This episode of tHEORetically Speaking features real-world evidence experts Alexandrina Balanean, Parisa Asgarisabet, and Danielle Gentile from Cardinal Health discussing the problems caused by lack of diverse representation in clinical trials.
In 1983, the US Congress passed the Orphan Drug Act (ODA) that offers financial incentives for developing drugs for rare diseases that have limited, if any, approved therapeutics. A new study published in the Orphanet Journal of Rare Diseases explores the impact that the ODA has had on the field of rare diseases over the past 40 years.
The Institute for Clinical and Economic Review has received industry backlash over changes to its value-assessment process it proposed in June. The changes, if implemented, would lower the threshold ranges for cost-effectiveness down to $50,000 to $100,000 a year, a net drop of $50,000 at each end of the range. The outgoing ICER president Steve Pearson noted that these changes are not going to be immediately implemented and are still under consideration.
A newly published study in the BMJ finds that almost 60% of drugs receiving new indications from the US Food and Drug Administration (FDA) do not add high therapeutic value to patients. As a result, the researchers argue that patients and providers should be aware and that prices should be set accordingly.
Eisai and Biogen’s Alzheimer’s treatment Leqembi (lecanemab) has received full approval by the US Food and Drug Administration (FDA). This marks the first full approval for an Alzheimer’s disease treatment in decades and clears the path to Medicare reimbursement, which previously required providers contribute data to a patient registry.
US Senator Bernie Sanders (I – VT) is looking to include price caps on drugs developed with federal funding into a reauthorization of the 2006 Pandemic and All-Hazards Preparedness Act. The proposal would place a ceiling price that matches the lowest price for a given drug in France, Germany, Japan, Canada, the UK, or Italy.
The Institute for Clinical and Economic Review (ICER) will perform a value-assessment on KarXT (xanomeline tartrate/trospium chloride), Karuna Therapeutics’ experimental therapeutic for schizophrenia. Public discussion of the assessment will take place next February at the 2024 New England CEPAC meeting.
Amicus Therapeutics has received approval from the European Commission (EC) for Opfolda (miglustat), its treatment for Pompe disease, a rare disorder that causes progressive muscle weakness. The approval specifically covers the drug in combination with Pombiliti (cipaglucosidase alfa). Results from a recent phase 3 study showing positive changes in mobility and breathing, both affected by the disorder, supported the EC’s decision.
BioMarin Pharmaceuticals has received full approval by the US Food and Drug Administration (FDA) for its gene therapy Roctavian (valoctocogene roxaparvovec-rvox) for severe hemophilia A in adults. This marks the first gene therapy for patients with severe cases of the disease, which causes bleeding and progressive joint damage.
India’s National Pharmaceutical Pricing Authority (NPPA) has announced its latest round of ceiling prices for medications, this time capping the prices of 51 medications and 2 formulations for anti-coagulant therapeutics. The other drugs covered include 24 diabetes treatments. The NPPA is India’s key drug pricing regulatory body, in charge of setting ceiling prices for medications and formulations.
The Institute for Clinical and Economic Review (ICER) has announced that its founder, Steve Pearson, MD, will step down from his role as president later this year. ICER’s COO Sarah Edmond will take over the position as president, while Pearson will move to an advisory position for the organization, which performs value-assessments on therapeutics.
OPKO Health and Pfizer’s Ngenla, a treatment for childhood growth hormone deficiency, has received approval by the US Food and Drug Administration (FDA). The treatment for the rare disease, which causes short stature and delayed puberty, had been rejected once by the agency with a request for more information.
One of the key therapeutic targets in Alzheimer's disease is amyloid, a sticky protein that builds up in clumps outside neurons, causing cell death and cognitive decline. However, new therapeutics targeting the protein have only shown mild efficacy in delaying disease progression. As a result, drug developers are looking for other biological targets.
Drug shortages have rocked the US, imperiling access to therapeutics for millions of patients with a wide array of diseases and conditions. A new Becker’s Hospital Review article lays out 7 of the most prominent drug shortages to watch. One is the attention deficit hyperactivity disorder (ADHD) drug Vyvanse (lisdexamphetamine dimesylate).
The UK’s National Institute for Health and Care Excellence (NICE) has sent a request to Eli Lilly asking for more data regarding Mounjaro (tirzepatide) before it will cover it for NHS patients. The decision comes based on data comparing the drug’s efficacy to Novo Nordisk’s rival drug Ozempic and standard insulin therapy. While the data showed Mounjaro was more effective at weight loss and glucose control, NICE needs more information before making a coverage decision.
UCB’s Rystiggo (rozanolixizumab-noli), a treatment for two subtypes of generalized myasthenia gravis (gMG), has been approved by the US Food and Drug Administration (FDA). Muscle weakness episodes in the rare autoimmune disease is caused by antibodies that cling to proteins at the connection site between nerves and muscles. Rystiggo, an antibody-based treatment, reduces circulating antibodies and reduces symptoms.
The US Food and Drug Administration (FDA) has approved Pfizer’s Litfulo (ritlecitinib) for use in adolescents with severe alopecia areata, an autoimmune condition that causes hair loss. The approval marks the first treatment for the psychologically damaging condition in teens. Positive results from a phase 2b/3 study, which included over 700 patients, supported the FDA’s decision..
The US Food and Drug Administration (FDA) has granted Roche accelerated approval for Columvi, its antibody-based therapeutics for two aggressive forms of lymphoma. Results from a phase 1/2 study found the drug provided durable remission for over half of patients and a complete response for 43%, with 66% continuing to respond for 9 months or longer.
Amgen’s supplemental biologics license application (sBLA) for its leukemia treatment Blincyto has received full approval by the US Food and Drug Administration (FDA). The move comes based on new data from two phase 3 trials, upgrading the treatment’s accelerated approval to a full approval in the treatment of CD19-positive B-cell precursor acute lymphoblastic leukaemia (B-ALL).
GSK’s Arexy and Pfizer’s Abrysvo vaccines for respiratory syncytial virus (RSV) have received recommendations from the US Centers for Disease Control and Prevention’s (CDC’s) Advisory Committee on Immunization Practices (ACIP). While the committee voted almost unanimously in support of the vaccines for adults aged 60-64, the recommendation for adults over 65 was far more contentious, sitting at 9-5 for and against, respectively.
The US Food and Drug Administration (FDA) has accepted bluebird bio’s application for priority review for its sickle cell gene therapy lovotibeglogene autotemcel (lovo-cel). The decision was made based on efficacy data from 36 patients and safety results from 50. Sickle cell disease (SCD) is an inherited blood disorder caused by a mutation in the gene encoding for hemoglobin that sticks together inside red blood cells.
New real-world evidence (RWE) presented at this year’s European Renal Association Congress explored hypokalemia care, finding that reduction or discontinuation of renin-angiotensin-aldosterone system inhibitor (RAASi) worsens outcomes of patients admitted for hyperkalemia (HK), high calcium levels in blood. RWE from the ZORA study shows that few patients have their RAASi medications reinitiated after a hyperkalemia event, which is associated with a two times increase in mortality.
Colchicine, an ancient treatment for gout, has been approved by the US Food and Drug Administration (FDA) for treating vascular inflammation present in patients with heart disease. The therapy, now produced by Agepha Pharma, is combined with a statin and reduces risk of serious cardiovascular events like stroke or heart attack.
Pharmacy benefit managers (PBM), which act as middlemen that help set formularies and drug prices, have faced a wave of US congressional scrutiny in recent months. This includes hearings, committees, and even legislation. In a new Mintz article, catch up on all the latest updates on federal activities targeting the industry and its practices.
In a study newly published in Health Affairs, authors use CMS figures to project US healthcare spending to exceed $7.2 trillion by 2031. This is based on an expected annual growth in spending of 5.4 percent by year. Although recent economic growth exceeded recent increases in healthcare spending, this trend is not expected to hold up long-term.