Recommended
More Related Content
What's hot
What's hot (20)
Similar to Rare disease stakeholder dynamics pmrg institute fall 2014
Similar to Rare disease stakeholder dynamics pmrg institute fall 2014 (20)
Recently uploaded
Recently uploaded (20)
Rare disease stakeholder dynamics pmrg institute fall 2014
- 1. 1 Rare Disease Stakeholder Dynamics Paul Allen Camm Epstein
- 2. NUMBERS
- 3. Definition of a rare disease: <200,000 Americans Number of rare diseases: >6,800 Americans with a rare disease: 25-30 million NIH Office of Rare Disease Research http://rarediseases.info.nih.gov/about-ordr/pages/31/frequently-asked-questions 3 11/12/2014
- 4. Rising prices, additional options and increasing use of combination therapy drive costs higher. 11/12/2014 Before: High $ * Very Low Vol = Low $ After: High $ * Low Vol = Medium $
- 5. Specialty medications are growing in absolute dollars and percent of drug spend $235 billion (50%) 2018 $92 billion (30%) 2012 CVS Caremark, Insights 2014 http://info.cvscaremark.com/insights2014/INSIGHTS%20Trend%202014-v2.pdf 5 11/12/2014
- 6. METHODS 6
- 7. Physicians Pulmonary Arterial Hypertension Payers Patients Payers Physicians Cystic Fibrosis Patients Physicians Hereditary Angiodema Payers Patients
- 8. STAKEHOLDERS
- 9. • Want to feel better, improved quality of life – Fewer symptoms of condition and fewer side effects of therapy – Easier to administer products • Willing to make tradeoffs between efficacy and convenience • Cost is often not a concern – Depends upon coverage, needs/wants and disposable income • PAPs and charitable foundations typically address any financial difficulties • Very well-informed – Often better informed than physicians not specializing in the condition • Share information with and supportive of other patients • Often emotional – Life-threatening – Familial impacting multiple family members 9 Patients
- 10. Physicians • Appeal denials when necessary • Test and treat aggressively • Prefer to prescribe proven therapies – Yet, sensitive to patient preferences • Receptive to tradeoffs between efficacy and improved QOL 10
- 11. Nurses • Work through patient assistance programs • Provide most patient education • Work through most access issue 11
- 12. Payers • They generally do not manage rare conditions aggressively, but… – Willing to select preferred products, increase restrictions and contract when there are multiple, poorly differentiated options • Especially when there are large cost differences and/or multiple options in the same class • Willing to relax, but not abandon, requirements for evidence • Encourage care delivered outside of centers and discount ‘center of excellence’ designations – Especially when there is a lack of evidence • Encourage use of oral formulations – Particularly when the costs are similar • Discourage use of combination therapy – Particularly when there is a lack of evidence • Critically evaluate clinical guidelines/consensus statements – Reject those not based on evidence • Frustrated by manufacturer pricing 12
- 13. Medical Societies • Develop consensus statements/clinical guidelines • Advocate what they believe to be appropriate care – Cost-effective care is an emerging consideration 13
- 14. Manufacturers • Charge high prices for some therapies – Particularly when the target patient population is very small – Particularly when there is a genetic marker • Find it difficult to enroll sufficient numbers of patients in clinical trials • Very supportive of Patient Assistance Programs (PAPs) – Work hard to ensure patient out-of-pocket costs are not a barrier for those unable to pay 14
- 15. Purchasers • Watching specialty pharmacy spend rise • Asking for tighter controls – Want to avoid negative public relations • Shifting costs to patients 15
- 16. Advocacy Organizations • Celebrate the entry of new options – Interpret new entrants as a sign of progress towards a cure • Supporting clinical guideline/consensus statement development • Supporting center of excellence designations 16
- 17. INTERACTIONS
- 18. • Share intense bonds – Often social, informal relationships “I feel like they are family” • Frequent and sometimes long conversations – Patients ask questions and make requests 18
- 19. 19 • Regularly bringing diverse stakeholders together • Facilitating dialogue and collaboration
- 20. DYNAMICS
- 21. 21 Access Restrictions Multiple Therapies Combination Therapy Multiple Drugs in Same Class Prescribing by non-KOLs Payer Preferences Multiple Routes of Administration Patient Preferences Physician Preferences Efficacy Safety Cost Patient Costs Diffusion of Care Sites Oral Therapies Centers of Excellence Designations Public Relations Very Low Prevalence Significant Unmet Needs Awareness/ Knowledge Consensus Statements/Clinical Guidelines Earlier Diagnosis/Treatment Better Outcomes Increased Utilization Higher Drug Prices Growing Spend/Trend Clinical Trial Enrollment Difficulties Lack of Comparative Effectiveness Research Reliance on Patient Assistance Enhanced Role of Nurses Patient-Physician Conversations Hope Benefit Design Patient-Provider Relations
- 22. PREDICTIONS
- 23. • Halo effect across rare conditions – Acceleration towards access restrictions tipping points • Debate and research on oral therapies – Cost-effectiveness – Patient preferences • New methods for studying combination therapies 23
- 24. • Patient registries to aggregate experience • Growth in supply of and demand for patient assistance • Enhanced social media and collaboration capabilities – Enriching interpersonal communications 24
- 25. IMPLICATIONS
- 26. • Success in crowded markets – Novel mechanisms of action – Improved ROA differentiation – Genetic markers/companion diagnostics – Premium pricing that is justified and sustainable • Price reductions when indications expand 26
- 27. • Success in new markets – New therapies – Sufficient unmet need to yield ROI 27
- 28. Currant Insights, LLC 33 East Avenue Saratoga Springs, NY 12866 Camm Epstein Managing Director (518) 429-0875 camm@currantinsights.com Olson Research Group, Inc 1020 Stony Hill Road, Suite 300 Yardley, PA, 19067 Paul Allen Executive Vice President (215) 499-0244 pallen@olsonreserchpa.com
Editor's Notes
- These costs are now on purchasers’ and payers’ radar screens
- Specialty medications are the fastest-growing - and the most expensive - segment of pharmacy care.
- Comprehensive solution, features
- Many patients avoid infused therapy The desire to avoid IV is so intense, that many relax their informational needs/wants and will try unproven therapies and mechanisms of action with preferred routes of administration
- When dealing with payers, nurses are typically on the ‘font lines’
- Denying combination therapy lacking evidence is driven by efficacy, safety and cost concerns – Payers do not want members to be guinea pigs Some payers have been frustrated with the markup some specialty pharmacy providers have been charging
- By definition, the universe is small Hard to find patients that meet the inclusion criteria Patients may be reluctant to switch – concerns about getting covered if the need/want to switch back, may be experiencing success with current therapy
- Many have close social relationship with one another
- Here’s a model of how many of the dynamics among stakeholders are interrelated for rare diseases.