FDA Approves $3.5 Million Gene Therapy to Treat Adults with Hemophilia B

FDA Approves $3.5 million gene therapy, Hemgenix, to treat adults with Hemophilia B. Following approval by the U.S. Food and Drug Administration, Australian pharmaceutical company CSL Ltd on November 22, 2022, set the list price of its one-time gene therapy for hemophilia B at $3.5 million, making it the costliest treatment in the world. Hemgenix (etranacogene dezaparvovec), an adeno-associated virus vector-based gene therapy, has been approved by the U.S. Food and Drug Administration for the treatment of adults with hemophilia B (congenital factor IX deficiency) who are currently receiving Factor IX prophylaxis therapy, have experienced recent or past life-threatening hemorrhage, or have had recurrent, severe spontaneous bleeding episodes.

FDA Approves $3.5 Million Gene Therapy. Certified Pharmacy Benefit Specialist (CPBS).
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Blood clotting factor IX, a protein required to build blood clots to halt bleeding, is either absent or present in insufficient amounts in people with hemophilia B, a genetic bleeding condition. After an injury, surgery, or dental operation, there may be prolonged or substantial bleeding as a symptom. In more serious circumstances, spontaneous bleeding episodes may happen without a known reason. Serious problems, such as bleeding into joints, muscles, or internal organs, including the brain, can result from protracted bleeding episodes.

Men are more likely than women to develop hemophilia B and show symptoms. Hemophilia B affects roughly 15% of hemophilia patients, with an incidence of one in 40,000 in the general population. Many female carriers of the illness don’t exhibit any symptoms. However, it’s thought that 10–25% of female carriers may experience mild symptoms; very rarely, women may experience moderate or severe symptoms.

In order to strengthen the body’s capacity to stop bleeding and encourage healing, the clotting factor that is inadequate or lacking is routinely replaced. To maintain enough amounts of clotting factor to stop bleeding episodes, patients with severe hemophilia B often need a routine treatment schedule of intravenous (IV) infusions of Factor IX replacement medicines. Hemgenix is a one-time gene therapy treatment administered by intravenous infusion. A viral vector called Hemgenix carries a gene for clotting factor IX. In order to manufacture more Factor IX protein, raise blood levels of the substance, and lessen bleeding episodes, the gene is expressed in the liver.

Two studies with 57 adult men aged 18 to 75 with severe or moderately severe hemophilia B examined the safety and efficacy of Hemgenix. On the basis of drops in the men’s annualized bleeding rate (ABR), effectiveness was determined. A 54-person study found that the subjects had higher levels of Factor IX activity, less need for routine Factor IX replacement prophylaxis, and a 54% drop in ABR from baseline. Hemgenix side effects that were most frequently reported were liver enzyme increases, headaches, minor infusion-related responses, and flu-like symptoms. Blood liver enzyme increases (transaminitis) and unfavorable infusion reactions should be watched in patients.

What do Ticketmaster and Pharmacy Benefit Managers have in common? [Weekly Roundup]

What do Ticketmaster and Pharmacy Benefit Managers have in common and other notes from around the interweb:

  • Accumulators And Maximizers: A New Front in the Battle Over Drug Costs. At the time the patient presents to the retail pharmacy and uses the copayment coupon, the retail pharmacy (which contracts with the PBM) enters the amount of the coupon in the adjudication system. The PBM notes this part of the transaction and deducts the value of the coupon so that it does not “accumulate” against the deductible. (The pharmaceutical assistance program also notes the value of the coupon, as the programs do put limits on the amount of assistance available.) The beneficiary still avoids the copayment cost at least until she hits the limits on the copayment program, when the coupon ceases to become available and the patient must begin to pay, reinstating the original incentives. From the payer’s point of view—either the self-insured employer or the insurer—the effect is financially salutary. Some pharmaceutical firm funds help pay for the initial costs of the medication. But eventually, the deductible and copayments come back into play and promote consumerism. Yet, as the coupon limits come into play, the beneficiary faces challenges from out-of-pocket spending, and adherence can drop.
  • What do Ticketmaster and Pharmacy Benefit Managers have in common? Popular American singer and songwriter, Taylor Swift, released her newest album ‘Midnights’ in October. The album quickly became the most-streamed album in 24 hours on Spotify, with 184.6 million streams, according to Guinness World Records. Following the release, the artist sought to work with Ticketmaster, a company who arguably has a monopoly on all ticket sales in the country, for pre-sale tickets for her new ‘Eras’ tour. But shortly after online pre-sale started for the tour on Tuesday, November 15th, Ticketmaster canceled the public on-sale that was supposed to take place on Friday, after the site had sold two million tickets and saw long wait times and temporary outages. How is Ticketmaster getting away with this? Pharmacy benefit managers (PBMs), like Ticketmaster, are third-party administrators of prescription drug programs that are primarily responsible for processing and paying prescription drug claims. As Wayne Winegarden notes in his study, “The Economic Costs of Pharmacy Benefit Managers,” due to their government sponsored ‘near-monopoly’ position, PBMs can charge fees that are high and retrospective.
  • Getting off the PBM Merry-go-Round: How Late-stage Offer Tricks Take Employers for a Ride. Monies offered by PBMs come in many forms – a general allowance credit that the client can use to pay for claims and expenses during open enrollment and implementation, an administrative credit that the client can use to pay back to the PBM for administrative fees, a clinical credit that the client can use to pay for voluntary clinical programs administered by the PBM or, at times, slightly improved pricing. If it seems odd that two of these credits go towards paying back the PBM for additional services, that’s because it is. Employers should think of these credits like getting tokens at a carnival – the tokens are valuable, but only if the employer uses them at the proverbial PBM carnival (i.e., invests them back into the PBM). If PBMs would manage their costs appropriately to begin with, employers wouldn’t be taken for this ride.
  • ERISA-Covered Companies Must Disclose Health Plan Costs. Starting in 2022, an estimated 2.5 million employer-sponsors of health plans are required to adopt new fee and pricing transparencies due to amendments made to ERISA. While ERISA has focused on retirement service fees in the past, the spotlight is now on healthcare costs. The requirements fall under the Affordable Care Act (ACA) and Consolidated Appropriations Act, 2021 (CAA 2021), and affect organizations covered by the Employment Retirement Income Security Act of 1974 (ERISA). ERISA applies to most private companies that offer healthcare and retirement plans to employees. Complying with ERISA fiduciary duties for group health plans has been challenging due to the lack of fee transparency in the industry. However, the new transparency rules put more fee and pricing information into the hands of health plan fiduciaries and other stakeholders to shed light on these fees.

FTC reaffirms commitment to unfair business practices

In a statement released last week, the FTC reaffirms commitment to unfair business practices. Beyond what is covered by the other antitrust provisions, Congress granted the Federal Trade Commission (FTC) the exclusive power to detect and enforce this conduct. However, the agency hasn’t always consistently fulfilled this duty in recent years. By limiting its oversight to a more limited range of situations, the FTC’s previous policy made it more difficult for the agency to address the whole spectrum of anticompetitive behavior in the market.

With the recent announcement, this restriction is lifted, and the agency states its intention to use all its statutory power to take legal action against businesses that take unfair advantage of their competitors rather than engaging in fair competition. Congress created the Federal Trade Commission Act in 1914 as a result of dissatisfaction with the Sherman Act’s enforcement, the first antitrust law. The FTC Act’s Section 5 prohibits “unfair methods of competition” and directs the Commission to uphold this rule.

FTC reaffirms commitment to unfair business practices. Certified Pharmacy Benefit Specialist (CPBS).
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According to the policy statement, unfair methods of competition are strategies that aim to acquire an advantage without engaging in a merit-based competition and that are likely to lessen market competition. The Commission’s strategy for policing them is outlined in the Policy Statement. It is the outcome of several months’ worth of collaboration between agency departments. Staff members combed through hundreds of Commission decisions, consent orders, and court rulings—including more than a dozen Supreme Court opinions—to examine the legislative background of Section 5 and its interpretation. The agency will follow the rich case history as it applies Section 5 to its operations. The Commission will warn firms about how to compete fairly and legally through enforcement and rulemaking.

This is without a doubt a prelude to the FTC’s inquiry into pharmacy benefit manager (PBM) practices. Those stakeholders seeking change, for the better, in pharmacy benefit manager practices are eagerly awaiting the commission’s final report. PBMs are a crafty bunch. They’ve already made moves to protect margins from any FTC decision that might curtail PBM revenue streams that are bad for customers.

Consolidated Appropriations Act shifts more responsibility to employers next year [Weekly Roundup]

Consolidated Appropriations Act shifts more responsibility to employers next year and other notes from around the interweb:

  • Centene gives politicians millions as it courts contracts and settles overbilling allegations. On Nov. 2, 2021, Nevada Gov. Steve Sisolak’s reelection campaign received ten separate $10,000 contributions from unrelated health insurance plans from across the country. The Buckeye Community Health Plan of Ohio, Louisiana Healthcare Connections, and Peach State Health Plan of Georgia were among the companies that sent money to the Democrat, according to state campaign finance records, even though only one, SilverSummit Healthplan, provided insurance in the Silver State. But a thread connects the companies: Each is a subsidiary of Centene Corp., ranked 26th on the Fortune 500 list, and the nation’s largest private managed-care provider for Medicaid, the government insurance program for people with low incomes or disabilities. Centene had already sealed Medicaid deals in Nevada through its SilverSummit subsidiary — yet a potential new line of business was on the horizon. Sisolak, who is up for reelection Nov. 8, had just approved a new public health plan option that would later open to bidding from contractors such as SilverSummit. And then, less than two months after Centene’s subsidiary contributions were made, Nevada settled with the company over allegations the insurer overbilled the state’s Medicaid pharmacy program. The state attorney general’s office did not announce the $11.3 million settlement but disclosed it in response to a public records request from KHN.
  • 4 questions to ask before signing your next PBM service agreement. The very idea of managing pharmacy benefits might make you nauseous. That’s because drug prices are skyrocketing, creating significant challenges for your company’s bottom line. You may think that your pharmacy benefit manager (or PBM) – who is responsible for handling contractual relationships between drug manufacturers, health insurance providers, pharmacies, and patients – would negotiate the best possible deals for everyone involved. Unfortunately, recent reports from the PBM Accountability Project show otherwise: PBMs often misuse their immense power by adding secret streams of revenue for themselves. The Federal Trade Commission (FTC) has noted this trend too and announced plans in 2022 to investigate the inner workings of PBMs. But some states are taking it upon themselves to crack down on PBM business dealings, too. For example, Florida and Iowa joined Michigan in passing legislation in 2022 that regulates certain PBM practices – while Ohio’s Medicaid department is also conducting audits.
  • Is prescription copay assistance contributing to rising drug prices? Why buyers should beware. Drug manufacturers say they offer coupon programs to help patients offset the rising out-of-pocket cost of prescriptions due to health insurance plan design changes. “Every day there are patients who show up at the pharmacy and find their commercial insurance won’t cover the cost of their medicine,” said Brian Newell, spokesman for the drugmaker trade group Pharmaceutical Research and Manufacturers of America. “To help fill these gaps in insurance coverage, biopharmaceutical companies offer coupons and other forms of patient assistance.” Pharmacy benefit managers say drugmakers only offer coupons to entice people to take more expensive brand-name drugs. When these programs started, they were really mostly for uninsured people, said Robert Popovian, chief science officer of the Global Healthy Living Foundation, which advocates for patients with arthritis and other chronic illnesses. But the need for drug copay assistance among the insured has grown as health plans have shifted more of the cost burden onto patients via high deductible health plans, said Kollet Koulianos, vice president of payer relations at the National Hemophilia Foundation.
  • Self-funded plans ignore the Consolidated Appropriations Act at their peril. The CAA places a range of new fiduciary responsibilities on individuals who manage health plans and on select employer-sponsored health care providers. These regulations aren’t just for plan fiduciaries, but also for trustees and other payers that provide health benefits, including pharmacy benefits. CAA provides plan sponsors with a framework to better evaluate and manage health plan spend and gain greater visibility into compensation arrangements between brokers and providers. That’s the good news. However, it also creates an element of risk because organizations must comply. Plan sponsors must familiarize themselves with the costs of health care, with how their broker or consultant partners are paid, and they must be able to disclose this information to the Department of Labor and Health and Human Services. Those who eschew this responsibility and fail to develop a defensible process or run afoul of CAA regulations could face fines and significant liability in the form of legal action from employees.

PBM Revenue Streams that are Bad for Customers

The list of methods in which bad PBM revenue streams are created is long. They include but are not limited to:

  1. Clawbacks
  2. Ballooning
  3. Drug reclassification
  4. Back-billing
  5. Rebate spreads
  6. Ingredient cost spreads
  7. DIR fees
  8. Differential contracting
  9. Poor utilization management
  10. Poor product mix

I could go on, but you get the idea. Kick back, increase the playback speed to 1.5x or 2x and watch as I go into detail about PBM revenue streams. To learn more about bad PBM revenue streams, join the next Certified Pharmacy Benefits Specialist course. It starts on January 12, 2023!

Learn about PBM revenue streams in the Certified Pharmacy Benefits Specialist (CPBS) program.
Only three online courses per year!

The Certified Pharmacy Benefits Specialist (CPBS) program offers three different instructional formats: live online classrooms, in-person Knowledge Camps, and self-study. Every Thursday at 6 PM ET, the online class meets for roughly 1.5 hours. Every class is taped for later viewing. PBIA is approved by the Society for Human Resource Management (SHRM), HR Certification Institute (HRCI), American Council of Pharmacy Education (ACPE), and forty-six states to provide up to 20 recertification credit hours to licensed pharmacists, pharmacy technicians, life and health professionals, and human resources specialists. Since we began offering our courses in 2014, more than 1,000 students have earned the CPBS credential.

“Thanks for everything throughout the course. The firm I work for is trying to get more involved in the Plan Sponsor space, and in doing so, we have been reviewing a lot of PBM/Plan Sponsor contracts. I can’t tell you how much more confident and comfortable I am working through these PBM contracts now and what I need to be looking out for.” – Adam Farkas, Esq. Associate Attorney

How PBMs Make Money and What to Do About It [Free Webinar]

Because plan sponsors don’t know how to calculate how much money pharmacy benefit managers (PBM) make, it gives PBMs all the incentive they need to overcharge. How many businesses do you know want to cut their revenues in half? That’s why traditional pharmacy benefit managers, and their stakeholders, don’t offer a fiduciary standard of care and instead opt for hidden cash flow opportunities to generate their service fees. Want to learn more?

Here is what some participants have said about the webinar.

“Thank you, Tyrone. Nice job, good information.” David Stoots, AVP

“Thank you! Awesome presentation.” Mallory Nelson, PharmD

“Thank you, Tyrone, for this informative meeting.” David Wachtel, VP

“…Great presentation! I had our two partners at the presentation as well. Very informative.” Nolan Waterfall, Agent/Benefits Specialist

A snapshot of what you will learn during this 30-minute webinar:

  • Hidden cash flows in the PBM Industry
  • Basic to intermediate level PBM terminologies
  • Specialty pharmacy cost-containment strategies
  • Examples of drugs that you might be covering that are costing you
  • The #1 metric to measure when evaluating PBM proposals

Understanding how PBMs make money and how much you pay them for their services is a key element in running an efficient pharmacy benefits program. Join us to learn more.

See you Tuesday, 11/8/22 at 2 PM ET!

Sincerely,
TransparentRx
Tyrone D. Squires, CPBS  
10845 Griffith Peak Drive, Suite 200  
Las Vegas, NV 89135 
Office: (866) 499-1940
Mobile: (702) 803-4154

P.S. Yes, it’s recorded. I know you’re busy … so register now and we’ll send you the link to the session recording as soon as it’s ready. 

Get ready for hemophilia gene therapies at $2M-$3M [Weekly Roundup]

Get ready for hemophilia gene therapies at $2M-$3M and other notes from around the interweb:

  • ERISA-Covered Companies Must Disclose Health Plan Costs. Starting in 2022, an estimated 2.5 million employer-sponsors of health plans are required to adopt new fee and pricing transparencies due to amendments made to ERISA. While ERISA has focused on retirement service fees in the past, the spotlight is now on healthcare costs. The requirements fall under the Affordable Care Act (ACA) and Consolidated Appropriations Act, 2021 (CAA 2021), and affect organizations covered by the Employment Retirement Income Security Act of 1974 (ERISA). ERISA applies to most private companies that offer healthcare and retirement plans to employees. Complying with ERISA fiduciary duties for group health plans has been challenging due to the lack of fee transparency in the industry. However, the new transparency rules put more fee and pricing information into the hands of health plan fiduciaries and other stakeholders to shed light on these fees.
  • 4 questions to ask before signing your next PBM service agreement. The very idea of managing pharmacy benefits might make you nauseous. That’s because drug prices are skyrocketing, creating significant challenges for your company’s bottom line. You may think that your pharmacy benefit manager (or PBM) – who is responsible for handling contractual relationships between drug manufacturers, health insurance providers, pharmacies, and patients – would negotiate the best possible deals for everyone involved. Unfortunately, recent reports from the PBM Accountability Project show otherwise: PBMs often misuse their immense power by adding secret streams of revenue for themselves. The Federal Trade Commission (FTC) has noted this trend too and announced plans in 2022 to investigate the inner workings of PBMs. But some states are taking it upon themselves to crack down on PBM business dealings, too. For example, Florida and Iowa joined Michigan in passing legislation in 2022 that regulates certain PBM practices – while Ohio’s Medicaid department is also conducting audits.
  • Payers and PBMs are restricting access to birth control options, report finds. Several large health insurers and pharmacy benefit managers (PBM) are limiting access to birth control medication, according to a report issued by the House Oversight and Reform Committee this week. There are more than 30 birth control products that health insurers and pharmacy benefit managers are placing cost-sharing requirements on, the report found. In other words, these healthcare stakeholders are making patients pay to cover part of the prescription drug cost — or else restricting coverage. The Affordable Care Act (ACA) requires that private health insurance plans cover birth control options that are approved by the Food and Drug Administration without asking for copays. Among the 34 birth control products identified in the report, 12 don’t have equivalent options on the market.
  • Get ready for hemophilia gene therapies at $2M-$3M. ICER says it’s fair for BioMarin and CSL to price incoming hemophilia gene therapies at $2M-$3M. Get ready for million-plus-dollar gene therapies to become the norm rather than the exception, and in some cases, the Boston-based drug pricing watchdog ICER is coming on board. The nonprofit ICER (Institute for Clinical and Economic Review) on Wednesday unveiled a new report finding that CSL Behring’s potential gene therapy etranacogene dezaparvovec for hemophilia B, which is due for an FDA approval decision by the end of this month, could be priced at around $3 million. ICER also updated its previous assessment of BioMarin’s EU-authorized hemophilia A gene therapy Roctavian, which ICER said could be fairly priced at about $2 million. “The new gene therapies can result in successfully treated patients appearing ‘cured’ for at least a period of time,” ICER chief medical officer David Rind said in a statement. “During this period, these gene therapies will eliminate the need for expensive prophylactic treatment. However, the duration of this ‘cure’ and the safety of therapies remains important uncertainties.”