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Deriving more value from real world evidence to ensure timely access of medicines by Patients

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Dr Sarah Wamala Andersson, Consultant, Real world evidence and value-based medicines

Pharma Pricing & Market Access Congress 2017 22 February 2017 London

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Deriving more value from real world evidence to ensure timely access of medicines by Patients

  1. 1. Deriving more value from Real world evidence (RWE) to ensure timely access of medicines by patients Dr. Sarah Wamala Andersson Consultant, Real World Evidence and Value-based medicines EVIDENCE CONFERENCE February 22, 2017, LONDON
  2. 2. Deriving more value from RWE to ensure timely access of medicines by patients 1. Why RWE now? 2. Enhancing value-based medicines through RWE 3. RWE as a means to develop strategic partnerships and alliances 4. Challenges of realising the real value of RWE
  3. 3. Why increasing interest for RWE?
  4. 4. Potential cost-savings with RWE Development Launch In-market Clinical development (100-200m USD) - Pricing, market access (100m USD) - Planning & tracking reimbursement process (150m USD) - Safety & value demonstration (200- 600m USD) - Effectiveness commercialization (200- 300m USD) (RWE) Productivity & cost saving (100-200m USD) Ref. Hughes, Kessler, 2013
  5. 5. Deriving more value from RWE to ensure timely access of medicines by patients 1. Why RWE now? 2. Enhancing value-based medicines through RWE 3. RWE as a means to develop strategic partnerships and alliances 4. Challenges of realising the real value of RWE
  6. 6. RCTs and RWE studies 1. Demonstrates effectiveness in the real world of clinical practice 2. Generalizable study finding on facts about patient journeys and outcomes 3. Broader & more representative of the patient population over a longer time frame 4. Real world setting (Busy practices, uncontrolled patients) 5. Demonstrates value for patients and benefits within health systems 1. Demonstrates clinical efficacy and safety 2. Randomization as an effective tool to minimize bias and confounding 3. Limited segment of the population is eligible for inclusion - questionable external validity & generalizability 4. Ideal, controlled setting (”Good” patient adherence and compliance) 5. Limited ability to investigate costs and value within health systems Refs. SVM Pharma PWC 2013 i
  7. 7. Population in the RCTs, Do they represent the real world? Utilizationinreallife Population studied Phases I-III Post-approval research Variances in populations using therapies in the real world Vs that studied • Age-groups • Socio-demography • Comorbidities • Lifestyle • Severity of disease • Level of compliance Ref. Czirakym & Pollock, Applied Clinical Trials, 2015 RCTs
  8. 8. Mind the efficacy - effectiveness gap?
  9. 9. Deriving more value from RWE to ensure timely access of medicines by patients 1. Why RWE now? 2. Enhancing value-based medicines through RWE 3. RWE as a means to develop strategic partnerships and alliances 4. Challenges of realising the real value of RWE
  10. 10. Framework for Developing RWE Encourages cross-functional working & external partnerships Design Research questions/ Hypothesis Identify RWD sources Conduct Statistical Analyses RWE SOLUTIONS Identify Gaps Cross-functional RWE teams External partnerships
  11. 11. Trends towards adaptive licensing -A push for more RWE and strategic partnerships and alliances?
  12. 12. Clinical Pharmacology & TherapeuticsVolume 91, Issue 3, Version of Record online: 15 FEB 2012 Current situation Future? Patients in RWE studies
  13. 13. Pharmaceuticals Licensing and Reimbursement in the European Union, United States, and Japan PAST FUTURE Clinical Pharmacology & Therapeutics Volume 100, Issue 6, pages 626-632, 17 OCT 2016 DOI: 10.1002/cpt.505 http://onlinelibrary.wiley.com/doi/10.1002/cpt.505/full#cpt505-fig-0001
  14. 14. Introduction of new medicines in Sweden (collaborations between county councils, governmental agencies and the pharmaceutical industry)
  15. 15. Deriving more value from RWE to ensure timely access of medicines by patients 1. Why RWE now? 2. Enhancing value-based medicines through RWE 3. RWE as a means to develop strategic partnerships and alliances 4. Challenges of realising the real value of RWE
  16. 16. Challenges of RWE 1. The enthusiasm level of RWE not matched with how to actually put it into practice 2. Poor reputation of RWE studies due to lower hierarchy of scientific evidence 3. Lack of capabilities 4. DATA • Size & complexity of data • Fragmented data • Transparency & quality of data • Industry access to data • Limited skills to evaluate big data with sophisticated methodology 5. Traditional design of research • Scientists with little understanding of the real world environment • Epidemiologic research methods • Limited engagement of patients and relevant stake-holders in research design Ref. PWC 2013
  17. 17. Concluding Remarks 1. RWE provides a huge potential to contribute to value- based medicines. 2. RWE enables access of medicines to patients effectively, timely and safely. 3. RWE encourages cross-functional working within companies and external strategic partnerships and alliances. 4. The real value of RWE will be realised by increasing capabilities and building the scientific reputation of RWE.
  18. 18. RWE is here to stay! Start with RWE now! To ensure timely access of medicines by patients
  19. 19. THANK YOU!

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