8 dƌŝĂůͲďĂƐĞĚĞĐŽŶŽŵŝĐĞǀĂůƵĂƟŽŶƐŝŶŽĐĐƵƉĂƟŽŶĂů ŚĞĂůƚŚ͗ƉƌŝŶĐŝƉůĞƐ͕ŵĞƚŚŽĚƐ͕ĂŶĚƌĞĐŽŵŵĞŶĚĂƟŽŶƐ :ŽŚĂŶŶĂ D ǀĂŶ ŽŶŐĞŶ DĂƌŝĞŬĞ & ǀĂŶ tŝĞƌ Emile Tompa WaƵlieŶ D oŶŐeƌƐ llaƌĚ : ǀaŶ Ěeƌ eeŬ DaƵƌiƚƐ t ǀaŶ TƵlĚeƌ :ƵĚiƚŚ E oƐmaŶƐ :KĐĐƵƉŶǀŝƌŽŶDĞĚ͕ĂĐĐĞƉƚĞĚĨŽƌƉƵďůŝĐĂƟŽŶ Chapter 8 ABSTRACT ZeƐoƵƌĐeƐ Ĩoƌ oĐĐƵpaƟoŶal ŚealƚŚ aƌe ƐĐaƌĐe͘ To alloĐaƚe aǀailaďle ƌeƐoƵƌĐeƐ aƐ eĸĐieŶƚlLJ aƐ poƐƐiďle͕ ĚeĐiƐioŶͲmaŬeƌƐ ŶeeĚ iŶĨoƌmaƟoŶ oŶ ƚŚe ƌelaƟǀe eĐoŶomiĐ meƌiƚƐ oĨ oĐĐƵpaƟoŶal ŚealƚŚ aŶĚ ƐaĨeƚLJ ;K,^Ϳ iŶƚeƌǀeŶƟoŶƐ͘ EĐoŶomiĐ eǀalƵaƟoŶƐ ĐaŶ pƌoǀiĚe ƚŚiƐ iŶĨoƌmaƟoŶ ďLJ ĐompaƌiŶŐ ƚŚe ĐoƐƚƐ aŶĚ ĐoŶƐeƋƵeŶĐeƐ oĨ ƚǁo oƌ moƌe alƚeƌŶaƟǀe iŶƚeƌǀeŶƟoŶƐ͘ ,oǁeǀeƌ͕ oŶlLJ a Ĩeǁ oĨ ƚŚe ƐƚƵĚieƐ ƚŚaƚ ĐoŶƐiĚeƌ ƚŚe eīeĐƟǀeŶeƐƐ oĨ K,^ iŶƚeƌǀeŶƟoŶƐ ƚaŬe ƚŚe edžƚƌa Ɛƚep oĨ ĐoŶƐiĚeƌiŶŐ ǁŚeƚŚeƌ ƚŚeLJ aƌe eĸĐieŶƚ iŶ ƚeƌmƐ oĨ ƚŚeiƌ ƌeƐoƵƌĐe impliĐaƟoŶƐ͘ Doƌeoǀeƌ͕ ƚŚe meƚŚoĚoloŐiĐal ƋƵaliƚLJ oĨ eĐoŶomiĐ eǀalƵaƟoŶƐ iŶ ƚŚe oĐĐƵpaƟoŶal ŚealƚŚ liƚeƌaƚƵƌe iƐ ŐeŶeƌallLJ poor. EīeĐƟǀeŶeƐƐ ƚrialƐ are ĐommoŶlLJ ƵƐeĚ aƐ a ǀeŚiĐle Ĩor eĐoŶomiĐ eǀalƵaƟoŶƐ͕ ƐiŶĐe ƚŚeLJ proǀiĚe a ƵŶiƋƵe opporƚƵŶiƚLJ ƚo reliaďlLJ eƐƟmaƚe ƚŚe reƐoƵrĐe impliĐaƟoŶƐ oĨ a Ŷeǁ iŶƚerǀeŶƟoŶ ǁiƚŚoƵƚ ƐƵďƐƚaŶƟallLJ ŚiŐŚer reƐearĐŚ edžpeŶƐeƐ. TŚe preƐeŶƚ paper aimƐ ƚo Śelp oĐĐƵpaƟoŶal ŚealƚŚ reƐearĐŚerƐ ĐoŶĚƵĐƚ ŚiŐŚ ƋƵaliƚLJ ƚrialͲďaƐeĚ eĐoŶomiĐ eǀalƵaƟoŶƐ ďLJ ĚiƐĐƵƐƐiŶŐ ƚŚe ƚŚeorLJ aŶĚ meƚŚoĚoloŐLJ ƚŚaƚ ƵŶĚerlie ƚŚem͕ aŶĚ ďLJ proǀiĚiŶŐ reĐommeŶĚaƟoŶƐ Ĩor ŐooĚ praĐƟĐe reŐarĚiŶŐ ƚŚeir ĚeƐiŐŶ͕ aŶalLJƐiƐ͕ aŶĚ reporƟŶŐ. TŚe preƐeŶƚ paper ǁill alƐo Śelp ĐoŶƐƵmerƐ oĨ ƚŚiƐ liƚeraƚƵre ǁiƚŚ ƵŶĚerƐƚaŶĚiŶŐ aŶĚ ĐriƟĐallLJ appraiƐiŶŐ ƚrialͲďaƐeĚ eĐoŶomiĐ eǀalƵaƟoŶƐ oĨ K,^ iŶƚerǀeŶƟoŶƐ. ϮϰϬ WrŝŶĐŝpůeƐ͕ ŵethŽĚƐ͕ aŶĚ reĐŽŵŵeŶĚaƟŽŶƐ INTRODUCTION ZeƐoƵrĐeƐ Ĩor oĐĐƵpaƟoŶal ŚealƚŚ are ƐĐarĐe ;ϭ͖ϮͿ. TŚereĨore͕ ĚeĐiƐioŶͲmaŬerƐ iŶ ƚŚiƐ ĮelĚ iŶĐreaƐiŶŐlLJ Đall ƵpoŶ aĚǀiƐorƐ aŶĚ reƐearĐŚerƐ ƚo Ŷoƚ oŶlLJ ĚemoŶƐƚraƚe ƚŚaƚ oĐĐƵpaƟoŶal ŚealƚŚ aŶĚ ƐaĨeƚLJ ;K,^Ϳ iŶƚerǀeŶƟoŶƐ are eīeĐƟǀe͕ ďƵƚ alƐo eĸĐieŶƚ iŶ ƚermƐ oĨ ƚŚeir reƐoƵrĐe impliĐaƟoŶƐ. EĐoŶomiĐ eǀalƵaƟoŶƐ proǀiĚe iŶĨormaƟoŶ oŶ ƚŚe relaƟǀe eĸĐieŶĐLJ oĨ ƚǁo or more alƚerŶaƟǀe iŶƚerǀeŶƟoŶƐ aŶĚ are ĚeĮŶeĚ aƐ ͞the ĐŽŵparaƟǀe aŶaůLJƐŝƐ ŽĨ aůterŶaƟǀe ĐŽƵrƐeƐ ŽĨ aĐƟŽŶ ŝŶ terŵƐ ŽĨ ďŽth theŝr ĐŽƐtƐ aŶĚ ĐŽŶƐeƋƵeŶĐeƐ͟ ;ϭͿ. TŚe maiŶ aƐpeĐƚƐ oĨ aŶLJ eĐoŶomiĐ eǀalƵaƟoŶ are ƚo iĚeŶƟĨLJ͕ meaƐƵre͕ ǀalƵe͕ aŶĚ Đompare ƚŚe ĐoƐƚƐ aŶĚ ĐoŶƐeƋƵeŶĐeƐ oĨ alƚerŶaƟǀeƐ ;ϭͿ. /Ŷ ƚŚe ŚealƚŚĐare ƐeĐƚor͕ eĐoŶomiĐ eǀalƵaƟoŶƐ are iŶĐreaƐiŶŐlLJ ďeiŶŐ ĐoŶĚƵĐƚeĚ aŶĚ plaLJ aŶ imporƚaŶƚ role iŶ maŶLJ ĐoƵŶƚrieƐ ǁŚeŶ ĚeĐiĚiŶŐ ǁŚeƚŚer ;ŶeǁͿ ƚreaƚmeŶƚƐ ƐŚoƵlĚ ďe ĐoǀereĚ ďLJ pƵďliĐ ĨƵŶĚiŶŐ ;ϭͿ. ,oǁeǀer͕ oŶlLJ a Ĩeǁ oĨ ƚŚe ƐƚƵĚieƐ ƚŚaƚ ĐoŶƐiĚer ƚŚe eīeĐƟǀeŶeƐƐ oĨ K,^ iŶƚerǀeŶƟoŶƐ ƚaŬe ƚŚe edžƚra Ɛƚep oĨ ĐoŶƐiĚeriŶŐ ǁŚeƚŚer ƚŚeLJ are eĸĐieŶƚ iŶ ƚermƐ oĨ ƚŚeir reƐoƵrĐe impliĐaƟoŶƐ ;ϯͿ. Doreoǀer͕ ƚŚe meƚŚoĚoloŐiĐal ƋƵaliƚLJ oĨ ƚŚoƐe ƚŚaƚ Ěo iƐ ŐeŶerallLJ poor ;ϰͲϳͿ. ZeaƐoŶƐ Ĩor ƚŚiƐ maLJ ďe ƚŚe ĚiƐƟŶĐƚ ĐŚalleŶŐeƐ ƚŚaƚ ĐoŶĨroŶƚ reƐearĐŚerƐ ǁŚeŶ ƚrLJiŶŐ ƚo iĚeŶƟĨLJ ƚŚe reƐoƵrĐe impliĐaƟoŶƐ oĨ K,^ iŶƚerǀeŶƟoŶƐ aƐ ǁell aƐ a laĐŬ oĨ reĐommeŶĚaƟoŶƐ oŶ Śoǁ ƚo Ěeal ǁiƚŚ ƚŚeƐe iƐƐƵeƐ ;ϯͿ. DaŶLJ eĐoŶomiĐ eǀalƵaƟoŶ ƚedžƚ ďooŬƐ aŶĚ arƟĐleƐ are ĚeƐiŐŶeĚ Ĩor ƵƐe iŶ ŚealƚŚĐare ƐeƫŶŐƐ aŶĚ maLJ ƚŚereĨore ďe ĚiĸĐƵlƚ ƚo aĚapƚ ƚo ƚŚe oĐĐƵpaƟoŶal ŚealƚŚ ĐoŶƚedžƚ ;ϰͿ. EīeĐƟǀeŶeƐƐ ƚrialƐ are a ĐommoŶlLJ ƵƐeĚ ǀeŚiĐle Ĩor eĐoŶomiĐ eǀalƵaƟoŶƐ͕ aƐ ƚŚeLJ proǀiĚe a ƵŶiƋƵe opporƚƵŶiƚLJ ƚo reliaďlLJ eƐƟmaƚe ƚŚe reƐoƵrĐe impliĐaƟoŶƐ oĨ a Ŷeǁ iŶƚerǀeŶƟoŶ ǁiƚŚoƵƚ ƐƵďƐƚaŶƟallLJ ŚiŐŚer reƐearĐŚ edžpeŶƐeƐ. lƚŚoƵŐŚ Ɛome eīorƚƐ Śaǀe ďeeŶ ƵŶĚerƚaŬeŶ ƚo improǀe ƚŚe ƋƵaliƚLJ oĨ ;ƚrialͲďaƐeĚͿ eĐoŶomiĐ eǀalƵaƟoŶƐ iŶ oĐĐƵpaƟoŶal ŚealƚŚ ;ϯ͖ϴ͖ϵͿ͕ more ŶeeĚƐ ƚo ďe ĚoŶe ƚo aĐĐompliƐŚ ƚŚiƐ. TŚereĨore͕ ƚŚe preƐeŶƚ paper aimƐ ƚo Śelp oĐĐƵpaƟoŶal ŚealƚŚ reƐearĐŚerƐ ĐoŶĚƵĐƚ ŚiŐŚ ƋƵaliƚLJ ƚrialͲďaƐeĚ eĐoŶomiĐ eǀalƵaƟoŶƐ ďLJ ĚiƐĐƵƐƐiŶŐ ƚŚe ƚŚeorLJ aŶĚ meƚŚoĚoloŐLJ ƚŚaƚ ƵŶĚerlie ƚŚem͕ aŶĚ ďLJ proǀiĚiŶŐ reĐommeŶĚaƟoŶƐ Ĩor ŐooĚ praĐƟĐe reŐarĚiŶŐ ƚŚeir ĚeƐiŐŶ͕ aŶalLJƐiƐ͕ aŶĚ reporƟŶŐ. Ϯϰϭ 8 Chapter 8 DESIGN OF AN ECONOMIC EVALUATION <ŝŶĚŽĨĞĐŽŶŽŵŝĐĞǀĂůƵĂƟŽŶƐ ŚooƐiŶŐ ƚŚe appropriaƚe ŬiŶĚ oĨ eĐoŶomiĐ eǀalƵaƟoŶ Ĩor a parƟĐƵlar oĐĐƵpaƟoŶal ŚealƚŚ ĚeĐiƐioŶ ĐoŶƚedžƚ ĐaŶ ďe a ĐŚalleŶŐe aƐ a reƐƵlƚ oĨ ƚŚe relaƟǀe ĐompledžiƚLJ oĨ ƚŚe ĚeĐiƐioŶͲmaŬiŶŐ ĐoŶƚedžƚ ƚŚaƚ ŐeŶerallLJ iŶĐlƵĚeƐ mƵlƟple ƐƚaŬeŚolĚerƐ ;e.Ő. ǁorŬerƐ͕ emploLJerƐ͕ iŶƐƵraŶĐe ĐompaŶieƐ͕ pƵďliĐ poliĐLJ maŬerƐͿ. &oƵr ŬiŶĚƐ oĨ eĐoŶomiĐ eǀalƵaƟoŶƐ are ĚiƐƟŶŐƵiƐŚeĚ. TŚere are ƐimilariƟeƐ aĐroƐƐ ƚŚe ĨoƵr ŬiŶĚƐ. TŚe maiŶ ĚiīereŶĐe iƐ ƚŚe meƚriĐ ƵƐeĚ ƚo meaƐƵre ƚŚe ŬeLJ oƵƚĐome ;ŚealƚŚ aŶĚͬor ƐaĨeƚLJ͕ iŶ ƚŚe ĐaƐe oĨ K,^ iŶƚerǀeŶƟoŶƐͿ ;ϭϬͿ. ϭͿ oƐƚͲeīeĐƟǀeŶeƐƐ aŶalLJƐiƐ ;EͿ͗ oƐƚƐ aŶĚ Ɛome ĐoŶƐeƋƵeŶĐeƐ ;e.Ő. proĚƵĐƟǀiƚLJ͕ ŚealƚŚĐare ƵƟlinjaƟoŶ impliĐaƟoŶƐͿ are meaƐƵreĚ iŶ moŶeƚarLJ ƵŶiƚƐ͕ ǁŚereaƐ ƚŚe ŬeLJ oƵƚĐome iƐ meaƐƵreĚ iŶ ŶaƚƵral ƵŶiƚƐ ;ϭͿ. ϮͿ oƐƚͲďeŶeĮƚ aŶalLJƐiƐ ;Ϳ͗ oƚŚ ĐoƐƚƐ aŶĚ ĐoŶƐeƋƵeŶĐeƐ are meaƐƵreĚ iŶ moŶeƚarLJ ƵŶiƚƐ. /Ŷ ďƵƐiŶeƐƐ aĚmiŶiƐƚraƟoŶ͕ Ɛ are ƐomeƟmeƐ ĚeƐĐriďeƐ aƐ reƚƵrŶͲoŶͲiŶǀeƐƚmeŶƚ aŶalLJƐeƐ. ϯͿ oƐƚͲƵƟliƚLJ aŶalLJƐiƐ ;hͿ͗ oƐƚƐ aŶĚ Ɛome ĐoŶƐeƋƵeŶĐeƐ are meaƐƵreĚ iŶ moŶeƚarLJ ƚermƐ͕ ǁŚereaƐ ƚŚe ŬeLJ oƵƚĐome iƐ meaƐƵreĚ iŶ ƵƟliƚLJ ƵŶiƚƐ. hƟliƟeƐ are oŌeŶ edžpreƐƐeĚ iŶ ƚermƐ oĨ YƵaliƚLJ ĚũƵƐƚeĚ >iĨe zearƐ ;Y>zƐͿ ;ϭͿ. ϰͿ oƐƚͲmiŶimiƐaƟoŶ aŶalLJƐiƐ ;DͿ͗ KŶlLJ ĐoƐƚƐ are ĐoŶƐiĚereĚ aĐroƐƐ alƚerŶaƟǀeƐ͕ aƐ iƚ iƐ aƐƐƵmeĚ ƚŚaƚ ƚŚe ĐoŶƐeƋƵeŶĐeƐ are Ɛimilar. DƐ are ĐoŶƐiĚereĚ iŶappropriaƚe iĨ ƚŚere iƐ ƵŶĐerƚaiŶƚLJ reŐarĚiŶŐ a poƐƐiďle ĚiīereŶĐe iŶ ƚŚe maŐŶiƚƵĚe oĨ ĐoŶƐeƋƵeŶĐeƐ ;ϭͿ. tŚiĐŚ ŬiŶĚ oĨ eĐoŶomiĐ eǀalƵaƟoŶ iƐ moƐƚ appropriaƚe ĚepeŶĚƐ oŶ ƚŚe ƐƚaŬeŚolĚerƐ iŶǀolǀeĚ aŶĚ ƚŚe ƋƵeƐƟoŶ ďeiŶŐ aƐŬeĚ. 'eŶerallLJ͕ emploLJerƐ are moƐƚ iŶƚereƐƚeĚ iŶ Ɛ ƚŚaƚ ĐaŶ proǀiĚe iŶƐiŐŚƚ iŶƚo ƚŚe impaĐƚ oĨ aŶ iŶƚerǀeŶƟoŶ oŶ a ĐompaŶLJ͛Ɛ ďoƩomͲliŶe͕ ǁŚereaƐ pƵďliĐ poliĐLJ maŬerƐ maLJ ďe more iŶƚereƐƚeĚ iŶ EƐ aŶĚ hƐ͕ parƟĐƵlarlLJ iĨ moŶeƚarLJ meaƐƵreƐ Ěo Ŷoƚ aĚeƋƵaƚelLJ ĐapƚƵre imporƚaŶƚ ŚealƚŚ oƵƚĐomeƐ ;ϭ͖ϴ͖ϭϭͿ. TŚereĨore͕ iƚ iƐ reĐommeŶĚeĚ ƚŚaƚ reƐearĐŚerƐ ĐoŶĚƵĐƚ ǀarioƵƐ ŬiŶĚƐ oĨ eĐoŶomiĐ eǀalƵaƟoŶƐ ǁiƚŚiŶ ƚŚe Ɛame ƐƚƵĚLJ iŶ orĚer ƚo iŶĨorm all releǀaŶƚ ƐƚaŬeŚolĚerƐ ;ϯͿ. ϮϰϮ WrŝŶĐŝpůeƐ͕ ŵethŽĚƐ͕ aŶĚ reĐŽŵŵeŶĚaƟŽŶƐ tŚĞŶƚŽƵŶĚĞƌƚĂŬĞĂŶĞĐŽŶŽŵŝĐĞǀĂůƵĂƟŽŶ EĐoŶomiĐ eǀalƵaƟoŶƐ are oŌeŶ ĐoŶĚƵĐƚeĚ aloŶŐƐiĚe ;͞piŐŐLJďaĐŬeĚ͟ oŶƚoͿ ƚrialƐ eǀalƵaƟŶŐ ƚŚe eīeĐƟǀeŶeƐƐ oĨ K,^ iŶƚerǀeŶƟoŶƐ. sarioƵƐ ĚeƐiŐŶ aƐpeĐƚƐ are ƚŚereĨore ƚLJpiĐallLJ ĚeƚermiŶeĚ ďLJ ƚŚe reƋƵiremeŶƚƐ oĨ ƚŚe eīeĐƟǀeŶeƐƐ ƚrial ;e.Ő. alƚerŶaƟǀeƐ͕ oƵƚĐome meaƐƵreƐͿ. ,oǁeǀer͕ ƚo eŶƐƵre ƚŚaƚ all releǀaŶƚ eĐoŶomiĐ Ěaƚa iƐ ĐolleĐƚeĚ iŶ a ǀaliĚ͕ reliaďle͕ aŶĚ eĸĐieŶƚ ǁaLJ͕ iƚ iƐ imporƚaŶƚ ƚo ĐoŶƐiĚer ƚŚe reƋƵiremeŶƚƐ Ĩor ƚŚe eĐoŶomiĐ eǀalƵaƟoŶ aƚ ƚŚe earlieƐƚ poƐƐiďle ƐƚaŐe ;ϭϮͲϭϰͿ. eďaƚe edžiƐƚƐ aƐ ƚo ǁŚeƚŚer aŶ eĐoŶomiĐ eǀalƵaƟoŶ ƐŚoƵlĚ ďe iŶĐlƵĚeĚ iŶ a ƚrial ďeĨore ƚŚe eīeĐƟǀeŶeƐƐ oĨ a Ŷeǁ iŶƚerǀeŶƟoŶ iƐ eƐƚaďliƐŚeĚ. ,oǁeǀer͕ Ŷoƚ iŶĐlƵĚiŶŐ aŶ eĐoŶomiĐ eǀalƵaƟoŶ ǁoƵlĚ riƐŬ loƐiŶŐ ƚŚe opporƚƵŶiƚLJ ƚo ƐimƵlƚaŶeoƵƐlLJ ĐolleĐƚ ĐoƐƚ aŶĚ eīeĐƚ Ěaƚa ;ϭϰͿ. lƐo͕ ƚŚe aďƐeŶĐe oĨ ƐƚaƟƐƟĐallLJ ƐiŐŶiĮĐaŶƚ ĐoŶƐeƋƵeŶĐeͬ eīeĐƚ ĚiīereŶĐeƐ ďeƚǁeeŶ ƚŚe alƚerŶaƟǀeƐ ďeiŶŐ ĐompareĚ ĚoeƐ Ŷoƚ ŶeĐeƐƐarilLJ implLJ ƚŚaƚ ƚŚe Ŷeǁ alƚerŶaƟǀe iƐ Ŷoƚ ĐoƐƚͲeīeĐƟǀe aŶĚͬor ĐoƐƚͲďeŶeĮĐial. EĐoŶomiĐ eǀalƵaƟoŶƐ are aďoƵƚ ƚŚe ũoiŶƚ ĚiƐƚriďƵƟoŶ oĨ ĐoƐƚƐ aŶĚ ĐoŶƐeƋƵeŶĐeƐ͕ aŶĚ ĐoƵlĚ ĚemoŶƐƚraƚe Đlear ĐoƐƚͲeīeĐƟǀeŶeƐƐͬĐoƐƚͲďeŶeĮƚ ǁŚeŶ ŶeiƚŚer ĐoƐƚ Ŷor ĐoŶƐeƋƵeŶĐe ĚiīereŶĐeƐ are iŶĚiǀiĚƵallLJ ƐiŐŶiĮĐaŶƚ ;ϭϰͿ. lƐo͕ ĐoƐƚ ƐaǀiŶŐƐ miŐŚƚ oĐĐƵr iŶ ƚŚe aďƐeŶĐe oĨ ŚealƚŚ improǀemeŶƚƐ aŶĚ ĐoƵlĚ ƚŚƵƐ ďe miƐƐeĚ iĨ aŶ eĐoŶomiĐ eǀalƵaƟoŶ is not performed. dƌŝĂůĚĞƐŝŐŶ WraŐmaƟĐ randominjed Đontrolled trials ;ZTsͿ are ŐenerallLJ aĐŬnoǁledŐed as tŚe ďest ǀeŚiĐle for eĐonomiĐ eǀalƵaƟons͕ ďeĐaƵse tŚeLJ enaďle tŚe eǀalƵaƟon of tŚe resoƵrĐe impliĐaƟons of K,^ interǀenƟons Ƶnder ͞real life͟ ĐondiƟons. TŚis setƵp inĐreases tŚe edžternal ǀaliditLJ of resƵlts͕ ǁŚile tŚe internal ǀaliditLJ is ŐƵaranteed ďLJ tŚe randominjaƟon of parƟĐipants ;ϰ͖ϭϰͿ. titŚin tŚe oĐĐƵpaƟonal ŚealtŚ seƫnŐ͕ Śoǁeǀer͕ parƟĐipantͲleǀel randominjaƟon maLJ not alǁaLJs ďe feasiďle ;e.Ő. ǁŚen interǀenƟons inĐlƵde orŐaninjaƟonal ĐomponentsͿ. /n sƵĐŚ Đases͕ randominjaƟon at tŚe leǀel of departments or loĐaƟons miŐŚt proǀide a more feasiďle approaĐŚ ;i.e. ĐlƵsterͲZTsͿ ;ϯͿ. To ensƵre tŚat tŚe resƵlts of an eĐonomiĐ eǀalƵaƟon are Őeneralinjaďle to oĐĐƵpaƟonal ŚealtŚ praĐƟĐe͕ trial ĐondiƟons sŚoƵld resemďle dailLJ praĐƟĐe as mƵĐŚ as possiďle. &or edžample͕ parƟĐipants sŚoƵld ďe similar to tŚose ǁŚo ǁill edžperienĐe tŚe Ϯϰϯ 8 Chapter 8 interǀenƟon if it is implemented ďroadlLJ͕ monitorinŐ sŚoƵld ďe done Ƶnder roƵƟne ĐirĐƵmstanĐes͕ and interǀenƟons sŚoƵld ďe Đompared to ƵsƵal praĐƟĐe. WĞƌƐƉĞĐƟǀĞ n essenƟal aspeĐt of an eĐonomiĐ eǀalƵaƟon is its perspeĐƟǀe. WerspeĐƟǀe refers to tŚe ͞point of ǀieǁ͟ taŬen to idenƟfLJ releǀant Đosts and ĐonseƋƵenĐes for inĐlƵsion in tŚe eǀalƵaƟon. TŚe ĐŚosen perspeĐƟǀe maLJ ďe tŚat of anLJ releǀant staŬeŚolder or an aŐŐreŐate of staŬeŚolders sƵĐŚ as a soĐietal perspeĐƟǀe. TŚe perspeĐƟǀe determines ǁŚiĐŚ Đosts and ĐonseƋƵenĐes are inĐlƵded. /n tŚe soĐietal perspeĐƟǀe͕ for edžample͕ all Đosts and ĐonseƋƵenĐes are Đonsidered irrespeĐƟǀe of ǁŚo paLJs or ďeneĮts͕ ǁŚereas onlLJ tŚose ďorne ďLJ emploLJers are inĐlƵded ǁŚen tŚe emploLJer͛s perspeĐƟǀe is applied. 'iǀen tŚis faĐt͕ tŚe perspeĐƟǀe is a ĐriƟĐal element in an analLJsis and sŚoƵld tŚerefore ďe stated edžpliĐitlLJ ;ϭͿ. K,^ interǀenƟons are tLJpiĐallLJ iniƟated ďLJ ĐompanLJ manaŐement͖ eitŚer to ĐomplLJ ǁitŚ tŚe laǁ͕ in an eīort to saǀe moneLJ ;i.e. redƵĐed siĐŬness aďsenĐe ĐostsͿ͕ or for moral reasons ;ϭϭͿ. onseƋƵentlLJ͕ most eĐonomiĐ eǀalƵaƟons of sƵĐŚ interǀenƟons are performed from tŚe emploLJer͛s perspeĐƟǀe ;ϰͲϳ͖ϭϱͿ͕ ďƵt otŚer perspeĐƟǀes maLJ also ďe releǀant͖ e.Ő. ǁorŬer͛s͕ insƵrer͛s͕ and soĐietal perspeĐƟǀe. tŚen tŚe emploLJer͛s perspeĐƟǀe is applied͕ ŬeLJ ǁorŬer oƵtĐomes͕ sƵĐŚ as tŚe ǀalƵe of ǁorŬer ŚealtŚ͕ are oŌen not inĐlƵded in tŚe analLJsis͕ ďƵt simplLJ tŚe ŚealtŚͲrelated edžpenses inĐƵrred ďLJ an emploLJer ;e.Ő. prodƵĐƟǀitLJ impliĐaƟonsͿ. TŚis is a ĐriƟĐal oǀersiŐŚt͕ as oĐĐƵpaƟonal ŚealtŚ is essenƟallLJ aďoƵt ǁorŬer ŚealtŚ. soĐietal perspeĐƟǀe is parƟĐƵlarlLJ ƵsefƵl to Đonsider as tŚe perspeĐƟǀe in a stƵdLJ͕ as it proǀides insiŐŚt into tŚe net eīeĐt aĐross all staŬeŚolders. ,ereďLJ͕ it ďeƩer ensƵres tŚat tŚe soĐietal Đosts of an interǀenƟon are less tŚan tŚe ďeneĮts edžperienĐed ďLJ all staŬeŚolders͕ ratŚer tŚan simplLJ tŚe ĐompanLJ͛s Đosts ďeinŐ less tŚan its ďeneĮts ;ϯͿ. TŚis informaƟon ǁill ensƵre tŚat tŚere is a net soĐietal ďeneĮt͕ ratŚer tŚan simplLJ Đost sŚiŌinŐ from one staŬeŚolder to anotŚer. /n addiƟon͕ tŚe disaŐŐreŐated informaƟon on Đosts and ĐonseƋƵenĐes from a soĐietal perspeĐƟǀe proǀides a Őood sense of tŚeir distriďƵƟon aĐross staŬeŚolders. ^ƵĐŚ informaƟon Đan ďe tŚe laƵnĐŚ pad for ďarŐaininŐ ďetǁeen tŚem ;ϭͿ. TŚis maLJ ďe of parƟĐƵlar importanĐe in ĐoƵntries ǁitŚ dƵalͲpaLJer ;e.Ő. TŚe EetŚerlandsͿ and Ƶniǀersal ŚealtŚĐare sLJstems ;e.Ő. TŚe hnited <inŐdomͿ͕ sinĐe Ϯϰϰ WrŝŶĐŝpůeƐ͕ ŵethŽĚƐ͕ aŶĚ reĐŽŵŵeŶĚaƟŽŶƐ emploLJers ŐenerallLJ ďear most of tŚe Đosts of K,^ interǀenƟons͕ ǁŚereas in sƵĐŚ ũƵrisdiĐƟons tŚe ŚealtŚĐare sLJstem andͬor Őoǀernment reaps a larŐe part of tŚeir ďeneĮts ;i.e. redƵĐed mediĐal spendinŐͿ ;ϭϲͿ. TŚerefore͕ it is reĐommendaďle to sƵpplement ĮndinŐs from tŚe emploLJer͛s perspeĐƟǀe ǁitŚ tŚose from otŚer releǀant perspeĐƟǀes͕ parƟĐƵlarlLJ tŚe soĐietal one. ŶĂůLJƟĐƟŵĞĨƌĂŵĞ ZesearĐŚers also need to deĐide aďoƵt tŚe Ɵme frame oǀer ǁŚiĐŚ Đosts and ĐonseƋƵenĐes are analLJsed. TŚe analLJƟĐ Ɵme frame oƵŐŚt to Đoǀer tŚe enƟre period oǀer ǁŚiĐŚ Đosts and ĐonseƋƵenĐes Ňoǁ from tŚe alternaƟǀes Ƶnder ĐonsideraƟon ;ϭϮͿ. TŚis Ɵme frame ŐenerallLJ edžtends ďeLJond tŚe folloǁͲƵp needed to estaďlisŚ tŚe eīeĐƟǀeness of a neǁ interǀenƟon. To illƵstrate͕ tŚe folloǁͲƵp of an eīeĐƟǀeness trial maLJ ďe terminated aŌer tŚe oĐĐƵrrenĐe of tŚe ĐliniĐal eǀent of interest ;e.Ő. inĐidenĐe of ZepeƟƟǀe ^train /nũƵrLJ ;Z^/ͿͿ. /f tŚis folloǁͲƵp ǁoƵld ďe Ƶsed for tŚe eĐonomiĐ eǀalƵaƟon͕ all Đosts and ĐonseƋƵenĐes inĐƵrred dƵrinŐ tŚe ĐoƵrse of tŚe disorder or its reĐƵrrenĐes ǁoƵld not ďe taŬen into aĐĐoƵnt ;e.Ő. Z^/Ͳrelated mediĐaƟon andͬor operaƟon ĐostsͿ͕ leadinŐ to an ƵnderesƟmaƟon of tŚe total Đosts and ĐonseƋƵenĐes. ltŚoƵŐŚ tŚe opƟmal folloǁͲƵp period is ŐenerallLJ ƵnŬnoǁn͕ researĐŚers and readers sŚoƵld at least feel ĐonĮdent tŚat tŚe most important Đosts and ĐonseƋƵenĐes are Đoǀered ďLJ tŚe ĐŚosen analLJƟĐ Ɵme frame. ddiƟonallLJ͕ fƵtƵre Đosts and ĐonseƋƵenĐes tŚat oĐĐƵr aŌer tŚe measƵrement period Đan ďe esƟmated ƵsinŐ informaƟon and data from ǀarioƵs soƵrĐes. TŚis is parƟĐƵlarlLJ important to do if fƵtƵre Đosts and ĐonseƋƵenĐes are edžpeĐted to ďe sƵďstanƟal ;e.Ő. manLJ of tŚe ;ŚealtŚͿ ďeneĮts of preǀenƟǀe interǀenƟons are tŚoƵŐŚt to oĐĐƵr in tŚe fƵtƵreͿ. /ĚĞŶƟĮĐĂƟŽŶ͕ŵĞĂƐƵƌĞŵĞŶƚ͕ĂŶĚǀĂůƵĂƟŽŶŽĨƌĞƐŽƵƌĐĞƵƐĞ /n eĐonomiĐ eǀalƵaƟons͕ Đosts and some ĐonseƋƵenĐes are edžpressed in monetarLJ Ƶnits. &or tŚis pƵrpose͕ releǀant resoƵrĐe Ƶse ĐateŐories sŚoƵld ďe idenƟĮed͕ measƵred͕ and ǀalƵed. s disĐƵssed earlier͕ releǀant resoƵrĐe Ƶse ĐateŐories for inĐlƵsion in an eĐonomiĐ eǀalƵaƟon depend on its perspeĐƟǀe. KtŚer faĐtors tŚat miŐŚt determine tŚe releǀanĐe of a resoƵrĐe Ƶse ĐateŐorLJ are͕ amonŐst otŚers͕ tŚe ĐoƵntrLJ or ũƵrisdiĐƟon in ǁŚiĐŚ tŚe stƵdLJ is ƵndertaŬen and tŚe natƵre of tŚe Ϯϰϱ 8 Chapter 8 alternaƟǀes ďeinŐ Đompared. Ōer releǀant resoƵrĐe Ƶse ĐateŐories are idenƟĮed͕ researĐŚers sŚoƵld determine Śoǁ to Đost tŚem. osƟnŐ ŐenerallLJ inǀolǀes tŚree steps͖ ϭͿ tŚe measƵrement of ƋƵanƟƟes of resoƵrĐes ĐonsƵmed ;YͿ͕ ϮͿ tŚe assiŐnment of Ƶnit priĐes ;WͿ͕ and ϯͿ tŚe ǀalƵaƟon of resoƵrĐes ĐonsƵmed ďLJ mƵlƟplLJinŐ tŚeir ƋƵanƟƟes ďLJ tŚeir respeĐƟǀe unit prices ;YΎWͿ ;ϭͿ. TŚese esƟmates sŚould ďe reported separatelLJ so tŚat readers can ũudŐe tŚe releǀance of tŚese measures to Śis or Śer seƫnŐ ;ϭϳͿ. DeaƐƵreŵeŶt ŽĨ ƋƵaŶƟƟeƐ ŽĨ reƐŽƵrĐeƐ ĐŽŶƐƵŵeĚ Zesource use data are ideallLJ collected prospecƟǀelLJ tŚrouŐŚ a data collecƟon process tŚat is fullLJ inteŐrated into tŚe eīecƟǀeness trial ;ϭ͖ϭϯͿ. lso͕ ǁŚen collecƟnŐ selfͲreported resource use data͕ researcŚers Śaǀe to ďalance recall ďias aŐainst completeness of informaƟon. ^Śorter recall periods reduce tŚe risŬ of parƟcipants forŐeƫnŐ important informaƟon. ,oǁeǀer͕ collecƟnŐ data ǁitŚ relaƟǀelLJ sŚort recall periods ;e.Ő. a couple of ǁeeŬsͿ oǀer a lonŐer period of Ɵme maLJ ďe oǀerlLJ ďurdensome to parƟcipants and maLJ tŚus increase tŚe risŬ of missinŐ data and dropͲ outs. TŚerefore͕ it maLJ ďe ďeƩer to madžiminje completeness at tŚe cost of some recall ďias ;ϭϰͿ͖ e.Ő. ďLJ usinŐ ϮͲ to ϯͲmontŚ recall periods in a trial ǁitŚ a lonŐͲterm folloǁͲup ;шϭϮ montŚsͿ ;ϭϴͿ. lso͕ care sŚould ďe taŬen to collect resource use data conƟnuouslLJ durinŐ folloǁͲup and to aǀoid tŚe need for edžtrapolaƟon of resource use esƟmates ďetǁeen measurement periods. ƐƐŝŐŶŵeŶt ŽĨ ƵŶŝt prŝĐeƐ hnit prices used for ǀaluinŐ resource use ouŐŚt to reŇect opportunitLJ costs͖ i.e. ͞the ǀaůƵe ŽĨ a reƐŽƵrĐe ŝŶ ŝtƐ ŵŽƐt hŝŐhůLJ ǀaůƵeĚ aůterŶaƟǀe ƵƐe͟ ;ϴͿ. In a world of perfect marŬets͕ sucŚ costs are reǀealed ďLJ tŚe marŬet price of a Őood or serǀice. ,oweǀer͕ if a compeƟƟǀe marŬet does not edžist for a Őood or serǀice͕ marŬet prices oŌen are an inaccurate measure of its ǀalue. &or edžample͕ if a premium is paid for a Őood or serǀice due to restricted marŬet entrLJ͕ marŬet prices maLJ oǀeresƟmate tŚe opportunitLJ costs at tŚe societal leǀel. tŚen tŚe societal perspecƟǀe is applied͕ an adũustment sŚould tŚerefore ďe made to tŚe marŬet price͖ e.Ő. ďLJ usinŐ tŚe price of a comparaďle Őood or serǀice ;ϴͿ. &or tŚe emploLJer͛s perspecƟǀe͕ tŚe actual purcŚase Ϯϰϲ WrŝŶĐŝpůeƐ͕ ŵethŽĚƐ͕ aŶĚ reĐŽŵŵeŶĚaƟŽŶƐ costs incurred ďLJ tŚe emploLJer maLJ ďe more appropriate͕ as tŚeLJ ďeƩer represent tŚe sum of moneLJ tŚat is not aǀailaďle to tŚe emploLJer for its ďest alternaƟǀe use ;ϭϮ͖ϭϵͿ. TŚus͕ appropriate unit prices maLJ ǀarLJ ďetween perspecƟǀes͕ and researcŚers sŚould ensure tŚat tŚeLJ reŇect tŚe true resource implicaƟons to tŚe decisionͲmaŬer at Śand ;ϴͿ. ďrief descripƟon of tŚe metŚods used for measurinŐ and ǀaluinŐ tŚe most freƋuentlLJ used resource use cateŐories in economic eǀaluaƟons of K,^ interǀenƟons is proǀided ďelow. TŚe most freƋuentlLJ used resource use cateŐories are͖ interǀenƟon͕ ŚealtŚcare͕ producƟǀitLJ͕ and worŬer͛s compensaƟon costs ;ϰͲϳ͖ϭϱͿ. /ŶterǀeŶƟŽŶ ĐŽƐtƐ InformaƟon on tŚe marŬet price of an interǀenƟon maLJ ďe deriǀed from ǀendors or companLJ andͬor researcŚ proũect records. DanLJ trials͕ Śoweǀer͕ assess noǀel interǀenƟons tŚat eitŚer Śaǀe no predeĮned price weiŐŚts associated witŚ tŚem or for wŚicŚ tŚe use of marŬet prices is inappropriate ;e.Ő. wŚen tŚe societal perspecƟǀe is appliedͿ ;ϭϮͿ. In sucŚ cases͕ tŚe actual interǀenƟon costs can ďe assessed usinŐ a ďoƩomͲup microͲcosƟnŐ approacŚ͕ in wŚicŚ detailed data reŐardinŐ tŚe ƋuanƟƟes of resources consumed as well as tŚeir unit prices are collected per interǀenƟon component separatelLJ. ^ucŚ resources maLJ include interǀenƟon staī Śours͕ materials used͕ depreciaƟon͕ oǀerŚead acƟǀiƟes͕ sƋuare feet of oĸce space͕ and traǀelinŐ ;ϭ͖ϯ͖ϭϮͿ. lso͕ worŬers maLJ ďe taŬen awaLJ from tŚeir reŐular producƟon acƟǀiƟes to parƟcipate in tŚe interǀenƟon and tŚis sŚould ďe accounted for as well. osts associated witŚ tŚe interǀenƟon͛s eǀaluaƟon sŚould not ďe included unless it is a condiƟon of implementaƟon ;ϴͿ. YuanƟƟes of resources consumed can ďe measured usinŐ administraƟǀe dataďases͕ edžpert panels͕ surǀeLJs or interǀiews witŚ interǀenƟon parƟcipants andͬor proǀiders͕ interǀenƟon operaƟon loŐs͕ or oďserǀaƟons ;ϮϬͿ. hnit prices maLJ ďe collected from administraƟǀe dataďases͕ scienƟĮc literature͕ ǀendors͕ andͬor cosƟnŐ manuals ;e.Ő. ;ϮϭͿͿ. Ϯϰϳ 8 Chapter 8 ,eaůthĐare ĐŽƐtƐ IdeallLJ͕ all ŚealtŚcare serǀice use is measured to reduce tŚe liŬeliŚood tŚat ;unedžpectedͿ sŚiŌs in ŚealtŚcare uƟlinjaƟon rates are missed. ltŚouŐŚ tŚis approacŚ will increase tŚe ǀaliditLJ of tŚe results͕ it maLJ not alwaLJs ďe feasiďle. n alternaƟǀe strateŐLJ is to limit data collecƟon to tŚose ŚealtŚcare serǀices tŚat are related to tŚe alternaƟǀes andͬor condiƟon under studLJ ;ϭϮͿ. descripƟon of tŚe care patŚ for tŚe condiƟon under studLJ miŐŚt proǀide researcŚers witŚ a clear picture of wŚat tŚose ŚealtŚcare serǀices are. In all cases͕ care sŚould ďe taŬen to include tŚe most important cost driǀers. ,ealtŚcare uƟlinjaƟon can ďe measured tŚrouŐŚ a ǀarietLJ of means͕ includinŐ retrospecƟǀe ƋuesƟonnaires͕ prospecƟǀe resource use diaries ;i.e. cost diariesͿ͕ and insurance or Śospital dataďases. ataďases͕ Śoweǀer͕ maLJ not alwaLJs contain all reƋuired data͕ and tŚeir ǀaliditLJ and reliaďilitLJ maLJ not ďe ǀerLJ ŚiŐŚ ;ϭϬͿ. Doreoǀer͕ ŚealtŚcare costs ďorne ďLJ parƟcipants ;e.Ő. coͲpaLJments͕ oǀerͲtŚeͲcounter medicaƟonͿ are tLJpicallLJ not included in tŚese dataďases. TŚerefore͕ researcŚers are oŌen dependent on selfͲreport data to measure tŚese ŚealtŚcare uƟlinjaƟon items. To ǀalue ŚealtŚcare uƟlinjaƟon͕ unit prices maLJ eitŚer ďe esƟmated usinŐ a microͲcosƟnŐ approacŚ͕ or ďased on predeĮned price weiŐŚts͕ prices accordinŐ to professional orŐaninjaƟons͕ or tariīs. TLJpicallLJ͕ seǀeral metŚods are used simultaneouslLJ ;ϭϬ͖ϭϵͿ. WrŽĚƵĐƟǀŝtLJ ĐŽƐtƐ &or emploLJers͕ an important ďeneĮt of K,^ interǀenƟons are tŚe resulƟnŐ cŚanŐes in producƟǀitLJ loss. WroducƟǀitLJ loss can ďe deĮned as tŚe companLJ͛s output loss correspondinŐ to reduced laďour input ;i.e. Ɵme and eīortsͬsŬills of tŚe worŬforceͿ. ccordinŐ to tŚis deĮniƟon͕ to ǀalue producƟǀitLJ loss is to ǀalue tŚe output loss ;ϮϮͿ. hnfortunatelLJ͕ Śoweǀer͕ oďũecƟǀe measurement of tŚe true impact of reduced laďour input on a companLJ͛s output is oŌen impossiďle to esƟmate. TŚerefore͕ researcŚers tLJpicallLJ use prodžies of producƟǀitLJ loss͕ wŚicŚ are oŌen esƟmated usinŐ ;selfͲreportedͿ data on tŚe parƟcipants͛ leǀel of aďsenteeism ;i.e. sicŬness aďsenceͿ andͬor presenteeism ;i.e. reduced performance wŚile at worŬͿ. TŚe metŚodoloŐies used for measurinŐ and ǀaluinŐ aďsenteeism and presenteeism are a ĮercelLJ deďated topic in tŚe Įeld of economic eǀaluaƟons. elow͕ a ďrief descripƟon of tŚe Ϯϰϴ WrŝŶĐŝpůeƐ͕ ŵethŽĚƐ͕ aŶĚ reĐŽŵŵeŶĚaƟŽŶƐ most freƋuentlLJ used metŚods is proǀided. &or more informaƟon aďout tŚe main deďates and deǀelopments reŐardinŐ tŚe idenƟĮcaƟon͕ measurement͕ and ǀaluaƟon of producƟǀitLJ we refer to otŚer puďlicaƟons͖ ;ϮϮ͖ϮϯͿ. TŚe two main metŚods for esƟmaƟnŐ aďsenteeism costs are tŚe ,uman apital pproacŚ ;,Ϳ and tŚe &ricƟon ost pproacŚ ;&Ϳ. &or ďotŚ metŚods͕ tŚe numďer of sicŬness aďsence daLJs Śas to ďe collected͕ for wŚicŚ administraƟǀe dataďases͕ selfͲ report ;ƋuesƟonnairesͿ͕ or reports ďLJ otŚers can ďe used ;ϵͿ. &or tŚe &͕ it is also important to idenƟfLJ tŚe numďer and duraƟon of diīerent aďsence periods. ccordinŐ to tŚe ,͕ aďsenteeism costs are eƋual to tŚe amount of moneLJ parƟcipants would Śaǀe earned Śad tŚeLJ not ďeen inũured or ill ;ϰ͖ϮϭͿ. TŚerefore͕ in tŚe ,͕ sicŬness aďsence daLJs are tLJpicallLJ ǀalued usinŐ actual waŐe rates of parƟcipants ;includinŐ emploLJment oǀerŚeads and ďeneĮtsͿ and represent losses for tŚe enƟre duraƟon of aďsence ;ϭ͖ϭϵ͖ϮϰͿ. It is arŐued tŚat tŚe , oǀeresƟmates tŚe true societal cost of sicŬness aďsence͕ as tŚe possiďle replacement of worŬers witŚ lonŐͲterm sicŬness aďsence is not taŬen into account ;ϭ͖ϰͿ. TŚerefore͕ tŚe & was deǀeloped͕ in wŚicŚ producƟon losses are assumed to ďe conĮned to tŚe ƟmeͲspan companies need to replace a sicŬ worŬer ďLJ a formerlLJ unemploLJed person to restore tŚe companLJ͛s iniƟal producƟon leǀel ;i.e. fricƟon periodͿ ;ϮϯͿ. In tŚe &͕ aďsenteeism is tLJpicallLJ ǀalued usinŐ aŐeͲ͕ ŐenderͲ andͬor educaƟonͲspeciĮc price weiŐŚts ;ϮϱͿ. TŚe lenŐtŚ of tŚe fricƟon period depends on tŚe state ;i.e. tŚe unemploLJment rateͿ and eĸciencLJ of tŚe laďour marŬet. s sucŚ͕ fricƟon periods tLJpicallLJ diīer ďetween countries and sŚould ďe esƟmated per countrLJ separatelLJ ;ϭͿ. If tŚere are important cŚanŐes in tŚe economic climate͕ it maLJ ďe necessarLJ to esƟmate tŚe fricƟon period anew. In tŚe EetŚerlands͕ a fricƟon period of Ϯϯ weeŬs is currentlLJ assumed ;ϮϭͿ. TŚus͕ if a sicŬness aďsence period edžceeds Ϯϯ weeŬs͕ aďsenteeism costs are truncated at tŚe costs of Ϯϯ weeŬs. &urtŚermore͕ as a reducƟon of laďour input is oŌen assumed to cause a less tŚan proporƟonal reducƟon in producƟǀitLJ͕ <oopmanscŚap et al. ;ϭϵϵϱͿ also proposed tŚe applicaƟon of an elasƟcitLJ factor of Ϭ.ϴ͕ wŚicŚ is oŌen used in economic eǀaluaƟons tŚat applLJ tŚe &. TŚis elasƟcitLJ factor implies tŚat a ϭϬϬй loss of laďor input corresponds witŚ an ϴϬй reducƟon in producƟǀitLJ ;ϮϱͿ. In tŚe economic eǀaluaƟon literature͕ tŚe need to consider presenteeism as a component of tŚe costs incurred from producƟǀitLJ loss is increasinŐlLJ ďeinŐ Ϯϰϵ 8 Chapter 8 recoŐninjed ;ϵͿ. Wresenteeism is tLJpicallLJ esƟmated usinŐ parƟcipant selfͲreport or report ďLJ otŚers. &or tŚis purpose͕ ǀarious instruments Śaǀe ďeen deǀeloped͕ includinŐ ďotŚ Őeneric ;ϮϲͲϮϵͿ and diseaseͲspeciĮc ƋuesƟonnaires ;ϯϬ͖ϯϭͿ. Dost of tŚese ƋuesƟonnaires measure worŬ performance in terms of points͕ percentaŐes͕ or proporƟons ;ϯϮͿ. TŚese responses can tŚen ďe used to esƟmate tŚe total numďer of worŬinŐ daLJs lost due to presenteeism ďLJ usinŐ tŚe eƋuaƟon͗ W с ; ʹ Ϳ Ύ p where W is full worŬinŐ daLJs lost due to presenteeism͕ is total worŬinŐ daLJs͕ A is sicŬness aďsence daLJs͕ and p is the proporƟon of lost worŬ performance esƟmated ďLJ the instrument used in the studLJ ;ϮϮͿ. To ǀalue the numďer of lost worŬinŐ daLJs due to presenteeism͕ actual waŐe rates of parƟcipants͕ or aŐeͲ͕ ŐenderͲ͕ andͬor ũoďͲ speciĮc price weiŐhts can ďe used. Zesearchers should ďe aware͕ howeǀer͕ that the esƟmated numďer of worŬ daLJs lost due to presenteeism maLJ ǀarLJ widelLJ ďetween instruments. This suŐŐests a lacŬ of comparaďilitLJ amonŐ instruments͕ ďut it is sƟll unclear which instrument proǀides the ďest presenteeism esƟmate ;ϮϮͿ. 'iǀen its siŐniĮcance͕ howeǀer͕ iŐnorinŐ presenteeism maLJ lead to seǀere underesƟmaƟons ;ϮϮͿ. Therefore͕ researchers are recommended to include this resource use cateŐorLJ wheneǀer possiďle. To assess the possiďle inŇuence of the choice of instrument͕ sensiƟǀitLJ analLJses can ďe performed ;^ee ďelowͿ. tŽrŬerƐ͛ ĐŽŵpeŶƐaƟŽŶ ĐŽƐtƐ torŬers͛ compensaƟon is an insurance proŐram͕ oīered in some countries ;e.Ő. anada͕ hnited ^tatesͿ͕ throuŐh which worŬers maLJ receiǀe waŐe replacement andͬ or medical ďeneĮts in the eǀent of an occupaƟonal inũurLJ or disease. &undinŐ usuallLJ comes from premiums paid ďLJ emploLJers ;ϴͿ. To esƟmate worŬers͛ compensaƟon costs͕ total claim costs per parƟcipant can ďe oďtained from companLJ andͬor worŬplace insurance records. It is ŐenerallLJ inadeƋuate͕ howeǀer͕ to use worŬers͛ compensaƟon costs as the sole cost cateŐorLJ͕ as theLJ do not reŇect the full edžtent of worŬͲrelated inũuries and illnesses ;ϰͿ. DanLJ compensaďle inũuries and illnesses Őo unreported and others are not compensaďle ;ϰͿ. then supplemenƟnŐ healthcare ϮϱϬ WrŝŶĐŝpůeƐ͕ ŵethŽĚƐ͕ aŶĚ reĐŽŵŵeŶĚaƟŽŶƐ andͬor producƟǀitLJ costs with worŬers͛ compensaƟon costs͕ douďle counƟnŐ should ďe aǀoided. lso͕ insurance premiumͲrelated waŐe replacement ďeneĮts should ďe edžcluded for the societal perspecƟǀe͕ as theLJ consƟtute ͞transfer paLJments͟ from the emploLJer ǀia the insurer to the worŬer rather than depleted sources ;ϭ͖ϰͿ /ĚĞŶƟĮĐĂƟŽŶ͕ŵĞĂƐƵƌĞŵĞŶƚ͕ĂŶĚǀĂůƵĂƟŽŶŽĨŽƵƚĐŽŵĞƐ s noted ďefore͕ Es haǀe the ŬeLJ outcome measured in natural units. The most appropriate outcome used for this purpose depends on the nature of the alternaƟǀes ďeinŐ compared͕ the condiƟon under studLJ͕ andͬor the applied perspecƟǀe. ^omeƟmes͕ there maLJ ďe some concern aďout whether the chosen outcome captures all releǀant conseƋuences. If this is a concern͕ it is adǀisaďle to conduct mulƟple Es usinŐ diīerent outcomes ;ϴͿ. In hs͕ the ŬeLJ outcome is measured in uƟlitLJ units͕ ŐenerallLJ Ŭnown as Y>zs. TheLJ capture ďoth the duraƟon of surǀiǀal and healthͲrelated ƋualitLJ of life in a sinŐle measure ;ϭ͖ϭϮ͖ϭϰͿ. n adǀantaŐe of Y>zs is that theLJ proǀide a Őeneral indedž score that allows decisionͲmaŬers to compare the conseƋuences of a ranŐe of interǀenƟons for diīerent health issues ;ϭ͖ϭϬͿ. ,oweǀer͕ eǀen thouŐh Y>zs are the preferred outcome measure when healthcare interǀenƟons for paƟents are eǀaluated from the societal perspecƟǀe ;ϭϯ͖Ϯϭ͖ϯϯͿ͕ theLJ haǀe not LJet ďeen freƋuentlLJ used in economic eǀaluaƟons of K,^ interǀenƟons ;ϰ͖ϲ͖ϳ͖ϯϰͿ. This maLJ ďe due to the fact that Y>zs maLJ not reŇect what occupaƟonal health decisionͲmaŬers feel is most important in terms of outcomes. In the case of a worŬplace safetLJ proŐrams͕ for edžample͕ outcomes such as worŬer safetLJ maLJ ďe more meaninŐful to decisionͲmaŬers than a uƟlitLJͲweiŐhted health measure ;ϭϭͿ. Doreoǀer͕ occupaƟonal health decisionͲmaŬers are ŐenerallLJ unfamiliar with Y>zs͕ and Y>zs seem to lacŬ sensiƟǀitLJ to mild condiƟons that are oŌen the focus of K,^ interǀenƟons ;e.Ő. of worŬsite health promoƟon proŐramsͿ ;ϯϱͿ. Therefore͕ more sensiƟǀe uƟlitLJ measures are warranted for economic eǀaluaƟons of K,^ interǀenƟons andͬor uƟlitLJ measures that are more applicaďle to the occupaƟonal health seƫnŐ͖ e.Ő. the recentlLJ conceptualinjed ͞iseaseͲdũusted torŬinŐ zears͕͟ which aims to edžpress the amount of worŬinŐ LJears lost due to poor worŬinŐ condiƟons and associated illness ;ϯϲ͖ϯϳͿ. Ϯϱϭ 8 Chapter 8 ANALYSIS OF AN ECONOMIC EVALUATION elow͕ we discuss some important issues in the analLJsis of trialͲďased economic eǀaluaƟons. To illustrate some of them͕ data is used from an economic eǀaluaƟon that was preǀiouslLJ performed alonŐside a ϭϮͲmonth praŐmaƟc ZT͕ in which construcƟon worŬers at risŬ for cardioǀascular disease either receiǀed a lifestLJle interǀenƟon or usual pracƟce. E in terms of ŬiloŐram ďodLJ weiŐht loss was performed from the societal perspecƟǀe and a from that of the emploLJer. Zesource use cateŐories included interǀenƟon͕ healthcare͕ aďsenteeism͕ and sports costs and were edžpressed in ϮϬϬϴ Euros. Dore detailed informaƟon aďout this trialͲ ďased economic eǀaluaƟon can ďe found elsewhere͖ ;ϯϴͿ. ^ĂŵƉůĞƐŝnjĞ IdeallLJ͕ economic outcomes are used in the sample sinje calculaƟon of a trial ;ϭϯͿ. ,oweǀer͕ althouŐh ǀarious techniƋues haǀe ďeen proposed to esƟmate the appropriate sample sinje for economic endpoints ;ϯϵͲϰϮͿ͕ sample sinje calculaƟons are tLJpicallLJ performed ďased on primarLJ outcomes ;ϭϬ͖ϭϯ͖ϭϰͿ. This is due to the fact that cost data are riŐht sŬewed and therefore reƋuire larŐer sample sinjes to detect releǀant diīerences than ;healthͿ outcome data. ,oweǀer͕ a larŐe sample sinje maLJ neither ďe feasiďle nor ethicallLJ acceptaďle ;ϭϰ͖ϰϯͿ. lso͕ a larŐe numďer of parameters has to ďe speciĮed to perform sample sinje calculaƟons for economic endpoints ;e.Ő. ǀariance parameters of eīecƟǀeness measures͕ cost measures͕ incremental costͲeīecƟǀeness raƟosͿ͕ manLJ of which are hard to predict a priori ;ϯϵ͖ϰϭ͖ϰϮͿ. onseƋuentlLJ͕ trialͲ ďased economic eǀaluaƟons are tLJpicallLJ underpowered for economic outcomes ;ϭϬͿ. >ow powered studies haǀe imprecise and uncertain cost esƟmates and should ďe interpreted with cauƟon ;ϰϯͿ. Doreoǀer͕ if studies are liŬelLJ to ďe underpowered͕ researchers are recommended to use esƟmaƟon rather than hLJpothesis tesƟnŐ ;i.e. ďLJ usinŐ conĮdence interǀals rather than pͲǀaluesͿ ;ϰϳͿ. ĚũƵƐƟŶŐĨŽƌĚŝīĞƌĞŶƟĂůƟŵŝŶŐ InterǀenƟons maLJ haǀe diīerent Ɵme proĮles of costs and conseƋuences. tithin occupaƟonal health͕ interǀenƟon costs are ŐenerallLJ incurred immediatelLJ͕ while ϮϱϮ WrŝŶĐŝpůeƐ͕ ŵethŽĚƐ͕ aŶĚ reĐŽŵŵeŶĚaƟŽŶƐ conseƋuences such as producƟǀitLJ costs miŐht edžtend into the future ;ϰϰͿ. Two tLJpes of adũustments should ďe made to account for these diīerences in ƟminŐ. The Įrst concerns the adũustment of cost data for inŇaƟon͖ i.e. “the general upward prŝĐe ŵŽǀeŵent ŽĨ gŽŽdƐ and ƐerǀŝĐeƐ͟ ;ϭϮͿ. ue to inŇaƟon͕ prices drawn from diīerent LJears are ŐenerallLJ not comparaďle ;ϴͿ. ll prices should therefore ďe adũusted to the same reference LJear usinŐ consumer price indices and the applied reference LJear should ďe stated edžplicitlLJ ;ϭϳͿ. The second adũustment concerns the adũustment of cost and outcome data for Ɵme preferences of indiǀiduals when theLJ are collected oǀer a period of more than one LJear ;ϭϮͿ. Eǀen within a world with njero inŇaƟon͕ indiǀiduals haǀe a preference for receiǀinŐ ďeneĮts todaLJ rather than in the future ;ϭͿ. Therefore͕ costs and conseƋuences incurred in diīerent LJears haǀe to ďe discounted at some rate to esƟmate their present ǀalue ;ϰϰͿ. The appropriate discount rate depends on the ďorrowinŐ cost of moneLJ and other contedžtual factors. 'uidelines for discount rates used in puďlic sector proũects are proǀided ďLJ some ũurisdicƟons. &or edžample͕ in the Eetherlands͕ cost data should ďe discounted at ϰй and health outcomes at ϭ.ϱй͕ while ďoth should ďe discounted at ϯ.ϱй in the hnited <inŐdom ;Ϯϭ͖ϯϯͿ. /ŶƚĞŶƟŽŶͲƚŽͲƚƌĞĂƚĂŶĚŵŝƐƐŝŶŐĚĂƚĂ 'uidelines for conducƟnŐ trials prescriďe that all parƟcipants should ďe included in the analLJses͕ all retained in the Őroup to which theLJ were allocated ;i.e. intenƟonͲ toͲtreat analLJsisͿ ;ϰϱͿ. ,oweǀer͕ true intenƟonͲtoͲtreat analLJses are oŌen hampered ďLJ missinŐ data͕ which are ŐenerallLJ ineǀitaďle in trials. &or economic eǀaluaƟons͕ this proďlem is eǀen more pronounced͕ ďecause total costs are tLJpicallLJ the sum of numerous cost components. s such͕ cost data will alreadLJ ďe incomplete if one component is missinŐ ;ϭϯͿ. DissinŐ data itself maLJ haǀe no relaƟon to oďserǀed and unoďserǀed factors amonŐ parƟcipants ;DZ͗ DissinŐ ompletelLJ t ZandomͿ͕ maLJ onlLJ haǀe a relaƟonship to oďserǀed factors ;DZ͗ DissinŐ t ZandomͿ͕ or maLJ also haǀe a relaƟonship to unoďserǀed factors ;DEZ͗ DissinŐ Eot t ZandomͿ ;^ee odž ϭ for a more detailed descripƟonͿ ;ϰϲͿ. ,istoricallLJ͕ completeͲcase analLJses ;i.e. eliminaƟnŐ cases with missinŐ dataͿ were used to deal with missinŐ data and this is sƟll an oŌen used approach in trialͲďased economic eǀaluaƟons ;ϰϳͿ. ,oweǀer͕ Ϯϱϯ 8 Chapter 8 completeͲcase analLJses reduce the power of a studLJ and lead to ďiased esƟmates if missinŐ data are not DZ ;ϭϮ͖ϭϯͿ. If the rate of missinŐ data is smaller than ϱй͕ completeͲcase analLJses maLJ ďe considered. If more than ϱй of data are missinŐ͕ researchers should use imputaƟon techniƋues to Įll in missinŐ ǀalues. EowadaLJs͕ mulƟple imputaƟon is ŐenerallLJ recommended to impute missinŐ data ;ϭϯ͖ϭϰͿ. then usinŐ mulƟple imputaƟon͕ mulƟǀariate reŐression techniƋues are used to predict missinŐ ǀalues on the ďasis of oďserǀed factors ;ϭϮ͖ϭϰͿ. To account for the uncertaintLJ aďout the missinŐ data͕ seǀeral diīerent imputed datasets are created ;ϰϲͿ. s a rule of thumď͕ thite et al. ;ϮϬϭϭͿ suŐŐested that the numďer of datasets should at least ďe eƋual to the percentaŐe of incomplete cases ;ϰϴͿ. The imputed datasets are suďseƋuentlLJ analLJsed separatelLJ to oďtain a set of parameter esƟmates͕ which can then ďe pooled usinŐ Zuďin͛s rules to oďtain oǀerall esƟmates͕ ǀariances͕ and ϵϱй conĮdence interǀals ;ϵϱйIsͿ ;ϰϲ͖ϰϴ͖ϰϵͿ. DulƟple imputaƟon leads to unďiased esƟmates if missinŐ data are DZ ;ϭϮͿ. Zesearchers should ďear in mind͕ howeǀer͕ that cost and conseƋuence esƟmates deriǀed usinŐ mulƟple imputaƟon are less reliaďle and precise than those ďased on a ϭϬϬй complete dataset ;ϭϰͿ. EǀerLJ endeaǀor should therefore ďe made to miniminje the amount of missinŐ data. Ždžϭ͗dLJƉĞƐŽĨŵŝƐƐŝŶŐĚĂƚĂ;ϰϲͿ ϭͿ DissinŐ ompletelLJ t Zandom ;DZͿ͗ The ͞missinŐness͟ of data has no relaƟonship to oďserǀed and unoďserǀed factors amonŐ parƟcipants. &or edžample͕ sicŬness aďsence data maLJ ďe missinŐ ďecause of proďlems with the reŐistraƟon of this data due to a temporarLJ computer proďlem. ϮͿ DissinŐ t Zandom ;DZͿ͗ The ͞missinŐness͟ of data has a relaƟonship to oďserǀed factors amonŐ parƟcipants͕ ďut not to unoďserǀed factors. &or edžample͕ missinŐ sicŬness aďsence duraƟons maLJ ďe lonŐer than aǀailaďle sicŬness aďsence duraƟons ďut onlLJ ďecause older emploLJees maLJ ďe more liŬelLJ to haǀe missinŐ sicŬness aďsence data. ϯͿ DissinŐ Eot t Zandom ;DEZͿ͗ Eǀen aŌer the oďserǀed data are taŬen into account͕ sLJstemaƟc diīerences remain ďetween the missinŐ ǀalues and the oďserǀed ǀalues. This means that the ͞missinŐness͟ of data also has a relaƟonship to unoďserǀed factors. &or edžample͕ in trials relLJinŐ on selfͲreported sicŬness aďsence͕ parƟcipants with lonŐer sicŬness aďsence duraƟons maLJ ďe more liŬelLJ to forŐet to return their cost diaries ďecause theLJ are not feelinŐ well. Ϯϱϰ WrŝnĐŝpleƐ͕ ŵethŽdƐ͕ and reĐŽŵŵendaƟŽnƐ /ŶĐƌĞŵĞŶƚĂůĂŶĂůLJƐŝƐŽĨĐŽƐƚƐĂŶĚĐŽŶƐĞƋƵĞŶĐĞƐ Ōer costs and conseƋuences haǀe ďeen ƋuanƟĮed͕ their mean diīerences ďetween the interǀenƟon and control Őroup;sͿ as well as the staƟsƟcal siŐniĮcance of these diīerences need to ďe assessed ;ϭϮͿ. s menƟoned aďoǀe͕ cost data are tLJpicallLJ riŐht sŬewed. This is caused ďLJ the fact that onlLJ a small proporƟon of parƟcipants incur hiŐh costs and costs are naturallLJ ďound ďLJ njero ;^ee &iŐure ϭͿ ;ϭͿ. 8 &ŝŐƵƌĞ ϭ͗ ŝƐƚƌŝďƵƟŽŶ ŽĨ ƚŚĞ ƐŽĐŝĞƚĂů ĐŽƐƚƐ ƉĞƌ ƉĂƌƟĐŝƉĂŶƚ ŝŶ Ă ƚƌĂŝůͲďĂƐĞĚ ĞĐŽŶŽŵŝĐ ĞǀĂůƵĂƟŽŶ ŽĨ Ă ůŝĨĞƐƚLJůĞ ŝŶƚĞƌǀĞŶƟŽŶ ĨŽƌ ĐŽŶƐƚƌƵĐƟŽŶ ǁŽƌŬĞƌƐ Ăƚ ƌŝƐŬ ĨŽƌ ĐĂƌĚŝŽǀĂƐĐƵůĂƌ ĚŝƐĞĂƐĞĐŽŵƉĂƌĞĚƚŽƵƐƵĂůƉƌĂĐƟĐĞ;ϯϴͿ The sŬewed cost distriďuƟon complicates the analLJsis of cost data͕ as it ǀiolates the assumpƟons of standard staƟsƟcal tests͕ such as independent tͲtests and linear reŐression analLJses. standard approach to descriďe sŬewed data is to proǀide a summarLJ measure of the distriďuƟon in the form of a median. ,oweǀer͕ this is inappropriate for cost data as decisionͲmaŬers need to ďe aďle to esƟmate the total Ϯϱϱ Chapter 8 cost of implemenƟnŐ a new interǀenƟon ;total implementaƟon costs с mean costs per parƟcipant Ύ numďer of parƟcipantsͿ. s such͕ the arithmeƟc mean is ŐenerallLJ ǀiewed as the most informaƟǀe measure to descriďe cost data ;ϭ͖ϭϰ͖ϱϬͿ. sarious methods are currentlLJ used to compare cost data ďetween studLJ arms͕ includinŐ standard nonͲparametric tests ;e.Ő. DannͲthitneLJ hͿ͕ tͲtests on loŐͲtransformed data͕ and nonͲparametric ďootstrappinŐ. ^tandard nonͲparametric tests compare the distriďuƟon of the data instead of means and are therefore inappropriate. TransformaƟons to normalinje the distriďuƟon are not straiŐhƞorward and are oŌen sensiƟǀe to departures from distriďuƟonal assumpƟons ;ϭϯͿ. Doreoǀer͕ ďacŬͲ transformaƟons are oŌen complicated. Therefore͕ researchers increasinŐlLJ faǀour the nonͲparametric ďootstrap ;ϭϯ͖ϱϬͿ͕ which can ďe used to esƟmate ϵϱйIs around mean cost diīerences while aǀoidinŐ distriďuƟonal assumpƟons ;odž ϮͿ ;ϱϭͿ. ŽdžϮ͗EŽŶͲƉĂƌĂŵĞƚƌŝĐƚƐƚƌĂƉƉŝŶŐ tith nonͲparametric ďootstrappinŐ͕ staƟsƟcal analLJses are ďased on repeatedlLJ samplinŐ with replacement from the oďserǀed data. In short͕ a sample of N parƟcipants is repeatedlLJ drawn with replacement from ďoth the interǀenƟon and control Őroup separatelLJ͕ where N eƋuals the numďer of parƟcipants per studLJ arm. EǀerLJ resample ;i.e. ďootstrap sampleͿ is the eƋuiǀalent of a repeƟƟon of the trial. ^ince resamples haǀe ďeen drawn with replacement ;i.e. per sample͕ parƟcipants can ďe drawn more than onceͿ͕ these ďootstrap samples diīer from one another. Wer ďootstrap sample͕ the staƟsƟcs of interest is calculated ;e.Ő. the diīerence in arithmeƟc mean costs and eīects͕ incrementalͲcost eīecƟǀeness raƟos͕ costͲ ďeneĮt esƟmatesͿ. LJ doinŐ so mulƟple Ɵmes͕ a distriďuƟon for the staƟsƟcs of interest is Őenerated that proǀides an approdžimaƟon of its populaƟon samplinŐ distriďuƟon͕ which can then ďe used to esƟmate conĮdence interǀals ;ϭϮͿ. t least ϮϬϬϬ ďootstrap samples are recommended͕ and preferaďlLJ more ;ϱϮͿ. sarious methods haǀe ďeen proposed to esƟmate ďootstrapped ϵϱй conĮdence interǀals͕ of which the ďias corrected and accelerated method is currentlLJ the preferred one ;ϱϭ͖ϱϯͿ. EonͲparametric ďootstrappinŐ is aǀailaďle in manLJ soŌware pacŬaŐes͕ includinŐ ^W^^͕ ^^͕ ^TT͕ and Z. ŽŵƉĂƌŝŶŐŝŶĐƌĞŵĞŶƚĂůĐŽƐƚƐĂŶĚĐŽŶƐĞƋƵĞŶĐĞƐ The core of anLJ economic eǀaluaƟon is the analLJsis of the relaƟon ďetween the costs and conseƋuences of alternaƟǀes. The preferred methods for conducƟnŐ such analLJses diīer ďetween the tLJpes of economic eǀaluaƟons and are discussed ďelow. Ϯϱϲ WrŝnĐŝpleƐ͕ ŵethŽdƐ͕ and reĐŽŵŵendaƟŽnƐ CŽƐtͲeīeĐƟǀeneƐƐ analLJƐŝƐ and ĐŽƐtͲuƟlŝtLJ analLJƐŝƐ In Es and hs͕ an incremental costͲeīecƟǀeness raƟo ;IEZͿ is calculated ďLJ diǀidinŐ the mean diīerence in cost ;ѐ ostͿ ďetween studLJ arms ďLJ that in eīect ;ѐ EīectͿ. The IEZ indicates the addiƟonal costs of a new interǀenƟon in comparison with a control condiƟon per unit of eīect Őained ;ϭ͖ϭϮͿ. ostinterǀenƟon ʹ ostcontrol ѐ ost с EīectinterǀenƟon ʹ Eīectcontrol с IEZ ѐ Eīect To illustrate͕ a descripƟon of the calculaƟon and interpretaƟon of the edžample trial͛s IEZ is proǀided in odž ϯ. Ždžϯ͗ĂůĐƵůĂƟŽŶĂŶĚŝŶƚĞƌƉƌĞƚĂƟŽŶŽĨƚŚĞŝŶĐƌĞŵĞŶƚĂůĐŽƐƚͲĞīĞĐƟǀĞŶĞƐƐƌĂƟŽ;/ZͿŽĨĂ ůŝĨĞƐƚLJůĞŝŶƚĞƌǀĞŶƟŽŶĨŽƌĐŽŶƐƚƌƵĐƟŽŶǁŽƌŬĞƌƐĂƚƌŝƐŬĨŽƌĐĂƌĚŝŽǀĂƐĐƵůĂƌĚŝƐĞĂƐĞĐŽŵƉĂƌĞĚ ƚŽƵƐƵĂůƉƌĂĐƟĐĞ;ϯϴͿ urinŐ followͲup͕ interǀenƟon Őroup parƟcipants siŐniĮcantlLJ decreased their ďodLJ weiŐht ďLJ Ϯ.ϬϮ ŬiloŐram compared to the control Őroup ;ѐ EīectͿ. Dean societal costs per parƟcipant were nonͲsiŐniĮcantlLJ hiŐher in the interǀenƟon Őroup compared to the control Őroup ďLJ ΦϮϵϯ ;ѐ ostͿ. hsinŐ this informaƟon͕ the IEZ can ďe calculated͖ IEZ͗ ΦϮϵϯͬϮ.ϬϮ с Φϭϰϱ This IEZ indicates that societLJ has to paLJ Φϭϰϱ per parƟcipant in the interǀenƟon Őroup for each addiƟonal ŬiloŐram ďodLJ weiŐht loss compared to usual pracƟce. 8 IEZs are ŐenerallLJ hard to interpret. &or edžample͕ neŐaƟǀe IEZs miŐht represent reduced costs and posiƟǀe eīects indicaƟnŐ a winͲwin situaƟon or increased costs and neŐaƟǀe eīects indicaƟnŐ a loseͲlose situaƟon ;ϭϰͿ. Therefore͕ IEZs are oŌen ŐraphicallLJ illustrated on costͲeīecƟǀeness planes ;EͲplanesͿ͕ in which incremental eīects are ploƩed on the yͲadžis and incremental costs on the zͲadžis ;&iŐure ϮͿ ;ϱϰ͖ϱϱͿ. If an IEZ is located either in the ^outh East Yuadrant ;^EͲYͿ or the Eorth test Yuadrant ;EtͲYͿ͕ the choice ďetween alternaƟǀes is clear ;assuminŐ there is no uncertaintLJ surroundinŐ the IEZͿ. In the ^EͲY͕ the new interǀenƟon is more eīecƟǀe and less costlLJ than the control condiƟon and is therefore said to dominate Ϯϱϳ Chapter 8 the control condiƟon. In the EtͲY͕ the opposite is true and the new interǀenƟon is dominated ďLJ the control condiƟon. If a new interǀenƟon is more eīecƟǀe and more costlLJ ;EEͲY͗ Eorth East YuadrantͿ or less eīecƟǀe and less costlLJ ;^tͲY͗ ^outh test YuadrantͿ͕ the decision whether or not to adopt it depends on the soͲcalled ͞willinŐnessͲtoͲpaLJ͟ ;ʄͿ. That is͕ the madžimum amount of moneLJ decisionͲmaŬers are willinŐ to paLJ for an addiƟonal unit of eīect ;ϭͿ. To illustrate͕ a hLJpothesinjed ʄ is depicted as the diaŐonal line in &iŐure Ϯ and diǀides the EͲplane into a costͲeīecƟǀe and a nonͲcostͲeīecƟǀe halǀe. IEZs located to the riŐht of this line are considered acceptaďle͕ whereas IEZs located to the leŌ are considered inacceptaďle ;ϭϰ͖ϱϰ͖ϱϱͿ. The more decisionͲmaŬers are willinŐ to paLJ for an addiƟonal unit of eīect͕ the steeper the slope of this line ;ϭϰͿ. EĞǁƚƌĞĂƚŵĞŶƚ ŵŽƌĞĐŽƐƚůLJ EŽƌƚŚ ǁĞƐƚ ƋƵĂĚƌĂŶƚ ,LJƉŽƚŚĞƐŝnjĞĚ ǁŝůůŝŶŐŶĞƐƐͲƚŽͲƉĂLJ ;ȜͿ EĞǁŝŶƚĞƌǀĞŶƚŝŽŶ ŝƐŵŽƌĞĞĨĨĞĐƚŝǀĞ ďƵƚ ŵŽƌĞĐŽƐƚůLJ ŽŶƚƌŽů ĐŽŶĚŝƚŝŽŶ ĚŽŵŝŶĂƚĞƐ EĞǁƚƌĞĂƚŵĞŶƚ ůĞƐƐ ĞĨĨĞĐƚŝǀĞ EĞǁƚƌĞĂƚŵĞŶƚ ŵŽƌĞĞĨĨĞĐƚŝǀĞ EĞǁŝŶƚĞƌǀĞŶƚŝŽŶ ŝƐůĞƐƐ ĐŽƐƚůLJ ďƵƚ ůĞƐƐ ĞĨĨĞĐƚŝǀĞ ^ŽƵƚŚ ǁĞƐƚ ƋƵĂĚƌĂŶƚ &ŝŐƵƌĞϮ͗ŽƐƚͲĞīĞĐƟǀĞŶĞƐƐƉůĂŶĞ Ϯϱϴ EŽƌƚŚ ĞĂƐƚ ƋƵĂĚƌĂŶƚ EĞǁŝŶƚĞƌǀĞŶƚŝŽŶ ĚŽŵŝŶĂƚĞƐ ^ŽƵƚŚ ĞĂƐƚ ƋƵĂĚƌĂŶƚ EĞǁƚƌĞĂƚŵĞŶƚ ůĞƐƐ ĐŽƐƚůLJ WrŝnĐŝpleƐ͕ ŵethŽdƐ͕ and reĐŽŵŵendaƟŽnƐ tith parƟcipantͲleǀel data͕ it is natural to consider represenƟnŐ the uncertaintLJ surroundinŐ IEZs usinŐ ϵϱйIs. ,oweǀer͕ as a raƟo measure͕ esƟmaƟnŐ ϵϱйIs around IEZs is not straiŐhƞorward and͕ more importantlLJ͕ ϵϱйIs around IEZs suīer from the same interpretaƟon proďlem as IEZs ;ϱϱͿ. Therefore͕ alternaƟǀe methods haǀe ďeen proposed to esƟmate the uncertaintLJ surroundinŐ IEZs. urrent Őuidelines recommend usinŐ the ďootstrap method descriďed in odž Ϯ. In this case͕ ďoth incremental costs and eīects are calculated per ďootstrap sample. The uncertaintLJ surroundinŐ an IEZ can then ďe ŐraphicallLJ illustrated ďLJ ploƫnŐ these ďootstrapped incremental costͲeīect pairs ;EͲpairsͿ on a EͲplane. s indicated ďLJ the edžample trial͛s EͲplane proǀided in &iŐure ϯ͕ EͲpairs commonlLJ coǀer more than one Ƌuadrant. 8 &ŝŐƵƌĞϯ͗ŽƐƚͲĞīĞĐƟǀĞŶĞƐƐƉůĂŶĞĨŽƌĂůŝĨĞƐƚLJůĞŝŶƚĞƌǀĞŶƟŽŶĨŽƌĐŽŶƐƚƌƵĐƟŽŶǁŽƌŬĞƌƐĂƚƌŝƐŬ ĨŽƌĐĂƌĚŝŽǀĂƐĐƵůĂƌĚŝƐĞĂƐĞĐŽŵƉĂƌĞĚƚŽƵƐƵĂůƉƌĂĐƟĐĞ;ϯϴͿ ďďreǀiaƟon͗ EtͲY͗ Eorth test Yuadrant͕ EEͲY͗ Eorth East Yuadrant͕ ^tͲY͗ ^outh test Yuadrant͕ ^EͲY͗ ^outh East Yuadrant͕ IEZ͗ Incremental ostͲEīecƟǀeness ZaƟo lthouŐh EͲplanes Őiǀe a Őood impression of the uncertaintLJ surroundinŐ the IEZ͕ theLJ do not proǀide a summarLJ measure of the ũoint uncertaintLJ of costs and eīects ;ϱϲͿ. Therefore͕ costͲeīecƟǀeness acceptaďilitLJ curǀes ;EsͿ were introduced Ϯϱϵ Chapter 8 that proǀide insiŐht into the proďaďilitLJ that a new interǀenƟon is costͲeīecƟǀe compared to the control condiƟon. This proďaďilitLJ can ďe esƟmated ďLJ determininŐ what proporƟon of EͲpairs is located in the costͲeīecƟǀe half of the EͲplane ;i.e. to the riŐht of the preǀiouslLJ menƟoned line with the slope eƋual to ʄͿ;&iŐure ϮͿ. ^ince it is ŐenerallLJ unŬnown what decisionͲmaŬers are willinŐ to paLJ for an addiƟonal unit of eīect͕ ʄ is ǀaried ďetween its natural ďounds ;ranŐe͗ Ϭ to ьͿ and the proďaďilitLJ that the new interǀenƟon is costͲeīecƟǀe compared to the control condiƟon is esƟmated for a ranŐe of ʄs. These ǀalues can then ďe ploƩed on Es that show the proďaďilitLJ of costͲeīecƟǀeness ;zͲadžisͿ for ǀarious ʄs ;yͲadžisͿ ;ϱϱͲϱϳͿ. To illustrate͕ the E of the edžample trial is proǀided in &iŐure ϰ. &ŝŐƵƌĞϰ͗ŽƐƚͲĞīĞĐƟǀĞŶĞƐƐĂĐĐĞƉƚĂďŝůŝƚLJĐƵƌǀĞĨŽƌĂůŝĨĞƐƚLJůĞŝŶƚĞƌǀĞŶƟŽŶĨŽƌĐŽŶƐƚƌƵĐƟŽŶ ǁŽƌŬĞƌƐĂƚƌŝƐŬĨŽƌĐĂƌĚŝŽǀĂƐĐƵůĂƌĚŝƐĞĂƐĞĐŽŵƉĂƌĞĚƚŽƵƐƵĂůƉƌĂĐƟĐĞ;ϯϴͿ Eote͗ This costͲeīecƟǀeness acceptaďilitLJ curǀe corresponds with the costͲeīecƟǀeness plane in &iŐure ϯ and indicates the proďaďilitLJ of the interǀenƟon ďeinŐ costͲeīecƟǀeness for diīerent ǀalues of willinŐnessͲtoͲpaLJ per ŬiloŐram ďodLJ weiŐht loss. ϮϲϬ WrŝnĐŝpleƐ͕ ŵethŽdƐ͕ and reĐŽŵŵendaƟŽnƐ This E indicates that if decisionͲmaŬers are not willinŐ to paLJ anLJthinŐ to oďtain an addiƟonal ŬiloŐram ďodLJ weiŐht loss ;i.e. ʄсϬͿ͕ there is a Ϭ.ϯϯ proďaďilitLJ that the new interǀenƟon is costͲeīecƟǀe compared to the control condiƟon. If decisionͲmaŬers are willinŐ to paLJ ΦϮϬϬϬ ;i.e. ʄсϮϬϬϬͿ͕ this proďaďilitLJ is Ϭ.ϵϱ. then interpreƟnŐ Es͕ two approaches can ďe used ďLJ decisionͲmaŬers. If their willinŐness to paLJ is Ŭnown͕ theLJ haǀe to ũudŐe whether the proďaďilitLJ of costͲeīecƟǀeness at this ceilinŐ raƟo is acceptaďle. If their willinŐness to paLJ is unŬnown͕ theLJ should consider whether the ceilinŐ raƟo at an acceptaďle proďaďilitLJ of costͲeīecƟǀeness is acceptaďle to them. The laƩer miŐht depend on the scale of the outcome measure and the preǀalence of the condiƟon under studLJ. CŽƐtͲďeneĮt analLJƐŝƐ In health economics and ďusiness administraƟon͕ ǀarious measures edžist for comparinŐ costs and ďeneĮts. Kf them͕ the Eet eneĮts ;EͿ͕ eneĮt ost ZaƟo ;ZͿ͕ and ZeturnͲKnͲInǀestment ;ZKIͿ are the most freƋuentlLJ used measures in occupaƟonal health research and can ďe esƟmated usinŐ the followinŐ eƋuaƟons ;ϲͿ͗ N с eneĮtƐ ʹ CŽƐtƐ CZ с eneĮtƐ ͬ CŽƐtƐ ZK/ с ;eneĮtƐ ʹ CŽƐtƐͿ ͬ CŽƐtƐ ΎϭϬϬ where CŽƐtƐ are deĮned as interǀenƟon costs and eneĮtƐ as the diīerence in moneƟnjed outcomes ďetween the interǀenƟon and control Őroup ;e.Ő. diīerence in producƟǀitLJ costsͿ. eneĮts are esƟmated ďLJ suďtracƟnŐ the mean edžpenses incurred ďLJ the interǀenƟon Őroup parƟcipants from those of the control Őroup. ,ereďLJ͕ posiƟǀe ďeneĮts indicate reduced spendinŐ. The E indicates the amount of moneLJ Őained aŌer costs are recoǀered ;i.e. net loss or net saǀinŐsͿ. The Z indicates the amount of moneLJ returned per monetarLJ unit inǀested. The ZKI indicates the percentaŐe of proĮt per monetarLJ unit inǀested ;ϱϴ͖ϱϵͿ. InterǀenƟons can ďe reŐarded as cost saǀinŐ if the followinŐ criteria are met͗ EхϬ͕ Zхϭ͕ and ZKIхϬй. To illustrate͕ a descripƟon of the calculaƟon and interpretaƟon of the edžample trial͛s costͲďeneĮt esƟmates is proǀided in odž ϰ. Ϯϲϭ 8 Chapter 8 Ždžϰ͗ĂůĐƵůĂƟŽŶĂŶĚŝŶƚĞƌƉƌĞƚĂƟŽŶŽĨƚŚĞĐŽƐƚͲďĞŶĞĮƚĞƐƟŵĂƚĞƐŽĨĂůŝĨĞƐƚLJůĞŝŶƚĞƌǀĞŶƟŽŶ ĨŽƌĐŽŶƐƚƌƵĐƟŽŶǁŽƌŬĞƌƐĂƚƌŝƐŬĨŽƌĐĂƌĚŝŽǀĂƐĐƵůĂƌĚŝƐĞĂƐĞŝŶĐŽŵƉĂƌŝƐŽŶƚŽƵƐƵĂůƉƌĂĐƟĐĞ (38) Dean interǀenƟon costs per parƟcipant were ΦϲϬϱ. urinŐ followͲup͕ aǀeraŐe aďsenteeism costs per parƟcipant were ΦϯϯϬϮ in the interǀenƟon Őroup and ΦϯϲϬϰ in the control Őroup. Thus͕ the aďsenteeism ďeneĮts per parƟcipant were ΦϯϬϮ ;ΦϯϲϬϰ Ͳ ΦϯϯϬϮͿ. hsinŐ this informaƟon͕ costͲďeneĮt esƟmates can ďe calculated͖ E͗ ΦϯϬϮ Ͳ ΦϲϬϱ с ΦͲϯϬϯ Z͗ ΦϯϬϮ ͬ ΦϲϬϱ с Ϭ.ϱϬ ZKI͗ ;;ΦϯϬϮ ͬ ΦϲϬϱͿͬ ΦϯϬϮͿΎϭϬϬ с ͲϱϬй These costͲďeneĮt esƟmates indicate that the interǀenƟon resulted in a net loss to the emploLJer of ΦϯϬϯ. lso͕ per Euro inǀested͕ the emploLJer Őained ΦϬ.ϱϬ and suīered a loss of ϱϬй. Thus͕ the interǀenƟon cannot ďe reŐarded as costͲďeneĮcial in terms of aďsenteeism costs. ostͲďeneĮt esƟmates͕ and Zs and ZKIs in parƟcular͕ are tLJpicallLJ presented without an indicaƟon of their uncertaintLJ. If uncertaintLJ is suďstanƟal and this is not taŬen into account͕ wronŐ conclusions could ďe drawn. Therefore͕ we recommend the use of the preǀiouslLJ descriďed ďootstrap method ;odž ϮͿ to esƟmate the uncertaintLJ surroundinŐ costͲďeneĮt esƟmates. In this case͕ the E͕ Z͕ andͬor ZKI are calculated per ďootstrap sample ;i.e. ďootstrapped Es͕ Zs͕ and ZKIsͿ. ^uďseƋuentlLJ͕ ϵϱйIs can ďe esƟmated usinŐ the ďias corrected and accelerated method ;ϱϭ͖ϱϯͿ. Eǀen thouŐh Zs and ZKIs are raƟo measures͕ esƟmaƟnŐ their ϵϱйIs is straiŐhƞorward as the denominator ;i.e. interǀenƟon costsͿ is tLJpicallLJ posiƟǀe. DanLJ occupaƟonal health decisionͲmaŬers͕ howeǀer͕ maLJ lacŬ the necessarLJ staƟsƟcal ďacŬŐround to interpret ϵϱйIs ;ϭϭͿ. possiďle waLJ to deal with this issue is to determine what proporƟon of ďootstrapped Es͕ Zs͕ andͬor ZKIs indicate cost saǀinŐs ;i.e. ͞the proďaďilitLJ of Įnancial return͟Ϳ. KccupaƟonal health decisionͲ maŬers can suďseƋuentlLJ use this informaƟon to consider whether the estaďlished proďaďilitLJ of Įnancial return is acceptaďle to them. then reporƟnŐ results͕ economists and policLJ maŬers prefer the E͕ whereas the Z and ZKI are more familiar to ďusiness manaŐers. s such͕ it is recommendaďle to report at least two of them ;i.e. E and ZͬZKIͿ͕ so that the results can ďe easilLJ interpreted ďLJ all staŬeholders. nother adǀantaŐe of this approach is that it maŬes the results easilLJ comparaďle with those of other studies͕ ďecause diīerent metrics ϮϲϮ WrŝnĐŝpleƐ͕ ŵethŽdƐ͕ and reĐŽŵŵendaƟŽnƐ are used in the literature to esƟmate whether K,^ interǀenƟons Őenerate cost saǀinŐs ;ϲͿ. ^ĞŶƐŝƟǀŝƚLJĂŶĂůLJƐŝƐ Economic eǀaluaƟons are oŌen conducted in the contedžt of incomplete informaƟon and uncertaintLJ͕ which necessitates the use of prodžLJ measures͕ and inǀariaďlLJ͕ the need to maŬe assumpƟon aďout the methods and unit prices used for ǀaluinŐ resource use͕ the methods used for dealinŐ with incomplete data͕ and the waLJ in which adũustments are made for diīerenƟal ƟminŐ ;ϰ͖ϴͿ. Therefore͕ sensiƟǀitLJ analLJses should ďe undertaŬen to assess how studLJ results would chanŐe for diīerent ŬeLJ assumpƟons and parameter ǀalues ;i.e. the roďustness of studLJ resultsͿ ;ϭϳ͖ϲϬͿ. The ranŐes of ǀalues tested͕ and arŐuments for selecƟnŐ these ranŐes͕ must ďe clearlLJ descriďed ;ϭϬ͖ϭϳͿ. sarious Ŭinds of sensiƟǀitLJ analLJses edžist. KneͲwaLJ sensiƟǀitLJ analLJses assess the impact of chanŐes to a sinŐle parameter at a Ɵme͕ while mulƟple parameters are ǀaried simultaneouslLJ in mulƟͲwaLJ sensiƟǀitLJ analLJses ;ϲϭͿ. DISCUSSION Zesources for occupaƟonal health are scarce. This maŬes it necessarLJ for decisionͲ maŬers to haǀe informaƟon on the relaƟǀe eĸciencLJ of K,^ interǀenƟons in order to allocate aǀailaďle resources to their ďest use. s such͕ economic eǀaluaƟons of K,^ interǀenƟons are ďecominŐ increasinŐlLJ important͕ manLJ of which are conducted alonŐside eīecƟǀeness trials. TrialͲďased economic eǀaluaƟons proǀide a uniƋue opportunitLJ to reliaďlLJ esƟmate the resource implicaƟons of K,^ interǀenƟons at low incremental cost ;ϭϬ͖ϭϰͿ. ,oweǀer͕ it is criƟcal that hiŐh ƋualitLJ trialͲďased economic eǀaluaƟons are performed when this informaƟon is used to inform allocaƟon decisions. esiŐninŐ a hiŐh ƋualitLJ trialͲďased economic eǀaluaƟon reƋuires close collaďoraƟon ďetween occupaƟonal health specialists͕ indiǀiduals edžecuƟnŐ the trial͕ and health economists ;ϭϰͿ. areful consideraƟons must ďe made reŐardinŐ the perspecƟǀe͕ the analLJƟc Ɵme frame͕ the idenƟĮcaƟon͕ measurement͕ and ǀaluaƟon of resource use and outcomes͕ as well as the methods used for calculaƟnŐ sample sinjes͕ comparinŐ Ϯϲϯ 8 Chapter 8 costs and conseƋuences͕ and handlinŐ missinŐ data and uncertaintLJ. The laƩer is of parƟcular importance͕ as few economic eǀaluaƟons in occupaƟonal health report on the uncertaintLJ surroundinŐ their incremental costͲconseƋuence esƟmates ;ϰͲϳ͖ϭϱͿ. &ailinŐ to esƟmate ǀalues under uncertaintLJ maŬes it impossiďle to determine the certaintLJ of results and could thus lead to inappropriate decisionͲmaŬinŐ. To ƋuanƟfLJ precision͕ nonͲparametric ďootstrappinŐ can ďe used as a staƟsƟcal techniƋue for dealinŐ with the riŐht sŬewed nature of cost data ;ϭ͖ϳͿ. n oǀerǀiew of our core recommendaƟons for trialͲďased economic eǀaluaƟons in occupaƟonal health can ďe found in ppendidž ϭ. TrialͲďased economic eǀaluaƟons maLJ also haǀe shortcominŐs͕ includinŐ limited sample sinjes͕ limited comparators͕ and truncated Ɵme horinjons ;ϭϰͿ. To deal with the laƩer͕ researchers miŐht consider edžtrapolaƟnŐ economic eǀaluaƟon results ďeLJond the followͲup of a trial ďLJ usinŐ decision analLJƟc modelinŐ͕ in which edžpected costs and conseƋuences ďetween alternaƟǀes are compared ďLJ sLJnthesinjinŐ informaƟon from mulƟple sources ;e.Ő. scienƟĮc literature͕ studLJ resultsͿ ;ϭ͖ϭϯ͖ϭϰͿ. &or more detailed informaƟon aďout decision analLJƟc modelinŐ we refer to other puďlicaƟons͖ ;ϭϰ͖ϲϮͿ. lso͕ eǀen thouŐh we recommend a praŐmaƟc ;clusterͲͿZT desiŐn for economic eǀaluaƟons͕ we are aware that randominjaƟon itself maLJ not alwaLJs ďe feasiďle andͬor desired in the occupaƟonal health seƫnŐ. In those cases͕ well edžecuted nonͲrandominjed studies maLJ proǀide ǀaluaďle informaƟon͕ ďut it is criƟcal that eīorts ďe made to control for selecƟon ďias ;e.Ő. ďLJ usinŐ propensitLJ score matchinŐͿ ;ϲϯ͖ϲϰͿ. then interpreƟnŐ economic eǀaluaƟons of K,^ interǀenƟons͕ it is important to ďear in mind that their results maLJ not ďe directlLJ applicaďle to other countries and ũurisdicƟons due to diīerences in healthcare͕ social securitLJ sLJstems͕ and other factors. serďeeŬ et al. ;ϮϬϭϬͿ demonstrated that economic eǀaluaƟon results can ďe Őeneralinjed from one countrLJ to another. ,oweǀer͕ to enaďle the necessarLJ calculaƟons͕ researchers need to proǀide an edžtensiǀe descripƟon of the interǀenƟon͕ a detailed list of resource use as well as informaƟon of the healthcare sLJstem in the oriŐinal studLJ and the allocaƟon of costs to ǀarious staŬeholders ;ϲϱͿ. Ϯϲϰ WrŝnĐŝpleƐ͕ ŵethŽdƐ͕ and reĐŽŵŵendaƟŽnƐ LJ simultaneouslLJ proǀidinŐ recommendaƟons for Őood pracƟce in the economic eǀaluaƟon of K,^ interǀenƟons and discussinŐ the methods and principles that underlie them͕ the present paper aimed to help researchers in conducƟnŐ and reporƟnŐ hiŐh ƋualitLJ trialͲďased economic eǀaluaƟons. ^uch studies are edžpected to contriďute to the deǀelopment of a sound eǀidence ďase on the resource implicaƟons of K,^ interǀenƟons ;ϯ͖ϰͿ͕ which is a necessarLJ prereƋuisite for eǀidenceͲďased pracƟces occurrinŐ in occupaƟonal health ;ϭϭͿ. The present paper maLJ also ďe helpful to consumers of this literature with understandinŐ and criƟcallLJ appraisinŐ trialͲďased economic eǀaluaƟons of K,^ interǀenƟons͕ which miŐht help improǀe the uptaŬe of their results. ĐŬŶŽǁůĞĚŐĞŵĞŶƚƐ te would liŬe to thanŬ all authors of the edžample trial for the proǀision of their data. 8 Ϯϲϱ Chapter 8 REFERENCES ϭ. Ϯ. ϯ. ϰ. ϱ. ϲ. ϳ. ϴ. ϵ. ϭϬ. ϭϭ. ϭϮ. ϭϯ. ϭ4. ϭϱ. 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