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Relapsed or refractory acute leukemia with a KMT2A translocation currently has an overall survival rate of less than one year when treated with frontline therapies. 

FDA Approves First and Only Drug for Rare Leukemia 

Researchers are currently enrolling participants to test the effectiveness of a rectal douche containing the antiretroviral drug tenofovir in a study that is expected to finish in January 2026. 

NIH Tests New PrEP Delivery Method 

Breast cancer treatment settings prove to be a good opportunity to talk about financial toxicity. These conversations can also happen in generalized healthcare, according to Laila Gharzai, M.D., LLM, from the Department of Radiation Oncology at Northwestern University.

How Financial Toxicity Screening Can Be Incorporated Into Everyday Healthcare in America

Laura is the vice president of content of Managed Healthcare Executive

 (MHE) and all its brands, including Formulary Watch

. She has been working on MHE since 2019 and has been with MHE's parent company, MJH Life Sciences

, since 2011. She has an MA in business and economic reporting from New York University.

In a phase 2 trial, a gene therapy reduced angioedema attacks, which could significantly change the quality of life for patients with hereditary angioedema.

A Single CRISPR Treatment Could Replace a Lifetime of Daily Treatment in Hereditary Angioedema

Managed Healthcare Executive provides C-suite executives at health plans and provider organizations with news, analysis, insights, and strategies to pursue value-driven solutions.

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Healthcare finance sub-trends that will make a significant impact on healthcare in the new year.

4 Key Trends to Affect Healthcare Significantly in 2020

 the biologics license application (BLA) for Upstaza (eladocagene exuparvovec). The application has been granted priority review with a target regulatory action date of Nov. 13, 2024.

Developed by PTC Therapeutics, Upstaza is a one-time gene therapy to treat patients 18 months and older with aromatic L-amino acid decarboxylase (AADC) deficiency, a fatal, rare genetic disorder that typically causes severe disability. It leads to metabolic disturbance of dopamine, norepinephrine, epinephrine, and serotonin. Children with AADC deficiency can experience seizure-like oculogyric crises (involving the eyes), frequent vomiting, behavioral problems, and difficulty sleeping.

AADC deficiency is caused by mutations of the DOPA decarboxylase (DDC) gene in which the AADC enzyme cannot perform its function in the formation neurotransmitters epinephrine and norepinephrine, dopamine and serotonin.

Upstaza is a recombinant adeno-associated virus serotype 2 (AAV2)-based gene therapy, containing the human DDC gene. It is designed to deliver a functioning DDC gene directly into the putamen, the part of the brain involved in learning and motor control. Administration of Upstaza is designed to increase the AADC enzyme and restore dopamine production.

"The data collected to date continue to support the transformative benefit of Upstaza, this highly innovative gene therapy directly infused into the brain,” Matthew B. Klein, M.D., CEO of PTC Therapeutics, said in a news release.

 of 30 children demonstrated that administration of Upstaza directly to the brain led to dopamine being made and released. The three trials were open label and patients were evaluated monthly for safety and efficacy. Efficacy was based on the results of scores from several developmental scales through the first year after treatment with Upstaza and five years after treatment.

Compared with baseline, at one year and five years after Upstaza, patients had evidence of dopamine productions visualized using PET imaging of the brain, as well as in motor milestones of head control, sitting unassisting, standing with support and walking with assistance. There were no serious adverse events. The most frequent adverse event was dyskinesia (involuntary movements), which resolved over time.

Upstaza was part of the pipeline of Agilis Biotherapeutics, which 

Upstaza is approved in both the European Union and in the United Kingdom to treat AADC deficiency. Upstaza generated revenue in 2023; but PTC’s documents only indicated that sales from Tegsedi, Waylivra and Upstaza contributed $22.7 million in 2023. The company will be required to pay $20 million in regulatory milestone payments to the former equity holders of Agilis, as well as an additional $.4.5 million upon approval of Upstaza in the United States.

Administration of Upstaza occurs through a stereotactic surgical procedure, a minimally invasive neurosurgical procedure used for the treatment of a number of pediatric and adult neurological disorders. It uses a technology developed by ClearPoint Neuro for infusion into the brain. The Upstaza administration procedure is performed by a qualified neurosurgeon in centers specialized in stereotactic neurosurgery.

In October 2022, PTC Therapeutics had discussed the FDA its BLA for Upstaza. At that time, regulators had asked for additional bioanalytical data in support of comparability of the therapy used in trials with the commercial product, according to 

. The company completed that analysis, but regulators said it was deficient. However, the FDA indicated it could use data that supports the safety of the drug delivery cannula to support an accelerated approval based on biomarker data that demonstrated an increase in dopamine production.

Upstaza is a one-time gene therapy to treat patients 18 months and older with AADC deficiency. It has a review date of Nov. 13, 2024.

Formulary Watch

FDA Sets Review Date for Gene Therapy for Enzyme Deficiency

By Reneé Buckingham, President of Humana’s Primary Care Organization, including CenterWell Senior Primary Care and Conviva Care Center

The senior population in the U.S. is rapidly expanding, as is the demand for comprehensive health care to address their specific needs. There are now approximately 

 and older living in America, and that number is expected to balloon up to 84 million in the next 30 years. At the same time, there is a severe shortage of geriatricians – only about one for every 10,000 seniors in the U.S. – who specialize in treating the prevalent chronic and complex conditions affecting older adults.

Access to quality care for seniors is a growing crisis, potentially leading to poorly managed and untreated chronic conditions and spiraling costs from worsening disease, disability and more frequent emergency department visits and hospitalizations. This is particularly true 

among people of color and low-income seniors,

 for whom health disparities severely impact outcomes and 

While the outlook may seem grim, there are care models that provide solutions to these access challenges and health inequities. One effective approach is the value-based, senior-focused primary care, like that practiced at 

 from Harvard University and Humana Healthcare Research shows that, compared to traditional fee-for-service primary care settings, this model expands access to care, especially for Black and low-income patients. Primary care visits increased 17% among Medicare Advantage (MA) beneficiaries overall, and among Black and low-income MA members by 39% and 21%, respectively. The research, which examined six different senior-focused primary care organizations and more than 462,000 patients, also suggests that those utilizing these healthcare practices may have better compliance with recommended cancer screenings and medications, better controlled blood pressure and diabetes, and lower hospital use. 

These results confirm the benefits we see patients experience daily at our more than 300 primary care centers, many of which are located in underserved areas where the need for accessible and quality health care is great. Using a value-based, care team approach, our physicians have smaller patient panels and spend more time with patients – 50% longer than typical doctor visits. That extended time builds trust and communication, enabling a better understanding of the circumstances impacting the patient’s health and a personalized treatment plan based on the patient’s life and health goals. This team-oriented model also allows us to address the socioeconomic and mental health factors impeding their well-being, with social workers available to connect patients to community services, such as food and housing assistance, and to mental health practitioners.

The need for and success of senior-focused primary care is evident in the expanding network of CenterWell and Conviva clinics which now deliver care to approximately 345,000 seniors across 15 states, making them the largest senior-focused, value-based primary care providers in the country. To increase access to primary care services specifically designed for older adults even further, we have plans to open CenterWell and Conviva clinics at 23 Walmart Supercenter retail stores in Florida, Georgia, Missouri and Texas.

With the dramatic increase in our senior population, the importance of accessible, quality health care for older adults has never been more critical. Value-based, senior-focused primary care is proving to be an effective approach, delivering the comprehensive care and support services seniors need, especially those living in underserved communities.

Value-Based, Senior-Focused Primary Care is Transforming Healthcare for Older Adults

Immigration, the economy and character of the candidates dominated the presidential election that former president Donald Trump won handily yesterday. Healthcare issues stayed largely in the background, with the exception of abortion and, over the final weekend, some statements made by Robert F. Kennedy Jr. about fluoridation.

“With no big health reform debate to command the attention of the nation and no big health proposal from either candidate, this is not a ‘healthcare election,’” Drew Altman, president and CEO of KFF, wrote in mid-October.

But now that Trump has won and the Republicans have gained control of the Senate (the House is still in doubt), attention is turning to how the new administration might reshape federal healthcare policies and programs and revisit long-held predicates of public health priorities and policies.

The futures of the Affordable Care Act (ACA), the signature achievement of the Obama administration, and the Inflation Reduction Act (IRA), the signature healthcare achievement of the Biden administration, are now cloudier than they would have been in a Harris administration, although that may less true for the  IRA than the ACA. One near-term issue is the enhanced ACA premium subsidies that greatly reduced the premium cost of individual coverage purchased on the ACA exchanges. Congress must decide next year whether to continue them or not. 

In the final week of the campaign, Republican House Speaker Mike Johnson made a remark in an exchange at one campaign event that seemed to suggest he was in favor of replacing the ACA. Several days later in an interview he said that was a misinterpretation. “We are laser-focused on improving healthcare, as we are every area. We need higher quality care in many parts of the country, we need greater access, we need lower costs,” Johnson said. 

Johnson, a Louisiana Republican, referenced the 

 as a source of healthcare ideas. In the current Congress, the caucus consists of 16 Republicans physicians, pharmacists and dentists and is chaired by Rep. Andrew Harris, Maryland’s only Republican congressman and an obstetric anesthesiologist.

 on the what the Trump presidency will mean for healthcare next Thursday, Nov. 14, at 12 noon ET. 

In the Senate, Sen. Bill Cassidy, a gastroenterologist, is expected to play a prominent role in the development of any healthcare legislation. He is expected to chair the Committee on Health, Education, Labor and Pensions (HELP), a position currently held by Sen. Bernie Sanders, the progressive independent from Vermont.

Lindsay Bealor Greenleaf, J.D., MBA, vice president and a state and federal health policy expert for ADVI Health, said in an interview today that the Republicans have moved on from outright repeal of the ACA. "It is way too entrenched," she said. She foresees, though, curtailment of federal spending on the program, which might include a trimming of premium subsidies, and more flexible benefit design. 

Any influence that Cassidy and Harris may have on Trump administration changes to the ACA may pale in comparison to the disruptive, attention-grabbing role of Kennedy if Trump follows through on statements he made on the campaign trail. “I am going to let him go wild on health. I am going to let him go wild on the food. I am going to let him go wild on medicine,” Trump said about Kennedy at his campaign at a Madison Square Garden rally on Oct. 27.

In a video call with his supporters early last week, Kennedy said Trump had promised him “control of the public health agencies, which are HHS and its subagencies, CDC, FDA, NIH and few others and then also USDA.” Trump campaign officials pushed back against a promise and cabinet position for Kennedy. 

In an interview conducted in a townhall format on NewsNation, a cable television network, Kennedy said Trump had asked him “to reorganize” the federal agencies that deal with human health, including the CDC, NIH, the FDA, and as well as some agencies in the USDA.” 

Regardless of his exact role, Kennedy seems positioned to be a high-profile figure in the Trump administration. In the NewsNation townhall, Kennedy said Trump had asked him to clear up the corruption and end the conflicts of interest and “return these agencies to their rich tradition of gold-standard, empirically based, evidence-based science” and to “measurably reduce chronic disease in our children in two years.”

"I think it is the hot topic to watch," Greenleaf said of Kennedy's role in the administration. The Trump-Kennedy relationship is new and one that healthcare interest group and policy experts are still trying to figure out. A position requiring Senate confirmation may be out of the question, she noted, but even having a role in the White House, Kennedy could be significant. Kennedy might be put in charge of a commission or committee investigating the causes of chronic disease, particularly among children, she said.

Kennedy, who endorsed Trump in August after suspending his own independent bid to become president, is championing a “Make America Healthy Again,” or MAHA, campaign that plays off of Trump’s Make America Great Again slogan and movement. On his MAHA website, Kennedy says the goal is to rid public health agencies of the pharmaceutical industry influence, ban food additives and change polices to reduce ultraprocessed food. The website has merchandise for sale, including a $35 embroidered baseball cap that says "Make America Healthy Again." 

On Nov. 2, Kennedy caused a stir with a social media post that revived fluoridation of public water supplies as a controversy. On the first day of a Trump administration, the White House would advise U.S​. water systems to remove fluoride from the public water supply, Kennedy wrote. “Fluoride is an industrial waste associated with arthritis, bone fractures, bone cancer, IQ loss, neurodevelopmental disorders, and thyroid disease,” his post said. 

Public health and dental organizations have defended fluoridation as important to oral health.

Patrick Cooney, of The Federal Group who has decades of Washigton, D.C. experience as lobbyist and as a congressional staff earlier in his career, noted that the power has swung back and forth between Democrats and Republicans for several elections. The 2024 result highlights that the "public continues to want significant change in the economy to impact their daily lives," Cooney said.
"Over the next several months the new Trump Administration and the 119th Congress will get on with the business of governing," Cooney said, "As that happens, we will have a clearer picture of where healthcare regulation, cost, and access to care issues will head."

The future of the Affordable Care Act looks cloudier but the hot topic is the role that Robert F. Kennedy Jr. may play in the new administration. 

A Hot Take: What the Trump Win Will Mean for U.S. Healthcare

The FDA has proposed that nonprescription drugs that contain phenylephrine be removed from pharmacy shelves. Phenylephrine is a nasal decongestant used in nonprescription cold products, such as Mucinex and Sudafed and other products, that the regulatory agency says is not effective.

“Based on our review of available data, and consistent with the advice of the advisory committee, we are taking this next step in the process to propose removing oral phenylephrine because it is not effective as a nasal decongestant,” Patrizia Cavazzoni, M.D., director of the FDA’s Center for Drug Evaluation and Research (CDER), said in a 

The agency conducted a review of all available data on the safety and efficacy of oral phenylephrine, including the historical data that were used to support the determination made 30 years ago that oral phenylephrine was effective.

The use of phenylephrine in nonprescription cold medications has increased since 2006, when sales of any products containing pseudoephedrine were placed behind the pharmacy counter. Pseudoephedrine can be used to make methamphetamine. Congress, as an amendment to the Patriot Act, the 

, began requiring photo identification and a log of sales of pseudoephedrine-containing products.

In response, many manufacturers of cold products reformulated their products with phenylephrine instead of phenylpropanolamine, ephedrine, and pseudoephedrine.

But research over many years has questioned the effectiveness of phenylephrine as a nasal decongestant. In fact, 

 on research done in the 1970s suggested that phenylpropanolamine and pseudoephedrine were effective decongestants but that oral phenylephrine was not. (

 is no longer available because it was found to be associated with strokes.)

, researchers found that oral phenylephrine was not significantly more effective than a placebo in alleviating nasal congestion in adults. Researchers conducted a systematic review involving searches on PubMed, Cochrane, and Scopus databases and identified four articles that compared oral phenylephrine with placebo or standard care and their effect on congestion and nasal airway resistance.

Two pharmacists, Randy C. Hatton, Pharm.D., and Leslie Hendeles, Pharm.D., both from the College of Pharmacy at the University of Florida, began their research of phenylephrine in 2005 and eventually filed two petitions with the FDA for a review of the evidence and additional studies. Their efforts led the FDA to convene two advisory committee meetings, the most recent being in October 2023. All 16 members of the 

 on the question of whether the data support that orally administered phenylephrine is effective as a nasal decongestant.

, Hatton pointed out that phenylephrine was first approved before 1962 and later available for over-the-counter use in 1976. The modern FDA was created by the 1962 Kefauver-Harris Amendments, which created the structure for well-controlled clinical trials in people with a focus on safety and efficacy. Prior to this, manufacturers did not have to prove a new drug was effective, and chemistry studies were enough to show safety.

“But what about the drugs that were approved before 1962? This window has become a loophole that some OTC drugs fall through,” Hatton wrote in the 

 article. “For nonprescription drugs, 10 years after the 1962 amendment to the Food, Drug and Cosmetic Act, the 

FDA established the OTC monograph process

, which required products that hadn't been proved effective to be reconsidered.”

For now, the FDA says products that contain phenylephrine are still available. But the agency is seeking comment on the proposed order through May 7, 2025, and will then issue a final order on phenylephrine.


An FDA advisory committee last year agreed that the data do not support phenylephrine as an effective nasal decongestant.


Brian Robertson

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