Most breast cancer patients who obtained the government’s approval for compassionate use of non-reimbursable drugs had gBRCA or PIK3CA mutations, signaling that the government should expand access to novel medicines.
According to the Ministry of Food and Drug Safety’s integrated drug information system, the MFDS approved 61 cases for compassionate use of therapies to treat breast cancer this year as of Monday.
Nearly half (28) of the compassionate use involved Pfizer’s PARP inhibitor Talzenna (talazoparib), and 22, Novartis’ PIK3Caα inhibitor Piqray (alpelisib).
Pfizer’s Talzenna won the marketing approval on July 30, 2020, but national health insurance does not cover it.
Pfizer applied for reimbursement for Talzenna immediately after obtaining the license, but the Health Insurance Review and Assessment Service (HIRA) refused to grant it in November.
AstraZeneca’s PARP inhibitor Lynparza (olaparib) is available in Korea, but it is not reimbursable in breast cancer treatment.
The company applied for reimbursement for Lynparza in breast cancer when it changed the drug from capsule to tablet last year, but the HIRA did not approve it.
Piqray is not reimbursable in Korea, either.
On May 13 last year, the treatment won the nod as combination therapy with fulvestrant in advanced or metastatic breast cancer in postmenopausal women and men who are hormone receptor-positive (HR+), human epidermal growth factor receptor 2 (HER2)-negative, and PIK3CA mutation-positive with disease progression after prior endocrine therapy.
Piqray is the only targeted therapy for PIK3CA mutated patients, who account for 40 percent of HR+/HER2- patients. But winning reimbursement has become more difficult because the standard treatment changed during the development of Piqray.
As the first-line standard of care for HR+/HER2- patients was changed to a combo of endocrine therapy and CDK4/6 inhibitor, there is no way for Novartis to establish evidence for a phase 3 clinical trial in patients previously treated with CDK4/6 inhibitors.
Instead, Novartis continuously produces data that proved Piqray’s efficacy in patients who used CDK4/6 inhibitors in phase-2 trials. Still, whether the HIRA will recognize the data as reliable evidence is unclear.
Under such circumstances, if Korean breast cancer patients with gBRCA or PIK3CA mutation want to get those targeted therapies, they should use them without insurance coverage or get compassionate use approval with the help of doctors.
To get the nod for compassionate use, a hospital has to spend a considerable amount of money on paperwork and manpower for follow-up. If a hospital does not have such resources, applying for compassionate use approval isn't easy. Thus, health experts said only a certain number of large hospitals in Korea could use this system.
As the MFDS’ latest drug information showed a clear demand for targeted therapies in breast cancer patients with gBRCA or PIK3CA mutation, the health authorities will be under pressure to make targeted therapies reimbursable to manage those patients, observers said.