Preparing for success with comparative effectiveness research.

Correspondence: Thomas G. Roberts, Jr., M.D., Farallon Capital Management, L.L.C., One Maritime Plaza, ite , an Francisco, Cali ornia . Mail: troberts arcap.com Recei ed Ma , accepted or p blication J ne , . lphaMed Press . http: d doi.org . theoncologist. . Decades o p blic and pri ate in estment in cancer biolog research has led to an enhanced nderstanding o the ndamental genetic changes that lead to the ormation and progres sion o cancer . Clinical scientists ha e or ed iterati el o er this same period to de elop n anced chemotherap regimens that ha e either e tended s r i al e pectations or most patients ith common t pes o cancer, or in the cases o pediatric cancers, germ cell t mors, and some hematologic malignancies, e ected c res. The last decade in partic lar has itnessed a period o intense prod cti it in the de elop ment o molec larl targeted therapies man o these agents ha e prod ced impressi e and s stained tho gh not et c rati e bene its in de ined s bgro ps o patients, at least ntil resistance de elops. Most recentl , therapies rooted in no ledge o cancer imm nolog ha e sho n promising res lts in s bgro ps that are not ell ser ed b e isting c toto ic and tar geted therapies these ad ances are highl li el to pla a more meaning l role in the oncolo gist s t re armamentari m . n alone, the FD appro ed ne molec lar entities or biologic agents or the treatment o cancer, rep resenting a third o FD appro als across all ther ape tic classes . O er the last t o ears, the n mber o cancer appro als ar e ceeded those in all other ields. t is no possible or all b t the most s eptical obser ers to en ision an enco r aging path or ard. One estion that arises implicitl rom this positi e d namic is hether the oncolog com m nit , partic larl in the nited tates, has prepared or s ccess. a e e de eloped s s tems to e al ate comprehensi el ne thera pies in real orld settings re e ro tinel generating the e idence that can help red ce practice ariations and impro e health o t comes in lnerable gro ps re e appl ing the ne therapies in the most appropriate set tings, directing them primaril to patients most li el to recei e a bene it a e e re ined methods that can estimate the al e both clini cal and economic o ne therapies compared to the clinical pro iles and costs o re erence standards Complicating the matter rther is the realization that oncolog s er s ccess, no and in the t re the direct res lt o one o the most prod cti e p blic pri ate partner ships in merican histor is threatened b iscal b dget constraints at the er same time that cancer care costs threaten the iscal health o the nited tates. The ational Cancer nsti t te estimates, sing conser ati e ass mp tions, that the real costs o cancer care in the nited tates ill increase at least o er the period rom to and ma e ceed billion constant c rrenc b the end o the decade . These cancer care costs, hich are gro ing aster than costs in other areas o medicine, ill comprise a s bstantial portion o the Medicare and Medicaid b dgets that ha e been cited b at least one ma or ratings agenc as sti ication or the loss o merica s credit rating ith rther do ngrades possible. Comparati e e ecti eness research C R , the latest name gi en to e orts that began in the s to impro e alit and ma imize the al e o health ser ices, o ers a s stem atic approach to addressing the conundrum brought upon b oncolog s success. C R has been used in most de eloped countries to a greater or lesser e tent to ma e e idence based unding decisions and to ormulate treat ment guidelines , . ndeed, the current en ironment should be ielding enthusiasm or the implementation o C R, et the ield has struggled to de ine its role ithin the merican cancer communit . The challenges or C R ha e been mani old. First, the enormit o the tas re uired to de elop e ecti e treatments has di erted attention understandabl rom some o the more conte tual uestions that C R see s to address. Critical in uir into the relati e eco nomic alue o ne treatments has not neces saril been encouraged as oncologists right ull pushed or greater inno ation. econd, until relati el recentl , the cost o cancer care has been minor hen compared to other