Evaluation of pharmacists' knowledge and attitudes regarding rare diseases and orphan drugs

Abstract

Rare diseases (RD) are very heterogenic group of disorders affecting less than 5 out of 10.000 people in the European Union (EU), at the same time putting them in danger or disabling them chronically. It is estimated that only in Serbia almost half a million people suffer from some RD. In spite of rarity, they represent an important medical and social problem. The aims of this pilot project were to evaluate the pharmacists' general knowledge and specific knowledge regarding RD, regulatory requirements and availability of drugs for the RD in the Republic of Serbia as well as pharmacists' attitudes and understanding of the health public importance of RD and drugs' availability. The prospective cross­sectional KAP study was conducted during 2012, on a convenient sample of the community pharmacists from the territory of the Niš branch of Pharmaceutical Chamber of Serbia. The questionnaire was fully completed by 139 pharmacists; 89.2% were females with mean age of 43.4±9.1 years. More than half of the respondents (66.9%) knew that there was no Register of RD in Serbia, but did not know the estimated percentage of the EU population suffering and the prevalence of RD (67%, 51.8%, respectively). Insufficient information about the problem points to insecurity in basic epidemiology and regulatory knowledge. The majority of the respondents supported the establishment of the regulatory instruments for the promotion of the research and development of the orphan drugs for RD.

Retke bolesti su heterogena grupa životno ugrožavajućih ili hronično onesposobljavajućih oboljenja, koja se prema kriterijumima evropske regulative javljaju sa učestalošću od najmanje jednog prema 2000 stanovnika. Uprkos tome što su retke u populaciji, one predstavljaju važan medicinski i socijalni problem sa kojim se susreću zdravstveni sistemi širom sveta. Procene su da samo u Republici Srbiji od retkih bolesti boluje skoro pola miliona ljudi. Ciljevi ovog pilot projekta bili su da se procene opšta i specifična znanja farmaceuta o retkim bolestima, regulativi u vezi sa lekovima za lečenje retkih bolesti i njihovoj dostupnosti u Republici Srbiji i utvrde stavovi farmaceuta o značaju retkih bolesti i dostupnosti terapije za bolesnike. Prospektivna studija preseka obuhvatila je farmaceute koji rade na teritoriji niškog ogranka Farmaceutske komore Srbije. Prikupljanje podataka obavljeno je tokom 2012. godine pomoću strukturiranog, anonimnog upitnika, posebno konstruisanog prema ciljevima istraživanja, a za obradu podataka korišćene su metode deskriptivne statistike i korelaciona analiza. Upitnik je potpuno popunilo 139 farmaceuta, pretežno ženskog pola (89,2%), prosečne starosti 43,4 ±9.1 godine. Više od polovine ispitanih farmaceuta (67%) ne zna da 6-8% populacije u EU boluje od neke retke bolesti, dok 51,8% smatra, što je pogrešno, da je prevalenca u Evropi manja od 5 na 10 000 ljudi. Većina ispitanika (66,9%) zna da u Srbiji ne postoji Registar retkih bolesti. Nedovoljna informisanost farmaceuta ukazuje na nesigurnost u poznavanju osnovnih regulatornih zahteva u vezi sa retkim bolestima i lekovima za njihovo lečenje. Većina ispitanika se u svojim stavovima zalagala za uspostavljanje regulatornih instrumenata za promovisanje istraživanja i razvoja lekova za retke bolesti.