RARE-Bestpractices Conference. 24 November 2016, Istituto Superiore di Sanità, Rome, Italy

Transcription

1 RARE-Bestpractices Conference 24 November 2016, Istituto Superiore di Sanità, Rome, Italy

2 valuing patients and public preferences Francis Arickx Dir. Pharmaceutical Policy National Institute for Health and Disability Insurance 2

3 valuing patients and public preferences 3

4 HTA serves a purpose 4

5 to support informed decision making for allocating limited resources to give access to therapies 5

6 access = availability + affordability 6

7 number of available innovative medicines sales per inhabitants vs. number of available innovative medicines ,5 2 1,5 1 0,5 sales per inhabitants (in mio EURO) 5 0 FR DK ES BE IR DE AU SE IT SK GR FI SI UK CZ NL LU HU RO EE PT PO BG LT LV 0 7

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10 # number of high cost drugs, complexity and price will continue to grow Oncology (targeted therapies, biomarkers, etc.) Auto-immune conditions Orphan Medicinal Products Source: IMS

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13 Belgium NIHDI expenditures OMP 13

14 ETA / ETR Early Temporary Access/Reimbursement 14

15 Supply driven reimbursement: Industry proposes, payer disposes Unmet medical needs: even with continuously growing health expenditure, a lot of medical needs remains unmet UMN / ETR procedure 15

16 Demand driven reimbursement: what? To-reimburse list made by payer, to be fulfilled by industry Demand driven reimbursement: why? Meet the unmet medical needs Reimbursement management But also: Regulation for the reimbursement of Off-label use Sustain research/small patients populations diseases Sustain small companies/ risky investments Demand driven reimbursement: how? Introduce authorities assessment on targeted diseases/treatment Listen to needs: physicians, patients, regulators

17 Unmet Medical Needs: priorities? Unmet Medical Needs: theoretically infinite Budget Health Insurance : in practice limited Prioritization Multi Criteria Decision Analysis Objective: Development of tool for ranking therapeutic and societal needs CAIT (UMN Commission) KCE (Belgian health care knowledge centre) King Baudouin Foundation

18 Source: King Baudouin Foundation 18

19 Total weighted score n on o on on Total weighted score KCE (Belgian health care knowledge centre) A multi-criteria decision approach for the appraisal of unmet needs: A pilot study de e assessme t Therapeutic need OUTPUT: 2 rankings : 1 for therapeutic need 1 for societal need List of ranked needs Societal need o o o o o o a erapeut eed o o o o a o etal eed 19

20 9 questions 5 criteria MEDICAL VULNERABILITY Max score Impact on life expectancy? 3 Impact on quality of life? 5 Rarity? 2 Disadvantages actual treatment? 2 SOCIAL VULNERABILITY Gender: Pregnant women 1 criteria weighting Age: children 1 Pat e t s d ty u derm ed? 1 SOCIETAL IMPACT Societal resources (human/financial) required? 1 Public health issue? 1 20

21 UMN list 2016: applications appraisal results Spinal muscular atrophy (SMA) Amyotrophic lateral sclerosis Recurrent glioblastoma Pancreatic cancer BRCA+ Mesothelioma (L2) Duchenne muscular dystrophy Selective early removal of eschar in children with deep thermal burns Acute Lymphocytic Leukemia (ALL) Metastatic uveal melanoma Acute Myeloid Leukemia (AML) FLT3 + Metastasized Non-Small Cell Lung Cancer T790m+ (2L) Recurrent and/or metastatic squamous cell carcinoma of the head and neck (SCCHN) Advanced metastatic gastric cancer HER2 + (L2 and + ) Serious bleedings and coagulopathy with fibrinogen deficiency Primary progressive multiple sclerosis Osteogenesis imperfecta Non-Small Cell Lung Cancer ALK + in progression after crizonitib Metastatic breast cancer ER+/HER2- Non-Small Cell Lung Cancer PD-L1+ (L3) Metastatic breast cancer BRCA+ Non-Small Cell Lung Cancer KRASm+ Recurrent or refractory chronic lymphocytic leukemia (17p del R/R CLL) Aggressive systemic mastocytosis (ASM) Non-Small Cell Lung Cancer (L2) Myasthenia gravis Lysosomal acid lipase deficiency Lupus nephritis Sporadic inclusion body myositis (sibm) Periodic fever syndromes (HIDS, TRAPS, crfmf) Alzheimer Non-Hodgkin Lymphoma (L1) Chronic hepatitis C with stage 4 5 chronic kidney disease Emergency treatment of known or suspected opioid overdose Alpha-1 antitrypsin deficiency (AATD) Refractory partial onset epilepsy with or without secondarily generalized tonic-clonic seizures (SGTCS) Clostridium difficile infection (recurrence prevention) Hypogammaglobulinemia (HGG) by solid organ transplantation Hidradenitis Suppurativa Non-infectious uveitis Hypoparathyroidism life threatening result QOL result rarity result social vulnerability result societal impact result

22 BeNeLuxA initiative 22

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24 the Collaboration Initiative Resulting from a need, identified and expressed by several Member States, European Commission and Council, partners, stakeholders,.. WPPHSL Working Party on Public Health at Senior Level NM CAPR Network Meeting Competent autorities on Pricing and reimbursement Multi-stakeholders Workshop on Pharmaceuticals Industry Process on Corporate Responsibility in the Field of Pharmaceuticals A call to make valuable innovative medicines accessible in the European Union (Belgium) Council Conclusions on Innovation and Solidarity (Belgium) Council Conclusions on Access to Innovation for the Benefit of Patients (Italy) Commission Staff Working Document: Pharmaceutical Industry: A Strategic Sector for the European Economy.. 24

25 the Collaboration Initiative A proof of concept for to collaborate on coalition of the willing Health Technology Assessment Horizon Scanning Exchange of information on pharmaceutical markets, prices and disease specific cross border registries Pricing and reimbursement including joint negotiation 25

26 the Collaboration Initiative A proof of concept for coalition of the willing Letter of intent signed by four ministers Mandate to deliver proposals for cooperation on HTA, Horizon Scanning and Pricing and Reimbursement topics To perform pilots on these topics Expensive medicines High cost per patient High budget impact Voluntary Consensus based cooperation Reimbursement decisions are national competence 26

27 the Collaboration Initiative A proof of concept for coalition of the willing Main goal: To ensure access to innovative drugs at affordable cost Cooperation is part of the policy-mix Information gathering on global markets benefits from joint approach National context determines course of action Joint negotiations in select cases What works? Setting clear, common goals Mutual benefit needs to be clear Pragmatic approach Focus on desired outcomes Lean organisational structure 27

28 the Collaboration Initiative where are we now? Objective is to examine and test in practice true collaboration Feasibility and added value (lessons learned) of the collaboration in the different areas will be documented (terms of reference and communication), evaluated and shared with stakeholders and other interested Member States Therefore need to be able to work n n nv onm n (discretion equals NOT intransparency), based on mutual understanding and trust Commitment to play an active role on EU level (EUNetHTA JA3, MoCA, Senior Party, EU presidency, etc..) 28

29 the Collaboration Initiative where are we now? Coop a on n a l only 37,5 million inhabitants Large interest from Industry Cooperation could lead to higher, instead of lower prices Interest of many individual companies Commitment to success Only one chance to build a solid cooperation Cooperation is not the solution to all problems Joint negotiations require Proof of concept Terms of reference Review of current pilots 29

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