferons. This process activates many parts of the immune system and can trigger major inflammation. Saphnelo dampens the excessive type I interferon signature found in up to 80% of adults with lupus and as many as 90% of children with the disease.

Combined data from two large-scale phase III clinical trials showed that more patients who received Saphnelo, in addition to standard therapy, had improvement than those who received placebo in addition to standard therapy. Saphnelo showed benefits on overall lupus disease activity, skin lupus and joints and the ability to taper down steroid doses.

Shannon Lee, a Lupus Foundation of America ambassador diagnosed with lupus 11 years ago, welcomed the news of a new treatment for lupus that can cut down on the use of oral corticosteroids, which can damage the body over time. “Steroids can cause many serious and life-threatening side effects, some of which I experienced while taking them before having to stop due to the issues they caused me,” said Lee. “Having another potential treatment option like Saphnelo is extremely exciting.”

“The approval of Saphnelo is the culmination of years of clinical development,” said Susan M. Manzi, MD, MPH, Lupus Foundation of America Board Chair and Medical Director and Chair, Allegheny Health Network Medicine Institute and Director, Lupus Center of Excellence. “Arriving at this important treatment milestone required the involvement of hundreds of people with lupus from around the world, who volunteered to participate in multiple clinical trials. We are grateful for their unselfish efforts, as well as those of basic and clinical physician-scientists who work tirelessly to support lupus drug development. Without them, we could not bring new life-changing therapies to those who need them.”

The Lupus Foundation of America’s research during the 1980s contributed to the development of Saphnelo through its support of research on interferons. In more recent years, the Foundation helped by educating people with lupus about the importance of participating in clinical trials for this new therapy. As part of its efforts to continue engaging people with lupus in ongoing research, the Foundation created an online data platform, RAY™ (Research Accelerated by You). This platform enables people with lupus and caregivers to share their lupus experiences to help researchers accelerate new treatments and improve disease outcomes.

The FDA recently approved a new ORAL therapy, Bylvay (odevixibat) from Albireo Pharma, with an indication for treating pruritus in all subtypes of progressive familial intrahepatic cholestasis (PFIC). It will be the first agent approved to treat this condition. Bylvay is a non-systemic ileal bile acid transport (IBAT) inhibitor that decreases the reabsorption of bile acids into the distal portion of the small intestine.

This ultra-rare group of autosomal recessive diseases is caused by mutations in genes that produce proteins involved in biliary epithelial transport. When these proteins are deficient, high levels of sBAs accumulate in the liver and blood and lead to severe itching, nutritional imbalances, and eventual cirrhosis. Onset of signs and symptoms of PFIC typically occurs during infancy.

Therapy may slow disease progression, prevent liver damage, and relieve pruritus in some patients. Surgical procedures (e.g., biliary diversion, nasobiliary drainage) may be required to prevent sBA accumulation in the liver. Ultimately, most patients will need a liver transplant and the original disease may recur.

PFIC affects approximately 1 in 50,000 to 100,000 births. There are fewer than 600 young adults and children living with the condition in the US. Given the US birth rate, it is expected that there will be fewer than 100 potential new patients annually diagnosed with PFIC.

Albireo has confirmed that the annual cost of therapy will be $385,000. Given the very small patient base and the added challenge of weight based dosing for infants, it is very likely that access to Bylvay will be through specialty pharmacy limited distribution. In an unusual move, Albireo has announced that it will hire regional care coordinators who will investigate benefits and financial assistance, facilitate dosing changes, lab work, refill reminders, re-authorization, and more. As we know, these activities would normally be handled by the specialty pharmacy.

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FDA Approves Odevixibat, the First Drug Treatment for Progressive Familial Intrahepatic Cholestasis

July 21, 2021 — The FDA approved odevixibat (Bylvay, Albireo Pharma Inc.), for treating pruritus in all subtypes of progressive familial intrahepatic cholestasis (PFIC).

Albireo announced that it is launching odevixibat immediately to accelerate availability for the patients and families affected by PFIC, a rare and devastating disorder affecting young children that causes progressive, life-threatening liver disease.

Patients with PFIC have impaired bile flow, or cholestasis, caused by genetic mutations. The resulting bile builds up in the liver and can lead to cirrhosis and liver failure within the first 10 years of life. Pruritus is the most prominent and chronic manifestation of PFIC, often resulting in a severely diminished quality of life.

Before the approval of odevixibat, severe cases of PFIC had to be managed surgically, including biliary diversion surgery and liver transplantation. Without those interventions, most PFIC patients do not survive past 30 years of age.

“Bylvay gives us a non-surgical option and will change how we treat PFIC,” said Richard Thompson, a professor of molecular hepatology at King’s College London and principal investigator of PEDFIC 1 and PEDFIC 2, the two key trials that led to the drug’s approval. “With this approval, my colleagues and I now have the opportunity to revisit how PFIC patients are being managed and we are hopeful for better outcomes for these children.”

PEDFIC 1 and PEDFIC 2 are the largest global phase 3 trials ever conducted in PFIC, according to an Albireo press release. In PEDFIC 1, a randomized, double-blind, placebo-controlled study, odevixibat met both its pruritus (P=0.004) and serum bile acid (P=0.003) primary end points.

PEDFIC 2, a long-term, open-label, phase 3 extension study, showed that odevixibat delivered sustained reductions in serum bile acid as well as improvements in pruritus assessments, growth and other markers of liver function in patients treated up to 48 weeks.

Across both studies, odevixibat was well tolerated, with diarrhea/frequent stools being the most common treatment-related gastrointestinal adverse events. (In PEDFIC 1, diarrhea/frequent bowel movements occurred in 9.5% of treated patients vs. 5.0% of placebo patients.) There were no serious treatment-related adverse events in either study.

Emily Ventura, the leader of the PFIC Advocacy and Resource Network (www.pfic.org), welcomed the approval of odevixibat. “Parents [now] may find hope in having a less invasive treatment option available,” said Ms. Ventura, who is a mother to a PFIC patient. “As a community, we experience extreme challenges and diminished quality of life for children and families with PFIC. Managing the symptoms can be extremely difficult—the burden is unimaginable with our kids suffering physically, emotionally and developmentally.”

The recommended dosage of odevixibat is 40 mcg/kg once daily in the morning with a meal. If there is an inadequate clinical response after three months, the dosage may be increased in 40-mcg/kg increments up to 120 mcg/kg once daily, not to exceed a total daily dose of 6 mg.

Ready for Odevixibat Launch
Odevixibat is expected to be packaged and shipped within the coming days, Albireo announced. With the immediate launch, the company said it is ready “with a focus on access and reimbursement, sales promotion, and patient support.” To support payor decision making, the company said it is submitting PEDFIC phase 3 data, which include long-term data with patients on the drug for more than two years, along with a caregiver study to reflect the burden of PFIC.

Once odevixibat is prescribed, health care providers and families will have the option to use Albireo Assist, which is a customized patient support program built with input from patient advocates. The program features dedicated, U.S.-based regional care coordinators employed by Albireo who will investigate benefits and review financial assistance options to help ensure optimal patient access. They will also proactively assist with facilitating dosing changes, lab work, refill reminders, reauthorization and other activities, the company noted.

One of my favorite topics…. Hospitals and health systems opening their own specialty pharmacies (HSSPs). We’ve written about this growing trend for several years, but much of the detail behind this trend was anecdotal. For perhaps the first time, the article below details findings from an American Society of Health-System Pharmacists (ASHP) survey, with more than 100 HSSP providers responding. The insights are enlightening.

The lead finding is that these HSSPs really only differentiate themselves in just two areas… “partly to integration with specialty clinics and hospital providers and access to electronic health records.” That’s it. One has to wonder what happens with non-hospital staff provider integration.

Yes, there is no doubt that the ability to directly interface with the hospital’s staff clinical team as well as the ability to access real-time patient clinical status via EMRs could benefit patient care management . However, those features fade quickly once a patient is release from the hospital. Unless the pharmacy captures patient reported health info ongoing, a traditional SP with a tight patient follow up model should be able to react faster than the HSSP.

We were not overly surprised to see that average volume at the HSSPs averages only 45,000 specialty scripts annually….. with gross revenues of less than $100 million. That works out to an average script cost of only $2200, quite low for an SP script these days.

74% of the respondents said that their SP was opened for six years or less, consistent with what we’ve previously reported.

One big weakness for HSSPs is limited geographical access…. most hold five or fewer state licenses with one-third holding only one. Only a handful have pursued national license access.

Not surprisingly, almost all HSSPs target self-administered drugs. However, only 30% also support certain specialty drugs under the medical benefit. That is not surprising as the legacy hospital pharmacy likely wants to continue to own that piece of the business.

The survey confirmed access to limited distribution drugs – the big competitive issue.
HSSPs have had only moderate success in gaining access to limited distribution (LD) drugs.
38% said they were successful with 76% to 100% of the LD drugs they pursued,
38% were successful with 51% to 75% and,
25% were successful with less than 50%.
The primary reasons cited for lack of access…..
82% Pharmaceutical manufacturer refusal to engage
72% Frozen out or blocked by payors.

All that being said, HSSPs are making inroads and are well on their way to steal market share from independent SPs.

Read the full article for more insights from the survey

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ASHP Releases Inaugural Survey of Health-System Specialty Pharmacies

By Gina Shaw
Health-system specialty pharmacies (HSSPs) employ a practice model that exceeds industry standards, thanks partly to integration with specialty clinics and providers and access to electronic health records. The top challenge facing HSSPs is access to payor networks, which limits the health system’s ability to deliver an exceptional level of service and coordinated care to all health-system patients.

Those are several of the key findings from ASHP’s inaugural National Survey of Health-System Specialty Pharmacy Practice, presented at its Specialty Pharmacy Conference on July 14, 2021.

Surveys were sent to 230 contacts at 206 different health systems with known specialty pharmacies, and 114 unique organizations completed the survey, for a 53% response rate. The survey included 99 questions over eight domains, including demographics, workforce issues, operations, payor access and financial management, patient care services, quality and outcomes, staff activities, and the future of specialty pharmacy.

Most HSSPs dispense fewer than 45,000 specialty prescriptions per year and have an annual gross revenue of less than $100 million, the survey found. Most of these specialty pharmacies are relatively new, with 73.8% of survey respondents saying their organization has offered specialty pharmacy services for six years or less.

“Based on our findings, we can say that most HSSPs can be considered small to midsized. We have a lot of work to do and are still growing,” said JoAnn Stubbings, BSPharm, a clinical associate professor emerita and the former associate director of specialty pharmacy at the University of Illinois-Chicago College of Pharmacy, who served on the advisory committee for the development of the survey.

“We learned from this survey that HSSPs are primarily regional in their approach,” said Craig Pedersen, PhD, RPh, a pharmacy manager at Virginia Mason Medical Center, in Seattle, who conducted survey development and analysis. “The majority have five state licenses or fewer; slightly over one-third only have one, although some have many—even up to 50 if they want to serve all 50 states. But most are like my health system: We are located in Washington and have licensees in Alaska, Arizona, California and Oregon, states that are somewhat proximal to us. But we’re not reaching into the Midwest or the East because those aren’t patients our medical center serves.”

Some of the survey’s other takeaways:
Integration. The HSSP practice model is integrated into specialty clinics, with 64.9% of respondents reporting that they have HSSP pharmacists dedicated to specific clinics and involved in treatment decisions and drug therapy selection prior to prescriptions being written. “This finding is consistent across specialty pharmacies regardless of size,” said Ms. Stubbings, a member of the Pharmacy Practice News editorial advisory board. “This upstream involvement of HSSPs allows for appropriate drug selection before PA [prior authorization] is submitted, as well as management of safety parameters, leading to faster medication access and better patient outcomes.”

Pharmacy versus medical benefit. The HSSP business model is focused on self-administered (96.2%) and clinic-administered medications (80.2%) under the pharmacy benefit. Only a small number of HSSPs provide self-administered medications (31.1%) or clinic-administered medications (22.6%) under the medical benefit. “What I’ve seen in most health systems is that the medical benefit is typically managed by another area of the pharmacy enterprise, but that is changing as we speak,” Ms. Stubbings said. “There is significant overlap, and we are starting to see HSSPs building infusion suites and enter home infusion, and this is becoming increasingly important. We hope to identify these trends in a future survey.”

Access still a challenge. HSSPs have had only moderate success in gaining access to limited distribution (LD) drugs, with 37.7% of respondents reporting they were successful with 76% to 100% of the LD drugs they sought access to, while another 37.7% were successful with 51% to 75% and 24.5% were successful with less than 50%. The primary reasons cited for lack of access included “pharmaceutical manufacturer refusal to engage” (82.1%) and “frozen out or blocked by payors” (71.7%).

A wide range of services offered. HSSPs offer a variety of services, with more than 90% stating that they offer PA support, side effect identification and management, medication refill reminders, copay assistance programs, and adherence support.

Leading conditions. The most common therapeutic categories served were inflammatory conditions and hematology/oncology (both 92.4%), hepatology/hepatitis C (85.7%), neurology/multiple sclerosis (78.1%) and infectious disease/HIV (70.5%). More than half of all respondents also indicated that their HSSPs provided care in cardiology, endocrinology, cystic fibrosis, respiratory/pulmonary arterial hypertension and solid-organ transplant.

340B and shrinking reimbursements. HSSPs see access to payor networks (82.9%), 340B Drug Pricing Program changes (42.9%) and shrinking reimbursement from payors (40%) as their top challenges, with new populations to serve and new therapeutic categories rated as leading opportunities for growth.

“HSSPs are responding to these challenges by exploring new payment methods, new models such as value-based care and further integration of services,” Ms. Stubbings said. “Overall, I am optimistic about some of the changes taking place in the marketplace that could positively impact our model. Some are legislative, some market-based, some based on our advocacy. Provider status is one change that is getting closer, after all these years, at the state and federal level, and could offer new opportunities for clinic-based pharmacists to be recognized and bill for their services. Some states are banning white bagging, and we are also seeing legislation proposed at federal level on DIR [direct and indirect remuneration] reform.”

A Working Definition
The survey authors proposed the following definition of an HSSP that is informed by the results of the survey: “The health-system specialty pharmacy is an integrated, advanced practice model of care that incorporates specialty medication use management across the continuum of care.” Ms. Stubbings noted that “we intend for you to use this definition in describing and justifying our model as leaders in this movement. We have a powerful story to tell, and we are in the position to define specialty pharmacy and not be defined by payors or accrediting organizations.”

By Gina Shaw, Pharmacy Practice News

The FDA recently approved a new ORAL therapy, Rezurock (belumosudil) from Kadmon Pharmaceuticals, with an indication for adult and pediatric patients with chronic graft-versus-host disease (chronic GVHD).

There are about 14,000 patients with GVHD in the U.S. each year, and between 7,000 and 10,000 of them need systemic therapy. Roughly 60% of those patients have already tried and failed on two or more prior therapies.

Kadmon plans to launch Rezurock in late August at a monthly ‘bargain’ price of $15,500 per 30-count bottle….. that’s $186,000 annually.

Given the relatively small US patient population and cost, Kadmon has decided to launch this new therapy into limited distribution. Onco360 has announced that it will be a limited distribution partner.

Click here for patient information

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FDA approves Belumosudil for chronic graft-versus-host disease

On July 16, 2021, the Food and Drug Administration approved belumosudil (Rezurock, Kadmon Pharmaceuticals, LLC), a kinase inhibitor, for adult and pediatric patients 12 years and older with chronic graft-versus-host disease (chronic GVHD) after failure of at least two prior lines of systemic therapy.

Efficacy was evaluated in KD025-213 (NCT03640481), a randomized, open-label, multicenter dose-ranging trial that included 65 patients with chronic GVHD who were treated with belumosudil 200 mg taken orally once daily.

The main efficacy outcome measure was overall response rate (ORR) through Cycle 7 Day 1 where overall response included complete response (CR) or partial response (PR) according to the 2014 criteria of the NIH Consensus Development Project on Clinical Trials in Chronic Graft-versus-Host Disease. The ORR was 75% (95% CI: 63, 85); 6% of patients achieved a CR, and 69% achieved a PR. The median time to first response was 1.8 months (95% CI: 1.0, 1.9). The median duration of response, calculated from first response to progression, death, or new systemic therapies for chronic GVHD, was 1.9 months (95% CI: 1.2, 2.9). In patients who achieved response, no death or new systemic therapy initiation occurred in 62% (95% CI: 46, 74) of patients for at least 12 months since response.

The most common adverse reactions (≥ 20%), including laboratory abnormalities, were infections, asthenia, nausea, diarrhea, dyspnea, cough, edema, hemorrhage, abdominal pain, musculoskeletal pain, headache, phosphate decreased, gamma glutamyl transferase increased, lymphocytes decreased, and hypertension.

The recommended dosage of belumosudil is 200 mg taken orally once daily with food.

This review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence. Project Orbis provides a framework for concurrent submission and review of oncology drugs among international partners. For this review, FDA collaborated with Australia’s Therapeutic Goods Administration, Health Canada, Switzerland’s Swissmedic, and the United Kingdom’s Medicines and Healthcare products Regulatory Agency.

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 6 weeks ahead of the FDA goal date.

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

We’ve often written about the legal pitfalls that specialty pharmacies may encounter that end up costing them financially (HIPAA violations are top of mind). Today we take a more positive tack. The article below is an insightful review of a number of tactics that can navigate around some of those pitfalls.

Written by an attorney with the rights of specialty pharmacies in mind, we are alerted to options available to address several of the most irksome issues faced by specialty pharmacies today (some have been around for years).

Here is a short list to whet your appetite to read the full article:
How does my specialty pharmacy navigate through Network Access / Payer & PBM Obstacles / and Any Willing Provider Laws?
What are the emerging challenges related to White and Brown Bagging?
Can I do employ Fair Pharmacy Audit Laws/ and what are the legal implications related to Clawbacks?
What Prompt Payment Laws can safeguard my reimbursements?

These won’t cure all that ails specialty pharmacies in today’s complicated market, but it may help put out some of the fires.

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Legal Tools to Protect Specialty Pharmacies

JULY 16, 2021 — In today’s complicated pharmacy benefits landscape, with increasing dominance by a collection of vertically integrated payor and pharmacy benefit manager (PBM) networks, hospital and health-system specialty pharmacies have some legal tools that they can use to address challenges, such as network access, mandatory white-bagging policies and payor activities in the 340B space.

That was the message from Jesse Mr. Dresser, Esq, an attorney with the health care law firm Frier Levitt LLC, in a session on legal conundrums in specialty pharmacy held during the 2021 ASHP Specialty Pharmacy Conference, held virtually.

Mr. Dresser advised specialty pharmacies that no matter the issue—compliance, network admission, site-of-care policies or reimbursement—it’s important to start with the patient and their type of plan. “That will dictate what laws and rules apply and what your rights and obligations are,” he said.

Types of specialty pharmacy networks

1. Closed or exclusive. “These are the ones where the sponsors are not allowing anyone in except their own wholly owned specialty pharmacy,” Mr. Dresser said. “We typically see these arrangements in the employer-sponsored commercial market. Large plan sponsors with thousands or hundreds of thousands of employees, like Pepsi-Cola, typically self-insure rather than spend an extra 10% to 15% on premiums on behalf of their employees, but they will still contract with an insurance company to administer their claims and PBMs to administer their pharmacy benefits. In this context, state laws don’t really apply and federal rules like those involving Medicare or Medicaid [also] don’t really apply.” Plans like these, he noted, are subject only to the Employee Retirement Income Security Act (ERISA), which is silent on what pharmacies must be in a network.
2. “Open,” but with heightened admission criteria. Such criteria include requirements for multiple forms of accreditation or licensure in all 50 states.
3. Truly open specialty pharmacy networks. “These are typically found in Medicare networks, where there is a robust federal Any Willing Provider law and a prohibition on payors from limiting who can be in the network,” Mr. Dresser said.

White and Brown Bagging
The growing trend among PBMs to move claims processing from the medication side to the pharmacy side, requiring more white and brown bagging—a hot topic throughout the meeting—has had a major impact on hospital infusion, Mr. Dresser said. “About this time last year, several large payors took virtually identical steps to begin to require that in-office infused medications be filled at their wholly owned specialty pharmacies, and placing limitations, or removing the ability altogether, on providers’ ability to source and seek reimbursement for medications administered in their facilities.”

There are laws being proposed in some states that would make mandatory white bagging illegal, but what can hospital and health-system specialty pharmacies do in the meantime?

“Depending on the type of plan involved, you might be able to deploy Any Willing Provider,” he said. “All 50 states and the District of Columbia, through Medicare Part D, are subject to the federal Any Willing Provider law, meaning that any willing pharmacy able to participate in a network’s terms and conditions has to be allowed in. The law is fairly robust, and its guidance requires that those terms and conditions have to be reasonable and relevant. It has been used successfully to protect not only network access, but fair and appropriate reimbursement for specialty pharmacies to participate in Medicare Part D programs.”

There also are some state-based Any Willing Provider laws as well as state laws banning mandatory mail-order pharmacy. “About 33 states have some level of this kind of protection,” Mr. Dresser said. “I would encourage anyone facing any kind of exclusion to figure out what type of network the patients you are not being able to fill for are in, and then see if you can use some of these legal tools to your advantage.”

Other legal tools available to specialty pharmacies include:
Fair pharmacy audit laws.
“These laws apply at the state level, typically in the context of commercial insurance and not necessarily ERISA or Medicare,” Mr. Dresser said. “They provide time limits on PBM audits, as well as audit appeal procedures. They often limit the number of prescriptions that can be looked at in a given audit, and helpfully, prohibit recoupment for clerical errors or things that can be ‘cured.’”

Prompt payment laws
“These include look-back periods limiting PBM audits,” Mr. Dresser explained. “Florida, for example, says you can’t go back more than 30 months in terms of a repayment demand. They also prohibit PBMs from unilaterally offsetting claims to recoup on audits, saying, ‘You owe us $100,000, and we’re going to recoup it immediately. You can appeal but we’re going to start now.’ If you’re facing an audit and potential recoupment, this is a good tool to have in your arsenal.

Mr. Dresser also spotlighted recent actions by PBMs in the 340B space. “They are trying to retain the spread between the costs of the 340B drug and the PBM reimbursement,” he said. “They send out notices to pharmacies requiring them to submit 340B claims with a submission clarification code to signal to the PBM that it is a 340B claim.”

This typically involves the use of a Submission Clarification Code of “20” in the NCPDP Field 420-DK. If a claim is 340B, the PBM then reimburses the pharmacy at a lower rate, for example, average wholesale price (AWP) – 30%, compared with a rate of AWP – 15% for non-340B claims.

“Essentially, PBMs are looking to usurp that savings for themselves,” Mr. Dresser said. “This is not necessarily limited to Medicaid programs managed by PBM; it could include commercial plans and often does. They are also using third-party administrators to get access to this information. Some PBMs own their own third-party administrators, so they might have the ability to make the determination, even if the pharmacy didn’t submit the clarification code at the point of sale.”

Mr. Dresser noted that there has been some success in pushing back against these efforts. “The tides have turned a bit in state legislation,” he said. “Some states, including most recently Ohio, have passed laws prohibiting PBMs from differentiating 340B and non-340B pricing. So, if you are facing mandates from a PBM that you use the clarification code, or otherwise encountering 340B-specific pricing, I encourage you to speak to counsel who can guide you on some of the recent tools that are available.”

Click Here to access the article in Specialty Pharmacy Continuum

Earlier this month the FDA approved a ‘new’ combination therapy, Darzalex Faspro (daratumumab and hyaluronidase-fihj), from Janssen Biotech. Darzalex was originally approved in 2015 with an indication for multiple myeloma. The combo therapy is ‘new and improved’ for the same indication.

Darzalex Faspro is a subcutaneous combination injection (over 5 minutes) and is used together with bortezomib, melphalan, and prednisone to treat newly diagnosed multiple myeloma (a type of bone marrow cancer) in patients who cannot receive autologous stem cell transplant (transplant that uses their own stem cells).

Dosing Schedule:
Once weekly Weeks 1-8, Once every 2 weeks from Weeks 9 to 24, Once every 4 weeks starting with Week 25 — until disease progression or unacceptable toxicity (see prescribing information for full details).

The standard dose schedule requires 23 doses over the first 52 weeks on therapy. Published price for the 15ml dose is $8296. The annual cost tips in at $190,800.

The ‘improved’ formulation is a lot more expensive than the early version Darzalex as well as its leading competitors, Pomalyst at$147,000 and Kyprolis at $114,000 per year.

Janssen did not announce details for distribution. Patients may be burdened with frequent office visits in the early stage of therapy, but that shifts to once monthly after the first nine weeks. As such, it is likely that patients will receive therapy in the office vs. home injection. That will diminish the need for a specialty pharmacy distribution model, but Janssen is very prone to SP limited distribution. Time will tell.

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FDA approves daratumumab and hyaluronidase-fihj with pomalidomide and dexamethasone for multiple myeloma

On July 9, 2021, the Food and Drug Administration approved daratumumab and hyaluronidase-fihj (Darzalex Faspro, Janssen Biotech, Inc.) in combination with pomalidomide and dexamethasone for adult patients with multiple myeloma who have received at least one prior line of therapy including lenalidomide and a proteasome inhibitor.

Efficacy was evaluated in APOLLO (NCT03180736), an open-label, active-controlled trial with 304 patients randomized (1:1) to Darzalex Faspro with pomalidomide and dexamethasone (Pd) vs Pd alone.

The main efficacy outcome measure was progression-free survival (PFS). The median PFS was 12.4 months in the Darzalex Faspro-Pd treatment group and 6.9 months in the Pd treatment group (HR 0.63; 95% CI: 0.47, 0.85; p=0.0018), representing a 37% reduction in the risk of disease progression or death for patients treated with Darzalex Faspro-Pd versus Pd.

The most common adverse reactions (≥20%) in patients with multiple myeloma who received Darzalex Faspro-Pd are fatigue, pneumonia, upper respiratory tract infection, and diarrhea.

The recommended dosage of Darzalex Faspro is 1,800 mg/30,000 units (1,800 mg daratumumab and 30,000 units hyaluronidase) administered subcutaneously into the abdomen over approximately 3 to 5 minutes according to the recommended schedule.

View full prescribing information for Darzalex Faspro.

The FDA approved this application 2 months ahead of the FDA goal date.

It’s about time.
It has been quite a while since we’ve heard about a specialty pharmacy acquisition. Over recent years there was a race to the top to acquire the remaining ‘gems’ that were primed for acquisition. Suddenly that well ran dry.

The news detailed below isn’t quite on par with those earlier big acquisitions. Two ‘relatively’ small companies will result in BioMatrix growing again. They have had several other small add-ons to bolster size and scale. The most recent acquisition of InfuCare will open new therapeutic avenues for BioMatrix, especially with infusion services. Those with any hands-on experience in the infusion market know that running an infusion shop is much more complicated than traditional (pick-pack-ship-service) specialty pharmacy.

So, turn back the clock quite a few years to when specialty pharmacies grew both organically and by scooping up relatively small organizations especially if they were acretive. It would not be surprising, therefore, to see BioMatrix emerge as a much larger organization should it continue to follow that strategy and become a gem in its own right.

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BioMatrix Specialty Pharmacy Acquires InfuCare Home Infusion Pharmacy

PLANTATION, Fla., July 6, 2021 /PRNewswire/ — BioMatrix Specialty Pharmacy announced today the acquisition of InfuCare LTD, in Tyler, Texas. This infusion focused specialty pharmacy adds a brick-and-mortar location in a key geographic area, expands the organization’s national footprint, and provides access to additional covered lives in one of the nation’s largest states. The acquisition is the latest of several transactions that have positioned BioMatrix as one of the largest privately held specialty pharmacies in the United States.

The staff at InfuCare have extensive experience in key therapeutic categories for BioMatrix. The organization is well versed in chronic, complex conditions requiring home infusion including IVIG, anti-infectives, parenteral nutrition and more. The organization has been recognized for providing exemplary home infusion and specialty pharmacy services in Texas. InfuCare is accredited by the National Association of Boards of Pharmacy.

BioMatrix CEO Nick Karalis commented, “We are pleased to welcome the InfuCare team as part of the BioMatrix family. A Texas pharmacy location is part of our strategic plan to expand our infusion services and increase access to covered lives. With complimentary core competencies and shared values of exceptional patient care, together we will improve health and shape a more promising future for the patients we serve,” Karalis said.

“The leadership and staff at InfuCare are delighted to begin our partnership with BioMatrix,” stated Gary Wyatt, Partner of InfuCare, LTD. “Our goal over the last 21 years has been to provide an exceptional and personal level of infusion care to our patients in the east Texas region. Having interacted with BioMatrix leadership over the last several months, we are confident that our focus and theirs will be complementary and will lead to an even greater level of care for both infusion and specialty patients in our region and beyond. We are excited to join such a highly respected company and look forward to a long and impactful relationship with BioMatrix.”

Additional information on BioMatrix and InfuCare can be found at www.biomatrixsprx.com and https://infucare.net

About BioMatrix Specialty Pharmacy
BioMatrix Specialty Pharmacy, an Inc. 5000 company, offers comprehensive, nationwide specialty pharmacy services and digital health technology solutions for patients with chronic, difficult to treat conditions. Our commitment to every patient is to provide individualized pharmacy services, timely access to care, and focused education and support. We offer a tailored approach for a wide range of therapeutic categories, improving health and empowering patients to experience a higher quality of life.

The public relations battle surrounding White Bagging is heating up.
Who’s on each side?
Depends on who you ask….!
On one side, hospitals are claiming that their very existence is being threatened due to White Bagging policies.
On the other side are the PBMs and the payers.
But, is there yet a third side? We will get to that.

First, what is White Bagging?
As defined by the hospitals in the article link below…..
White Bagging consists of “policies that require hospitals administering certain high-cost medications in an outpatient setting [actually inpatient as well] to receive those medications from third parties contracted with the health plan, instead of providing those medications directly from the hospital pharmacy inventories.” Increasingly, those third parties are specialty pharmacies acting as specialty distributors, under a distributor’s license (no prescription required) or as the pharmacy (with a prescription).

What’s at stake? Money and control.
The number of rare therapies approved over the past two years has mushroomed. Most of the therapies have been $HUGE$ dollar meds that are hospital administered, often at leading national medical centers. These providers are very concerned about money and control. If they can buy directly and bill under often outdated, traditional contract terms (which never envisioned meds costing in the hundreds of thousands of dollars for a one-time infusion and may still pay them at U&C rates) then chances are they will realize a larger Net profit. If a specialty pharmacy is inserted into that scenario, they may not be able to realize any profit if the SP directly bills the PBM as a pharmacy claim or payer via a medical claim.

It is no surprise that the hospitals are now upping the ante and turning to the courts and even state legislatures, where they may have significant influence, to seek relief and regain control.

But…. who is the third party to this battle? Manufacturers.
The pharmaceutical manufacturers proliferated the “White Bagging” war by selecting one or a handful of specialty pharmacies to be their preferred distributor(s) for these new infused therapies. Traditional distributors can’t perform all the functions of a specialty pharmacy as they are prohibited from making direct patient contact. Manufactures realized that SPs can help them out with managing patient care, ensuring compliance, tracking outcomes for FDA reporting, in addition to all the traditional SP patient stuff such as clearing prior authorizations and financial assistance. When payers and PBMs realized what was happening they got creative and saw a way to insert utilization review and tighter benefits management into the mix, things that SPs are also adept at juggling.

It is also noteworthy to mention that SPs are also inventorying their meds for distribution. Traditional distributors charge manufacturers a percentage for their limited services…. better to pay an SP for the full-service package vs. just “park, pack, and ship.”

So, it is surprising that the hospital lobby is focusing its ire only on the payers and PBMs, allowing the manufacturers off the hook. Then again, there are a lot of big research dollars that flow from manufacturers to the same big medical centers in new drug trials….. just sayin’.

So, read the article below for a good update on what the leading payers are doing related to “White Bagging”.

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Payers Attempt to Impose “White-Bagging” Policies on Hospitals

CLICK HERE TO READ THE FULL ARTICLE