Rare diseases are a heterogenic group of disorders with a little in common except of their rarity affecting by less than 5 : 10.000 people. In the world is registered about 6000-8000 rare diseases with 6–8% suffering population only in the European Union. In spite of rarity, they represent an important medical and social problem due to their incidence. For many rare diseases have no treatment, but if it exists and if started on time as being available to patients, there is a good prognosis for them to be able for normal life. The problems of patients affected by rare diseases are related to the lack of diagnosis and timely undergoing as well as their treatment or prevention. Orphan drugs are products intended for treatment, diagnosis or prevention of rare diseases, but for their development and marketing the industry has not been interested in yet because of their marketing reasons. Patients suffering from a rare disease although belonging to the vulnerable group for their specific health needs,is becoming invisible in the health care system due to their additional needs unproperly recognized. Ethical problems faced by patients, but also health care professionals are related to the allocation of medical diagnostics, unequal approach to health care, inappropriately specialized social services as well as therapy and rare orphan drugs unavailability. Ethical questions related to clinical trails on orphan drugs, population screening and epidemiology testing on rare diseases will also be discussed in this paper.
Thalidomide disaster was one of the biggest disasters in pharmaceutical industry. Since mid fifties of the twentieth century, more than 10.000 children have been born with stunted arms and legs, and damaged internal organs. Today, many questions sill stand: Was the tragedy preventable? What were the main reasons for borning more than 4.000 thalidomide children in West Germany, and 17 in United States? We tried to answer these questions, by comparing thalidomide stories in West Germany and United States. In West Germany, law regulations were very flexible. Federal Health Office registered new drugs, but had no authority to withdraw a drug that was approved dangerous on the market. Analyzing the stories he heard from his patients and colleagues, pediatrician dr. Widunkind Lenz realized that Contergan® (brand name for thalidomide) was responsible for birth defects. Pharmaceutical company, Chemie Grünenthal developed thalidomide, but re- fused to withdraw drug from the market. It took years, until the trial started. Dr. Lenz was dismissed from trial, as biased. As a result of two years long trial, company and federal government established the fund for children injured by Contergan®. In United States, pharmacologist dr. Frances Kelsey had been employed in Food and Drug Administration (FDA). She was the one who received a new drug application for thalidomide. Realized that application was in- complete, she refused to approve application. Unlike dr. Lenz, law regulations and FDA stood behind her. Thalidomide has never been approved in United States, and Dr. Kelsey was promotioned. She was also awarded for preventing thalidomide to reach the market. By comparing these stories, we realize how are important ethics and regulations in approving new drugs and their withdrawing. ; Talidomidska tragedija, sa preko 10 000 novorođenčadi sa oštećenim ekstremitetima širom sveta, predstavlja jednu od najvećih katastrofa u istoriji industrije lekova. Iako je od početaka ove priče prošlo gotovo pola veka i dalje stoji pitanje da li je i kako, bilo moguće sprečiti katastrofu? Uporednom analizom okolnosti pod kojima se tragedija dešavala u Sjedinjenim Američkim Državama (SAD) i Zapadnoj Nemačkoj u ovom radu je pokušano da se utvrdi da li su poštovani etički principi tokom uvođenja ovog leka u terapiju i koliko su zakonski propisi u oblasti registracije lekova doprineli da se u SAD rodi 17 dece sa anomalijama, naspram 4.000 dece u Zapadnoj Nemačkoj. Prema zakonu o lekovima u Zapadnoj Nemačkoj, procesi licenciranja, registracije i pojavljivanja leka na tržištu, bili su u nadležnosti Državnog zdravstvenog zavoda. Ipak, eventualno povlačenje leka sa tržišta niko nije mogao narediti. Pedijatar dr. Widukind Lenz je prvi ukazao na povezanost upotrebe talidomida kod trudnica i pojave malformacije na fetusima. Zbog manjkavosti u zakonskoj ragulativi, Contergan® (talidomid) je tek posle dugo vremena povučen iz terapije, a dr. Lenz je degradiran i kao stručnjak i kao čovek. Ishod sudskog procesa protiv farmaceutske kompanije bio je osnivanje fondacije za decu oštećenu talidomidom. U SAD-u su i zakon i Agencija za hranu i lekove (FDA) stajali uz dr. Frances Kelsey, koja je sprečila registraciju talidomida bez izvršenih kliničkih ispitivanja. Uporednom analizom talidomidskih priča u ove dve zemlje može se zaključiti da su pri uvođenju novog leka u terapiju, pored poštovanja strogih zakonskih propisa, neophodni savest i odgovornost pojedinaca u institucijama koje u tom procesu učestvuju ali i samih farmaceutskih kompanija koje lekove registruju.
Pharmacy profession has undergone through a period of intensive review and redefining of its professional roles, practice, and educational needs. Pharmacy graduates are now expected to be equipped with specific competencies necessary for provision of patient-centered and population-based pharmaceutical care, responsible use of medicines and management of limited healthcare resources. In line with other health professionals' education, pharmacy education progresses through several 'generations' of reforms which are faced with different professional and societal challenges. Thus, they have been implemented differently and to different extent in various regions. The aim of this communication is to point out the critical factors for further advancement of pharmacy education, with the emphasis on relevant societal and professional challenges in Serbia. Contemporary health professionals' education in developed countries is based on the four main paradigm including: (I) integrated interdisciplinary competency-based curriculum, (II) active learning approach involving clinical experience and interprofessional teaching and learning, (III) commitment to serve the community health needs and (IV) continuous teaching competencies development of academic staff and teacher practitioners in order to accommodate these innovations. The majority of developing countries, including Serbia, are in the initial phase of these reforms. Lack of integrated policy for healthcare and education system development, insufficient capacity of the professional regulatory bodies, and lack of motivation and commitment to change in the academic community were identified as major obstacles to progress. ; Farmacija kao profesija prolazi kroz period intenzivnih globalnih preispitivanja i redefinisanja profesionalnih uloga, prakse, kao i potreba znanja i sistema obrazovanja. Od farmaceuta se očekuje da, pored tradicionalnih znanja i veština, poseduju i specifične kompetencije potrebne za sprovođenje farmaceutske zdravstvene zaštite, racionalne i odgovorne primene lekova, kao i da učestvuju u upravljanju ograničenim resursima zdravstvenog sistema. U skladu sa reformama sistema obrazovanja drugih zdravstvenih profesionalaca, i promene u obrazovanju farmaceuta se odvijaju kroz nekoliko 'generacija' reformi koje su okarakterisane različitim profesionalnim i društvenim izazovima. Kao rezultat ovih razlika, promene u obrazovanju farmaceuta su u različitim delovima sveta sprovedene na različite načine i u različitom obimu. U ovom prikazu želimo da ukažemo na ključne faktore značajne za unapređenje obrazovanja farmaceuta, sa naglaskom na društvene i profesionalne izazove u Srbiji. Obrazovanje farmaceuta u razvijenim zemljama zasnovano je na četiri osnovne paradigme koje predstavljaju: (I) integrisani interdisciplinarni kurikulum zasnovan na jasno definisanim kompetencijama, (II) primena metoda aktivnog učenja, uključujući kliničku praksu i interprofesionalne nastavne aktivnosti, (III) posvećenost zdravstvenim potrebama stanovništva i (IV) kontinuirano unapređenje nastavničkih kompetencija nastavnog kadra koji učestvuje u inovaciji obrazovanja. Srbija, kao i većina zemalja u razvoju, tek je u početnoj fazi promena u obrazovanju farmaceuta, a kao osnovne prepreke uočavaju se nedostatak integrisanih politika razvoja zdravstvenog i obrazovnog sistema, nedovoljni kapaciteti profesionalnih i drugih regulatornih institucija, kao i nedovoljna motivisanost i posvećenost promenama u okviru akademske zajednice.
Background/Aim. Pharmacists' competence represents a dynamic framework of knowledge, skills and abilities to carry out tasks, and it reflects on improving the quality of life and on patients' health. One of the documents for the Evaluation and Competency Development of Pharmacists is the Global Competency Framework (GbCF). The aim of this study was to implement the GBCF document into Serbian pharmacies, to perform assessment and self assessment of the competencies. Methods. The assessment and self-assessment of pharmacists' competencies were performed during the period 2012-13 year in eight community pharmacy chains, in seven cities in Serbia. For assessment and self-assessment of pharmacists competencies the GbCF model was applied, which was adjusted to pharmaceutical practice and legislation in Serbia. External assessment was conducted by teams of pharmacists using the structured observation of the work of pharmacists during regular working hours. Evaluated pharmacists filled out the questionnaire about demographic indicators about the pharmacist and the pharmacy where they work. Results. A total of 123 pharmacists were evaluated. Pharmacists' Professional Competency Cluster (KK1) had the lowest score (average value 2.98), while the cluster Management and Organizational Competency (KK2) had the highest score (average value 3.15). The competence Recognition of the Diagnosis and Patient Counseling (K8), which belonged to the cluster KK1, had the lowest score (average value for assessment and self-assessment were 2.09, and 2.34, respectively) among the all evaluated competencies. Conclusion. GbCF might be considered as an instrument for the competencies' evaluation/self-evaluation and their improvement, accordingly. ; Uvod/Cilj. Kompetencije farmaceuta predstavljaju dinamični okvir i obuhvataju znanja, veštine i sposobnosti da se izvrše zadaci sa ciljem unapređenja kvaliteta života i zdravlja bolesnika. Jedan od dokumenata za ocenu i razvoj kompetencija farmaceuta je globalni okvir stručnosti - Global Competence Framework (GbCF). Cilj ovog rada bio je primena GbCF u apotekama u Srbiji i ocena i samoocena kompetencije farmaceuta. Metode. U periodu 2012-2013. godine izvršena je ocena i samoocena kompetencije farmaceuta u osam apotekarskih lanaca iz sedam gradova u Srbiji. Za ocenu i samoocenu stručnosti farmaceuta primenjen je model GbCF, prilagođen praksi i zakonodavstvu u Srbiji. Eksternu ocenu izvršili su timovi farmaceuta kroz strukturiranu opservaciju (upotrebom GbCF dokumenta) kompetencija farmaceuta tokom radnog vremena. Ocenjivani farmaceuti su popunili i upitnik o demografskim pokazateljima koji su se odnosili na podatke o samom farmaceutu i apoteci u kojoj radi. Rezultati. Ocenjena su ukupno 123 farmaceuta. Najviša ocena kompetencija farmaceuta zabeležena je za skup 'Upravljanje i organizacija' (KK2) (srednja vrednost 3,15), dok je skup 'Stručno znanje' (KK1) (srednja vrednost 2,98) ocenjen najnižom ocenom. Kompetencija K8 - 'Prepoznavanje dijagnoze i savetovanje bolesnika', koje pripadaju skupu KK1, imale su najniži skor (srednja vrednost u proceni i samoproceni iznosila je 2,09 i 2,34, respektivno) posmatrajući sve, kompetencije. Zaključak. Dokument za ocenu i razvoj kompetencija farmaceuta GbCF može služiti kao instrument za ocenu/samoocenu kompetencije, a samim tim i za njihovo poboljšanje.
Adherence is tremendously important for efficiency and safety of pharmacotherapy and has a significant influence on its clinical, economic and humanistic outcomes, which can be observed both on the individual level of the patient as well as on the overall healthcare system. The inadequate degree of adherence is a long-lasting and widely present problem which can have immense and long-term consequences. Therefore, the issue of adherence is a big challenge for medical professionals in clinical practice. The concept of adherence is based on cooperation between patients and medical team members in the process of therapy management. It is focused on patients and implies a two-way transfer of information, agreement and shared responsibility for success in realization of adequate therapy regime and defined goals. Although there are numerous methods for adherence assessment, the evaluation of this parameter is still considered problematic. The complexity of adherence can be seen in its multidimensionality since numerous factors which individually and in mutual interaction affect medicine-taking behavior and thus create a framework that determines the achieved level of adherence have been identified and analyzed. Considering the fact that poor adherence is one of the main preventable causes of unsatisfactory therapy results and excessive costs of medical care, it is evidently necessary to ensure a high quality base in the form of knowledge, skills and motivation at the healthcare system level for adherence evaluation and improvement, as well as a multidisciplinary approach based on coordinated activities of healthcare policy creators, researchers and medical professionals. ; Adherenca je izuzetno važna za efikasnost i bezbednost farmakoterapije i značajno utiče na njene kliničke, ekonomske i humanističke ishode, što se može pratiti kako na individualnom nivou bolesnika, tako i na celokupnom zdravstvenom sistemu. Neadekvatan stepen adherence odavno je prisutan i široko rasprostranjen problem koji može imati velike i dugoročne posledice. Zato pitanje adherence predstavlja veliki izazov za zdravstvene profesionalce u kliničkoj praksi. Koncept adherence je utemeljen na saradnji bolesnika i članova zdravstvenog tima u procesu upravljanja terapijom. U potpunosti je orjentisan ka bolesniku i podrazumeva dvosmerni transfer informacija, dogovor i podeljenu odgovornost za uspešnost u realizaciji adekvatnog terapijskog režima i definisanih ciljeva. Iako postoji veliki broj metoda za procenu ostvarenog stepena adherence, još uvek se evaluacija ovog parametra smatra problematičnom. Kompleksnost adherence ogleda se u njenoj multidimenzionalnosti, budući da je identifikovan i proučen veliki broj faktora koji pojedinačno i u međusobnoj interakciji utiču na ponašanje bolesnika u vezi sa terapijom i tako kreiraju okvir koji determiniše ostvareni stepen adherence. Budući da je nizak stepen adherence jedan od glavnih preventabilnih uzroka nezadovoljavajućih terapijskih ishoda i prekomernih troškova zdravstvene zaštite, evidentna je potreba da se obezbedi kvalitetna baza u formi znanja, veština i motivacije na nivou zdravstvenog sistema za evaluaciju i unapređenje adherence, kao i multidisciplinaran pristup, koji je zasnovan na koordinisanim aktivnostima kreatora zdravstvene politike, istraživača i zdravstvenih profesionalaca.
Access to orphan drugs (In EU regulation Orphan Drugs are referred as Orphan Medicinal Products (OMP)) is a key role in determining whether patients with rare diseases (RDs) will receive adequate and efficient treatment. The objective of this article is to identify differences in patient access to orphan drugs in 3 pharmaceutical markets: Serbia, Croatia and Macedonia. Patient access was defined: as the market access (availability) and affordability (financial accessibility). We analysed the legislative requirements for the authorisation process and made a cross country comparison. Retrospective cross-sectional analysis was done on drug lists in selected countries and a cross-comparison between the List of Orphan Drugs in Europe (LODE) for a six-month period (May 2014-October 2014). We included all 179 OMPs marketed in EU in our analysis, which had received market authorization in Croatia upon its membership in the EU. Total number of marketed drugs in Serbia was 59 (32.96%) drugs and in Macedonia 52 (29.05%) drugs. However, market authorization does not guarantee patient access to any given drug, so only 39.11% of OMPs could be accessed by Croatian patients (70 drugs).The number of refunded drugs in Serbia and Macedonia was smaller (32 and 20, respectively) which makes respectively, 17.88% and 11.17% of drugs on the LODE. The present study showed some variations between countries in selected indicators of availability and access to orphan drugs. Patients in Croatia had greater number of registered and refunded drugs, but in Serbia more than a half of registered OMPs could be refunded from National Health Insurance Fund. Macedonia had smaller number of inhabitants and also had the smaller number of patients from certain RDs which results in lower total number of OMPs. ; Dostupnost lekova za retke bolesti (orfan lekovi, u EU regulativi koristi se izraz na engleskom Orphan medicinal products, OMP) igra važnu ulogu u tome da li će bolesnici sa retkim bolestima imati pristup efikasnoj i adekvatnoj terapiji. Ciljevi ovog rada su da se identifikuju razlike u pristupu bolesnika orfan lekovima u tri odabrane zemlje: Srbiji, Hrvatskoj i Makedoniji. Pristup orfan lekovima definisana je kao: tržišna pristupačnost (dostupnost) i priuštivost (finansijska pristupačnost). Analizirali smo legislativne zahteve u procesu stavljanja leka u promet i uradili komparaciju među posmatranim zemljama. Retrospektivnom studijom preseka poredili smo nacionalne liste lekova posmatranih zemalja i Listu orfan lekova u EU, za period od šest meseci (maj-oktobar 2014.). Od ukupno 179 OMP, koliko ih je u tom periodu bilo sa dozvolom za stavljanje u promet u EU, u Srbiji je bilo registrovano 59 (32,96%), u Makedoniji 52 (29,05%), dok su u Hrvatskoj učlanjenjem u EU svi registrovani lekovi postali tržišno dostupni. Međutim, dozvola za stavljanje u promet nije i garancija da bolesnik ima pristup datom leku, pa je samo 39,11% lekova sa dozvolom za stavljanje u promet bilo na listi lekova koje se refundiraju u Hrvatskoj (70 OMP). Broj lekova kojima bolesnici imaju pristup preko nacionalnih fondova zdravstvenog osiguranja u Srbiji i Makedoniji su manji (32 OMP i 20OMP, respektivno), što čini da je priuštivost lekova u Srbiji tek 17,88% , a u Makedoniji 11,17% od liste orfan lekova u EU. Broj lekova, fizička i finansijska pristupačnost u analiziranim zemljama nije ista, dok je u Hrvatskoj bolesnicima dostupan najveći broj registrovanih lekova, u Srbiji se više od polovine registrovanih OMP može refundirati o trošku RFZO. Makedonija zbog manjeg broja stanovnika ima manji broj obolelih od RB, što rezultira manjim brojem OMP.
Access to orphan drugs (In EU regulation Orphan Drugs are referred as Orphan Medicinal Products (OMP)) is a key role in determining whether patients with rare diseases (RDs) will receive adequate and efficient treatment. The objective of this article is to identify differences in patient access to orphan drugs in 3 pharmaceutical markets: Serbia, Croatia and Macedonia. Patient access was defined: as the market access (availability) and affordability (financial accessibility). We analysed the legislative requirements for the authorisation process and made a cross country comparison. Retrospective cross-sectional analysis was done on drug lists in selected countries and a cross-comparison between the List of Orphan Drugs in Europe (LODE) for a six-month period (May 2014-October 2014). We included all 179 OMPs marketed in EU in our analysis, which had received market authorization in Croatia upon its membership in the EU. Total number of marketed drugs in Serbia was 59 (32.96%) drugs and in Macedonia 52 (29.05%) drugs. However, market authorization does not guarantee patient access to any given drug, so only 39.11% of OMPs could be accessed by Croatian patients (70 drugs).The number of refunded drugs in Serbia and Macedonia was smaller (32 and 20, respectively) which makes respectively, 17.88% and 11.17% of drugs on the LODE. The present study showed some variations between countries in selected indicators of availability and access to orphan drugs. Patients in Croatia had greater number of registered and refunded drugs, but in Serbia more than a half of registered OMPs could be refunded from National Health Insurance Fund. Macedonia had smaller number of inhabitants and also had the smaller number of patients from certain RDs which results in lower total number of OMPs.
Introduction Healthcare enlightenment and healthcare culture in Vranje and its region were under Turkish influence until the liberation in 1878, when the organisation of education, schooling and healthcare were initiated in the Vranje region Objective The aims of this study were to shed light on the beginning of the development of pharmaceutical personnel, and their contribution to healthcare culture development in Vranje and its region, with a focus on healthcare legislation and its impact on pharmaceutical service availability, and the analysis of pharmaceutical personnel development within the healthcare services. Methods We used the methods of documentation analysis and the desk analysis of the secondary information. Results At the beginning of the 19th century healthcare services in Vranje were still inexistent. After the liberation from the Turks, Naum's medical office with pharmacy was opened in Vranje. Although it did not comply with regulations, it presented the only formal source of supply of medications and sanitary material, and as such it gave its contribution to healthcare service at that time in Vranje. Mr ph. Stevan Varjacic, 1st class pharmacy assistant, worked at a temporary hospital from 1880 until January 1881, supplying with medications soldiers and residents of Vranje. The development of pharmaceutical schooling began in 1883, when a pharmacy was opened by the pharmacist Velimir Karic according to the Law on Healthcare Organization and People's Healthcare Maintenance. By the end of the 1930s there were ten civilian healthcare institutions in Vranje, even three of which were pharmacies. By the end of 1948, they were nationalized, and were renamed the City National Pharmacy of Vranje. Conclusion By the end of the 10th century, passing of the legislation on the regulations of pharmaceutical industry within healthcare services had an impact on Vranje that resulted in opening pharmacies with educated pharmaceutical personnel. Pharmacy owners changed, and the quality and continuity of medical supplies was always secured. ; Uvod Zdravstvena prosvećenost i zdravstvena kultura u Vranju i vranjskom kraju bili su pod turskim uticajem do 1878. godine, kada je nakon oslobođenja od Turaka počela organizacija prosvete, školstva i zdravstva u ovom kraju. Cilj rada Ciljevi rada su bili osvetljavanje početaka razvoja apotekarskog kadra i doprinos farmaceuta razvoju zdravstvene kulture u Vranju i vranjskom kraju s osvrtom na zdravstvenu regulativu i njen uticaj na dostupnost usluga apotekarske službe i analizu razvoja farmaceutskog kadra u okviru zdravstvene službe. Metode rada Korišćene su metode dokumentacione analize i desk-analiza sekundarnih podataka. Rezultati Početkom 19. veka zdravstvena služba u Vranju još nije postojala. Nakon oslobođenja od Turaka u Vranju je radila ordinacija s apotekom felčera Nauma, koja je, iako nije bila usklađena s propisima, bila jedini formalni izvor snabdevanja lekovima i sanitetskim materijalom i kao takva dala doprinos tadašnjoj zdravstvenoj službi u Vranju. U privremenoj vranjskoj bolnici od 1880. godine do januara 1881. godine radio je apotekarski pomoćnik prve klase magistar farmacije Stevan Varjačić, a lekovima su se snabdevali vojnici i stanovnici Vranja. Razvoj školske farmacije počinje 1883. godine otvaranjem apoteke farmaceuta Velimira Karića u skladu s tada važećim Zakonom o uređenju sanitetske struke i o čuvanju narodnog zdravlja. Krajem tridesetih godina 20. veka u vranjskom kraju je radilo deset civilnih zdravstvenih ustanova, od kojih su čak tri bile apoteke. One su krajem 1948. godine podruštvljene i promenile naziv u 'Gradska narodna apoteka u Vranju'. Zaključak Postojanje zakonske regulative o uređenju farmaceutske delatnosti u okviru zdravstvene službe uticalo je na to da Vranje krajem 19. veka dobije apoteke sa školovanim farmaceutskim kadrom. Vlasnici apoteka su se menjali, ali kvalitet i kontinuitet u medicinskom snabdevanju uvek je bio obezbeđen.
Aim To analyze the pharmacy network (structure and resources) in Bulgaria, Croatia, Serbia, and Slovenia and its relation to public expenditures for medicines. Methods We performed a cross-sectional study using the officially published data for the period 2003-2008 in four selected countries. Data sources were relevant national institutions. Results In 2008, Serbia had 27.5, Bulgaria 66.8, Croatia 59.5, and Slovenia 71.2 pharmacists per 100000 inhabitants. There was a significant difference in the number of pharmacists per 100000 inhabitants between all countries except between Bulgaria and Slovenia. The number of inhabitants per one pharmacy was significantly different between all observed countries. The expenditures for medicines per capita in 2008 were between (sic)30.34 in Bulgaria to (sic)137.03 in Slovenia, with a significant difference between all countries except between Bulgaria and Serbia. The number of pharmacists per 100000 inhabitants and expenditures for medicines per capita were positively correlated in all observed countries, except in Bulgaria. Conclusion There were significant difference in the structure and availability of the pharmacy service in all selected countries. Expenditures for medicines were positively correlated with the number of pharmacists in all countries, except in Bulgaria. Our findings could be valuable to national regulatory bodies for the creation of national drug policies.
The first state-owned pharmacy in Serbia dates back to 1836. At first its title was Dvorska i voena apoteka (Court and Military Pharmacy), which later changed to Pravitelstvena apoteka (Government Pharmacy). The pharmacy moved from Kragujevac to Belgrade on two occasions along with the government seat, to finally settle in Belgrade on 8 December 1841 Pravitelstvena Apoteka fostered the developement of the pharmaceutical science in the country and acted as a public healthcare institution. It had a state-of-the-art laboratory, equipped for chemical analysis of mineral water and for toxicological and biochemical analysis of water containers. It is the cradle of the applied chemistry in Serbia and it operated as a controlling centre for other pharmacies. In 1853, Pravitelstvena Apoteka opened a branch Filijala Pravitelstvene Apoteke, which was run by the Central Pharmacy in Belgrade and the appointed administrator D{stroke}ord{stroke}e Bogdanović, MPharm. On 10 June 1859, both pharmacies were privatised by the decree of the Prince of Serbia, Duke Miloš Obrenović.
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.
Introduction. Every clinical trial has to meet all ethical criteria in addition to the scientific ones. The basic ethical principles in the clinical trials are the following: nonmaleficence, beneficence, respect for autonomy and the principle of justice. Objective. The aim of the study was to analyze clinical cases with the outcomes leading to the changes in regulatoryethical framework related to the clinical trials, as well as the outcomes of key clinical trials that influenced the introduction of the ethical principles into clinical trials. Methods. This was a descriptive research (methods of analysis and documentation; desk analysis of the secondary data). Results. By analyzing the cases from the secondary sources as well as clinical and ethical outcomes, it may be noticed that the codes, declarations and regulations have been often preceded by certain events that caused their adoption. Moral concern and public awareness of the ethical issues have initiated not only the development of numerous guidelines, codes, and declarations, but also their incorporation into the legislative acts. Conclusion. It is desirable that ethical instruments become legally binding documents, because only in this way will be possible to control all phases of the clinical trials and prevent abuse of the respondents. ; Uvod. Svako kliničko ispitivanje, pored naučnih, mora da zadovolji i etičke kriterijume. Osnovni etički principi u kliničkim ispitivanjima su: neškodljivost, dobročinstvo, poštovanje autonomije i princip pravednosti. Cilj rada. Cilj rada je bio da se analiziraju klinički slučajevi čiji su ishodi doveli do promena u regulatornoetičkom okviru u vezi s kliničkim ispitivanjima i ishodi kliničkih ispitivanja koja su uticala na uvođenje etičkih principa. Metode rada. Istraživanje je bilo deskriptivno, a primenjene su metoda dokumentacione analize i deskanaliza sekundarnih podataka. Rezultati. Analizom slučajeva iz sekundarnih izvora, te kliničkih i etičkih ishoda, može se primetiti da su kodeksima, deklaracijama i pravilima prethodili događaji koji su uzrokovali njihovo donošenje. Moralna briga i svest javnosti o etičkim problemima pokrenule su razvoj brojnih smernica, kodeksa i deklaracija i njihovo uvrštavanje u zakonska akta. Zaključak. Ukoliko se regulativa svih faza kliničkih ispitivanja zasniva na etičkim postavkama, kontrola i ishod su bazirani na humanim osnovama i prava su mera dobrobiti društva i pojedinca.
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.
