Christine Blank

New drugs launched this year hit record high prices, according to a new analysis. The median annual price of 13 novel drugs approved for chronic conditions by the FDA so far in 2022 is $257,000, according to 

. Additionally, eight of 13 drugs are priced at more than $200,000 annually.

In the Reuter’s analysis, the drug with the highest annual or one-time price is Johnson & Johnson’s Carvykti (ciltacabtagene autoleucel) for multiple myeloma at $465,000, followed by Alnylam Pharmaceuticals’ Amvuttra (vutrisiran) for polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis, a rare and fatal disease, at $463,500.

Other novel drugs priced above $200,000 annually include: Immunocore Holdings’ Kimmtrak (tebentafusp-tebn) for metastatic uveal melanoma ($430,000), Agios Pharmaceuticals’ Pyrukynd (mitapivat) for hemolytic anemia in PK deficiency ($334,000), Sanofi’s Enjaymo (sutimlimab-jome) for cold agglutinin disease ($280,000), Bristol Myers Squibb’s Opdualag (nivolumab and relatlimab-rmbw) for metastatic melanoma ($273,885), CTI BioPharma’s Vonjo (pacritinib) for myelofibrosis ($257,160), and Novartis’ Pluvicto (lutetium Lu 177 vipivotide tetraxetan) for prostate cancer ($255,000).

report confirms what we already know: that drug manufacturers continue to launch new drugs at prices that reflect their monopoly power, not the actual value of the drug to patients,” Sean Dickson, JD, director of health policy at West Health Policy Center, a nonprofit focused on lowering healthcare costs, told 

“While manufacturers may claim that these prices reflect the small markets for drugs that target unique diseases, that’s still a price based on monopoly power — not on the incremental benefit to patients. Moreover, many of these drugs will likely be used for wider populations, but the pricing will still reflect the idea that the drug is limited to a small subset of patients,” Dickson added.

The recently passed Medicare negotiation provisions in the 

 will help lower drug prices in the future, Dickson acknowledged, “but the trend of ever-higher launch prices suggests the need for additional policies that limit monopoly power abuses.”

The Inflation Reduction Act allows Medicare, which President Joseph R. Biden signed into law on Tues., Aug. 16, 2022, to negotiate directly for prescription drugs and caps Medicare patients’ out-of-pocket costs at $2,000 annually on some drugs, among other provisions.

Related: Webinar: Inflation Reduction Act Will Be Positive for Medicare

Defending the cost of its Mounjaro (tirzepatide) treatment for diabetes ($12,700 annually), Eli Lilly & Co 

 that “each person’s individual (health) insurer and plan will determine the out-of-pocket costs.” In addition, the company offers savings cards to reduce costs to as little as $25 a month.

requested price data from all 15 of the companies that launched new drugs this year, six either did not respond to a request for price details or initially provided only partial information, such as a per vial cost, rather than an annual cost based on average patient use, the news outlet said.

Articles

Sean Dickson of West Health Policy Center discusses Reuters' analysis of drug launch prices.

 Analysis: 2022 Drug Launches Feature Record High Prices

The FDA issued an emergency use authorization (EUA) for the Bavarian Nordic’s Jynneos monkeypox vaccine intradermal injection for people, including those under the age of 18, who are at high risk for monkeypox infection. This will increase the total number of doses available for use by up to five-fold, FDA said in a 

“In recent weeks, the monkeypox virus has continued to spread at a rate that has made it clear our current vaccine supply will not meet the current demand,” said FDA Commissioner Robert M. Califf, M.D. “The FDA quickly explored other scientifically appropriate options to facilitate access to the vaccine for all impacted individuals. By increasing the number of available doses, more individuals who want to be vaccinated against monkeypox will now have the opportunity to do so.”

U.S. monkeypox cases have soared to 10,392 as of Aug. 10, 2022, according to the 

, and HHS Secretary Xavier Becerra declared the ongoing spread of monkeypox virus in the United States a 

Jynneos, the modified vaccinia ankara (MVA) vaccine, was 

 as a subcutanous injection in 2019 for prevention of smallpox and monkeypox disease in adults 18 years of age and older determined to be at high risk for smallpox or monkeypox infection.

While Jynneos is administered subcutaneously as two doses, four weeks (28 days) apart, the EUA now allows for a fraction of the Jynneos dose to be administered between the layers of the skin for adult patients.

Two doses of the vaccine given 4 weeks (28 days) apart will still be needed. There are no data available to indicate that one dose of Jynneos will provide long-lasting protection, which will be needed to control the current monkeypox outbreak, the FDA said.

Meanwhile, the Biomedical Advanced Research and Development Authority (BARDA), part of the office of the Administration for Strategic Preparedness and Response at the Department of Health and Human Services (HHS) is buying around $26 million worth of SIGA Technologies’ IV TPoxx treatment courses.

IV TPoxx, a treatment for both monkeypox and smallpox, is an important option for those who are unable to swallow, SIGA said in a 

. The manufacturer expects to deliver the product in 2023.

The EUA allows Jynneos to be used in children at high risk for monkeypox and as an intradermal injection, rather than subcutaneous, for adults.

FDA Issues EUA for Monkeypox Vaccine for Under 18; IV Treatment on the Way

One of the lasting effects of the COVID-19 pandemic has been an epidemic of COVID-19 “long-haulers,” those who have post-infection conditions that can last anywhere from months to years. An 

estimated 7.7 to 23 million Americans have developed long COVID

, and roughly 1 million people may be out of the workforce at any given time due to the condition — equivalent to about $50 billion in lost earnings annually, the Department of Health and Human Services (HHS) said in a 

Some of the symptoms include fatigue, fever, headache, brain fog, and joint and muscle pain, well as difficulty breathing, chest pain, or heart palpitations. Long COVID has even been identified as disability under the Americans with Disabilities Act.

“Long COVID can hinder an individual’s ability to work, attend school, participate in community life, and engage in everyday activities,” said HHS Secretary Xavier Becerra. “As our nation continues to make strides in the fight against COVID-19, these reports are critical to shine a light on long COVID’s impact and how to match people to resources.”

To that end, the Department of Health and Human Services has released two reports that “pave an actionable path forward to address long COVID and associated conditions.”

The National Research Action Plan on Long COVID

, created in coordination with 14 government departments and agencies, has introduced the first U.S. governmentwide national research agenda focused on advancing prevention, diagnosis, treatment, and provision of services and supports for individuals and families experiencing long COVID.

 details advances in current research and charts a course for future study to better understand prevention and treatment of long COVID. 

The Services and Supports for Longer-Term Impacts of COVID-19 report

 highlights resources for healthcare workers and those effected by broader effects of COVID-19.

Research priorities include building on extensive ongoing research, primarily led by CDC and the Veterans Affairs, to conduct additional studies of long COVID risk factors, health trajectories and outcomes that are inclusive of age, gender, race, ethnicity, geographic location, socioeconomic status, insurance coverage status, pregnancy status, and disability status — enabled by new capabilities in data analytics, HHS said.

HHS is also supporting studies that can “rapidly adapt to examine risk and protective factors as they emerge (i.e., new variants, vaccinations, repeat infections, and therapies for COVID-19) on risk of long COVID,” the agency said.

Then agency also wants to expand the portfolio of studies, including the RECOVER Initiative and others, on the effectiveness of therapeutics to prevent and treat long COVID to include antivirals, anti-inflammatory, immune modulators, and other existing or new treatments.

Meanwhile, long COVID researchers expect to have the results from three major trials later this year or sometime next year, per 

. Axcella Therapeutics, 9 MetersBiopharma'sAT1011 will likely have results in the second half of 2022, while Virios’s IMC-2 will convey results in the first half of 2023.

 topline results from its phase 2a randomized, double-blind, placebo-controlled investigation of AXA1125 in patients with fatigue related to long COVID.

“Long COVID is a persistent and growing challenge of the pandemic, affecting an estimated 100 million patients worldwide with fatigue as the most common symptom reported,” Axcella said in a statement.

In Axcella’s study, 41 subjects were enrolled and randomized to receive either 67.8 grams per day of AXA1125 or a matched placebo in two divided doses for 28 days, with a one-week safety follow-up period.

Endpoints included phosphocreatine recovery time following moderate exercise as assessed by 31P-magnetic resonance spectroscopy, which was included to assess mitochondrial function, and clinically relevant endpoints including self-reported mental and physical fatigue as assessed by the Chalder Fatigue Questionnaire, six-minute walk test, as well as serum lactate levels.

Subjects who received AXA1125 had improvements in measures of mental and physical fatigue that were both highly statistically significant and clinically relevant compared with those who received placebo, Axcella said.

“The results of this trial encourage us to further evaluate the multi-targeted effects of AXA1125 on mitochondrial and related biomarkers to advance our understanding of the benefits AXA1125 delivers to Long COVID patients,” said Karim Azer, Ph.D., vice president of platform and discovery at Axcella.

A rise in the number of long COVID is sparking research efforts by the HHS, the CDC and drug companies to understand the conditions and develop treatments.

Long COVID-19 a Priority for Biden Administration, Drug Developers

Two specialty medications to treat rare diseases, along with novel treatments for myeloma and plaque psoriasis, are highlighted in OptumRx’s new Drug Pipeline Insights report. The top four medications —teclistamab, deucravacitinib, betibeglogene autotemcel, and elivaldogene autotemcel— are expected to receive FDA approval in the third quarter.

 (brand name to be determined) is a novel oral drug being evaluated to treat adults with moderate-to-severe plaque psoriasis developed by Bristol Myers Squibb. Plaque psoriasis affects about 6 million people in the United States. Deucravacitinib is a novel tyrosine kinase 2 (TYK2) inhibitor that regulates overproduction of immune inflammatory compounds in immune-mediated diseases such as psoriasis, OptumRx said.

While deucravacitinib would provide a first-in-class oral treatment for a condition with limited oral options, it is still entering a crowded marketplace, according to OptumRx.

Injectable biologics are commonly used with high response rates in patients with moderate-to-severe plaque psoriasis. Immunosuppressants such as methotrexate and cyclosporine have historically been used, but their place in therapy is limited because of safety concerns, according to the PBM.

Celgene’s Otezla (apremilast), an oral phosphodiesterase 4 (PDE4) inhibitor, has been available since 2014 and has a safer adverse event profile versus historical oral treatments but its use has been limited because injectable biologics are more effective, OptumRx said. In trials, deucravacitinib demonstrated superior efficacy versus Otezla and was associated with numerically lower rates of discontinuations due to adverse events.

“Deucravacitinib is indeed entering a crowded marketplace with multiple treatments already available,” Bill Dreitlein, Pharm.D., senior director, pipeline and drug surveillance at OptumRx, told 

. “However, psoriasis is an $11 billion marketplace and deucravacitinib will aim to carve out a slice of that as a new oral option.”

An FDA decision is expected Aug. 19, 2022.

(brand name to be determined), administered subcutaneously, is a novel drug to treat relapsed or refractory multiple myeloma developed by Janssen. Teclistamab is a bispecific antibody that targets both B-cell maturation antigen (BCMA) and CD3, a T-cell receptor.

It enters a competitive field, with three similar therapies that target BCMA for relapsed or refractory multiple myeloma, OptumRx said. Existing medications include:GSK’s Blenrep(belantamab mafodotin), which is administered intravenously; along with CAR-T cell therapies Carvykti (ciltacabtagene autoleucel) from Janssen Biotech and Abecma (idecabtagene vicleucel) from Bristol Myers Squibb.

Indirectly, teclistamab appears more effective than Blenrep, but less effective versus the CAR-T cell therapies, according to OptumRx. “However, it appears that the CAR-T cell therapies see higher rates of CRS and neurotoxicity than teclistamab,” the PBM said.

A wholesale acquisition cost for teclistamab has not been announced, according to OptumRx.

An FDA decision is expected Sept.10, 2022.

, with a brand name of Zynteglo, is a gene therapy for transfusion-dependent beta-thalassemia developed by Bluebird Bio. If approved, Zynteglo could be a cure for the rare disease through a one-time intravenous infusion, according to OptumRx.

Beta-thalassemia is caused by genetic mutations in cells that carry oxygen throughout the body, which may produce classic signs of anemia and may be life-threatening if left untreated, according to OptumRx.

Current treatment for severe beta-thalassemia is mainly regular, life-long red blood cell transfusions. Patients also need iron chelation therapy to address excess levels of iron in the body due to the repeated blood transfusions.

Stem cell transplantation from a matched donor, ideally a sibling, is currently the only possible cure. However, stem cell transplants carry their own risks, and fewer than 25% of patients have access to a suitable match, Dreitlein said.

The only other FDA-approved therapy for treatment transfusion dependent beta-thalassemia is Reblozyl (luspatercept) developed by Bristol Myers Squibb and Acceleron, which only treats the anemia resulting from the disease and requires an injection every three weeks.

“If approved, Zynteglo would be a one-time treatment and the first gene therapy for a blood disorder,” Dreitlein said. As a result, it could “break the ice” for other treatments in the future for other blood disorders such as sickle cell disease, he added.

Of the estimated 3,000 patients with beta-thalassemia in the United States, about half of them are dependent on blood transfusions. “The target population for this treatment is extremely small. Bluebird bio estimates about 1,500 patients with beta-thalassemia in the United States would be eligible,” OptumRx said.

Analysts forecast a $2.1 million cost for a one-time dose of Zynteglo, according to OptumRx.

An FDA decision is expected Aug. 19, 2022

with the brand name Skysona, is a gene therapy to treat cerebral adrenoleukodystrophy (CALD) in young boys developed by Bluebird Bio. Patients with CALD lack a protein (ALDP) needed to break down fatty acids, which causes toxic acids to build up in the brain and leads to disability and an early death. Most patients with CALD will die within a decade of diagnosis if they are not treated with bone marrow (stem cell) transplantation.

There are around 40 patients with CALD in the United States annually, according to OptumRx.

“If approved, Skysona would be a one-time treatment that may stabilize disease progression and preserve neurological function in patients with early CALD,” OptumRx said.

While Bluebird bio has not announced a price for Skysona, the wholesale acquisition cost for Zolgensma (onasemnogene abeparvovec), a one-time gene therapy for spinal muscular atrophy, is $2.125 million, according to Optum Rx.

An FDA decision is expected Sept. 16, 2022.

OptumRx profiles four therapies that are expecting FDA decisions in the next few months.

Top 4 Drugs to Watch in the Third Quarter

More than 200 organizations expressed their strong support for the 

Pharmacy Benefit Manager Transparency Act

 (S. 4293), while an organization representing PBMs opposes the proposed legislation.

, the groups urged Sens. Chuck Schumer (D-N.Y.) and Mitch McConnell (R-Ky.) to quickly bring the Act to a vote in the full Senate. The bill, which was introduced in late May 2022 by Sens. Maria Cantwell (D-Wash.) and Chuck Grassley (R-Iowa), passed the Senate Committee on Commerce, Science, and Transportation in June 2022.

The diverse group of organizations and businesses supporting the legislation include: The AIDS Healthcare Foundation, the National Community Pharmacists Association, the American Pharmacists Association, FMI-The Food Industry Association, Food Lion, Hannaford, and the Coalition of State Rheumatology Associations.

The legislation “would bring needed transparency to and ultimately stop PBM-insurers’unjust and deceptive practices, especially as the three largest PBM-insurers now control at least 80% of the market,” the organizations wrote. “We hope the full Senate will soon take up this legislation to empower the FTC [Federal Trade Commission] to bring enforcement actions against the consolidated PBM-insurer industry and their anticompetitive practices, including spread pricing and clawbacks.”

The Act empowers the FTC to increase drug pricing transparency and ban “deceptive unfair pricing schemes like spread pricing and prohibit arbitrary clawbacks of payments made to pharmacies,” NCPA said in a 

It also includes provisions to allow other state officials to bring action to enforce the law if the state attorney general lacks jurisdiction to do so, and allow for a Government Accountability Office study of PBM practices.

Of every $100 in spending on drugs sold in retail pharmacies, about $41 goes to intermediaries in the supply chain such as PBMs, according to 

from the USC Leonard D. Schaeffer Center for Health Policy and Economics.

“These intermediaries are earning excess returns 

 with the average firm in the S&P 500, and these excess returns are rising over time,” Health Affairs 

But the Act will not reduce prescription drug costs, said JC Scott, president and CEO of the PBM industry association, Pharmaceutical Care Management Association (PCMA), in a 

. “Instead, the legislation is designed to award pharmacies and drug manufacturers an increase to their bottom lines, rather than lowering prescription drug costs for consumers,” he said.

The bill hinders the ability of PBMs to drive competition in the prescription drug marketplace and enables drug manufacturers to “tacitly collude to raise prices even higher,” Scott said.

The bipartisan bill introduced in the Senate in May would require PBMs to report to the FTC how much money they make through spread pricing and pharmacy fees.


Groups Urge Vote on Pharmacy Benefit Manager Transparency Act

Although seven biosimilars to the blockbuster rheumatoid arthritis (RA) drug AbbVie’s Humira (adulimumab) are expected to launch in 2023, they may not be available to patients, according to the Biosimilars Forum, which represents biosimilar manufacturers.

Leaders from the Forum and their member companies are concerned, based on past experiences, that the Humira biosimilars won’t be placed on formularies unless manufacturers are willing to be reimbursed less due to high rebate fee structure.

“The first-to-market biosimilars have been launched in general at a list price 30% lower than the brand,” Juliana M. Reed, executive director of the Biosimilars Forum, told

 “When each new biosimilar is launched, the competition drives down the price even further. Biosimilar developers prefer to have access to formularies at lower list price rather than have to add a high rebate on to the product in order to gain access to the market through PBMs. Forum member companies have experienced this with PBMs.”

Additionally, to ensure coverage for all biosimilars at their launch, it will be important for Centers for Medicare and Medicaid Services to classify the Humira biosimilars as maintenance changes, Reed said.“Without this change in policy, those biosimilars will not be able to be included on formularies until 2024,” she said.

Much of the future of the biosimilars industry depends on whether Humira biosimilars will be placed on formularies as they become available in the U.S. market.

 and currently faces no U.S. competition amid a 

The first biosimilar, Amgen’s Amjevita, is expected to launch in January 2023. The others expected to launch in the second half of the year include: the Merck’s Hadlima, Boehringer Ingelheim’s Cyltezo, Coherus BioSciences’ Yusimry, Viatris’ Hulio, Sandoz’ Hyrimoz, and Pfizer’s Abrilada.

PBMs' payment model has changed since Humira biosimilar manufacturers began developing the drugs, according to Reed. In order to gain exclusive formulary access, PBMs are requiring the lowest net cost, which is calculated using a high rebate from manufacturers.

Representatives for Express Scripts, United Health Group, and Optum did not respond to requests for comment from 

Reed said CMS and policymakers need to implement a clear and expedited pathway to add all biosimilars to Medicare Part D formularies when they become available.

Related: Biosimilars Can Save Money for Medicare and Beneficiaries

Medicare Part D and its beneficiaries would benefit if all biosimilars are made available and used more often, according to a recent 

 by the Office of Inspector General (OIG) at the U.S. Department of Health and Human Services. In 2019, biosimilars’ reference products were prescribed about five times more frequently than biosimilars in Part D. But if biosimilars had been used instead, spending on all biosimilars could have been reduced by $84 million, or 18%. Additionally, beneficiaries’ out-of-pocket costs for these drugs could have decreased by $1.8 million or 12%.

Next year, seven biosimilars of Humira are expected to launch, but leaders from the Biosimilars Forum are concerned not all will make it onto formularies.