Session 3: Communication of HTE to Key Stakeholders Daniel Arthur - - PowerPoint PPT Presentation

Daniel Arthur Caños, PhD, MPH Director, Evidence Development Division Coverage and Analysis Group Center for Clinical Standards and Quality CMS

Session 3: Communication

• f HTE to Key Stakeholders

Challenge Question

• Ho w do e s CMS pro mo te the a sse ssme nt o f he te ro g e ne ity
• f tre a tme nt e ffe c ts while c linic a l e vide nc e is b e ing

de ve lo pe d?

• Wha t sub -g ro ups a re o f inte re st to Me dic a re whe n

g e ne ra liza b ility is c o nside re d fo r Na tio na l Co ve ra g e De te rmina tio ns?

42 CFR § 405.212 Medicare Coverage IDE study criteria

(1) T he princ ipal purpo se o f the study is to te st whe the r the de vic e impro ve s he a lth o utc o me s o f a ppro pria te ly se le c te d pa tie nts. (2) T he ra tio na le fo r the study is we ll suppo rte d b y a va ilable sc ie ntific a nd me dic a l info rma tio n, o r it is inte nde d to c la rify o r e sta b lish the he a lth o utc o me s o f inte rve ntio ns a lre a dy in c o mmo n c linic al use . (3) T he study re sults a re no t a ntic ipa te d to unjustifia b ly duplic a te e xisting kno wle dg e . (4) T he study de sig n is me tho do lo g ic ally a ppro priate a nd the a ntic ipate d numb e r o f e nro lle d sub je c ts is a de q ua te to c o nfide ntly a nswe r the re se a rc h q ue stio n(s) b e ing a ske d in the study. (5) T he study is spo nso re d b y a n o rg a niza tio n o r individua l c a pa b le o f suc c e ssfully c o mple ting the study. (6) T he study is in c o mplianc e with a ll a pplic a ble F e de ra l re g ula tio ns c o nc e rning the pro te c tio n o f huma n sub je c ts fo und a t 21 CF R pa rts 50, 56, a nd 812 a nd 45 CF R pa rt 46. (7) Whe re a ppro pria te , the study is no t de sig ne d to e xc lusive ly te st to xic ity o r dise a se pa tho physio lo g y in he a lthy individua ls. Studie s o f a ll me dic a l te c hno lo g ie s me a suring the ra pe utic o utc o me s a s o ne o f the o b je c tive s ma y b e e xe mpt fro m this c rite rio n o nly if the dise a se o r c o nditio n b e ing studie d is life thre a te ning a nd the pa tie nt ha s no o the r via b le tre a tme nt o ptio ns. (8) T he study is re g iste re d with the Na tio nal Institute s o f He a lth's Na tio nal L ib ra ry o f Me dic ine 's Clinic alT rials.g o v. (9) T he study pr

• toc ol de sc r

ibe s the me thod and timing of r e le ase of r e sults on all pr e - spe c ifie d

• utc ome s, inc luding r

e le ase of ne gative outc ome s and that the r e le ase should be haste ne d if the study is te r minate d e ar ly. (10) T he study pr

• toc ol must de sc r

ibe how Me dic ar e be ne fic iar ie s may be affe c te d by the de vic e unde r inve stigation, and how the study r e sults ar e or ar e not e xpe c te d to be ge ne r alizable to the Me dic ar e be ne fic iar y population. Ge ne r alizability to populations e ligible for Me dic ar e due to age , disability, or

• the r

e ligibility status must be e xplic itly de sc r ibe d.

Evidence-based Medicare Coverage

Na tio na l Co ve ra g e De te rmina tio n (NCD):

• Suffic ie nt e vide nc e to c o nc lude tha t the ite m o r se rvic e

impro ve s c linic a lly me a ning ful he a lth o utc o me s fo r the Me dic a re po pula tio n

• Ba se d o n a c o mpre he nsive re vie w o f pub lishe d e vide nc e

Medicare Beneficiaries in Clinical Studies

• I

nitia l studie s o n ne w te c hno lo g ie s ma y no t inc lude ma ny

• lder adults ≥ 65 years of age for several reasons

inc luding :

• He te ro g e ne ity – ma y ha ve multiple c o mo rb iditie s a nd/ o r b e

ta king multiple me dic a tio ns

• No n-a dhe re nc e - ma y ha ve diffic ulty fo llo wing pro to c o ls a nd/ o r

ma king a ll study fo llo w-up visits

• Othe r c o nside ra tio ns – me a sure me nt issue s, c o g nitive func tio n

Coverage with Evidence Development (CED)

• Co ve ra g e in the c o nte xt o f a ppro ve d c linic a l studie s o r with the

c o lle c tio n o f a dditio na l c linic a l da ta

• Allo ws fo r po sitive c o ve ra g e whe n e vide nc e is insuffic ie nt fo r a

mo re fa vo ra b le de c isio n

• E

vide nc e g a ps ma y b e due to lo w numb e r o f b e ne fic ia rie s in c linic a l studie s, la c k o f me a ning ful he a lth o utc o me s, limite d g e ne ra liza b ility, inc o nsiste nc y o f study finding s.

• Witho ut CE

D, the ite m o r se rvic e wo uld b e no n-c o ve re d

• Ma y invo lve ra ndo mize d c o ntro lle d tria ls, o b se rva tio na l studie s

a nd/ o r re g istrie s

• spe c ific inte rve ntio n,
• b e ne fits a nd ha rms,
• he a lth o utc o me s

CMS & AHRQ General Requirements

a ) T he princ ipa l purpo se o f the study is to te st whe the r the ite m o r se rvic e me a ning fully impro ve s he a lth o utc o me s o f a ffe c te d b e ne fic ia rie s who a re re pre se nte d b y the e nro lle d sub je c ts. b ) T he ra tio na le fo r the study is we ll suppo rte d b y a va ila b le sc ie ntific a nd me dic a l e vide nc e . c ) T he study re sults a re no t a ntic ipa te d to unjustifia b ly duplic a te e xisting kno wle dg e . d) T he study de sig n is me tho do lo g ic a lly a ppro pria te a nd the a ntic ipa te d numb e r o f e nro lle d sub je c ts is suffic ie nt to a nswe r the re se a rc h q ue stio n(s) b e ing a ske d in the Na tio na l Co ve ra g e De te rmina tio n. e ) T he study is spo nso re d b y a n o rg a niza tio n o r individua l c a pa b le o f c o mple ting it suc c e ssfully. f) T he re se a rc h study is in c o mplia nc e with a ll a pplic a b le Fe de ra l re g ula tio ns c o nc e rning the pro te c tio n o f huma n sub je c ts fo und in the Co de o f Fe de ra l Re g ula tio ns (CFR) a t 45 CFR Pa rt 46. I f a study is re g ula te d b y the Fo o d a nd Drug Ad ministra tio n (FDA), it is a lso in c o mplia nc e with 21 CFR Pa rts 50 a nd 56. I n a dditio n, to furthe r e nha nc e the pro te c tio n o f huma n sub je c ts in studie s c o nduc te d unde r CE D, the study must pro vide a nd o b ta in me a ning ful info rme d c o nse nt fro m pa tie nts re g a rding the risks a sso c ia te d with the study ite ms a nd/ o r se rvic e s, a nd the use a nd e ve ntua l dispo sitio n o f the c o lle c te d da ta . g ) All a spe c ts o f the study a re c o nduc te d a c c o rding to a ppro pria te sta nda rds o f sc ie ntific inte g rity. h) T he study ha s a writte n pro to c o l tha t c le a rly de mo nstra te s a dhe re nc e to the sta nda rds liste d he re a s Me dic a re re q uire me nts. i) T he study is no t de sig ne d to e xc lusive ly te st to xic ity o r dise a se pa tho physio lo g y in he a lthy individua ls. Suc h studie s ma y me e t this re q uire me nt o nly if the dise a se o r c o nditio n b e ing studie d is life thre a te ning a s de fine d in 21 CFR §312.81(a ) a nd the pa tie nt ha s no o the r via b le tre a tme nt o ptio ns. j) T he c linic a l re se a rc h studie s a nd re g istrie s a re re g iste re d o n the www.Clinic a lT ria ls.g o v we b site b y the princ ipa l spo nso r/ inve stig a to r prio r to the e nro llme nt o f the first study sub je c t. Re g istrie s a re a lso re g iste re d in the Ag e nc y fo r He a lthc a re Qua lity (AHRQ) Re g istry o f Pa tie nt Re g istrie s (Ro PR). k) T he re se a rc h study pro to c o l spe c ifie s the me tho d a nd timing o f pub lic re le a se o f a ll pre spe c ifie d o utc o me s to b e me a sure d inc luding re le a se o f o utc o me s if o utc o me s a re ne g a tive o r study is te rmina te d e a rly. T he re sults must b e ma de pub lic within 12 mo nths o f the study’ s prima ry c o mple tio n da te , whic h is the da te the fina l sub je c t ha d fina l da ta c o lle c tio n fo r the prima ry e ndpo int, e ve n if the tria l do e s no t a c hie ve its prima ry a im. T he re sults must inc lude numb e r sta rte d/ c o mple te d, summa ry re sults fo r prima ry a nd se c o nda ry o utc o me me a sure s, sta tistic a l a na lyse s, a nd a dve rse e ve nts. Fina l re sults must b e re po rte d in a pub lic ly a c c e ssib ly ma nne r; e ithe r in a pe e r-re vie we d sc ie ntific jo urna l (in print o r o n-line ), in a n o n-line pub lic ly a c c e ssib le re g istry de dic a te d to the disse mina tio n o f c linic a l tria l info rma tio n suc h a s Clinic a lT ria ls.g o v, o r in jo urna ls willing to pub lish in a b b re via te d fo rma t (e .g ., fo r studie s with ne g a tive o r inc o mple te re sults).

l) T he study pr

• toc ol must e xplic itly disc uss be ne fic iar

y subpopulations affe c te d by the ite m or se r vic e unde r inve stigation, par tic ular ly tr aditionally unde r r e pr e se nte d gr

• ups in c linic al studie s, how the inc lusion and e xc lusion

c r ite r ia e ffe c t e nr

• llme nt of the se populations, and a plan for

the r e te ntion and r e por ting of said populations in the tr

• ial. If the inc lusion and e xc lusion c r

ite r ia ar e e xpe c te d to have a ne gative e ffe c t on the r e c r uitme nt or r e te ntion of unde r r e pr e se nte d populations, the pr

• toc ol must disc uss why the se c r

ite r ia ar e ne c e ssar y. m) T he study pr

• toc ol e xplic itly disc usse s how the r

e sults ar e or ar e not e xpe c te d to be ge ne r alizable to affe c te d be ne fic iar y subpopulations. Se par ate disc ussions in the pr

• toc ol may be ne c e ssar

y for populations e ligible for Me dic ar e due to age , disability or Me dic aid e ligibility. Guidance for the Public, Industry, and CMS Staff: Coverage with Evidence Development