Ovrednotenje sistemske ureditve in izvajanja programov zgodnjega dostopa do zdravil in donacij zdravil v Sloveniji

Morovič, Domen Ian (author), Kos, Mitja (mentor), Knez, Lea (comentor)

Programi zgodnjega dostopa do zdravil in donacije zdravil sta edina mehanizma, ki poleg kliničnih preskušanj bolnikom omogočata dostop do zdravil pred registracijo oziroma razvrstitvijo na listo financiranja iz naslova obveznega zdravstvenega zavarovanja. Namen magistrske naloge je bil pridobiti vpogled deležnikov na sistemsko ureditev in izvajanje programov zgodnjega dostopa do zdravil in donacij zdravil v Sloveniji. V okviru raziskave smo izvedli 12 intervjujev s ključnimi deležniki, ki so vključevali 7 predstavnikov zdravstvene dejavnosti, 2 predstavnika regulatornih organov (JAZMP in ZZZS) ter 3 predstavnike farmacevtskih družb. Dodatne udeležence smo v intervjuje vključevali, dokler ni prišlo do saturacije pridobljenih informacij. Od intervjuvancev smo želeli izvedeti, katere so pozitivne točke sedanje ureditve, s katerimi izzivi se najpogosteje srečujejo ter katere se jim zdijo ključne spremembe, ki bi pripomogle k optimizaciji trenutne ureditve programov zgodnjega dostopa in donacij zdravil. Intervjuje smo zvočno posneli ter pripravili prepise. Pridobljene podatke smo kvalitativno analizirali v programu NVivo 11 Pro. Na osnovi intervjujev smo kodirali zapise ter te razdelili na teme in podteme. Pozitivni pogled udeležencev na programe zgodnjega dostopa do zdravil je bil največkrat povezan s hitrejšim dostopom do inovativnih zdravljenj in s tem z izboljšanimi možnostmi zdravljenja bolnikov. Več intervjuvancev je kot razloge za izvajanje programov zgodnjega dostopa in donacij zdravil navedla pomanjkanje kliničnih preskušanj zdravil ter dolg čas od registracije zdravil do razvrstitve na listo ZZZS. Skoraj vsi udeleženci so izrazili mnenje, da so zadovoljni, da možnosti zgodnjega dostopa do zdravil v Sloveniji obstajajo, so pa pri tem izpostavili tudi nekaj možnosti za izboljšave. Več udeležencev je omenilo pomanjkanje možnosti dostopa do zdravil med registracijo zdravila in njegovo razvrstitvijo na listo. Poslovnih donacij, ki omogočajo dostop do zdravil v tem obdobju, večina intervjuvancev ne sprejema pozitivno, saj se nekaterim zdijo sporne in neetične, nekateri pa imajo težave s tem, da ne omogočajo takšne sledljivosti kot programi zgodnjega dostopa. Deležniki so prav tako izpostavili željo po vzpostavitvi javno dostopnega seznama programov zgodnjega dostopa do zdravil. Tak seznam bi po mnenju udeležencev pripomogel k boljši dostopnosti informacij o programih, saj te trenutno niso enostavno dosegljive. Prav to otežuje delo zdravnikov, ki niso pogosto vpleteni v vzpostavljanje in izvajanje programov. Po besedah več deležnikov je drug otežujoč dejavnik za zdravnike njihova preobremenjenost v redni praksi. Intervjuvani deležniki so v splošnem izrazili zadovoljstvo s trenutno ureditvijo programov zgodnjega dostopa do zdravil, pri tem pa so podali tudi veliko predlogov za izboljšave. Ker gre tako v Sloveniji kakor tudi v svetovnem merilu za precej pionirsko področje zdravstva, menimo, da je izrednega pomena sprotno izobraževanje vseh vpletenih v ureditev in izvajanje programov zgodnjega dostopa do zdravil, ki bi omogočalo ohranitev dobrih lastnosti programov in uvedbo izpopolnitev v dobrobit bolnikov.

• Language: Slovenian
• Keywords: program zgodnjega dostopa do zdravil, donacije zdravil, sočutna uporaba zdravil, registracija zdravila, razvrstitev zdravila na listo
• Work type: Master's thesis/paper
• Organization: FFA - Faculty of Pharmacy
• Year: 2022
• PID: 20.500.12556/RUL-143259
• Publication date in RUL: 10.12.2022

Secondary language

• Language: English
• Title: Evaluation of systemic regulation and performance of early access programs and donations of medicines in Slovenia

Abstract

In the absence of access to clinical trials, early access programs and donations of medicines are the only mechanisms that provide access to medicines prior to their registration or reimbursement. The aim of this master's thesis was to gain insight into the systemic regulation and performance of early access programs and donations of medicines in Slovenia. To this end, 12 interviews were conducted with key stakeholders, including 7 representatives of healthcare providers, 2 representatives of regulatory authorities (JAZMP and ZZZS), and 3 representatives of pharmaceutical companies. Additional interviewees were included in the study until saturation of the data, collected in the interviews was evident. Audio recordings were made during the interviews, from which transcripts were prepared. Qualitative analysis of the collected data was conducted using the NVivo 11 Pro program. Based on the interviews, coded references from the interviews were assigned themes and subthemes. Participants perceived early access programs as positive, particularly because they provide faster access to innovative treatments, thereby improving patients’ treatment options. Several interviewees cited the lack of clinical trials of medicines and the long time between drug registration and reimbursement as the main incentives for early access programs and donations. Almost all participants expressed satisfaction that early access to medicines exists in Slovenia, but also pointed to some areas for improvement. In particular, the lack of options between registration of a medicinal product and its reimbursement is problematic. Donations of medicines that provide access to treatment during this period, are not viewed very favourably by most participants, as some consider them controversial and unethical, while others have problems with them not providing the same level of traceability as early access programs. Most stakeholders also emphasized their desire to see a publicly available list of early access programs. This would help increase the availability of information about the programs that is not currently not widely available. It also hinders the work of physicians, who are not often involved in setting up and running the programs. Another obstacle for physicians, according to several stakeholders, is their overload in regular practice. Since this is a pioneering area of health care, both in Slovenia and worldwide, we believe that continuous education is of utmost importance to preserve what is good and improve it for the benefit of patients.

• Keywords: early access programs, donations of medicines, compassionate use, registration of medicines, drug reimbursement