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
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
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
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
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
Notas do Editor
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.