The UK’s National Institute for Health and Care Excellence (NICE) has issued a draft guidance recommending the approval of Upstaza, PTC Therapeutic’s gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency.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 treatment for the ultra-rare disorder.
Next-generation sequencing (NGS) is essential for advances in individualized medicine, but getting it to patients requires convincing insurers to pay for the technique. For this goal, care must be taken to ensure an accurate value-assessment process. In a new article in Value and Outcomes Spotlight, Lieven J.P. Annemans, PhD, discusses the barriers patients face accessing NGS and proposes a plan of action to reduce them.
Cell and gene therapies are set to explode over the next decade. However, reaching their full potential will take careful consideration to avoid the pitfalls in drug development, manufacturing, and market access. Tomorrow, August 24th, Endpoints News and Precision ADVANCE will host Cell & Gene Day, featuring a stellar lineup of talks from industry leaders. The first of three panels is entitled, “Future technologies for cell & gene therapy.”
Beta Thalassemia is a blood disorder that results in dangerously low levels of functional hemoglobin and requires expensive lifelong medical care, including regular blood transfusions. Beti-cel is a gene therapy by Bluebird Bio that can potentially cure the disorder by genetically modifying a patient's own hematopoietic stem cells (HSCs). The Institute for Clinical and Economic Review (ICER) has just released its assessment of Beti-cel, finding that it is effective and could save money on treatment, but more comprehensive long-term data would be necessary to determine the degree of the net health benefit over the current standard of care.
Despite the global pandemic, cancer was still the leading cause of death in 2020. This pressing therapeutic need underlies the growth projected by Kenneth Research in their new report entitled, “Global Gene Therapy Market.” The report predicts that the gene therapy market will earn around $6 billion USD from 2022-2031 with a CAGR of ~34%.