The National Institute for Health and Care Excellence (NICE) has issued a final draft guidance recommending the approval of ruxolitinib (Jakafi) as a treatment for adult patients with polycythemia vera that is intolerant or resistant to hydroxycarbamide or hydroxyurea, according to a press release from Novartis.1 This may help to make the agent available for patients residing in England and Wales.
“We welcome this recommendation from NICE, as polycythemia vera can be an extremely debilitating illness that has a significant impact on patients’ lives in terms of day-to-day symptoms,” Jon Mathias, co-chair of MPN Voice, said in the press release. “Ruxolitinib addresses a significant unmet need in patients who cannot tolerate or no longer respond to [hydroxycarbamide/hydroxyurea].”
According to a previous report, treatment with hydroxycarbamide or hydroxyurea leads to the development of resistance or intolerance in 24% of patients with polycythemia vera, which correlates with a higher risk of disease progression.2
In an international MPN Landmark survey, 72% of patients with polycythemia vera reported that they experienced reduced quality of life (QOL) due to disease symptoms.3 Additionally, 14.0% of surveyed patients with polycythemia vera reported that they were experiencing emotional hardship due to their condition, and 29.0% stated that they felt worried or anxious about their disease. A further 33.0% of the surveyed polycythemia vera population stated that they experienced impairment at work, and 40.3% reported impairment with respect to overall activity.
“There is a significant unmet need for people with polycythemia vera in England and Wales, who live with a large symptom burden as a result of their condition,” Claire Harrison, MD, FRCP, FRCPath, consultant hematologist at Guy’s and St Thomas’ NHS Foundation Trust in London, said in the press release.1 “Today’s decision is a step in the right direction for providing additional treatment options that reduce the burden of these symptoms and improve disease progression, in this under-represented patient population.”
Investigators sent the MPN LANDMARK survey to patients with polycythemia vera, myelofibrosis, and essential thrombocythemia, and physicians across the United Kingdom, Australia, Germany, Canada, Japan, and Italy from April 2016 to October 2016. The survey was designed to evaluate how MPNs affected QOL, patients’ ability to work, and implementation of disease-management strategies.
Patients 18 years and older diagnosed with myelofibrosis, polycythemia vera, or essential thrombocytopenia were able to respond to the survey. Those enrolled on clinical trials were not eligible to take the survey.
Overall, 219 physicians and 699 patients—including 174 with myelofibrosis, 223 with polycythemia vera, and 302 with essential thrombocythemia—completed the survey.
The FDA approved ruxolitinib as a treatment for patients with polycythemia vera who are intolerant to hydroxyurea in December 2014.4 Supporting data for the FDA approval came from the phase 3 RESPONSE trial (NCT01243944), in which treatment with ruxolitinib produced improvements in hematocrit control and spleen volume reductions compared with best available therapy. Additionally, a higher number of patients receiving ruxolitinib experienced complete hematologic remission. Investigators reported that the most common hematologic adverse effects included thrombocytopenia and anemia.
References
- NICE recommends Novartis Jakavi® (ruxolitinib) for patients living with polycythaemia vera (PV). News release. Novartis. September 14, 2023. Accessed September 15, 2023. https://shorturl.at/hixAD
- Alvarez-Larran A, Pereira A, Cervantes F, et al. Assessment and prognostic value of the European LeukemiaNet Criteria for clinicohematologic response, resistance, and intolerance to hydroxyurea in polycythemia vera. Blood. 2012;119(6):1363-1369. Doi:10.1182/blood-2011-10-387787
- Harrison CN, Koschmieder S, Foltz L, et al. The impact of myeloproliferative neoplasms (MPNs) on patient quality of life and productivity: results from the international MPN Landmark survey. Ann Hematol. 2017;96(10):1653-1665. doi:10.1007/s00277-017-3082-y
- FDA approves Jakafi® (ruxolitinib) for the treatment of patients with uncontrolled polycythemia vera. News release. Incyte Corporation. December 4, 2014. Accessed September 15, 2023. https://shorturl.at/aARU3