ORPHAN DRUGS & RARE
DISEASES GLOBAL
CONGRESS 2015 EUROPE
Ultra-orphan drugs: what is the long term strategy to support ultra-orphan drugs &
how commercially viable are these drugs to stakeholders
Presenter: Christian Girard, MiM
Chief Editor, Orphan Drugs Industry Insider
Founder & CEO, ABCrowdFunding Advisors
Orphan disease prevalence: < 1/2.000
Ultra-rare disease prevalence: < 1/50.000
Picture: Alexion Pharmaceuticals
ULTRA-ORPHAN DISEASES: WHAT ARE WE TALKING ABOUT?
Atypical Hemolytic Uremic Syndrome (aHUS)
N-acetylglutamate synthase deficiency (NAGS deficiency)
Hutchinson-Gilford progeria syndrome (Progeria)
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Ribose-5-phosphate isomerase
deficiency (RPI deficiency)
& many others…
http://www.orpha.net/orphacom/cahiers/docs/GB/Prevalence_of_rare_diseases_by_decreasing_prevalence_or_cases.pdf
1-9/1.000.000 prevalence
<1/1.000.000 prevalence
1/8.000.000 prevalence
1-9/1.000.000 prevalence
1 case identified (1999)
ANY DRUGS FOR THESE DISEASES? YES, AT LEAST FOR A FEW ONES Atypical Hemolytic Uremic
Syndrome (aHUS)
N-acetylglutamate synthase deficiency (NAGS
deficiency)
Hutchinson-Gilford progeria syndrome (Progeria)
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Ribose-5-phosphate isomerase deficiency (RPI
deficiency)
Soliris (eculizumab)
Carbaglu (carglumic acid)
Pravastatin / zoledronic acid (not approved yet)
Soliris (eculizumab)
Not to our knowledge
ULTRA-RARE DRUGS PRICING (YEARLY) Carbaglu Glybera Soliris Orfadin Vimizim Juxtapid Cinryze Procysbi
Up to 2.000k$ Up to 1.600k$ Up to 410k$ Up to 400k$ Up to 380k$ Up to 380k$ Up to 300k$ Up to 250k$
VERY SMALL PREVALENCE & DRUG PRICING IMPACT ON PAYOR BUDGET
PATIENTS $50 000 $100 000 $250 000 $500 000 $1 000 000US 6 000 $300 000 000 $600 000 000 $1 500 000 000 $3 000 000 000 $6 000 000 000EU 10 000 $500 000 000 $1 000 000 000 $2 500 000 000 $5 000 000 000 $10 000 000 000
US+EU 16 000 $800 000 000 $1 600 000 000 $4 000 000 000 $8 000 000 000 $16 000 000 000
Assumptions: 100% patients treated; disease prevalence = 1/50.000
POTENTIAL SALES (INCREASING PRICING VALUE)
ULTRA-RARE DRUGS SALES
2014 SALESSoliris $2 146 000 000Cinryze $503 000 000Procysbi $70 000 000Vimizim $37 000 000
ULTRA-RARE DRUGS = ULTRA-RARE TECHNOLOGY? Small molecules Biologics Gene therapies
Ultra-rare drugs do not necessarily imply advanced technology. Some are « simply » repurposed small molecules
THREAT: EVOLVING PAYORS POLICIES Global cost of orphan drugs under scrutiny:
120B$ (ever increasing) – more than 10% of the overall pharmaceutical market in 2014
Ultra-rare drugs = high pricing (in investors’ opinion at least)
Trend: increasing number of ultra-rare drugs Ultra-rare drugs efficacy? Room for negotiation with payors: very low
prevalence – patients registries – staged payment terms
As a result, stellar pricing belongs to the past
BUT STILL, AN ATTRACTIVE MARKET OPPORTUNITY 7.000+ orphan diseases identified ca. 500 drugs approved ca. 6.500 diseases without dedicated
treatment About 50% of them are ultra-rare disorders Orphan regulations incentives (7 to 10 year
market exclusivity starting at approval) Clinical trials cost lower (very few patients in
trials due to very low prevalence) Small-sized sales forces Historically, premium pricing (will this last?) Little or no competition… blue ocean strategy
GOVERNMENTAL SUPPORT OR PATIENTS EMPOWERMENT? National plans or strategies for rare
diseases EUROPLAN Budgets for ultra-rare diseases, but
drops in the ocean
National & international patients organizations much more active and motivated
Patients association offer support (initial financing and in clinical trials)
VALUE CREATION IN ULTRA-RARE DRUGS Leading orphan drugs players’ products
& R&D portfolio include ultra-rare drugs and candidates
Genzyme (Sanofi group) Shire Alexion Pharmaceuticals BioMarin Pharmaceutical Ultragenyx Pharmaceutical SOBI
ULTRA-RARE DRUGS PLAYERS MARKET CAPITALIZATION
Alexion Pharmaceuticals: 37B$
BioMarin Pharmaceutical: 22B$
Ultragenyx Pharmaceutical: 3.7B$
Raptor Pharmaceuticals: 1B$
Aegerion Pharmaceuticals: 0.5B$
M&A IN ULTRA-RARE DRUGS
Alexion / Synageva: 8.4B$
Horizon Pharma / Hyperion: 1.1B$
AbbVie / Pharmacyclics: 21B$
Sanofi / Genzyme: 20B$
Shire / NPS Pharma: 5.2B$
Alexion / Enobia: 1.1B$
INVESTMENT IN ULTRA-RARE PLAYERS PROVED SUCCESSFUL (1)
INVESTMENT IN ULTRA-RARE PLAYERS PROVED SUCCESSFUL (2)
REGIONAL COMPANIES VS. ULTRA-RARE DRUGS INVOLVEMENT Any orphan drugs regulatory incentives
in the country (region)? If not, opt for development where these
incentives exist (US, EU, JP, AU, KR…) Ultra-rare diseases markets are not
local: they are global Out-licensing is an option
ULTRA-RARE VS. «ONLY» ORPHAN
Ultra-low prevalence justified (in industry’s and investors’ view) high pricing for ultra-rare drugs
Non-ultra-rare drugs price tags can also be stellar (eg. Vertex’ Kalydeco for Cystic Fibrosis- 294k$)
PTC Therapeutics’ Translarna (for Duchenne Muscular Dystrophy – not an ultra-rare disease) pricing questioned by payors
Ultra-rare and «only» orphan drugs face the same issues: can the payor (ie. the citizens) accept to pay and how long?
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