Phacilitate Cell  Gene Therapy 2012   Faulkner Worldwide Hta Analysis
Upcoming SlideShare
Loading in...5
×
 

Phacilitate Cell Gene Therapy 2012 Faulkner Worldwide Hta Analysis

on

  • 920 views

 

Statistics

Views

Total Views
920
Views on SlideShare
900
Embed Views
20

Actions

Likes
0
Downloads
6
Comments
0

2 Embeds 20

http://www.linkedin.com 16
https://www.linkedin.com 4

Accessibility

Categories

Upload Details

Uploaded via as Microsoft PowerPoint

Usage Rights

© All Rights Reserved

Report content

Flagged as inappropriate Flag as inappropriate
Flag as inappropriate

Select your reason for flagging this presentation as inappropriate.

Cancel
  • Full Name Full Name Comment goes here.
    Are you sure you want to
    Your message goes here
    Processing…
Post Comment
Edit your comment

Phacilitate Cell  Gene Therapy 2012   Faulkner Worldwide Hta Analysis Phacilitate Cell Gene Therapy 2012 Faulkner Worldwide Hta Analysis Presentation Transcript

  • Analysis of regenerative medicinetechnologies worldwide: How havethey been assessed by HTAs on a global basis? Plenary Session Eric Faulkner, MPH Director, Global Market Access, Quintiles Adjunct Assistant Professor, Eshelman School of Pharmacy, University of North Carolina at Chapel Hill Executive Director, Genomics Biotech and Emerging Medical Technology Institute, NAMCP January 2012
  • The Value of Innovation is a Matter ofDEGREE in Healthcare ReimbursementINNOVATION: a new idea or method: a new invention or way of doing something* What evidence of comparative effectiveness? What value vs. alternatives? Is it cost-effective? Short vs. long term effect? Is it safe? Marginal vs. transformative? Does it work? New mechanism of action? *Source: Encarta World English Dictionary. Image source: www.bing.com 2
  • Value: The Devil is in the Detail Building Value: The Devil is in the Detail How much is Easy to According to enough? say…harder whom? to measure VALUE = outcomes What are the costs best models & approaches? coststhe What is right balance? What benefits vs. alternatives? How do we get there? *Image source: www.bing.com
  • Value: A Gem is the Sum of its Facets Value: A Gem is the Sum of its Facets Because value is multi- dimensional…it is often difficult capture all facets Sustainability of Time to recovery health or recovery Budget impact & Survival and return to Unmet Need and nature of cost-effectiveness normal activities recurrences Disutility of care or treatment process Long-term Comparative Degree of health (e.g., diagnostic consequences of impact vs. or recovery errors, ineffective therapy alternatives care) PARTICULARLY true for regenerative medicine…Adapted from: Michael Porter. What is value in healthcare? NEJM 2010. Image source: www.bing.com
  • Health Decision Makers View RegenerativeMedicine as a “True Innovation” Regenerative medicine ranked comparably with molecular diagnostics and personalized medicine as a true innovation, but decision makers were less certain regarding cost offset potential Technology Type Greatest Potential to Greatest Potential to Improve Care Quality Provide Cost Offsets (% respondents; n=121) (% respondents; n = 120)Small molecule drugs 14.0 14.2 ThePersonalized medicine 27.3 17.5 Regenerative MedicineCellular therapies and 20.7 Dilemma… 9.2regenerative medicine Promise vs.Molecular diagnostics 24.0 Cost 15.0 ConcernsDiagnostic imaging 9.1 5.0Nanotechnology 16.5 10.0 Source: Survey of payers, hospital administrators and manufacturers from the National Association of Managed Care Physicians membership survey evaluation 2009-10.
  • Cell Therapies & Regenerative Medicine:What is Different? Issue Cell Conventional Implications Therapies & Biologics Regenerative Medicines Well understood & accepted Not well, at No • Payers and physicians may by payers and physicians present place higher scrutiny on value perceived as demonstration truly novel • Commercialization may involve additional educational efforts and/or complexities Involves multiple procedural Often Rarely • More like reimbursement for a steps that may be separately device/procedure reimbursable • Failure to achieve (including in cell extraction, reimbursement of any purification and processing, and component part may administration that may include jeopardize reimbursement of imaging) the entire procedure HTA will focus on the cost- Yes Not often • Requires HEOR data effectiveness of the entire applicable collection regarding the entire procedure procedure
  • Cell Therapies & Regenerative Medicine:What is Different? Issue Cell Conventional Implications Therapies & Biologics Regenerative Medicines May involve multiple billing Yes No • Lack of appropriate codes /tariffs and/or payment codes/tariffs or payment may centers for reimbursement of limit or preclude access and the full procedure uptake May involve requirements for Yes Sometimes • This is a top HTA criticism of longer-term data collection to most cell therapies in global demonstrate value (including markets to date post-market follow-up data) Strong potential to be more Often Sometimes • Higher cost means that costly than standard of care therapies must demonstrate (SOC) alternatives significant outcome improvements vs. SOC comparators May enable a disease cure or Yes Rarely • Can alter the balance of prolonged therapeutic effect benefit-cost tradeoffs in value assessment
  • Can we Learn from HTAs Re: ValueDemonstration for Regenerative Medicine? The Good*Image source: www.photobucket.com
  • Global Evaluation of Regenerative MedicineHealth Technology Assessments (HTAs)• Purpose of the study: Evaluate all available HTAs on cell and gene therapies in key global HTA markets to: – See how early regenerative medicine technologies have been handled – Identify key HTA criticisms that impacted reimbursement & commercial potential – Derive lessons for upcoming technologies in the space• Included review of 48 HTAs and reimbursement policies from Australia, Canada, France, Spain, Sweden, the UK and US – No HTAs available from Germany and Italy on the topic – Did not include evaluation of Brazil, Russia, S. Korea, China, Japan – Many are nonredundant…so harder to see trends on same technology across markets – Excluded traditional cell replacement therapies for cancer indications (e.g., bone marrow, peripheral blood)*Image source: www.bing.com
  • Evidentiary Criticisms in HTADifferences Among Markets Evidence Consideration AU CA FR DE IT ES SE UK US Insufficient evidence of value S S NA NA NA S Y S S Insufficient number/quality of studies S S NA NA NA Y Y S S Lack of comparative data S S NA NA NA Y Y S S Lack of long term data (>1 year) Y Y N NA NA Y Y Y S Inconclusive or inconsistent outcomes S N NA NA NA NA N S S Focus on surrogate outcomes S S NA NA NA S Y N S Inappropriate endpoints S S N NA NA Y Y S S Concerns regarding safety N N Y NA NA N N N S Insufficient efficacy S S NA NA NA Y Y S S Insufficient cost-effectiveness S NA N NA NA N Y N NY = yes, in all assessments (yellow); S = some (green); N = no (red); NA = not available. Abbreviations: AU = Australia, CA = Canada, FR =France, DE = Germany, IT = Italy, ES = Spain, SE = Sweden, UK = United Kingdom, and US = United States. *Note: due to the limited number ofassessments in some markets and variability of information reported in HTAs and reimbursement policies, results should be interpreted withcaution. N = 48 HTAs and reimbursement policies from Australia, Canada, France, Spain, Sweden, the UK and US
  • Evidentiary Criticisms in HTADifferences Among Markets Evidence Consideration AU CA FR DE IT ES SE UK US Insufficient evidence of value S S NA NA NA S Y S S Insufficient number/quality of studies S S NA NA NA Y Y S S Aside from the US, Lack of comparative data S Australia had the most S NA NA NA Y Y S S Spain and HTAs (n=15). Many Sweden had few Lack of long term data (>1 year) Y were horizon scanning Y N NA NA Y Y Y S HTA reviews on reports and virtually regenerative Inconclusive or inconsistent outcomes S N all rejected the NA NA NA NA N medicineS S and technology based on premature to Focus on surrogate outcomes S S level of NA NA NA S Y N S draw conclusions evidence/maturity and on payer Inappropriate endpoints S S some due to N NA NA Y Y S S position + insufficient marginal perspectives improvement to Concerns regarding safety N N Y NA NA N N N S address unmet need Insufficient efficacy S S NA NA NA Y Y S S Insufficient cost-effectiveness S NA N NA NA N Y N NY = yes, in all assessments (yellow); S = some (green); N = no (red); NA = not available. Abbreviations: AU = Australia, CA = Canada, FR =France, DE = Germany, IT = Italy, ES = Spain, SE = Sweden, UK = United Kingdom, and US = United States. *Note: due to the limited number ofassessments in some markets and variability of information reported in HTAs and reimbursement policies, results should be interpreted withcaution. N = 48 HTAs and reimbursement policies from Australia, Canada, France, Spain, Sweden, the UK and US
  • Evidentiary Criticisms in HTADifferences Among Markets Evidence Consideration AU CA FR DE IT ES SE UK US Insufficient evidence of value S S NA NA NA S Y S S Many US HTAs were Insufficient number/quality of studies S S NA NA NA noncoverageY Y policies S S for technologies not yet on the market. Lack of comparative data S S NA NA NA Y Y S S Main reason is likely UK HTAs did not to prevent broader Lack of long term data (>1 year) Y Y N NA NA Y Y Y S generally include hospital use through traditional cost- existing Inconclusive or inconsistent outcomes S N NA NA NA NA N S S effectiveness analysis reimbursement seen in NICE review mechanisms for Focus on surrogate outcomes S S NA NA NA S Y N S (at this point). Many “unproven technologies were technologies” Inappropriate endpoints S S N NA NA Y Y S S early stage, “home Concerns regarding safety N Nbrew”,Y and/orNA NA N N N S required physician Insufficient efficacy S Sapproval to use NA NA NA Y Y S S Insufficient cost-effectiveness S NA N NA NA N Y N NY = yes, in all assessments (yellow); S = some (green); N = no (red); NA = not available. Abbreviations: AU = Australia, CA = Canada, FR =France, DE = Germany, IT = Italy, ES = Spain, SE = Sweden, UK = United Kingdom, and US = United States. *Note: due to the limited number ofassessments in some markets and variability of information reported in HTAs and reimbursement policies, results should be interpreted withcaution. N = 48 HTAs and reimbursement policies from Australia, Canada, France, Spain, Sweden, the UK and US
  • Case: Autologous Chondrocyte Implantationfor Knee Cartilage Defects Key Takeaways: RECOMMENDATION: Clinical Rationale: • Technologies reviewed asNOT recommended, except in a bundle context of ongoing clinical • Few RCTs; small sample • Limited discrimination studies sizes; poor trial design & between major controls technologies Economic Rationale: • Inconsistent study • Poor study design = designs and outcomes increased potential for • Insufficient evidence to reported rejection produce robust cost/QALY • Inconsistent evidence of • Methods for mapping QoL to clinical effectiveness Implications for economics not sound • Proportion of post- Regenerative Medicine: • Long-term NICE model took procedural complications into account cost of knee • Limited long-term • Value demonstration for replacement + QoL for 50 yrs outcomes data; when QoL-driven indications is • ICER for mosaicplasty available, similar to difficult; align with payer dominated ACI in modeled standard of care expectations sceanarios • No comparison with • Focus on quality design • Inconsistent model results conservative management • Prepare for long-term based on “poor” evidence follow-up
  • Case: Pancreatic Islet Cell Transplant AfterPancreatectomy RECOMMENDATION: Key Takeaways: Clinical Rationale: Recommended, but with • Poor study designs and stipulations for educating • Only case series were even safety can bepatients on procedure risk and available, with study overlooked if: stipulations on treatment selection designs that NICE would – Population is small with generally not consider high unmet need under “normal” – Outcomes are circumstances transformative or • 24% to 85% of patients exceptional Economic Rationale: were insulin-free at 6-18 months • Extremely small, niche • In one study 75% population with Implications for remained insulin-free up Regenerative Medicine: – High unmet need to 5 years – Limited budget impact • Accepted despite serious • Payers recognize clear, potential AEs, including • No economic review included vein thrombosis, liver transformative outcomes in the assessment failure, pancreatic • Expect greater scrutiny for infarct, and sepsis marginal, poorly differentiated outcomes
  • Case: Limbal Stem Cell Transplantation forLimbal Cell Deficiency Key Takeaways: RECOMMENDATION: Clinical Rationale: Recommended, for patients • Accepted despite high with nonpterygium limbal cell • Out of 873 citations, only procedural costs because:deficiency (ocular disorder) but 9 studies met inclusion – Population is limited withweak evidence for treatment of criteria, suggesting that high unmet need pterygium recurrence rigorous study design is – Therapy provided clear required (only RCTs) clinical benefits • Long-term defined as • Safety concerns Economic Rationale: follow-up at least 6 overlooked b/c of benefits months post-procedure (not as rigorous as most • Considered budget impact, regen. med assessments but not cost-effectiveness • Improvement in visual Implications for • Costs estimated at >$8,000 acuity and visual function Regenerative Medicine: to >$33,000 Cdn per eye, deemed clinically depending on requirements significant for follow-up and • Value scrutiny more like retransplantation, with overall • Concerns regarding risks drugs than medical devices costs of ~$58,000 of long-term • RCT-level data and immunosuppression with appropriate outcomes are allogeneic transplants key to acceptance
  • Case: Autologous Cell Transplant forMyocardial Infarction Key Takeaways: RECOMMENDATION: Clinical Rationale: Further research required to • Focus on surrogate • Review based on 10 RCTs outcomes = perception notdetermine long-term efficacy and and 4 noncomparative ready for “prime time” safety (horizon scan report trials 2007) • For high risk + high volume • All studies at the time + high cost = highest level evaluated surrogate of payer scrutiny outcomes (e.g., LVEF) vs. Economic Rationale: changes in morbidity and mortality • Limited cost impact analysis • Lack of long-term data on evaluated incremental Implications for “hard” and/or clinically costs of relevant procedure Regenerative Medicine: discernable health steps via codes/payment outcomes rates in the Australian • Understand what payers system • Insufficient evidence on view as most important short- and long-term • Did NOT account for cost value outcomes safety given risks of cells as a commercial associated with the • Evidence threshold for product, but as part of a procedure high risk/high cost hospital procedure indications will be HIGH
  • Case: Vaccines for Prostate Cancer Key Takeaways: RECOMMENDATION: • The pricing of ProvengeNo recommendation rendered; drew significant attention tomost patients on Provenge who Clinical Rationale: cellular therapies progressed received • Some payers may havechemotherapy, so overall value • All prostate cancer vaccines heighted concerns or proposition is not yet clear other than Provenge were sensitivities re: cost of Economic Rationale: viewed as investigational future therapies • Although Provenge did not• Not considered, although the prevent cancer progression, $93K cost of Provenge was Implications for it was noted to improve Regenerative Medicine: extremely overall survival (OS) controversial, involving a potential Medicare National • Outside of TEC assessment • The higher the cost of a Coverage Determination the level of OS improvement new therapy, the greater (NCD) and involvement of US was questioned, which is the level of payer Congress members common for many oncology scrutiny agents today as payers• In the US, despite costs, it is focus on the balance of • Similar acceptance difficult/impossible not to benefits vs. costs potential for non-oncology cover an oncology indications in the US is agents, but harsh less certain reimbursement limits can apply
  • Several US Payers Maintain Preemptive Noncoverage Policies for Regen. Therapies Sample target indications currently not covered by leading US payers: – Myocardial infarction – Polymyositis – Congestive heart failure – Autoimmune cytopenia – Multiple sclerosis – Diabetes mellitus (type I) – Systemic lupus erythematosus – Systemic vasculitis – Systemic sclerosis – Celiac disease – Rheumatoid arthritis – Crohns disease – Juvenile rheumatoid arthritis – Oncology (select indications/applications) – Idiopathic thrombocytopenic purpura – Dematomyositis Implications for Regenerative Medicine: • US payers are concerned about unproven, hospital-based cell therapies • Policies will change as evidence for tested products matures in the marketplaceSource: Indications identified through evaluation of multiple US MCO coverage policies.
  • Percentage of HTAs that Noted KeyEvidentiary Criticisms Consider Long-term Clearly comparator data will be characterize Rigorous required. safety outcomes are Plan for post- key market data collection Insufficient Lack of Lack of Inconclusive Inappropriate Concerns Insufficient Insufficient #/quality of comparative long-term or endpoints regarding efficacy cost- studies data data inconsistent safety effectivenessStudy design (>1yr) studiesquality is key. Device-like studies = N = 48 HTAs and reimbursement policies from Australia, Canada, France, Spain, Sweden, the UK and US high rejection N = 48 HTAs and reimbursement policies from Australia, Canada, France, Spain, Sweden, the UK and US potential
  • Percentage of HTAs that Noted KeyEvidentiary Criticisms Ex-US HTAs did not focus Almost 50% as much on of HTAs safety noted that studies did not include the right Some endpoints outcomes were either CE not unclear, mentioned surrogate or b/c some various were early outcomes HTAs and/or were technologies reported were subject to a less rigorous review Insufficient Lack of Lack of Inconclusive Inappropriate Concerns Insufficient Insufficient #/quality of comparative long-term or endpoints regarding efficacy cost- studies data data inconsistent safety effectiveness (>1yr) studies N = 48 HTAs and reimbursement policies from Australia, Canada, France, Spain, Sweden, the UK and US N = 48 HTAs and reimbursement policies from Australia, Canada, France, Spain, Sweden, the UK and US
  • Access Recommendations from HTA ReviewBodies/Payers Recommendations Recommendations from All HTAs Approximately from US HTAs & Reviewed 60% of all HTAs Coverage Policies recommended Reviewed either rejection of the technology due to one or 40% more of the evidentiary criticisms 40% presented The majority of 60% the noncoverage 60% policies in the US were for technologies that have not yet entered the Rejected Accepted market Rejected Accepted
  • What does the Climate Look Like for Value Demonstration in Regenerative Medicine? • Regenerative medicine is “on the payer radar” – The “storm” has been sighted & decision makers are watching carefully • Despite the promise, there is a “perfect storm” of factors converging that make global value demonstration more complex…particularly for ground breaking technologies – Driven by novelty, resource allocation pressures, and market health reform • Early indicators may suggest storms ahead, but the key to success is planning and preparation. Focus on: – Solid study designs; appropriate patient targeting and sample size – Understanding the outcomes that matter on an indication-by-indication basis – Plans for longer-term data collection beyond the pivotal study – Characterizing benefits/cost balance vs. alternatives; economics matter • Remember…for payers, saying “no” is easier than saying “yes” – Have your emergency kit well prepared to weather climate changes in HTA. Image source: www.bing.com 22
  • Many Other Issues Required To Understand Value Drivers for Regenerative Medicine •Are available codes//tariffs sufficient for key procedure •Payers will look at the balance of outcomes steps? relative to cost of entire procedure •Do we need a new code/tariff? If so, what timing and info •How will evidence development differ from required? conventional therapies? Coding/ Evidence Tariff & Outcomes • What outcomes are important in avoiding reimbursement rejection? • What are the key reimbursement Coverage •Autologous or allogeneic? limitations that we can expect for the therapy? •Type or cell and persistence? Cell Therapy • How are process for cell collection, preparation, and Approach Reimbursement administration reimbursed? and Market •Is payment level appropriate to Access support access for each step? Payment •What are our options for address• What are HTA agency and payers inappropriate payment? Payer &perspectives on cell therapies? HTA•How have payers and HTA agencies Trends handled initial cell therapy entrants? Site of •How does site of care influence•What key criticisms of the value Patient Care reimbursement potential for the therapy? proposition have been cited for cell Access therapies? •Is special expertise required to provide the therapy? • Are Centers of Excellence required? • How flexible will payers be regarding patient access? • Are there subpopulations that are best to target? •What information will physicians require for adoption vs. conventional therapies? 23
  • Need for Reimbursement Planning is MorePronounced for Regenerative Medicine If uncertainties exist/to Landscape, seconda confirm ry, & primary HEOR/reimbursement research approach Clinical & Economic Market Implement Pivotal Early Payer Access StudyValue Demonstration Environment Maker EngagementPlan (HEOR strategy) Research Ancillary clinical and Market Forecast & economic studies Business Plan Manage the Moving Target Not new…just more Market acute focus Access, Pricing & • Due to novelty, even more reason to characterize decision Reimbursement requirements Reimbursement Strategy • Understand endpoints,/outcomes trial requirements, patient Submissions & considerations Launch • Identify appropriate comparator and perspectives on available Launch/Commercial alternatives Strategy • Understand how the procedure will fit into market reimbursement system • Decision maker reactions to TPP and costs
  • Reimbursement and Market Access: Putting it All Together • Multiple regenerative medicine technologies are moving into clinical development, but: o They are truly novel and not yet understood/accepted by HTA, payer and physician decision makers: EDUCATE o Reimbursement and commercial strategy is more complex due to combination procedure/surgery and cellular component; technologies can touch more than one payment system; EVALUATE – “measure twice & cut once” o Manufacturing, distribution and market access channels are more complex than conventional biopharmaceuticals; NAVIGATE o Early analysis of HTAs suggests that regenerative medicines will have to demonstrate strong clinical, economic and long-term follow-up data; DEMONSTRATE o “Front-loaded” cost model and “episode of care” view can complicate economic and cost-effectiveness analysis; VENERATE – clearly understand economic drivers o Need for strong educational efforts and clear communication of value proposition; COMMUNICATE – “multifaceted value…let me count the ways…” • Reverse engineer product plans targeting reimbursement requirements to optimize commercial potential so the pieces fit. Image source: www.bing.com 25
  • Eric Faulkner Director, Global Market Access QuintilesThank You! 4820 Emperor Blvd. Durham, NC 13979 919-998-7266 (office) 919-381-8306 (mobile) Eric.Faulkner@quintiles.com Executive Director, Genomics, Biotech and Emerging Medical Technology Institute National Association of Managed Care Physicians Assistant Professor, Eshelman School of Pharmacy, Institute for Pharmacogenomics and Individualized Therapy University of North Carolina at Chapel Hill