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The number of biosimilars launching in 2023 will spike as legal challenges around patent infringement are finally expiring. That will only exacerbate the problem of biosim adoption that has been inherent in the marketplace since biosims started to be approved in 2015. In short, biosimilars are still struggling to gain traction….. and a recent survey says that communications are at the heart of the problem.

Some of the survey highlights include:

• Oncologists felt that switching decisions were typically initiated by pharmacies (29.0%) or hospital/treatment centers (19.4%). Note, there is only a handful of biosimilars with ‘an interchangeable’ designation. 
• Patients (55.2%) said they were given an option to switch to a biosimilar. 63.9% went through with the switch / 8.6% declined to switch.
• Patients reported (40.8%) that they were never notified of a payer driven biosimilar switch, and only 26.4% said their oncologist or physician briefed them.
• The top three most common reasons for switching, as reported by oncologists

   – Payer requirements (23.5%), 

   – Biosimilars were considered identical (14.1%), and 

   – Hospitals wanted to save money (12.9%).

• Only 9.3% of patients felt that the payers could be trusted to make the right decisions about switching! It is also noteworthy that only 12.1% of oncologists felt similarly!
• 35.3% of patients felt they had been given the opportunity to ask questions about biosimilars 
• Only 43.4% of patients felt that the biosimilar would be as effective in treating their cancer!
• And, only 79.4% of oncologists felt the biosimilar would be just as effective as the reference product!!!

As the researchers said, “If biosimilar acceptance is to grow, it’s going to take a great deal of work to improve the levels of trust between payers, patients, and oncologists.”

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Researchers Identify a Communications Breakdown Over Biosimilars

June 6, 2022Researchers have identified what they say is a critical lack of communication about biosimilars between patients with breast cancer and their oncologists, based on surveys conducted from 2020 to 2021.

They also said many switches from the reference product Herceptin (trastuzumab) are dictated by payers and much needs to be done to improve the levels of trust between payers, patients, and oncologists if biosimilar acceptance is to grow.

“There is a need for tailored and effective patient and oncologist information and education on trastuzumab biosimilars, along with improved health care communications regarding switching,” wrote lead author Elizabeth Lerner Papautsky, Ph.D., M.S., an assistant professor in the Department of Biomedical and Health Information Sciences at the University of Illinois at Chicago. Papautsky and her colleagues reported their results in May in the journal Breast Cancer Research and Treatment.

In two separate surveys, Papautsky and her colleagues collected responses from 143 patients with breast cancer and 33 medical oncologists. The researchers said that 63.9% of patients reported they had been switched to a trastuzumab biosimilar–most often Kanjinti (69.8%)–and of those, 40.8% reported they had been given no prior notice they would be switched.

In none of the responses did oncologists ever say that the switch to a biosimilar was initiated by them. It was most commonly the case that the switch was mandated by the payer (45.2%). Oncologists were much more likely than patients to report satisfaction with the way biosimilar information had been communicated.

“The discrepancy between patient-reported experiences and oncologists’ perceptions of the patient experience suggests a lack of adequate information that may be a challenge not only to the uptake of trastuzumab biosimilars, but to the patient oncologist relationship,” Papautsky and her fellow researchers wrote.

The authors expressed strong concern that these communication gaps be rectified because, they said, Herceptin is a costly drug and biosimilars represent an important opportunity to improve patient access and lower the cost of health care. Payers have latched onto the savings aspect, they said. “Literature suggests that with increasing availability of biosimilars, a variety of switching scenarios have become common across disease types.”

They cautioned that “with guidelines often being vague, the practice of switching is largely unregulated.”

Because of the above-described patient/doctor disconnect on biosimilars, many patients resorted to self-directed research to find out about these biologic alternatives, the report said.

Among patients participating in the survey, 58.1% were fully covered by private insurance and 99.3% and 91.4% were female and white, respectively. Responding oncologists were most likely to work in an urban setting (68.0%) or at an academic center or affiliate (35.3%).

Despite the communication problems, most patients (55.2%) said they were given an option to switch to a Herceptin biosimilar–it wasn’t forced on them. And of patients who responded, 63.9% went through with the switch. Papautsky said 8.6% declined to switch.

However, lack of prior notification about switching was a problem, patients reported (40.8%); and just 26.4% said their treating oncologist or physician briefed them beforehand about biosimilars. Smaller percentages of patients said they got that type of information instead from advanced practice practitioners (5.7%), chemotherapy nurses (15.5%), or others.

Oncologists said that if not dictated by payers, switching decisions were typically initiated by pharmacies (29.0%) or hospital/treatment center administrations (19.4%).

The three most common reasons for switching, as reported by oncologists, were payer requirements (23.5%), biosimilars were considered identical to Herceptin (14.1%), and hospitals wanted to save money (12.9%).

Few patients or oncologists felt that the payers could be trusted to make the right decisions about switching (9.3% vs 12.1%, respectively). Patients were less likely than oncologists to agree they had been given the opportunity to ask questions about biosimilars (35.3% vs 58.8%) or that the biosimilar would be as effective in treating the cancer (43.4% vs 79.4%).

Tony Hagen, Managed Healthcare 

The article below should be a learning moment for the specialty pharmacy industry. It is all about going digital…. but it does not get lost in techno babble. It makes the  argument that the specialty pharmacy industry is essentially just now emerging from its Paleolithic age….  yep, when the Neanderthals roamed. Heck, it is an industry that still depends heavily on the fax machine!

The article presents examples of other industries that benefitted from embracing  digital interoperability through open protocols. If you can’t define ‘digital interoperability’ then you really must read the article. Admittedly, achieving digital interoperability across the healthcare spectrum faces a host of obstacles especially when healthcare providers are reluctant to spend big bucks on the transition and competition among insurers is intense. 

The article stops short of stating the obvious….. that the specialty pharmacy industry is at a tipping point. The industry has wrung virtually the last drop of efficiency that is possible without a major breakthrough and digital interoperability seems to be a solution.

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Specialty Pharma’s Next Big Opportunity: It’s Time for Patient Access to Adopt an Open Protocol

In January 2020, the financial conglomerate Visa announced it was acquiring a relatively unknown startup, Plaid, for $5.3 billion. Corporate acquisitions like these are not uncommon, but someone at the United States Department of Justice took notice of this announcement. Visa had established a stranglehold on financial transactions. The Justice Department moved to stop the acquisition on grounds that Plaid posed “a threat to this monopoly: it has been developing an innovative new solution that would be a substitute for Visa’s online debit services.”

Plaid derives its power because of, not in spite of, its invisibility ― a power Visa and ultimately the government could not deny. When a consumer transfers funds electronically from one financial platform to another, or makes a deposit, or applies for a mortgage, there’s a decent chance Plaid is involved. It is the software that powers interoperability among various financial services brokers. It seamlessly patches together transactions that would otherwise be very complicated. Visa, which at the time reportedly held roughly 70 percent of the online debit market, had good reason to feel threatened. Plaid unearthed a way to charge merchants and consumers less for the convenience of online connectivity.

Ultimately, both parties backed out of the acquisition. Visa’s attempted $5.3 billion purchase price looked like a bargain when Plaid was valued at approximately $13.4 billion in a Series D funding round in April 2022.

A similar inflection point now faces the specialty pharmaceutical industry. Along their journey, specialty pharma patients interact with a series of siloed institutions: clinics, specialty pharmacies, copay program vendors, patient assistance programs, nurses, etc. Not unlike the financial industry, these institutions still exchange patient information via fax machines. Facilitating digital interoperability among these various stakeholders is paramount. So what lessons can be gained from the story of Plaid’s meteoric rise?

To diagnose the problem facing specialty pharma, a brief history lesson is in order. The healthcare industry earnestly embarked on its first major digital transformation in the 1990s, when the large-scale transition to electronic recordkeeping began. The industry preference for paper did not disappear overnight. Health systems were slow to digitize their filing cabinets full of charts and other patient data. The Institute of Medicine first advocated a shift from paper-based to electronic medical records in 1992, yet only 13 percent of U.S. healthcare facilities were found to have an EHR system fully implemented by 2004. Many are still making the transition.

Today, specialty drug sales represent more than half of all drug spending. This sector of the healthcare industry has both the incentive ― and the financial wherewithal ― to make the patient experience as seamless as possible. Yet in many ways, it is more backwards than the fintech industry before Plaid. When Plaid effectively forced financial institutions to take an open-protocol approach to digital interoperability, “Every bank (took) their five-year strategy on digitizing and brought it down into one or two years,” CEO Zach Perret said in an interview with Fortune magazine.

In healthcare in general, and specifically in patient services, the transition to an open-protocol approach has been slow and uneven. Open protocol, simply put, is a digital language that facilitates electronic transactions among prescribers, pharmacies, patient support vendors, data aggregators, insurers, and other stakeholders in the specialty pharmaceutical patient journey. Taking “an open protocol approach” means standardizing the open and shared application programming interfaces (APIs) within an industry, or a subsector of a larger industry. Widespread adoption of these protocols allows every stakeholder in the environment to expect a well-defined behavior when interfacing digitally with one another.

The power of the open-protocol approach has been realized across many industries. Look at the example of Twilio. In 2008, the startup launched its first API to make and receive phone calls entirely in the cloud. Now, six years following its IPO, the company is worth billions. Its suite of related tools includes platforms for data security, speech analytics, and customer relations management.

If the healthtech and pharmatech industries can harness this opportunity to standardize their most common digital tasks, integrating record-keeping and transactions among the various parties will be blazing-fast compared to today. This is the essential lesson pharmatech and healtech firms can glean from what Plaid did for the fintech industry.

If an open-protocol approach facilitates convenience, what’s the holdup? As in finance, traditional healthcare stakeholders tend not to share information very efficiently with their competitors. Interoperability has never been an explicit goal of the industry. Yet for specialty pharmaceutical patients, interacting with stakeholders in multiple silos is the norm. Routinely, these patients will need at least one transaction to obtain their prescriptions from the prescriber, then another with the office to provide their HIPAA, TCPA and hub consent, then interact with the hub regarding their coverage, then again with a specialty pharmacy regarding their shipments and out-of-pocket payment, then another to process their copay, another with a patient assistance program, another for adherence support, etc. The patient’s journey is typically long and complicated ― to say nothing of their own recovery from the condition for which they seek treatment.

You may ask: Can an open protocol be HIPAA compliant? HIPAA does not specifically prohibit using and sharing open protocol-based software. Neither does “open protocol” inherently mean “insecure.” Although the industry’s reticence toward sharing back-end software protocols is understandable, nothing is standing in the way of community problem-solving toward ensuring that any private health data transmitted via open protocols remains secure. On the contrary, an open protocol can easily enable such monitoring of the passed data, much better than fax machines and FTP transfers. For now, this reticence is making life more complicated for patients. Their patient journey typically requires engaging with discrete service providers who are often using antiquated means of communication. 

The discussion around how to integrate an open-protocol approach into a complicated system governed by HIPAA has been going on for years. Yet many key players in the industry are still on step zero: converting their primary mode of communication from paper to digital. 

The time to cross that bridge has passed. As the case study of Plaid makes clear, the time for the industry to embrace open protocol is now.

by Yishai Knobel, CEO and co-founder of RxWare 12/01/2022 

This week the FDA approved a new ORAL specialty drug, Skyclarys (omaveloxolone) from Reata Pharmaceutical, indicated for the treatment of Friedreich’s Ataxia in adults and adolescents aged 16 years and older. Friedreich’s Ataxia is a rare condition that affects about 5,000 patients in the United States. This oral med is administered once daily.

The company was granted orphan drug, fast track and rare pediatric disease designations from the FDA. With this approval, the agency granted a rare pediatric disease priority review voucher.

Friedreich’s ataxia causes progressive damage to the spinal cord, peripheral nerves, and the brain, resulting in uncoordinated muscle movement, poor balance, difficulty walking, changes in speech and swallowing, and a shortened lifespan. The condition can also cause heart disease. This disease tends to develop in children and teenagers and gradually worsens over time.

Reata confirmed that he annual WAC cost for Skyclarys will be $370,000.

Given its cost and the relatively small patient base, it is likely that Skyclarys will launch through limited distribution.

CLICK HERE to access prescribing information 

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FDA approves first treatment for Friedreich’s ataxia

FDA has approved Skyclarys (omaveloxolone) as the first treatment for Friedreich’s ataxia, a rare, inherited, degenerative disease that damages the nervous system, characterized by impaired coordination and walking.

Patients take Skyclarys capsules orally without food once a day at a recommended dosage of 150 mg.

The efficacy and safety of Skyclarys to treat Friedreich’s ataxia was evaluated in a 48-week randomized, placebo-controlled, and double-blind study [Study 1 (NCT02255435)] and an open-label extension.

Study 1 enrolled 103 individuals with Friedreich’s ataxia who received placebo (52 individuals) or Skyclarys 150 mg (51 individuals) for 48 weeks. Of the research participants, 53% were male, 97% were white, and the mean age was 24 years at study entry. Nine (18%) patients were younger than age 18.

The primary objective was to evaluate the change in the modified Friedreich’s Ataxia Rating Scale (mFARS) score compared to placebo at week 48. The mFARS is a clinical assessment that measures disease progression, namely swallowing and speech (bulbar), upper limb coordination, lower limb coordination, and upright stability. Individuals receiving Skyclarys performed better on the mFARS than people receiving placebo.

In a post hoc analysis, individuals who continued treatment with Skyclarys in an open-label extension for up to three years performed better on the mFARS compared to a matched set of untreated patients from a natural history study.

Safety Information

The most common side effects of Skyclarys were an increase in alanine transaminase and an increase of aspartate aminotransferase, which can be signs of liver damage, headache, nausea, abdominal pain, fatigue, diarrhea and musculoskeletal pain.

The FDA recently approved a new antihemophilic factor (recombinant) for Hemophilia A, Altuviiio (Fc-VWFXTEN fusion protein-ehtl) from Sanofi. It was approved for prophylaxis, on-demand treatment of bleeding episodes, and perioperative bleed management.

Sanofi touts the new treatment as a significant improvement over other factors on the market due to its once-weekly dosing, near-normal factor levels with prophylaxis, and overall reduced bleed levels during episodes. By comparison, other factor products indicated for prophylaxis require dosing every two days due to shorter half-life.

Sanofi will announce Altuviiio’s price when it becomes commercially available in the spring. It commented that it will likely be comparable to Eloctate which is now available at $12 per unit or a cost of $12,000 for a patient requiring 1,000 IUs weekly or, $625,000 annually. Some patients may require dosing as high as 3,000 IUs weekly resulting in an annual upper end cost of $1.8 mm.

Sanofi did not comment on how Altuviiio will be distributed. As virtually all other factor products have launched through limited distribution it is expected that Altuviiio will similarly follow suit.

CLICK HERE for prescribing information

The article below speaks to a variety of inefficiencies in Specialty Pharmacy Information Technology.

What an opportunity to create a new acronym…. SPIT.

The article makes the case that SPIT has followed the market rather than leading as IT has done in many other industries. In short, it has been slow on the uptake. 

The article makes the case that dispensing software is at the heart of the problem.  That’s true to a degree as many pharmacies are averse to replacing their dispensing platforms due to ‘transition angina’ and cost. 

But the article misses pointing out that real SPIT has blossomed and is now available from a number of developers. These very sophisticated platforms enable SPs to provide highly tailored patient journey management that is fully integrated with dispensing functionality. Customizable modules are available by disease state and even by the drug being dispensed. Of key importance, these platforms can also generate the kind of highly detailed reporting that makes pharma partners salivate. Now that’s impressive SPIT.

But is SPIT growing? 

The number of potential SP customers that can afford state-of-the-art SPIT platforms are microscopic compared to the number of retail pharmacies that get by using only traditional dispensing platforms. For an industry to grow more customers are needed and a couple of developments are making this possible. First, hospitals / health systems have more than doubled the potential customer base seeking SPIT in the last few years. They also have the funding to afford the best SPIT. Secondly, creative  vendors have developed software-as-a-service options that offer even retail pharmacies access to affordable online SPIT programs.

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Information Technology Inefficiencies Within Specialty Pharmacy

Although specialty pharmacy has mirrored health care IT’s explosive growth and market innovation over the past several decades, the industry has not advanced significantly in dispensing software.

Information technology (IT) has revolutionized the health care industry, allowing pharmacy services to expand dramatically over the past 30 years. Virtual dispensing platforms have improved efficiency and patient outcomes; automated prescription dispensing robots cut the time to fill scripts and improve accuracy of fills; pharmacy applications connect patients with their health care in ways that improve treatment engagement and adherence—the list can go on and on.

The specialty pharmacy market has experienced a similar transformation over the past few decades, beginning from a single pharmacy supporting dispensing………….

CLICK HERE to read the full article

Just about every specialty pharmacy has already obtained specialty pharmacy accreditation if they want to be a serious player in the SP market. The most serious SPs have not one, but two, or even three SP accreditations….. and even niche distinctions such as in rare diseases.

Accreditation is not easy but it is cathartic in practice. Well defined standards provide targeted guidelines for these companies to achieve, maintain, and nourish on a daily basis. 

Many SOs find it difficult to stay the course. But, even for those that may stray from the righteous path, the day of reconning will arrive when their accreditation expires, a new application is required, and the process needs to be repeated.

The several leading accrediting companies are aware of the challenges for all SPs and are working to make improvements and efficiencies on their end. The article below is a good read that details many changes that SPs can expect when they ramp up…. or resurrect…. their accreditation process.

CLICK HERE to access the full article

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Many Changes in Pharmacy Accreditation on the Horizon

New specialty pharmacy and infusion standards and a benefits-laden path for becoming certified as a center of excellence are just two noteworthy developments in accreditation and credentialling for specialty and health-system pharmacies, experts noted during two recent conferences.

URAC

On Oct. 3, URAC released Specialty Pharmacy Accreditation 5.0, the latest version of its accreditation standards for specialty pharmacy operations. In February 2023, the Accreditation Commission for Health Care (ACHC) will release the annual updates of its portfolio of standards, including specialty pharmacy and infusion pharmacy. These updates follow ASHP’s new credentialing program, the ASHP Certified Center of Excellence in Medication Use Safety and Pharmacy Practice, which it released in the fall of 2021.

Heather Bonome, PharmD, URAC’s director of pharmacy, described the process of developing version 5.0 in a session at the National Association of Specialty Pharmacy (NASP) 2022 Annual Meeting & Expo. “Our last major revision to this standard was in October of 2019, and we didn’t just update it for the sake of updating,” she said. “We have standards that work that specialty pharmacies are used to, and we didn’t change those. Instead, we combed through feedback from our applicants over a three-year period about what worked and what didn’t. We also consulted all accredited organizations, various industry associations, consultants and leaders in SP. Standard by standard, we asked questions, got feedback and revised again. It was a very iterative process.”

One change that should please most applicants is this: “There has been a lot of streamlining,” Dr. Bonome said. “When you submit the application, there are about 50% fewer uploads and linking that you will need to do.”

The sections of specialty pharmacy accreditation 5.0 now include:

• Foundational focus areas
• Pharmacy operations
• Medication distribution
• Patient services and communications
• Patient management

Dr. Bonome noted that a separate focus area has been created on medication distribution management, which is no longer a part of pharmacy operations. “We heard a lot about medication distribution in our feedback, and that it was a burden to always do ongoing testing the way it had been done in the past,” she said.

Dr. Bonome gave an example for medication distribution: To achieve full accreditation, a specialty pharmacy must demonstrate the ability to maintain distribution, even in the face of weather such as hurricanes.

In addition, rare disease management has been changed from a designation to a center of excellence certification, and mail service pharmacy accreditation also has received a new-version update. Its content mirrors that of specialty pharmacy, with the sole exception of patient management, which does not appear in mail service pharmacy accreditation.

If your specialty pharmacy has already begun getting accredited under version 4.0 of the standards, you don’t need to reconfigure your application for 5.0, Dr. Bonome stressed. “When you sign up for accreditation, you sign up under a specific version, and you can continue with the process under that version even though the new standards have come out,” she explained. “Then, when you apply for reaccreditation in three years, you will do it under the new version.”

A Deeper Dive Into URAC’s Upgrading Process

During the year that URAC spent revising its specialty pharmacy accreditation standards to the newly released version 5.0, “we really heard a lot from you that the accreditation process took too long,” Heather Bonome, PharmD, the director of pharmacy for the accrediting organization, told a group of webinar attendees in October.

“So we took a step back and asked ourselves, ‘How can we keep the vigor but shorten the review process?’“ The first move “was really to streamline the standards,” she said.

Streamlining meant reassessing every aspect of the accreditation process, she noted, from client application uploads to URAC’s months-long desktop reviews, to ensure the revised product continued to reflect specialty pharmacy’s exacting safety and quality measures.

The result of the examination was a six-month reduction in specialty pharmacy’s accreditation time line, according to Jennifer Richards, PharmD, JD, URAC’s product development principal. “We did this in several ways,” she said. For one, the number of supporting documents required in the application process “was reduced by up to 50%.” Part of the reduction came from eliminating standards that duplicated ones set forth by state boards of pharmacy, she added.

“This is what the time line looks like for the revised program,” said Dr. Bonome, pointing to a graphic in the slide presentation. “There is now a two-month period for submitting applications and four months for URAC to conduct the desktop review, validate the review and render its decision.”

Reducing the time it takes to complete the approval process isn’t new. “For the last year-plus, every new or revised program we’ve released has followed this same process,” Dr. Bonome said.

“With every revision,” she added, “we learned how to make the process better for pharmacies seeking accreditation and reaccreditation. It’s been an opportunity to make sure that the best practices of the industry are reflected in the standards so patients are getting the best high-quality care—because at the end of the day, that’s really what it’s all about.”

When to Move Up to 5.0 Among Key Questions

A question-and-answer portion of the webinar served as a forum for revealing more details about URAC’s accreditation updates. Drs. Bonome and Richards fielded the questions from attendees, of whom about 80% represented specialty pharmacies. Here is a sampling of their responses.

Q. When does an organization have to move up to the 5.0 version of the specialty pharmacy accreditation standards?

A. You need to remain compliant with your current standards until it is time to seek reaccreditation. The clock starts ticking on the new version on the day you submit your reaccreditation application.

Q. What if we were recently accredited under 4.0 and a standard in that version is no longer included in 5.0? Do we still need to comply with it?

A. Yes, because that was the version you committed to for the full three years of the accreditation cycle. There are a lot of really good, solid standards that remain the same, so we’ll look for continued compliance with them. When it comes time for reaccreditation, the program that is currently available—assuming it’s still 5.0—is what you’ll need to focus on.

Q. Do we have an option to apply for version 5.0, if we signed a contract for 4.0 within the last month or so?

A. Yes. If you recently signed a contract for 4.0 but haven’t started working on your application and desktop review process and you want to update to 5.0, you can reach out to your client relations manager and work through that with them.

Q. What crosswalks are available, and is there a 3.1 to 5.0 version?

A. Three crosswalks are currently available through AccreditNet (URAC’s client access portal). The first is the 4.0 to 5.0 crosswalk. The second one, 5.0 to 4.0, reverses the data exchange and allows people to use whichever one works for them, depending on their organization’s own internal operations. We also have a 3.1 to 4.0 crosswalk. Currently there is no 3.1 to 5.0 crosswalk, but we might consider one if there is a significant number of requests.

ACHC Updates Annually

ACHC’s 2023 updates are part of the group’s practice of releasing updates to its standards annually, said Ralph McBride, RPh, the interim program director for all ACHC pharmacy programs, during the NASP session. “[Pharmacy is] a dynamic industry that is changing rapidly, and we listen to feedback from providers as we revise our standards. We believe in a rigorous process, but in incremental steps.”

Beyond specialty pharmacy accreditation, ACHC offers other accreditations to set a pharmacy apart, including ambulatory infusion, infusion pharmacy, mail order pharmacy, and compounding pharmacy (both sterile and non-sterile). “We also have designations in HIV, oncology, and rare and orphan diseases that can add distinction to your accreditation, as well as nutritional support and hazardous drug handling,” Mr. McBride said.

ASHP Tailor-Made to Health Systems

ASHP’s new Certified Center of Excellence in Medication Use Safety and Pharmacy Practice designation differentiates high-performing hospital and health-system pharmacy departments, said Lynnae Mahaney, the senior director of pharmacy accreditation, in a separate interview. “No one had previously offered an accreditation or certification specifically designed for health-system pharmacy, be it for one hospital or multiple hospitals,” she noted. “Pharmacists in the health-system world have been talking about this for quite some time, and we are now able to offer a standard that recognizes innovative, high-quality, safe and effective hospital pharmacy services.”

The new ASHP accreditation program “enhances the credibility and the value of your pharmacy services in the eyes of healthcare leaders, administrators, payors, and ultimately policymakers and patients,” Ms. Mahaney added.

The standard is very rigorous, she said. “We are expecting best-practice excellence and preparation time can be significant, depending on the organization. A unique aspect of this process is that the evidence of quality must include data: How are you measuring yourself, and how are you verifying that you are practicing at an excellent level and continuing to improve?”

In addition, ASHP is launching an individual certification for pharmacy leaders, the Certified Pharmacy Executive Leader, which recognizes core competencies in professionalism, leading people, leading the pharmacy enterprise, and leading within and across complex healthcare systems. This designation is currently in its pilot phase with an initial cohort. Certification will be valid for seven years.

A Long Process but Worth It

Estimating that it takes between four to six months to apply for and hopefully obtain specialty pharmacy accreditation from URAC, “there are fruits of that labor,” said Maribeth Bettarelli, PharmD, the executive director for Quality & Accreditation at CVS Caremark. “Achieving accreditation truly demonstrates an organization’s commitment, leadership and initiative,” Dr. Bettarelli said during a session focused on accreditation at the Academy of Managed Care Pharmacy (AMCP) Nexus 2022 meeting, in National Harbor, Md.

Dr. Bettarelli, who has participated in many accreditation processes for URAC and other organizations, said she is grateful to be part of a team at CVS Caremark that’s solely focused on reaching accreditation goals. She recommended that organizations that are just beginning the accreditation process partner with accrediting bodies early to conduct a gap analysis, so that they are in good shape when the accreditation review occurs.

Even if a specialty pharmacy has complete access to limited distribution drugs, Dr. Bettarelli noted several benefits of accreditation, such as improved staff engagement and the development of more individualized patient care plans. “Accreditation can really elevate the effectiveness of an organization in multiple ways,” she said.

Clinical Trials….. are you ready to give even a five minute standup presentation to a group of new employees to orient them to this integral, yet not frequently considered and even less frequently discussed, step in the drug approval process?

Every specialty pharmacy should offer ongoing education on the SP model and the broader healthcare industry….. especially if employees are in a customer service role. Clinicians should have an even greater depth of orientation especially if they interact with physicians, health care organizations and manufacturers.

The attached article offers a very good overview on the topic of CLINICAL TRIALS and can be used, as is, as an employee in-service presentation. Even industry veterans might benefit from reading the article as a refresher.

So….. can you speak to each of the following points?

• What is a clinical trial (in 100 words or less)?
• What are common trial procedures?
• What are Behavioral interventions?
• What are the three phases of Medical interventions?
• What are Observational Studies?
• Who is eligible for a clinical trial?
• Are clinical trials free?
• What’s a placebo?
• Are clinical trials ethical?
• and More……

Less than 15 minutes invested in this article might be a beneficial investment of your time.

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What Are the Different Types of Clinical Trials & Other FAQs

By Simon Spichak, MSc | January 4th, 2023

Clinical trials investigate new treatments, lifestyle interventions, or risk factors for developing treatments for a disease. By becoming a participant, volunteers move forward new insights and treatments for all diseases. 

These trials and studies come in different shapes and sizes. Understanding the type of research you’re participating in will help you understand its purpose and whether it is right for you.  

Clinical trials and studies are run from research centers or universities. Each study is headed by a principal investigator who is usually a physician and researcher, along with many doctors, nurses, and other healthcare staff.

They may also be sponsored by pharmaceutical companies testing out new drugs. Descriptors for the trial provide information about how far along a drug is in its development, or whether the trial involves a placebo — a sham version of the treatment to help scientists test its effects — is involved. (We’ll take a closer look at that below.) 

In general, there are two main clinical studies and trials, with many different design variations and goals. The goals, protocols, and other information about a clinical trial or study is posted before the start of the trial on clinicaltrials.gov. 

Here’s a quick run-down.

What is a clinical trial?

In a clinical trial, the general public signs up to help scientists learn critical information about illnesses and the interventions that treat them. These interventions might be medical, surgical, or behavioral. In the Alzheimer’s research space, there is much discussion of a number of ongoing clinical trials testing whether experimental, new drugs can help slow down or stop Alzheimer’s disease progression. There are also clinical studies that look at Alzheimer’s effects on the brain, and the factors that may cause it, make it more likely, or help prevent it. 

The goal of such studies is to gather vast amounts of data that then helps researchers understand whether, for example, a particular dementia treatment is safe and effective for people living with dementia — and whether it is better than other available treatments. 

Some of the interventions that are tested in trials include:

Drugs. Currently, there are several clinical drug trials testing whether anti-amyloid drugs like lecanemab are “disease modifying.” In other words, these trials are testing whether a drug can actually change the course of the disease, as opposed to only treating its symptoms.

Medical devices. Medical device trials test devices. The devices category includes new diagnostics like Alzheimer’s blood tests which are currently being developed to provide earlier Alzheimer’s diagnosis for patients and, ultimately, better treatment and care.

Procedures

In the dementia research space, trials that test medical procedures might explore new ways of measuring cognitive function in people with Alzheimer’s and other forms of dementia. Tests that provide more accurate information about cognitive function could lead to improvements in diagnosis and treatment.

Behavioral interventions

There are also clinical trials that test certain “behavioral interventions,” like lifestyle changes, and their effects on health or illnesses. For example, a clinical behavioral intervention trial from Finland used a combination of interventions including dietary changes and exercise to examine whether these behaviors reduced participants’ risk of developing dementia. These clinical trials can last for several years.

Medical interventions

There are different phases of clinical trials. Each phase gathers information about safety and efficacy, which is then reviewed by regulating agencies. In the U.S., the agency is the Food and Drug Administration (FDA), which decides whether to approve new drugs and diagnostic tools so that they can become available to the general public. These are the different phases: 

• Phase 1. This phase is the earliest trial for any medical intervention. It is conducted in a small group of healthy participants to assess safety and, in the case of drug testing, appropriate dosage. 
• Phase 2. This is the middle phase of investigation and often the first phase where participants have a specific disease or condition. A Phase 2 trial provides information about preliminary effectiveness and dosages in a larger group of participants. 
• Phase 3. Phase 3 trials, sometimes combined with Phase 2 trials, test whether a drug works in a larger cohort of participants with a disease. Often, this pits a new intervention against the gold standard treatment for the condition or a placebo.*   

Observational studies

Observational studies are exactly what they sound like: They are very “hands-off.” The researchers do not introduce any new interventions or treatments to the participants. Instead, the researchers stand back, observe people over a long period of time, and gather data to answer questions like “Does drinking diet soda increase the risk of developing dementia?” or “Does Viagra cut dementia risk?” 

This can help explain the effects of certain interventions in the real world, sometimes providing evidence that drugs used for one indication may also reduce the risk or slow the progression of cognitive decline. If any promising drugs are found through this method, they are tested again in a more rigorous clinical trial.

Who is eligible for a clinical trial?

Each study or trial has a plan that outlines exactly what kind of participants a study is recruiting for. 

A list of necessary requirements for participating in such a trial is called the eligibility criteria. 

For example, in the case of an Alzheimer’s or dementia trial, eligibility criteria may include:

• Genetic risk factors like APOE4
• The stage of your disease and severity of the symptoms
• Presence of Alzheimer’s-associated biomarkers in your blood or brain
• Age
• Gender

There are also exclusion criteria for many clinical trials. For example, if you are taking other medications, such as blood thinners, or you have a co-occurring illness or condition, you may be excluded from participating in a clinical trial.

Are clinical trials free?

Typically, participants are reimbursed for any costs that might be incurred by participating in a clinical trial, such as the cost of procedures during the screening process, or travel to trial sites. Some clinical trials also pay compensation to the participants throughout the trial. 

However, this is not always the case. From a patient’s perspective, these trials often don’t cost them anything. Trial sponsors are typically reimbursed for procedures that are offered during the trial by Medicare if these procedures are eligible. The sponsor might still pay for the procedures so participants don’t incur additional costs.

The first stage of any trial involves a screening process which can sometimes last weeks or months, which helps trial administrators determine whether a potential participant may be eligible. Sometimes, a diagnosis is required to enter the screening process, according to Terrence Casey, director of communications and outreach at the Penn Memory Center. “It depends on the trial and the inclusion and exclusion criteria as determined by the study sponsor,” Casey told Being Patient.

Drug trials are often seeking people in a certain stage of a certain disease — for example, early-stage Alzheimer’s disease. However, Alzheimer’s and other forms of dementia are notoriously difficult to accurately diagnose. So, part of that eligibility determination depends on the confirmation of a patient’s diagnosis. This trial eligibility screening process usually doesn’t cost the patient anything. 

The bottom line: The situation may differ, from trial to trial, and sometimes, depending on diagnosis status, from patient to patient. While many trials do reimburse participants for the trial expenses, this is not always the case. Ask your trial administrator at the start of the eligibility screening process whether there will be out-of-pocket expenses for you in order to participate.

What’s a placebo?

To better understand whether drugs are effective without any bias, often, participants would be randomized to different treatment groups at the start of the trial. One group will receive a treatment while another group would receive a dummy that looks just like the treatment, which is called a “placebo” or “sham” treatment.

A placebo is a sugar-pill or other substitute given in lieu of a drug. In an Alzheimer’s drug trial, for example, all the participants in the study of a monoclonal antibody receive an infusion. But only one group of participants will receive an infusion with the drug being tested. A sham treatment is a simulation of a procedure, such as magnetic stimulation, that makes it hard for a participant to tell whether they receive the actual treatment. 

As a best practice, many Phase 2 and Phase 3 clinical trials will withhold this information from participants and clinicians until the very end. 

If you sign up for a single-blinded trial, you will not learn until the end whether you receive the treatment being tested, or the placebo. In a double-blinded trial, neither the clinicians nor the participants will know whether they receive the treatment or the placebo. 

These practices make the results of a clinical trial more robust by controlling for the placebo effect: Clinician and patient expectations that can influence the results of the study.

Are clinical trials ethical?

The United States has a dark history when it comes to medical experimentation. Events like the Syphilis Study at Tuskegee or the mistreatment of Henrietta Lacks and the cloning of her cancer cells were unethical and exploitative of people of color. 

Today, there are safeguards in place that protect participants in clinical trials more than ever before. Months or years before anyone is ever recruited, researchers who want to conduct a study must fill out forms and explanations of the study which are then reviewed by an institutional review board. 

These boards consist of doctors, researchers, and people outside the medical community. Their goal is to make sure the trial is well designed so that it does not cause any unnecessary harm to participants.

The second line of protection is called informed consent. This helps participants and their families weigh the risks and benefits of participating in the study. Throughout this process, the researchers also assess how well a participant understands the study. 

Once everything is clear, the participant can choose to sign a consent form and join the trial. Participants can withdraw their consent at any time, even if the trial has not yet ended.

Should I participate in a clinical trial?

There are some risks to participating in any clinical trial or study. If the trial is randomized and blinded, you might not even receive the treatment. You could also experience some side effects. There is the risk that the treatment may not work. 

However, everyone who participates furthers our understanding of Alzheimer’s disease and dementia. Even studies where a drug or intervention is ineffective provide valuable information for researchers. You could also receive a treatment for a disease before it is available anywhere else. You will receive more frequent checkups. Importantly, you can also feel empowered by taking an active role in your health.

Ultimately, deciding whether to participate in a study is a decision for each individual patient.

How Do Alzheimer’s Clinical Trials Work? & Other FAQs

The FDA recently approved a new ORAL therapy, Jaypirca (pirtobrutinib) from Eli Lilly and Company, indicated for mantle cell lymphoma (MCL) after at least two prior lines of therapy, including a BTK inhibitor.

Jaypirca is the  fourth BTK inhibitor for blood cancer treatment —but with a big difference from the three other approved BTK inhibitors — AbbVie and Johnson & Johnson’s Imbruvica, AstraZeneca’s Calquence, and BeiGene’s Brukinsa.

The three earlier products can’t be used sequentially. Jaypirca binds to BTK differently and therefore can be used after progression on another BTK inhibitor. This key difference gives Jaypirca a unique edge allowing treatment of  patients who have failed on another BTK inhibitor.

Lilly released pricing and the  U.S. wholesale acquisition cost of Jaypirca will be $21,000 per 30 days of therapy for the 200 mg daily dose. Jaypirca will be available in the United States later this quarter.

Lilly did not release details on distribution. All of the three competing BTK products launched through limited distribution. As such, it is likely that Jaypirca will launch similarly.

CLICK HERE for prescribing information

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FDA grants accelerated approval to pirtobrutinib for relapsed or refractory mantle cell lymphoma

On January 27, 2023, the Food and Drug Administration (FDA) granted accelerated approval to pirtobrutinib (Jaypirca, Eli Lilly and Company) for relapsed or refractory mantle cell lymphoma (MCL) after at least two lines of systemic therapy, including a BTK inhibitor.

Efficacy was evaluated in BRUIN (NCT03740529), an open-label, multicenter, single-arm trial of pirtobrutinib monotherapy that included 120 patients with MCL previously treated with a BTK inhibitor. Patients had a median of 3 prior lines of therapy, with 93% having 2 or more prior lines. The most common prior BTK inhibitors received were ibrutinib (67%), acalabrutinib (30%), and zanubrutinib (8%); 83% had discontinued their last BTK inhibitor due to refractory or progressive disease. Pirtobrutinib was administered orally at 200 mg once daily and was continued until disease progression or unacceptable toxicity.

The main efficacy measures were overall response rate (ORR) and duration of response (DOR), as assessed by an independent review committee using Lugano criteria. The ORR was 50% (95% CI: 41, 59) with a complete response rate of 13%. The estimated median DOR was 8.3 months (95% CI: 5.7, NE), and the estimated DOR rate at 6 months was 65.3% (95% CI: 49.8, 77.1).

The most common adverse reactions (≥15%) in patients with MCL were fatigue, musculoskeletal pain, diarrhea, edema, dyspnea, pneumonia, and bruising. Grade 3 or 4 laboratory abnormalities in ≥10% of patients were decreased neutrophil counts, lymphocyte counts, and platelet counts. The prescribing information includes warnings and precautions for infections, hemorrhage, cytopenias, atrial fibrillation and flutter, and second primary malignancies.

The recommended pirtobrutinib dosage is 200 mg orally once daily until disease progression or unacceptable toxicity.

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

This application was granted priority review and fast track designation. FDA expedited programs are described in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics. The application also was granted orphan drug designation.

The day has finally arrived.

The day when a biosimilar to Humira finally hits the market.

A total of 40 biosimilars have been approved since 2015, yet many were held up from marketing approval because of patent battles.  Amgen’s Amjevita was the fourth biosimilar ever approved but the first biosimilar to Humira in 2016.

Since then a total of eight….. yes 8….. Humira biosimilars have been approved. And why not? 

Humira has billions of $$$s of worldwide sales and even a one-eighth share would be a blockbuster for any of Humira’s offspring.

What is perhaps most curious is the way Amjevita is being launched. 

It will be available at two….. yes 2…..price points. 

You can buy Amjevita at a 5% discount to Humira….. or, you can buy adalimumab-atto at a 55% discount. To save you running the numbers, that translates into a price of $6,575 for Amjevita or $3100 for the non-branded product. No small difference!

Analysts are strongly suggesting that branded Amjevita will be the bigger seller. 

Why? 

In a word….. REBATES. Yes, PBMs are already pushing the more expensive, branded option so that scads of rebate dollars can be generated….. many of which may land in the coffers of the PBMs. What that means to patients (especially those with deductibles and coinsurance) appears to be irrelevant.

Acronyms….. they seem to be everywhere….. especially in healthcare. 

One acronym that has surfaced in the specialty pharmacy world is MIP. 

Scratching your head on what that one means? Well, it stands for Medically Integrated Specialty Pharmacy, one of the now hundreds of SPs that are owned and operated by Integrated HealthCare Systems (IHCSs) and hospitals. 

But wait…  there are more acronyms associated with MIPs….! How about DIRs? Yes, Direct and Indirect Remuneration (DIR) fees. Even MIPs are being hit by DIRs. 

But wait, there is now yet another acronym referencing the type of DIR fees being assessed by PBMs….. a GER which means Generic Effective Rate. Huh?  These new fees are calculated on a drug’s maximum allowable cost, wholesale acquisition cost, or average wholesale price (AWP).

Confused yet?

The article below is well worth a read even for those of us who think we are experts at understanding DIR fees. It covers a lot of ground and offers examples in fairly straightforward English. Any staff member who is less proficient on the topic would benefit from reading this article. 

An excerpt from the article follows…..

• Between 2010 and 2020, retroactive DIR fees increased by more than 100,000%.  DIR fees are charged by PBMs outside of administration fees and are often collected after the point of sale (POS) and do not reflect the pharmacy’s actual reimbursement at the time of dispensing. Traditionally, DIR fees are based on so-called pharmacy performance metrics.  DIRs have recoupment fees of up to 15% of the adjudicated price paid to the pharmacy for dispensing a prescription. PBMs develop and manage specific criteria used to score the contracted pharmacy. The scoring process varies from insurer to insurer and may include unachievable goals for specialty pharmacies focused on dispensing oncology medications [for example]. Unrealistic goals may include………….

CLICK HERE to read the full article — PBM Fees Put the “GER” in Danger for Specialty Pharmacies