christian girard presentation london 20150629
TRANSCRIPT
![Page 1: CHRISTIAN GIRARD PRESENTATION LONDON 20150629](https://reader035.vdocument.in/reader035/viewer/2022062320/55cce77bbb61eb9e3a8b4748/html5/thumbnails/1.jpg)
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
![Page 2: CHRISTIAN GIRARD PRESENTATION LONDON 20150629](https://reader035.vdocument.in/reader035/viewer/2022062320/55cce77bbb61eb9e3a8b4748/html5/thumbnails/2.jpg)
Orphan disease prevalence: < 1/2.000
Ultra-rare disease prevalence: < 1/50.000
Picture: Alexion Pharmaceuticals
![Page 3: CHRISTIAN GIRARD PRESENTATION LONDON 20150629](https://reader035.vdocument.in/reader035/viewer/2022062320/55cce77bbb61eb9e3a8b4748/html5/thumbnails/3.jpg)
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)
![Page 4: CHRISTIAN GIRARD PRESENTATION LONDON 20150629](https://reader035.vdocument.in/reader035/viewer/2022062320/55cce77bbb61eb9e3a8b4748/html5/thumbnails/4.jpg)
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
![Page 5: CHRISTIAN GIRARD PRESENTATION LONDON 20150629](https://reader035.vdocument.in/reader035/viewer/2022062320/55cce77bbb61eb9e3a8b4748/html5/thumbnails/5.jpg)
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$
![Page 6: CHRISTIAN GIRARD PRESENTATION LONDON 20150629](https://reader035.vdocument.in/reader035/viewer/2022062320/55cce77bbb61eb9e3a8b4748/html5/thumbnails/6.jpg)
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)
![Page 7: CHRISTIAN GIRARD PRESENTATION LONDON 20150629](https://reader035.vdocument.in/reader035/viewer/2022062320/55cce77bbb61eb9e3a8b4748/html5/thumbnails/7.jpg)
ULTRA-RARE DRUGS SALES
2014 SALESSoliris $2 146 000 000Cinryze $503 000 000Procysbi $70 000 000Vimizim $37 000 000
![Page 8: CHRISTIAN GIRARD PRESENTATION LONDON 20150629](https://reader035.vdocument.in/reader035/viewer/2022062320/55cce77bbb61eb9e3a8b4748/html5/thumbnails/8.jpg)
ULTRA-RARE DRUGS = ULTRA-RARE TECHNOLOGY? Small molecules Biologics Gene therapies
Ultra-rare drugs do not necessarily imply advanced technology. Some are « simply » repurposed small molecules
![Page 9: CHRISTIAN GIRARD PRESENTATION LONDON 20150629](https://reader035.vdocument.in/reader035/viewer/2022062320/55cce77bbb61eb9e3a8b4748/html5/thumbnails/9.jpg)
THREAT: EVOLVING PAYORS POLICIES Global cost of orphan drugs under scrutiny:
120B$ (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
![Page 10: CHRISTIAN GIRARD PRESENTATION LONDON 20150629](https://reader035.vdocument.in/reader035/viewer/2022062320/55cce77bbb61eb9e3a8b4748/html5/thumbnails/10.jpg)
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
![Page 11: CHRISTIAN GIRARD PRESENTATION LONDON 20150629](https://reader035.vdocument.in/reader035/viewer/2022062320/55cce77bbb61eb9e3a8b4748/html5/thumbnails/11.jpg)
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)
![Page 12: CHRISTIAN GIRARD PRESENTATION LONDON 20150629](https://reader035.vdocument.in/reader035/viewer/2022062320/55cce77bbb61eb9e3a8b4748/html5/thumbnails/12.jpg)
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
![Page 13: CHRISTIAN GIRARD PRESENTATION LONDON 20150629](https://reader035.vdocument.in/reader035/viewer/2022062320/55cce77bbb61eb9e3a8b4748/html5/thumbnails/13.jpg)
ULTRA-RARE DRUGS PLAYERS MARKET CAPITALIZATION
Alexion Pharmaceuticals: 37B$
BioMarin Pharmaceutical: 22B$
Ultragenyx Pharmaceutical: 3.7B$
Raptor Pharmaceuticals: 1B$
Aegerion Pharmaceuticals: 0.5B$
![Page 14: CHRISTIAN GIRARD PRESENTATION LONDON 20150629](https://reader035.vdocument.in/reader035/viewer/2022062320/55cce77bbb61eb9e3a8b4748/html5/thumbnails/14.jpg)
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$
![Page 15: CHRISTIAN GIRARD PRESENTATION LONDON 20150629](https://reader035.vdocument.in/reader035/viewer/2022062320/55cce77bbb61eb9e3a8b4748/html5/thumbnails/15.jpg)
INVESTMENT IN ULTRA-RARE PLAYERS PROVED SUCCESSFUL (1)
![Page 16: CHRISTIAN GIRARD PRESENTATION LONDON 20150629](https://reader035.vdocument.in/reader035/viewer/2022062320/55cce77bbb61eb9e3a8b4748/html5/thumbnails/16.jpg)
INVESTMENT IN ULTRA-RARE PLAYERS PROVED SUCCESSFUL (2)
![Page 17: CHRISTIAN GIRARD PRESENTATION LONDON 20150629](https://reader035.vdocument.in/reader035/viewer/2022062320/55cce77bbb61eb9e3a8b4748/html5/thumbnails/17.jpg)
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
![Page 18: CHRISTIAN GIRARD PRESENTATION LONDON 20150629](https://reader035.vdocument.in/reader035/viewer/2022062320/55cce77bbb61eb9e3a8b4748/html5/thumbnails/18.jpg)
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?