Characteristics and availability of medicine early access programs and donations in Slovenia

Abstract

Background: Early access programs (EAPs) and medicine donation programs (MDPs) enable patients to have access to new medicines prior to regulatory approval or national reimbursement, respectively. The objective of this study was to evaluate the characteristics and timeliness of EAPs and MDPs in Slovenia.

Methods: Originator medicines approved by the EMA through a centralized procedure from 2010 to 2019 were included in the study. Data on the EAPs and MDPs were obtained from the Agency for Medicinal Products and Medical Devices of Slovenia.

Results: The EMA approved 458 new indications for 324 medicines during the study period. In Slovenia, a total of 58 medicine indications (12.7%) became available before national reimbursement, including 35 (7.6%) by EAPs and 26 (5.7%) by MDPs (3 through both programs). Among these, 35 (60.3%) medicine indications were associated with oncology. EAPs facilitated access to medicines a median of 21.0 months before reimbursement (24.7 months for oncology medicines). Initiating EAPs at the time of the marketing authorization application could potentially improve the time of early access by an additional 5.5 months (5.2 months for oncology). MDPs enabled access to medicines 9.5 months prior to reimbursement (9.4 months for oncology), with potential further improvement by 7.9 months with the initiation of access at the time of marketing authorization (5.7 months for oncology).

Conclusion: Our findings highlight Slovenia's successful implementation of EAPs and MDPs, with oncology emerging as the predominant focal area. The potential exists for improvements in the scope, timeliness, and transparency of information on EAPs and MDPs accessible to patients in Slovenia.

Keywords: access time; antineoplastic and immunomodulating agents; compassionate use programs; early access programs; oncology.

Figure 1.

The timeline and the definition of time variables for EAPs (A) and MDPs (B).

Figure 2.

The time of early access (in months) and the potential time of additional early access for medicine indications available through EAPs (A) and MDPs (B) in Slovenia between 2010 and 2019 and median time of access. The zero presents the time of the EAP (A) or MDP (B) approval. The arrows indicate medicines that were still waiting for reimbursement at the end of year 2020. Abbreviations: ≥2L, relapsed or refractory to first-line treatment; 17p del, 17p deletion; 1L, treatment naive; a, advanced; ALK, anaplastic lymphoma kinase; ALL, acute lymphoblastic leukaemia; AML, acute myeloid leukaemia; B-ALL, B-precursor acute lymphoblastic leukaemia; BRCA, breast cancer gene; C.Difficile, Clostridium difficile; ca, cancer; CHF, chronic heart failure; CLL, chronic lymphocytic leukaemia; EAP, early access programme; EGFR, epidermal growth factor receptor; FLT3, fms-related receptor tyrosine kinase 3; HCV, hepatitis C virus; HER2, human epidermal growth factor receptor 2; HR, hormone receptor; IPF, idiopathic pulmonary fibrosis; la, locally advanced; MA, marketing authorization; MCL, mantle cell lymphoma; mCRPC, metastatic castration-resistant prostate cancer; MDP, medicine donation programme; MRD, minimal residual disease; non-m CRPC, non-metastatic castration-resistant prostate cancer; NSCLC, non-small-cell lung cancer; Ph, Philadelphia chromosome; Pt-sensitive, platinum sensitive; wo prior CT, without prior chemotherapy.