Observational real-world evidence update: prophylaxis of hereditary angioedema with Takhzyro and C1 inhibitors : effectiveness and value
- Collection:
- Health Policy and Services Research
- Author(s):
- Bloudek, Lisa, author Jaksa, Ashley, author McKenna, Avery, author Carlson, Josh, author Chen, Yilin, author Patrick, Amanda, author Campbell, Jon D., author
- Contributor(s):
- Institute for Clinical and Economic Review, issuing body.
- Publication:
- [Boston, Massachusetts] : Institute for Clinical and Economic Review, August 24, 2021
- Language(s):
- English
- Format:
- Text
- Subject(s):
- Angioedemas, Hereditary -- prevention & control Complement C1 Inhibitor Protein United States
- Genre(s):
- Technical Report
- Abstract:
- In 2018, The Institute for Clinical and Economic Review (ICER) conducted a review of lanadelumab (Takhzyro®, Takeda Pharmaceutical Company, Ltd.) and two C1 inhibitors (Haegarda®, CSL Behring, GmbH; and Cinryze®, Takeda Pharmaceutical Company, Ltd.) for long-term prophylaxis against acute attacks in patients with hereditary angioedema (HAE). The primary objective of this analysis is to update the prior estimation of the cost effectiveness of Takhzyro, Haegarda, and Cinryze using recent observational real-world evidence (RWE). This work is a pilot project to explore how ICER can update review topics using observational RWE, with an emphasis on therapies that have been approved through accelerated approval pathways and are in use for over two years. HAE is a rare genetic disorder that causes painful attacks of swelling in the face, hands, feet, and stomach, as well as potentially life-threatening swelling of the throat. Most HAE is caused by a deficiency (Type I HAE) or dysfunction (Type II HAE) of a protein called C1 inhibitor (C1 esterase inhibitor, C1-INH). Attacks can last for up to five days, and can be spontaneous or triggered by stress, medical procedures, and certain medications like oral contraceptives or ACE inhibitors. Attacks can occur rarely or as often as once every few days. Because of their severity and unpredictability, attacks can significantly reduce a patient’s functioning and ability to perform activities of daily living. The goal of HAE treatment is to reduce the duration, frequency, and severity of attacks. Ondemand treatments are used to reduce the duration and severity of a single attack. Long-term prophylactic treatments, the focus of the 2018 report1 and this update, are taken regularly to prevent attacks and reduce attack severity. ICER’s 2018 report found that long-term prophylaxis with either of the C1 inhibitors or Takhzyro resulted in fewer acute attacks and improved quality of life for people living with HAE, but 2018 pricing of all three treatments exceeded traditional costeffectiveness thresholds. The 2018 report identified uncertainties in the evidence and key model assumptions that influenced the cost-effectiveness findings. One of the most consequential uncertainties in the 2018 model was the frequency and severity of attacks at baseline among patients who would be prescribed prophylactic treatment. As demonstrated in the 2018 report, small differences in the assumed attack rate resulted in a wide range of cost-effectiveness results. This report was developed specifically to pilot-test the impact of leveraging observational RWE to update ICER reviews of drugs initially approved through the accelerated approval pathway. This report does not include an assessment of berotralstat, a recently approved prophylactic treatment that was not included in the 2018 Report.
- 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:
- 9918401082906676 (See catalog record)
- Permanent Link:
- http://resource.nlm.nih.gov/9918401082906676