Prior authorization is a complex, lengthy process that stands between patients and the drugs they need. In a panel at the Academy of Managed Care Pharmacy 2023, Heather Odem, PharmD, of UnitedHealthcare and Richard Ogletree, PharmD, of Alliant Health Solutions spoke about the need to shift measures […]
NICE Recommends Novel Gene Therapy for Rare Genetic Brain Disorder
The UK’s National Institute for Health and Care Excellence (NICE) has issued a draft guidance recommending the approval of Upstaza, PTC Therapeutic’s gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency. The gene therapy, if approved, would cost £3 million and would be the only […]
Patient Advocacy Groups Decry NIH Refusing to Exert March-In Rights for Prostate Cancer Drug Xtandi
Patient advocacy groups are speaking out after the US National Institutes of Health (NIH) decided against exercising march-in rights, which allows the government to share patents developed with public funds, on Pfizer’s and Astella’s prostate drug Xtandi. The move would’ve lowered prices for the […]
India Shoots Down Application for J&J’s Tuberculosis Drug Patent Extension
Johnson & Johnson notched a loss today as India’s patent office refused to extend the patent on bedaquiline, a drug for treatment-resistant tuberculosis. The company was looking to extend the patent to 2027, but the patent office’s refusal means J&J will lose the patent by July, ending the […]
Improving Clinical Trials for Rare Disease Therapeutics
The decades old Orphan Drug Act helped spur a renaissance in rare disease research, incentivizing development therapeutics that may otherwise be deemed unprofitable. However, factors like small, spread-out patient populations and funding considerations make running clinical trials for rare diseases […]
Cidara’s Anti-Fungal Receives Approval with Priority Review from US FDA
Rezzayo, Cidara Therapeutics’ newest anti-fungal drug, has received the nod from the US Food and Drug Administration (FDA) for approval under priority review for two kinds of candida infections, candidemia and invasive candidiasis. The drug is currently being reviewed by the EMA, which has already […]
Real-World Evidence Explores Inpatient and Outpatient Delivery of CAR T-Cell Therapy
Real-world evidence (RWE) presented at the 2023 Transplantation and Cellular Therapy Meeting compared and contrasted outcomes for patients receiving inpatient vs. outpatient CAR T-cell therapy for mantle cell lymphoma (MCL) and follicular lymphoma (FL). Patients with FL fared better with outpatient […]
Rare Skin Cancer Drug by Incyte Receives Accelerated Approval
Incyte notched a win today, March 23rd, with accelerated approval for retifanlimab, its treatment for a rare, aggressive skin cancer called Merkel cell carcinoma. The approval comes after patients single-arm study achieved a 52% response rate, with 18% seeing a complete response. These responses […]
Improving Clinical Trials for Rare Disease Therapeutics
With millions worldwide living with rare diseases that have few or no current therapeutic options, clinical trials are essential in treating the conditions faced by this diverse patient population. However, running these trials are expensive, hard to recruit for, and often require novel trial […]
Biogen’s ALS Drug Receives Unanimous Recommendation Accelerated Approval from FDA Adcomm
Tofersen, Biogen’s ALS Drug, has received a unanimous 9-0 recommendation for accelerated approval by the US Food and Drug Administration’s Peripheral and Central Nervous System Drugs Advisory Committee. The move comes in spite of results from a Phase III trial in which the drug failed to meet its […]
Value-Based Care as a Solution for Healthcare Workforce Shortages
Widespread healthcare workforce shortages and high employer-sponsored health insurance costs are impacting health systems and increasing healthcare costs. One potential solution for this problem is value-based care (VBC), which bases reimbursement on quality of care and patient outcomes. According […]
CMS Approves Coverage for VR Pain Relief Solution
In the first approval of its kind, the US Centers for Medicare and Medicaid Services (CMS) have approved AppliedVR’s RelieVRx immersive virtual reality (VR) platform as a durable medical device. This opens up funding for not only RelieVRx, but also other therapeutic immersive VR programs. With the […]
FTC Says Parkinson’s Drug Patent Fight is Major Case for Patients
A case between Supernus and Sage Chemical over the Parkinson’s disease drug Apokyn has far-reaching implications for patients, according to the US Federal Trade Commission. Sage’s generic version of the drug was approved in 2022, but patients could not access it because approval does not cover the […]
HHS Shoots Down Call to Use March-In Rights for Xtandi
In a win for Pfizer and Astellas, the US Department of Health and Human Services decided against using march-in rights to lower the price of Xtandi, a blockbuster prostate cancer drug. Exercising march-in rights would the government to share patents made using public funds with third-party […]
Questioning the Detrimental Impacts of QALY in Health Policy
While disability advocates and lawmakers have decried the use of quality-adjusted life years (QALY) in recent years, some argue that it is an indispensable measure that is unfairly maligned. For example, patient advocates argue that the focus on quality of life devalues the lives of disabled people […]
Study Offers Recommendations for China’s Biosimilar Regulations
A recently published paper in Frontiers in Pharmacology examines the regulatory framework surrounding biosimilars in China. The researchers noted that the regulatory system is not “clearly formed” and is ill suited to support the nearly 400 biosimilars in development in the country. The paper offers […]
Eisai CEO Not too Worried About Leqembi After CMS Keeps Anti-Amyloid Rule
Eisai’s CEO Ivan Cheung told Fierce Pharma he is not too worried about the long-term success of Leqembi in light of the US Centers for Medicare and Medicaid Services (CMS) declined to change its rule about reimbursement for drugs in its class. The rule states that drugs targeting amyloid, a key […]
Takeda Reveals Pricing for Dengue Vaccine
Fresh off approval of its dengue vaccine in Brazil, Takeda has announced its pricing strategy for the shot. Prices will be determined by endemic status of the disease in a given country. Indonesia, where the virus is endemic, will see the vaccine priced at $40 per dose, while travel markets like […]
Exploring the Systemic Causes of Maternal Mortality in the US
A 2022 report by the US Centers for Disease Control and Prevention (CDC) explored the underlying causes of high maternal mortality in the US. Over 75% of maternal deaths happen after returning from the hospital after delivery and 80% were preventable. A new article in Health Affairs, explains how […]
Ozempic and Mounjaro Back on Shelves
Novo Nordisk’s Ozempic and Eli Lilly’s Mounjaro, both diabetes drugs used off-label for weight loss, have been on a backlog for months due to an explosion of popularity. According to the US Food and Drug Administration, most doses of the two drugs are back in stock. This excludes the higher doses of […]
Using AI to Streamline Rare Disease Diagnosis
It can take years for patients with rare diseases to get a proper diagnosis, often worsening their condition in the process. Artificial Intelligence (AI) may represent a way to help streamline proper diagnoses of rare diseases, improving patient outcomes in the process. In a new pharmaphorum article […]
US Congress Preps for Health Spending Battle
As US Republicans ramp up their ongoing fight to cut government spending, Department of Health and Human Services (HHS) Secretary Xavier Beccera will stand before the Senate Finance Committee this Wednesday. Republicans have their sights set on a proposed policy by the Centers for Medicare and […]
Linda Goler Blount, MPH, to Join ICER Governance Board
Linda Goler Blount, MPH, has been elected to join the governance board of the Institute for Clinical and Economic Review. Blount has worked in health policy and is currently the president of the Black Women’s Health Imperative. She has built a career on addressing health disparities, especially […]
US FDA Still Mulling Over Biogen ALS Treatment Approval Despite Disappointing Phase III Results
Briefing documents from a US Food and Drug Administration (FDA) advisory committee show that Biogen’s ALS drug tofersen is still in the running for approval despite a recent failure in a Phase III trial. The favorable outlook is due, in part, due to its effects on reducing a key protein hallmark of […]
US FDA to Review Ipsen’s Rare Bone Growth Disease Therapy
The US Food and Drug Administration (FDA) has set a date to decide on Ipsen’s new drug application for its drug candidate palovarotene in the treatment of fibrodysplasia ossificans progressive (FOP), a rare disorder where extra-skeletal tissues like muscles and tendons are converted into bone […]
Connecting Real-World Data and Domain Expertise to Enhance Trial Design and Planning
The clinical trial landscape has evolved in such a way that it’s more challenging to find sites and patients than ever before. Data and analytics combined with deep therapeutic and operational expertise can bring therapies to patients faster. In this webinar, therapeutic and data science experts […]
FDA Adcomm Recommends Paxlovid for Mild-to-Moderate COVID-19
Pfizer’s Paxlovid has received a nod from the US Food and Drug Administration’s (FDA’s) Antimicrobial Drug Advisory Committee for use in mild-to-moderate cases of COVID-19 in adults. The 16-1 vote came after Pfizer’s previous request for full approval hit a roadblock last year when the agency […]
Exclusivity Loss Looms for Ten Key Drugs in 2023
As the year marches on, Fierce Pharma takes a look at 10 major drugs facing exclusivity losses in 2023. One of the most notable is Humira, AbbVie’s historically expensive blockbuster, which will face stiff competition from several upcoming biosimilars. Other drugs on the list include J&J’s […]
Housing Instability Worsens Health Outcomes
Astronomical rent and home prices are driving the ever-worsening affordable housing crisis. A newly published article in JAMA Network Open explores the link between housing instability and health outcomes, finding that displacement and unsafe living situations related to unaffordable housing are […]
Sanofi Cuts Insulin Prices and Sets Out-of-Pocket Price Cap
Hot on the heels of Novo Nordisk and Eli Lilly, Sanofi announced it will cut the price of its main insulin formulation, Lantus, in January of next year. Just outstripping the others, the cuts amount to a 78% price drop for the life-saving drug used by millions of people with diabetes. In addition, […]
