Abstract 81P
Background
Innovative targeted therapies are being developed rapidly, with new trials providing evidence almost continuously. Even though the European Medicines Agency (EMA) makes efforts to expedite their review and approval, individual countries have longer approval processes. Early Access Program (EAP) permits patients who have exhausted all treatment options to obtain innovative treatment.
Methods
Retrospective study of targeted therapy EAP requests submitted to INFARMED (National Authority of Medicines and Health Products I.P.) between 2020 and 2022 by an Oncology Department at a Portuguese Central Hospital.
Results
During the study period, 15 distinct targeted therapy drugs were approved from INFARMED: abiraterone, alpelisib, bevacizumab, cabozantinib, denosumab, encorafenib, everolimus, pembrolizumab/lenvatinib, neratinib, niraparib, pertuzumab, sacituzumab govitecan, trastuzumab-deruxtecan and tucatinib. Of the 107 approved requests, 53 (49.5%) were used in a neoadjuvant (NA), 2 (1.9%) in an adjuvant context, and 52 (48.6%) in metastatic disease. The most requested drugs were pertuzumab (n=53) for NA breast cancer (BC) with double HER2 blockade, trastuzumab-deruxtecan (n=7) for metastatic BC and abiraterone (n=23) in hormone-sensitive prostate cancer. Regarding NA double HER2 blockade, all patients were women, median age 52 (30-86) with median follow-up of 28 months. Two patients had recurrence: at 13 and 15 months. Median follow-up for metastatic patients was 14 months: 50% women, median age 63 (33-85). The impact of these therapies on patient survival was limited by the small number of patients treated for different indications, the lack of information regarding toxicity and impact on quality of life, making it difficult to compare them with clinical trials.
Conclusions
EAPs enable early access to therapies not yet funded by the national health system but approved byEMA, in situations with no therapeutic alternative, with risk to life or serious complications. Between 2020 and 2022, 107 patients benefited from EAPs, more than half with curative intent and 48.6% in metastatic disease. In an era where targeted therapy is constantly innovating, these programs play an important role in accessing these innovative, targeted, and personalized therapies.
Clinical trial identification
Editorial acknowledgement
Legal entity responsible for the study
The authors.
Funding
Has not received any funding.
Disclosure
All authors have declared no conflicts of interest.