New innovative HTA methods for supporting novel approaches to deliver better precision medicine

Transcription

1 New innvative HTA methds fr supprting nvel appraches t deliver better precisin medicine W. Gettsch, PhD 1,2 1. Natinal Health Care Institute (ZIN), Diemen, the Netherlands 2. Utrecht Institute fr Pharmaceutical Sciences, Utrecht University, Utrecht, the Netherlands

2 Defintin and (traditinal) fcus f HTA Health technlgy assessment (HTA) is a multidisciplinary prcess that summarises infrmatin abut the medical, scial, ecnmic and ethical issues related t the use f a health technlgy in a systematic, transparent, unbiased, rbust manner. Its aim is t infrm the frmulatin f safe, effective, health plicies that are patient fcused and seek t achieve best value. Traditinally fcus n: single technlgies, mstly pharma and medtech Natinal r reginal recmmendatins t supprt reimbursement and pricing plicies 2

3 Changes in fcus f HTA Internatinalizatin (increasing efficiency, quality & predictability) Medical vs nn-medical (jint REA by EUnetHTA) Alignment between stakehlders Within HTA (EUnetHTA) Outside HTA (regulatrs, health care prviders, patients etc.) Real wrld data Persnalized treatments Smaller ppulatins Cmbinatin technlgies Different sequences f treatments 3

4 Develpment f mre persnalized treatment cncepts CAR-T treatment in practice (USA) Franken, MG. et al. Plicymaker, Please Cnsider Yur Needs Carefully: Des Outcmes Research in Relapsed r Refractry Multiple Myelma Reduce Plicymaker Uncertainty Regarding Value fr Mney f Brtezmib? Value in Health 2014, Vlume 17, Issue 2, Schulthess D, Gassul D, Makady A etal. Assessment f Chimeric Antigen T-cell Therapy (CAR-T). Effectiveness in practice. Drug Infrmatin Assciatin (DIA) Cngress, Bstn, June

5 Mre fcus n later stages f life cycle f pharmaceuticals Use f technlgy in health care Presenting and discussing requirements studies in ED* Cllecting evidence in early develpment Initial HTA Assessment fr market authrizatin Time line f innvatin Cmparative r full HTA (supprting apprpriate use) Additinal data cllectin HTA STAKEH REGU *Early dialgue 5

6 NL Experiences with cnditinal funding (CF) and additinal data cllectin ( ) T=0: Assessment Therapeutic value & 1e CE/BIA Uncertainties identified Outcmes research prpsal discussed Outcmes Research Dutch clinical practice Eg. registries T=4: Re-assessment Cnclusins Advice t MH n reimbursement Feedback frm stakehlders n these experiences *Makady etal. Value in Health 2018 (accepted). 6

7 Internatinal Initiatives & Future Directins EUnetHTA WP5B: Cre datasets fr HTA registries EMA Registry initiatives: Fr instance qualificatin f disease registries IMI-GetReal I & II: RWE use fr clinical effectiveness f drugs IMI-BigData prjects, IMI-EHDEN: Using registries and big data generating RWE, cllect health data in cmmn data structure ISPOR/ISPE Special Task Frce: Gd prcedural & reprting practices fr RWE studies H2020 HTx (next generatin HTA, ) prject: 7 develp methds that can integrate RWE and RCT in rder t predict (cst-)effectiveness in small ppulatins T pilt implementatin f these methds in Eurpe with the HTA bdies (EUnetHTA) and their stakehlders Invlvement in HTx frm HTA, academics, patients, regulatrs etc.

8 Cnclusins New HTA methds are needed: supprt internatinalizatin adaptatin t a new era f persnalized medicine by cmbining RCT with RWD/big data alignment with regulatrs but als healthcare prviders, patients and technlgy prducers In the cming years this will be supprted by Interactin f HTA rganisatins in EUnetHTA Develpment f mre persnalized, methds thrugh IMI and H2020 prjects Implementatin f these mdels in HTA practice; EUnetHTA, IMI-GetReal, big data and H2020-HTx (internatinal) But again in cllabratin with regulatrs, clinicians, patients and technlgy prducers 8