Betibeglogene autotemcel for beta thalassemia: effectiveness and value : final evidence report
- Collection:
- Health Policy and Services Research
- Author(s):
- Beaudoin, Francesca L., author Richardson, Marina, author Synnott, Patricia G., author Lancaster, Victoria, author Fluetsch, Noemi, author Herce-Hagiwara, Belén, author Campbell, Jon D., author Pearson, Steven D., author Rind, David M., author
- Contributor(s):
- Institute for Clinical and Economic Review, issuing body. New England Comparative Effectiveness Public Advisory Council, issuing body.
- Publication:
- [Boston, Massachusetts] : Institute for Clinical and Economic Review, July 19, 2022
- Language(s):
- English
- Format:
- Text
- Subject(s):
- beta-Thalassemia -- therapy Biological Products -- therapeutic use Genetic Therapy United States
- Genre(s):
- Technical Report
- Abstract:
- Beta-thalassemia is a rare blood disorder with the potential for high morbidity and mortality when treated suboptimally. Transfusion-dependent thalassemia (TDT), the most severe form of this disease, is managed through lifelong regular blood transfusions and iron chelation therapy to avert the consequences of iron overload. There are likely between 1,000 – 1,500 people in the US living with TDT, but there are estimated to be about 1.25 million carriers of the genetic defect that is responsible for thalassemia. Until recently, the only curative option for TDT was allogenic hematopoietic (blood) stem cell transplantation (HSCT) from a matched donor, ideally a sibling. HSCT requires high doses of conditioning chemotherapy and places the recipient at risk of complications associated with HSCT itself (e.g., graft vs. host disease) and finding an HSCT match is difficult as fewer than 25% of patients have access to a suitable match. Life expectancy still lags far behind population norms even with improved treatments: from 2011 to 2021 the median age of death for a person in the US with TDT was 37. Additionally, patients with TDT still report decreased quality of life due to the impact on physical and mental health. Patients and clinicians reported to us that living with severe forms of beta thalassemia requires being “tethered to the health care system” and often to a specific region near a medical center that can provide thalassemia care. Some patients receive transfusions as often as every two weeks, and nearly all patients plan their lives around transfusions. Regular transfusions and chelation can be technically challenging in young children, causing stress in patients and caregivers. Betibeglogene autotemcel (beti-cel), manufactured by bluebird bio, is a gene therapy that provides an additional potentially curative option for patients with TDT. Beti-cel is manufactured ex vivo utilizing an individual’s own hematopoietic stem cells (HSCs). A lentiviral vector (BB305) is then used to add functional copies of the β-globin gene (βA-T87Q) to patients’ HSCs. The modified HSCs (beti-cel) are then infused intravenously back into the individual following conditioning chemotherapy. The Food and Drug Administration (FDA) accepted bluebird bio's Biologics License Application (BLA) of beti-cel for priority review on November 22, 2021, and the revised PDUFA date is set for August 19, 2022 (originally May 20, 2022). Public statements made by the manufacturer suggest that beti-cel pricing will be consistent with an outcome-based payment plan of five equal yearly payments totaling $2.1 million for individuals who achieve and maintain transfusion independence.
- Copyright:
- Reproduced with permission of the copyright holder. Further use of the material is subject to CC BY license. (More information)
- Extent:
- 1 online resource (1 PDF file (various pagings))
- Illustrations:
- Illustrations
- NLM Unique ID:
- 9918506283406676 (See catalog record)
- Permanent Link:
- http://resource.nlm.nih.gov/9918506283406676