Health outcomes assessment of public expenditure management for medicines

Podobno kot drugje po svetu se tudi v Sloveniji srečujemo z naraščajočimi izdatki za zdravstveno varstvo. Področje zdravil predstavlja pomemben segment obvladovanja izdatkov v zdravstvu. Slovenija tako, podobno kot druge države, izvaja nadgradnje obstoječih ukrepov, hkrati pa uvaja nove ukrepe na področju politike določanja cen zdravil in plačila zanje (ang. reimbursement). V preteklosti smo bili deležni številnih sprememb sistema zunanje primerjave cen zdravil, na osnovi katerega se v Sloveniji oblikujejo najvišje dovoljenje cene (NDC) zdravil. Na področju plačil za zdravila je Slovenija v letu 2003 uvedla sistem referenčnih cen zdravil, ki ga imenujemo tudi sistem najvišjih priznanih vrednosti, in sicer za medsebojno zamenljiva zdravila (MZZ). Sistem najvišjih priznanih vrednosti se je v letu 2013 razširil na terapevtske skupine zdravil (TSZ), temu je poleg naraščajočih izdatkov botrovala tudi globalna finančna kriza. Kljub temu da se sistemi določanja cen zdravil in plačil v splošnem kažejo kot smiseln in učinkovit način obvladovanja javnih izdatkov za zdravila, ni nujno, da ti prispevajo k načrtovanim prihrankom. Poleg tega so lahko takšni ukrepi nesprejemljivi za zdravstvene delavce in paciente, kar lahko vpliva na njihovo namembnost. Glavni namen doktorske disertacije je ovrednotenje sistemskih ukrepov, namenjenih obvladovanju izdatkov za zdravila v Republiki Sloveniji (RS). Osredotočili smo se na sistem zunanje primerjave cen zdravil in sistem najvišjih priznanih vrednosti ter pri tem predpostavili, da tako nadgradnje sistema zunanje primerjave cen zdravil, kot tudi izvajanje sistema najvišjih priznanih vrednosti za MZZ ter razširitev le-tega na TSZ učinkovito zmanjšujejo javne izdatke za zdravila. Poleg tega smo želeli ovrednotiti percepcijo pacientov do sistema TSZ. Predvideli smo, da pacienti vidijo ta sistem kot smiseln in racionalen sistemski ukrep, kateremu se bodo ustrezno prilagodili. V ta namen smo zastavili štiri raziskave. Vpliv sistema zunanje primerjave zdravil na učinkovito upravljanje javnih izdatkov za zdravila smo ovrednotili na osnovi retrospektivne raziskave. Izhajali smo iz nabora podatkov o cenah zdravil v referenčnih državah na presečni datum in ovrednotili finančni učinek nadgradenj sistema zunanje primerjave cen zdravil, ki so bile uvedene s spremembami Pravilnika o določanju cen zdravil za uporabo v humani medicini (v nadaljevanju Pravilnik) v obdobju od 2007 do 2012. Vrednotili smo še morebitne nekonsistentnosti pri določanju NDC zdravil na osnovi sistema zunanje primerjave cen zdravil in finančni učinek splošnih dogovorov o cenah zdravil. Ugotovili smo, da so vse nadgradnje sistema zunanje primerjave cen zdravil, z izjemo tiste v letu 2009, prispevale k zmanjšanju izdatkov za zdravila. Skupno so v obdobju 2007–2012 vse nadgradnje sistema pripomogle k 5,5 % znižanju izdatkov za zdravila. Vseeno pa so se kot učinkovitejši način zmanjševanja izdatkov izkazali splošni odgovori o cenah zdravil med Zavodom za zdravstveno zavarovanje Slovenije (ZZZS) in imetniki dovoljenja za promet. Splošni dogovor o 3 % znižanju cen originalnih zdravil iz leta 2009 je izničil porast izdatkov na račun spremembe v Pravilniku iz leta 2009. Splošna dogovora o dodatnem 3 % znižanju cen originalnih zdravil iz leta 2010 in 2012 pa sta pripomogla k večjemu znižanju izdatkov kot spremembe v Pravilniku iz leta 2010 in 2012. Iz raziskave je razvidno še, da obstoječa regulacija NDC zdravil dopušča nekonsistentnosti, ki večinoma izhajajo iz neupoštevanja cen v eni ali več od treh za Slovenijo referenčnih držav. Z retrospektivno raziskavo, ki temelji na analizi podatkovnih zbirk o cenah in porabi zdravil, smo ovrednotili dolgoročni učinek sistema najvišjih priznanih vrednosti za MZZ na zmanjševanje izdatkov za zdravila. Uporabili smo podatke o NDC zdravil, veleprodajnih oziroma dejanskih cenah zdravil, ki so lahko hkrati tudi dogovorjene cene, in referenčnih cenah oziroma najvišjih priznanih vrednostih (NPV), ki predstavljajo ceno zdravila, krito iz naslova obveznega in prostovoljnega zdravstvenega zavarovanja (OZZ + PZZ). Opredelili smo dvoletno obdobje raziskave, z začetkom osem let po uvedbi sistema MZZ, se pravi leta 2012, in do razširitve sistema najvišjih priznanih vrednosti na TSZ ob koncu leta 2013. V raziskavi smo ocenili še iniciativo imetnikov dovoljenja za promet za prilagajanje veleprodajnih cen zdravil na raven NPV ter vrednost doplačil za paciente. Sistem najvišjih priznanih vrednosti za MZZ je v približno dvoletnem obdobju raziskave v povprečju prispeval k 45 % znižanju NPV, kar pomeni 20 % znižanje izdatkov iz naslova OZZ + PZZ za zdravila, vključena v sistem MZZ. Poleg tega se učinek sistema MZZ kaže kot približno 30 % znižanje izdatkov, ki bi se generirali, če bi bila iz sredstev OZZ + PZZ namesto NPV krita NDC zdravil. Ugotovili smo, da imetniki dovoljenja za promet ob objavi novih NPV s popusti na ravni veledrogerije znižajo veleprodajne cene zdravil, in sicer blizu ali na raven NPV, ne pa nižje, kar prispeva k znižanju doplačil za paciente, nima pa vpliva na izdatke OZZ + PZZ. Kljub zniževanju cen zdravil na ravni veledrogerije, pa še vedno ostaja 9–33 % delež zdravil iz sistema MZZ, ki od pacienta zahtevajo doplačilo, odvisno od posameznega obdobja spremembe NPV. Poleg tega popusti na ravni veledrogerije pogosto niso sporočeni ZZZS, kar bi pomenilo uraden dogovor o ceni zdravila in večjo transparentnost veleprodajnih cen zdravil v sistemu MZZ. Dogovorjena cena je bila v času raziskave sicer sklenjena za skoraj polovico zdravil, financiranih iz OZZ + PZZ, kar vključuje tudi 54,6 % zdravil iz sistema MZZ. Ekonomske izide uvedbe sistema najvišjih priznanih vrednosti za TSZ smo ovrednotili v retrospektivni raziskavi, ki je vrednotila spremembe v izdatkih in tržni položaj učinkovin tri leta pred in dve leti po uvedbi sistema TSZ za prve tri terapevtske skupine (zaviralci protonske črpalke, zaviralci angiotenzinske konvertaze (zaviralci ACE) in zdravila za zniževanje ravni serumskih lipidov). V raziskavi smo izhajali iz anonimizirane evidence o izdanih zdravilih na recept, ki smo jo dobili od ZZZS. Vrednotili smo spremembe NPV zdravil, izdatke OZZ + PZZ, tržno koncentracijo imetnikov dovoljenja za promet, ocenjeno s Herfindahl-Hirschmanovim indeksom, porabo zdravil v definiranih dnevnih odmerkih (DDD), zamenjave predpisanih učinkovin in tržni položaj učinkovin, ocenjen na osnovi Boston Consulting Groups (BCG) matrike. Raziskava je pokazala, da je uvedba sistema TSZ pripomogla k takojšnjemu znižanju izdatkov, in sicer k 45 % znižanju izdatkov za zdravila za zniževanje ravni serumskih lipidov, 25 % znižanju izdatkov zaviralcev ACE in 10 % znižanju izdatkov zaviralcev protonske črpalke. Pri tem pa rezultati regresijskega modela kažejo, da je trend zmanjševanja izdatkov po uvedbi TSZ značilno manj strm kot pred uvedbo tega sistema. Iz navedenega lahko razberemo, da je bistvenemu enkratnemu znižanju sledila bolj ali manj izražena stagnacija izdatkov za zdravila. Po uvedbi sistema najvišjih priznanih vrednosti za TSZ ni bilo sprememb v skupni porabi zdravil v posamezni TSZ, povečala se je poraba referenčne učinkovine pantoprazol, medtem ko porasta v porabi referenčnih učinkovin simvastatin in ramipril ni bilo. Uvedba sistema TSZ je povezana z značilno večjim številom menjav učinkovine v izdanih zaviralcih protonske črpalke. Po uvedbi sistema TSZ pa ni bilo zaznati bistvenih sprememb v tržni koncentraciji imetnikov dovoljenja za promet v katerikoli izmed treh TSZ. Razumevanje in odnos pacientov do sistema najvišjih priznanih vrednosti za TSZ smo ovrednotili s pomočjo vprašalnika, ki smo ga razvili na osnovi informacij laične in strokovne javnosti. Vprašalnik je sestavljen iz dveh delov in ima potrjeno razvidno in vsebinsko veljavnost. Raziskavo so v naključno izbranih lekarnah po Sloveniji od 15. maja do 15. junija 2014 izvajali magistri farmacije, ki so ob prisotnosti pacientov izpolnili prvi del vprašalnika. Drugi del vprašalnika, namenjen samo-izpolnjevanju, so pacienti izpolnili doma in ga raziskovalcem vrnili po pošti. Raziskava je pokazala, da pacienti razumejo osnovno značilnost sistema TSZ, da je zdravilo treba doplačati, ko njegova cena presega NPV, medtem ko se je razumevanje drugih pravic in dolžnosti pacientov v okviru tega sistema izkazalo kot bistveno slabše. Pacienti so nehomogeno ocenili smiselnost uvedbe sistema TSZ in njegove koristi. Velik delež pacientov pa v tem sistemu vidi nepotrebno breme in razlog za nezaupanje v slovenski zdravstveni sistem. 50,4 % od 667 v raziskavo vključenih pacientov bi moralo zdravilo ob vključitvi v raziskavo doplačati. Doplačilo je sprejelo 46,7 % pacientov, 3,7 % pa ga je zavrnilo, kar kaže na veliko tendenco pacientov do doplačil. Ocena povprečnega doplačila za trimesečno zdravljenje z zdravili je v času raziskave znašala 6,92 EUR, medtem ko je raziskava pokazala, da je najvišji povprečni znesek doplačila, ki so ga pacienti pripravljeni doplačati za trimesečno zdravljenje z zdravilom, 10,4 EUR. Zaradi značilno slabšega razumevanja sistema najvišjih priznanih vrednosti za TSZ lahko ta predstavlja izziv za starejše paciente, tiste s slabšim splošnim zdravstvenim stanjem in za paciente z nižjimi dohodki. Rezultati raziskav omogočajo vpogled v preplet sistemskih ukrepov za upravljanje izdatkov za zdravila ter kažejo na njihovo ekonomsko upravičenost. Obenem pa predstavljajo izhodišče za urejanje področja cen zdravil in plačil za zdravila ter nadgradnjo obstoječih sistemov. Poleg tega rezultati raziskav razkrivajo potrebe pacientov v okviru sistema najvišjih priznanih vrednosti in predstavljajo izhodišče za načrtovanje izobraževanja in aktivnega vključevanja pacientov pri zdravljenju z zdravili in njihovem financiranju.Like other countries, Slovenia has been faced with increasing health-care spending. Medicines represent an important element of the health-care expenditure management. In this regard, upgrades of existing and introduction of new medicines pricing and reimbursement policies or systems have been carried out in many countries, including Slovenia. Namely, frequent upgrades of the external reference pricing system defining the methodology of formulating medicines maximum allowed prices have been performed in Slovenia. Further, in 2003 an internal reference pricing system, the generic reference pricing (GRP) system, was introduced in Slovenia as the reimbursement system, which was followed by the therapeutic reference pricing (TRP) system introduced in the end of 2013. Even though the medicines pricing policies and reimbursement systems are generally recognized as reasonable and effective cost-containment approaches, it is not necessary that they always contribute to the anticipated savings. Moreover, medicines cost-containment policies or systems should be unacceptable for the health professionals and patients, which may further affect their intended purpose. The purpose of the doctoral dissertation was to evaluate pricing and reimbursement systems aimed at controlling public expenditure on medicines in Slovenia. The focus was on the external reference pricing system and internal reference pricing systemit was assumed that upgrades of the external reference pricing, implementation of GRP system and TRP system introduction have contributed to the reduction in public expenditure on medicines. Additionally, the dissertation aimed to assess patients’ knowledge and attitude towards TRP system based on the assumption that patients perceive TRP system as a logical and rational measure to which they would adapt. To this end, four studies were designed. The impact of external reference pricing system on medicines expenditure control was evaluated based on the retrospective database analysis. The data on medicine prices in reference countries on cross-sectional date were used for the evaluation of the budget impact of five external reference pricing system updates, which were introduced by the changes in Rules on Determining Prices of Medicines for Human Use (Rules) during 2007–2012. Potential inconsistencies in medicines pricing based on the external reference pricing and the budget impact of specific price reduction agreements were also assessed. It was found that all the external reference pricing system updates with the exception of that introduced by the Rules from 2009 have contributed to the public medicine expenditure reduction. All the system updates together provided 5.5 % cost reduction. However, specific price reduction agreements concluded between the Health Insurance Institute of Slovenia (HIIS) and manufacturer authorisation holders were proven more effective in cost reduction. The first specific price reduction agreement on 3% reduction of originator medicines price negated the cost increase resulting from the Rules changes in 2009. The second and third specific price reduction agreements on additional 3% originator medicines price reduction introduced in 2010 and 2012 respectively, contributed to the greater cost decrease than changes of the Rules in 2010 and 2012. The study also showed that existing regulation on the medicines maximum allowed price (MAP) formulation allows pricing inconsistencies mostly resulting from the non-involvement of prices in one or more of the three for Slovenia reference countries. The long-term impact of GRP system on medicine expenditure reduction was assessed in retrospective database analysis using data on MAP and wholesale price (actual price including discounts) of medicines, and maximum reimbursable price (also called reference price) covered by the health-care payer from the compulsory and complementary health insurance. A two-year study period was defined, beginning approximately eight years after the GRP system introduction that is in 2012 and ending with the introduction of the TRP system in the end of 2013. The marketing authorisation initiative for the medicine price adjustments to the maximum reimbursable price level and the amount of patient out-of-pocket cost were also assessed. Within approximately two-year study, the GRP system contributed on average to the 45% maximum reimbursable price reduction, which means approximately 20% reduction in expenditure covered by the compulsory and complementary health insurance. The cost-saving effect of GRP was shown as approximately 30% reduction of the medicines expenditure that would be generated if the MAP of medicines would be covered from the compulsory and complementary health insurance instead of maximum reimbursable price. It was found that at the time of maximum reimbursable price updates manufacturers have decreased wholesale medicine prices by offering discounts and rabats to wholesalers, but only closer or at the level of the maximum reimbursable price and not lower. Despite the price reductions at the wholesale level, there were still 9–33% of medicines belonging to the GRP system, the wholesale price of which exceeds maximum reimbursable price. Meaning that for those medicines co-payment was required from the patients. Furthermore, it was found that discounts and rabats at wholesale level have usually not been communicated to the payer - HIIS, which would mean that official price reduction agreement is concluded. However, the official price reduction agreement was concluded for almost half of the all reimbursable medicines, including 54.6% of medicines included in GRP system. Economic outcomes of the TRP system introduction were assessed through the retrospective database analysis evaluating changes in medicine expenditure and medicines market position three years before and two years after introduction of this reimbursement system for the first three introduced therapeutic classes – proton pump inhibitors, angiotensin-converting-enzyme (ACE) inhibitors and lipid-lowering agents. The health claims data on all prescription-only medicines in Slovenia obtained from HIIS were used for this purpose. Changes in maximum reimbursable price of medicines, medicines expenditure covered by the compulsory and complementary health insurance, market competition of marketing authorisation holders assessed based on the Herfindahl-Hirschman index, medicine consumption in defined daily doses and market position of active substances assessed based on the Boston Consulting Groups (BCG) matrices were assessed. The study showed that the introduction of TRP system significantly reduced medicine expenditure covered by the compulsory and complementary health insurance. That is 45%, 25% and 10% expenditure reduction of medicines included in therapeutic class of ACE inhibitors, lipid lowering agents and proton pump inhibitors, respectively. However, results of the regression model showed that in the first two years after TRP introduction the slope of expenditure reduction was significantly less steep than in the last three years before TRP system. This means that the large one-off expenditure reduction was followed by the more or less expressed medicine expenditure stagnation. The introduction of TRP system did not affect total medicine consumption in any therapeutic classhowever, the new reimbursement system contributed to the increased use of the reference active substance pantoprazole, whereas there was no increase in consumption of the simvastatin and ramipril also representing reference active substances. TRP system introduction contributed to the increase in the number of medicine substitutions in dispensed proton pump inhibitors, whereas it did not cause essential changes in market competition of marketing authorisation holders. Patients’ knowledge and attitude towards TRP system was evaluated using the questionnaire, which was developed based on the professional and lay public information. The questionnaire consisted of two parts and it has ensured face and content validity. The survey was carried out in the randomly selected community pharmacies in Slovenia from 15 May to 15 June 2014 with the help of community pharmacists. The first part of the questionnaire was completed by the community pharmacist in the presence of the patients, whereas the second part was filled in by the patients at home and returned to researches by mail. The study showed that patients know about the basic feature of the TRP system, namely that co-payment is needed when medicine price exceeds reference or maximum reimbursable price, whereas other patient duties and rights were not as well known. Patients were inhomogeneous in their assessment of relevance and benefits of the TRP system. A large proportion of patients perceived this system as unnecessary burden and the reason for the reduced confidence in the Slovenian health-care system. Furthermore, 50.4% of the 667 patients included in the study would have to co-pay for their prescribed medicine. Co-payment was accepted in 46.7% patients, whereas only 3.7% of patients were not willing to accept out-of-pocket cost. This indicates the patients’ high tendency for the initially prescribed medicine and willingness to co-payment. During the study period, the average out-of-pocket cost for the three months treatment with medicines included in one of the first three introduced therapeutic classes amounted to 6.92 EUR, while the survey showed that the average maximum willingness to co-pay for the three months treatment with medicines included in the TRP system is 10.4 EUR. Due to the significantly worse knowledge about TRP system, its introduction and potential extension to other therapeutic classes could be a special challenge for the elderly, patients with worse general health condition and those with lower monthly net income. Results of the four present studies provide insight into the combination of medicines pricing policies and reimbursement systems as cost-containment measures and show that in the case of Slovenia they are effective in medicine expenditure reduction. In addition, the results represent the basis for the further improvements and upgrades in medicines pricing and reimbursement policies. In addition, our findings reveal patients’ needs within the TRP as a reimbursement system and suggest the need for the education and active involvement of the patients in the treatment with medicines and their financing