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Understanding Different Stakeholder Requirements Throughout Commercialization

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Learn how to use Real World Evidence (RWE) to bridge the evidence gap between regulators and payers.

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Understanding Different Stakeholder Requirements Throughout Commercialization

  1. 1. © 2018 PAREXEL INTERNATIONAL CORP. UNDERSTANDING DIFFERENT STAKEHOLDER REQUIREMENTS THROUGHOUT COMMERCIALIZATION Richard Macaulay
  2. 2. © 2018 PAREXEL INTERNATIONAL CORP. / 2 USING RWE TO BRIDGE THE EVIDENCE GAP BETWEEN REGULATORS AND PAYERS • The emerging evidentiary gap between payers and regulators • Overview: payers vs. regulators • Recent trends – Payers and regulators – New transformative therapy classes • The evidence divergence • How RWE can bridge this gap • Conclusion/summary AGENDA
  3. 3. © 2018 PAREXEL INTERNATIONAL CORP. / 3 DUBLIN, IRELAND COMPARING PAYERS AND REGULATORS
  4. 4. © 2018 PAREXEL INTERNATIONAL CORP. / 4 Quality1 Safety2 Efficacy3 Value4 Regulators Payers Key considerations of… Focus of… Hurdles to Patient AccessHurdles to patient access THERE ARE 4 HURDLES THAT A PRODUCT MUST CLEAR TO ACHIEVE PATIENT ACCESS
  5. 5. © 2018 PAREXEL INTERNATIONAL CORP. / 5 …HOWEVER, THE EVIDENTIARY DEMANDS OF PAYERS AND REGULATORS ARE DISTINCT… Regulators Payers KEYFOCUS Quality Comparative benefits Efficacy Real-world effectiveness Safety Economic value Product is safe and efficacious as demonstrated through trial data Product will offer real-world clinical value over standard of care and represents an optimal investment of limited funds Regulators Payers Evidence requirements
  6. 6. © 2018 PAREXEL INTERNATIONAL CORP. / 6 … AN ADDITIONAL ITEM OF COMPLEXITY IS THE NUMBER AND VARIETY OF PAYERS VERSUS REGULATORS Regulators Typically single trans-national bodies (eg, FDA/EMA) with similar foci Payers A range of national, regional and local payers by market with varying and distinct foci The wide range of payers can be split into archetypes with similar foci Payer archetype Market example Cost-effectiveness Comparative clinical effectiveness Budget optimisation Payer archetyping
  7. 7. © 2018 PAREXEL INTERNATIONAL CORP. / 7 LONDON, U.K. RECENT TRENDS REGULATORS
  8. 8. © 2018 PAREXEL INTERNATIONAL CORP. / 8 5 TRADITIONALLY, THE REGULATORY HURDLE WAS SEEN AS THE KEY ONE TO OVERCOME FOR PATIENT ACCESS New Expedited Access Pathways Regulatory Hurdle is Decreasing Breakthrough Status Adaptive Pathways EfficacySafetyQuality Regulators DRIVENBY FDA EMA Regulatory hurdle is decreasing
  9. 9. © 2018 PAREXEL INTERNATIONAL CORP. / 9 ZYKADIA® ceritinib KEYTRUDA® pembrolizumab … RESULTING IN THERAPIES BEING APPROVED AT VERY EARLY CLINICAL DEVELOPMENT STAGES FDA approval date Key efficacy data source Expedited FDA regulatory pathways 29 April 2014 Phase 1 trial Single-arm, n=163 Orphan designation Breakthrough Status Accelerated Approval 4 September 2014 Phase 1 trial Single-arm, n=173 Orphan designation Breakthrough Status Accelerated Approval Examples of drugs FDA approved on Phase 1 data
  10. 10. © 2018 PAREXEL INTERNATIONAL CORP. / 10 BERLIN, GERMANY RECENT TRENDS NEW THERAPY CLASSES
  11. 11. © 2018 PAREXEL INTERNATIONAL CORP. / 11 THERE ARE TRANSFORMATIONAL NEW CLASSES OF THERAPIES SUCH AS GENE THERAPIES… Eg. STRIMVELIS ® Gene therapies Gene inserted into a virus Curative potential for genetic diseases and long- term cost savings Indication: ADA-SCID SoC: ERT ~$500,000/yr or HSCT AD1115611 (Phase 1/2 trial, n=12) 100% survival (median f/u 7yrs) Replace faulty or missing genes in patients’ cells
  12. 12. © 2018 PAREXEL INTERNATIONAL CORP. / 12 … PLUS CAR-T CELL AGENTS THAT SHOW REMARKABLE EFFICACY IN ADVANCED CANCER… Eg. KYMRIAH® CAR-T cell therapies Genetically engineered T cells Express a synthetic receptor designed for a specific target Expand and persist in vivo with high potency Indication: r/r B cell ALL ELIANA (Phase 2 trial) 83% CR/CRi at 3 months 79% 12-month OS
  13. 13. © 2018 PAREXEL INTERNATIONAL CORP. / 13 .. AND IMMUNO-ONCOLOGY DRUGS WHICH OFFER LT SURVIVAL BENEFITS ACROSS MULTIPLE CANCERS Immuno-oncology (I-O) Harnessing the body’s immune system to fight cancer Five FDA-approved anti-PD-1/L1 Potential for LT survival benefits KEYTRUDA® (pembrolizumab) OPDIVO® (nivolumab) TECENTRIQ® (pembrolizumab) IMFINZI® (durvalumab) BAVENCIO® (avelumab) T cell Cancer cell I-O agents block immune inhibitory receptors PD-1 PD-L1 Combinations
  14. 14. © 2018 PAREXEL INTERNATIONAL CORP. / 14 NEW THERAPY CLASSES WITH TRANSFORMATIVE BENEFITS WILL BE EXPEDITED APPROVAL CANDIDATES New therapy classes Gene therapies CAR-T cell therapies Immuno- oncology Offer transformational patient benefits Candidates for expedited regulatory approval Approved on earlier clinical data packages Transformative therapy classes Key features Consequences For diseases with severe unmet needs Often in rare patient populations
  15. 15. © 2018 PAREXEL INTERNATIONAL CORP. / 15 BOSTON, MASSACHUSETTS RECENT TRENDS PAYERS
  16. 16. © 2018 PAREXEL INTERNATIONAL CORP. / 16 CONCURRENTLY, PAYERS ARE DEMANDING MORE EVIDENCE TO JUSTIFY PRICE PREMIUMS… Escalating costs Budgetary pressures Reimbursement authorities are becoming more demanding of the patient value of new healthcare technologies Reimbursement hurdle
  17. 17. © 2018 PAREXEL INTERNATIONAL CORP. / 17 … THIS IS A TREND THAT IS ESPECIALLY CLEAR OUTSIDE THE US… Ex-US pricing trends Structural P&R reforms Payer evidence acceptance trends e.g. AMNOG CDF Budget impact Clinically-meaningful benefits In patient-relevant endpoints From trials directly compared to the relevant comparator A trend for payers to increasing demand
  18. 18. © 2018 PAREXEL INTERNATIONAL CORP. / 18 … BUT THE PRICE OF MEDICINES IS INCREASINGLY A FOCUS OF DEBATE IN THE US… US pricing trends Value scorecards driven by provider organisations Cost-utility evaluations by ICER becoming prominent
  19. 19. © 2018 PAREXEL INTERNATIONAL CORP. / 19 … SUCH THAT IN MANY COUNTRIES THE PAYER HURDLE IS MORE DIFFICULT TO CLEAR THAN THE REGULATORY Payer hurdle increasingly key in many markets EfficacySafetyQuality Regulators Value4 Regulators Payers Marketing authorisation increasingly less seen as the major barrier to patient access… … compared with the challenges of translating this into approval by payer bodies (e.g. NICE, G-BA, PBAC)
  20. 20. © 2018 PAREXEL INTERNATIONAL CORP. / 20 TOKYO, JAPAN THE EMERGING EVIDENCE GAP
  21. 21. © 2018 PAREXEL INTERNATIONAL CORP. / 21 THERE IS AN EVIDENTIARY DIVERGENCE BETWEEN THE DEMANDS OF PAYERS AND REGULATORS… Evidentiary misalignment Comparative Clinical Benefits Robust Health Economic Analyses Patient-Relevant Endpoints Single-arm Phase I/II data Increasing Evidence Demands of Payers Evidence Provisions of Expedited Pathways Increasing demand for Increasing approvals on
  22. 22. © 2018 PAREXEL INTERNATIONAL CORP. / 22 Mock NICE technology appraisal of a CAR-T cell therapy Hettle et al. Health Technol Assess. 2017 Feb;21(7):1-20 … THESE NEW TRANSFORMATIVE THERAPIES WILL DEMAND HIGH PRICES IN LINE WITH THEIR VALUE… Prices of transformative therapies 10.07 Incremental QALY gain per patient Price per patient (on top of SoC)>£500k Assume NICE WTP of £50,000/QALY
  23. 23. © 2018 PAREXEL INTERNATIONAL CORP. / 23 … PLUS GENE & CAR-T THERAPIES ONLY NEED SINGLE TREATMENTS => SUBSTANTIAL UPFRONT INVESTMENT P&R issue of single treatment duration Many CAR-T and gene therapies Require a single treatment Expenditure is not spread across the duration of treatment Entire costs must be incurred upfront
  24. 24. © 2018 PAREXEL INTERNATIONAL CORP. / 24 … BUT HOW DO YOU APPROPRIATELY REIMBURSE THERAPIES WHERE UNCERTAINTY IS SO HIGH? Appropriate reimbursement? High costs High benefits High uncertainty High risk
  25. 25. © 2018 PAREXEL INTERNATIONAL CORP. / 25 SOME THERAPIES WITH SUBSTANTIAL CLINICAL GAINS HAVE NOT TRANSLATED INTO COMMERCIAL SUCCESS GLYBERA® – first EC-approved gene therapy1 Regulatory approval • EC-approved in Oct 2012 • For hereditary LPLD (incidence ~1 per million) Commercial launch • Launch price (in Germany) €1.1 million per patient Market withdrawal • May 2016 - only been used commercially once • Nov 2017 - UniQure did not renew their marketing authorisation 1ScienceBusiness.net (2017) World’s first gene therapy to be withdrawn in from market in Europe
  26. 26. © 2018 PAREXEL INTERNATIONAL CORP. / 26 SHANGHAI, CHINA USING RWE TO BRIDGE THIS GAP
  27. 27. © 2018 PAREXEL INTERNATIONAL CORP. / 27 GENERATING PAYER-RELEVANT CLINICAL TRIAL DATA MAY NECESSITATE TRADE-OFFS IN TIME, COST AND RISK Trade-offs in generating payer relevant clinical trial data Payer-relevant comparator Payer-relevant endpoints larger trial Time Cost Risk longer to read out Time Risk
  28. 28. © 2018 PAREXEL INTERNATIONAL CORP. / 28 …BUT COMPANIES CAN ALSO USE RWE TO BRIDGE THIS EVIDENCE GAP FOR OPTIMAL MARKET SUCCESS… Real world evidence Bridging the gap Payer evidence demands Evidence required for regulatory approval
  29. 29. © 2018 PAREXEL INTERNATIONAL CORP. / 29 RWE can be used to quantify and validate: … AND RWE CAN PROVIDE SUPPORT TO OPTIMISE THE VALUE OF A PRODUCT IN MANY WAYS… Real World Evidence Disease burden Clinical benefit Economic benefits Epidemiology Treatment patterns Unmet need Effectiveness Safety Performance-based pricing schemes Support cost- effectiveness and budget impact modelling
  30. 30. © 2018 PAREXEL INTERNATIONAL CORP. / 30 … WITH RWE ALREADY HAVING BEEN USED TO SUPPORT REIMBURSEMENT ACROSS MANY MARKETS Examples of directly using RWE in reimbursement Market Example(s)Scheme AIFA-Onco Registry established in 2005 – performance-based pricing well-established 2016 Cancer Drugs Fund reforms as a temporary reimbursement fund to collect RWE Since 2011, Managed Entry Schemes allow dynamic pricing based on RWE In the last 3-4 years, a trend towards performance-based pricing schemes TASIGNA® (nilotinib) TAGRISSO® (osimertinib) XALKORI® (crizotinib) REPATHA® (evolocumab) ENTRESTO (SAC/VAL) KYMRIAH (tisagenlecleucel)
  31. 31. © 2018 PAREXEL INTERNATIONAL CORP. / 31 TAIPEI, TAIWAN SUMMARY AND CONCLUSIONS
  32. 32. © 2018 PAREXEL INTERNATIONAL CORP. / 32 REIMBURSEMENT IS INCREASING BECOMING A KEY HURDLE FOR COMPANIES, RWE OFFERS A SOLUTION Regulators are reducing evidentiary demands for marketing authorization through a range of new expedited approval pathways There are a range of innovative therapies coming to market that will likely be approved through such pathways on very early clinical data packages, with the potential for transformational patient benefits but where substantial uncertainty remains Payers, whom are increasingly demanding more robust evidence to justify price premiums, will find it very challenging to appropriately price and reimburse such therapies How companies navigate this emerging evidentiary gap between regulators and payers to obtain faster reimbursement will be increasingly key to ensuring market success RWE offers an opportunity to fill these evidentiary gaps by examining how medicines are used and perform in the real world
  33. 33. © 2018 PAREXEL INTERNATIONAL CORP. / 33 ANY QUESTIONS
  34. 34. © 2018 PAREXEL INTERNATIONAL CORP. / 34 THANK YOU © 2018 PAREXEL INTERNATIONAL CORP. / 34

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