Which stakeholders to involve? Who is the Stakeholder? What are dilemmas in stakeholder involvement? Why to involve stakeholders? Regulators.

Transcription

1 A LACK OF EFFECTIEESS, COST AD EPIDEMIOLOGIC DATA: WHAT ARE THE OPTIOS FOR CODUCTIG PHARMACOECOOMIC STUDIES I CETRAL AD EASTER EUROPE? Basic economic data related to healthcare Epidemiology of cancer Financial resources allocated to and organization of cancer treatment Breast cancer project in 4 CE countries Jana Skoupa, MD ISPOR Prague, 9. ovember 200

2 Rapidly increasing due to new entries Formal CE and BI assessment since L02 L Mio CZK Source: GLOBOCA 2008 Source: ISPOR Poster HTA agency not in all countries in place Absence of official methodology for CEA WTP threshold absent in Czech Inaccessibility of data «Low lewel of co operation with payers Affordability Index = annual spending L0 per Inhabitants (IMS data) Czech Republic as reference =,0 Source: Local survey Czech Slovakia Poland Hungary Ca specialists/ M inhab () A Specialized centers/0 M inhab () Local guidelines available DRG system BEACIZUMAB COLORECTAL CA BEACIZUMAB RCC BEACIZUMAB BREAST CA BEACIZUMAB LUG CA Source: Local survey Aim of project: «Treatment sequence in breast Ca (un resectable, locally recurrent or metastatic in patients not suitable for hormonal therapy) «Resource use in these breast Ca patients (excl. chemotherapy) Included CZ, SK, HU and PL «Sample covered 30 % patients per country Healthcare/payers perspective (direct costs)

3 Common questionnaire Treatment initiation «Routine follow up during treatment period «Pre progression/after treatment follow up «Disease progression «Each position consisted of ««Percentage consuming and frequency per given period Hospitalization (if any) incl. LOS Assessment per disease period and total course Data derived from local IS/chart review Similar proportions (80 90%) proceed to second line treatment Differences are seen in proceeding to third line chemotherapy Lab testing Imaging Outpatient visits «Included medical and radiation oncologists, surgeons Hospital stays BSC/palliative/terminal care Grade 3 and 4 AEs CZECH SLOAKIA Proportion of patients completing 3 lines of treatment: HUGAR POLAD FAC=fluorouracil/doxorubicin/cyclofosfamid; AT=doxorubicin/paclitaxel; CMF=cyklofosfamid/fluorouracil/methitrexat; R=vinorelbine

4 Different organization of cancer care «Centers, specialists, reimbursement of care Differences in «Centers, Differences in Limited options/value of transferring CE «Treatment sequences data across CEE countries. Cost varies as of sequences «First line chemotherapy Cost varies as of «Hospitalization frequency and/or reimbursement «BSC and palliative care specialists, reimbursement of care «Treatment Different organization of cancer care «First line chemotherapy «Hospitalization frequency and/or reimbursement «BSC and palliative care A LACK OF LOCAL EFFECTIEESS, COST AD EPIDEMIOLOGIC DATA: WHAT ARE THE OPTIOS FOR CODUCTIG PHARMACOECOOMIC STUDIES I CETRAL AD EASTER EUROPE? Key Stakeholders for Local Pharmacoeconomic Studies and their Contribution to Overall Success. PharmDr. Martin išňanský, MBA, PhD. President, ISPOR Local Chapter, Slovakia SLOAHTA, n.o. skoupa@pharmaprojects.cz Agenda Who is the Stakeholder? Who is the Stakeholder? Which stakeholders to involve? Why to involve stakeholders? What are dilemmas in stakeholder involvement? Patients Regulators What we have learned? different groups and organizations bring key information and experience on both the producing of concrete Health Technology Assessments (HTAs) and on the various health interventions that could be subject to an HTA; the main goal is to facilitate a transparent, responsible, accountable, participative and responsive stakeholder involvement process; the stakeholder policy applies to the EUnetHTA Joint Action , and does not apply to stakeholder policies at a national level. Groups or organizations which potentially will be affected by, or have an interest in and may in a consultative role influence on the actions or aims of an HTA organization, project or policy direction. Source: EUnetHTA Join Action

5 Who is the Stakeholder? Patient and Healthcare Consumer Organizations Healthcare Providers Payers Industry Health Media Regulators (Ministry of Health, Drug Agency, etc.) Experts, Opinion Leaders, Academics Source: EUnetHTA Join Action Which stakeholders to involve? ) who to involve 2) how to involve and 3) what to involve different stakeholder groups in? Ideally ALL OF THEM, however, with proper BALACE Source: Garrido et al.: HTA and Health-Policy in Europe. Current Status, Challenges and Potential Why to involve stakeholders? The main objective of stakeholder involvement is to ensure representation of interests in and legitimacy of HTA and thereby enhance the possibility of utilizing HTA in policy making. legitimacy = reason giving Legitimacy concerns the ability to give reasons for policy decisions that are understood and perceived as sound by those who are to submit to that authority. Sources: Humphrey M. Democratic legitimacy, public justification and environmental direct action. Political Studies.; 2006;54: King LA. Deliberation, legitimacy, and multilateral democracy. Governance. 2003;6():-2. Parkinson J.. Legitimacy problems in deliberative democracy. Political Studies. 2003;5:80-96 Adopted from EUnetHTA JA & Stakeholders Forum What are dilemmas in stakeholder involvement? How to obtain a balanced representation of all potential interests? What to do if industry is overrepresented and patients, medical societies, health care managers interests hardly represented? It appears as if stakeholders with funding can be active, those without have difficulties! What level of agreement between all partners and stakeholders is needed on methodological issues and assessment of quality of studies and data? How to obtain sharing of data and other information between partners and stakeholders? Mutuality is desirable. Source: EUnetHTA Join Action Patients Regulators Dear policy maker: Have you made up your mind? A discrete choice experiment among policy makers and other health professionals Marc A. Koopmanschap, Elly A. Stolk Erasmus University Medical Center Xander Koolman Delft University of Technology Objectives: The aim of this study was to get insight in what criteria as presented in Health technology assessment (HTA) studies are important for decision makers in healthcare priority setting. Source: Bridges and Jones: Int J Ass of Tech in Healthcare; 23: (2007), Conclusions: Dutch policy makers and other healthcare professionals seem to have reasonably well articulated preferences: six of seven attributes were significant. Disease severity, budget impact, and cost effectiveness were very important. The results are comparable to international studies, but reveal a larger set of important decision criteria. Source: International Journal of Technology Assessment in Health Care, 26:2 (200),

6 What did we learn? It is important to know the interests of stakeholders Involve! Be interested! Know more! It is important to plan for stakeholder involvement: secure time and resources Be precise about who, how and when to involve stakeholders at different levels (local, regional, national, supranational ) Rafał Zyśk Rafał Zyśk MD, MBA HEC, Warsaw, Poland Polish HTA Agency Polish HTA Agency came into being in Before its existence no public health technology assessment process was in Poland. Reasons for negative recommendation by Polish HTA Agency An example of oncology treatment Agency is led by Agency president dr Wojciech Matusewicz. Agency consists of 3 crucial departments, led by directors. Mean number of issued recommendations per year 60. Essential advisory body Consultancy Board (0 professors). Rafał Zyśk MD, MBA, ISPOR, Praha 200 Polish HTA Agency Drug and non drug medical technologies Amount of recommendations Recommendations for drug technologies 90 Recommendations for non-drug technologies 65 Sum of all recommendations

7 Positive, negative and conditional recommendations for drugs egative recommendations in particular medicine areas egative recommendations for drugs in medicine areas Drug technologies recommendations (%) Positive recommendations 68 egative recommendations 83 Conditional recommendations 38 Abstention Sum of recommendations for drugs 90 Recommendations for oncological drugs Recommendations for oncological drugs Positive recommendations 5 egative recommendations 9 Conditional recommendations 8 Abstention All 43 egative recommendations and reasons in oncology umber of recommendations alergology rare deseases internal medicine dermatology diabetology gyneacology cardiology nephrology neonatology neurology ophtalmology oncology psychiatry pulmunology rheumatology all Crucial reasons for negative recommendations for oncological drugs Reasons for negative recommendations* umber of recommendati ons Indication in application was different then SCP indication o possibilities for strict monitoring after reimbursement decision Lack of cost effectiveness 8 Application filed after deadline Safety profile 3 Low clinical effectiveness 2 Incredible evidence 3 Incredible HTA report 0 * for recommendation or more reasons are possible Crucial reasons for negative recommendation for oncological drugs Weak clinical evidence In case of low quality clinical trials, giving unclear, uncertain outcomes, Agency takes negative recommendation until presenting more convincing evidence (eg. bexaroten in T-cell lymphoma , clofarabine, capecitabine, temozolomide 2008). Low clinical effectiveness - In case of poor therapeutical effects presented in clinical trials Agency takes negative recommendations (eg. erlotinib in SCLC , temozolomide 2008, sunitinib in renal cancer , bevacizumab in renal cancer 2009, temsirolimus in renal cancer 2009, erlotinib in pancreatic cancer - 200)

8 Crucial reasons for negative recommendation for oncological drugs Incredible HTA report In case of application of HTA report, against the Polish Guidelines for conducting HTA, Agency takes negative recommendation (e.g. bevacizumab in CRC 2008, clofarabine ) RZ Crucial reasons for negative recommendation for oncological drugs Lack of cost effectiveness In case of significant exceed of cost effectiveness threshold (3x GDP per capita) for QAL or LG, Agency takes negative recommendation (eg. lenalidomid in multiple myeloma , bevacizumab in renal cancer 2009, sorafenib in renal cancer 2009) Safety profile During process of reimbersment decision taking, one of the most important things is safety profile of new substance. This reason was deciding among others: zolendronic acid 2008, cetuximab Crucial reasons for negative recommendation for oncological drugs Conclusions o possibilities for strict monitoring of reimbursement after reimbursement decision. Positive HTA recommendations and positive conditional with major restrictions prevailed in Poland (eg. trastuzumab in BC application for open reimbursement in every pharmacies). Polish reimbursement system prefers financing of high-cost drugs within special therapeutical hospital programs with predefined inclusion and exclusion criteria than in pharmacies. Clinical rather than economic aspect was the most common reason for an appraisal decision (clinical aspects 60% of reasons, economic aspects 8% of reasons) In the nearest future ew project of Reimbursement Bill contains: Extension of Consultancy Board (20 members) Creation of Economical Board responsible for price negotiations Introduction of risk-sharing mechanisms Publishing - HTA reports and verification analysis on the HTA Agency webside ( Thank you for your attention Rafal Zyśk MD, MBA 48

9 Slide 43 RZ this reason doesn't require explanation HE Consulting, 0/26/200

10 A LACK OF EFFECTIEESS, COST AD EPIDEMIOLOGIC DATA: WHAT ARE THE OPTIOS FOR CODUCTIG PHARMACOECOOMIC STUDIES I CETRAL AD EASTER EUROPE?