dr peter arlett european commission orphan medicines: dg enterprise and industry perspective icord...
TRANSCRIPT
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Dr Peter Arlett
European Commission
Orphan medicines: DG Enterprise and Industry
perspective
ICORD February 2005
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In this talk
• Cooperation – a role for the EU
• Orphan regulation – impact so far
• Key role of market exclusivity
• Looking to the future
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Cooperation
• Multiple patients
• Multiple diseases
• Multiple stakeholders
• Multiple member states
• Multiple languages
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the way ahead can be confusing
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Cooperation
• EU working with stakeholders has provided some solutions
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EU solutions
• Public Health Programs – communication, networking, databases, patient groups
• Framework Programs – funding of research on rare diseases
• EU Orphan Regulation – incentives for developing orphan medicines
+ the vital role of Member State actions
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EU Orphan regulation
• In force since 2000:
– If medicine designated as ‘orphan’ – 10-years market exclusivity from ‘similar’ medicines
– Protocol assistance
– Fee reductions– Access to the centralised procedure
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EU Orphan Regulation
• > 250 designations
• 20 authorised medicines
• 24 indications
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The successes….so farNew authorisations• 2 x Fabrazyme & Replagal for
Fabry disease• Glivec for chronic myeloid
leukaemia• 2 x Tracleer & Ventavis for
pulmonary hypertension• Somavert for acromegaly• Zavesca for Gaucher disease• Carbaglu for hyperammonaemia• Aldurazyme for
Mucopolysaccharidosis• Busilvex (iv) for haematopoietic
progenitor cell transplantation• Onsenal for Familial Adenomatous
Polyposis• Photobarr for Barrett’s
Oesophagus
• Litak for Hairy Cell Leukaemia• Lysodren for adrenal cortical
carcinoma• Pedea for patent ductus
arteriosus Wilzin for Wilson’s disease
• Trisenox for acute promyelocytic leukaemia
• Xagrid for Thrombocythaemia• Orfadin for Hereditary tyrosinemia
type 1 • Prialt for chronic pain
Extensions of indication• Glivec for GIST• Glivec for first line use in CML• Glivec for paediatric use in CML
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The impact on industry
SMEs*:
• 15% of Marketing Authorisations
• 20% of Scientific Advice requests
• 40% of designations
Better data are required
*EMEA figures
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10-years market exclusivity is key
But derogations e.g. article 8(2) of the regulation states:
• “This period may however be reduced to six years if, at the end of the 5th year, it is established……that the product is sufficiently profitable not to justify maintenance of market exclusivity. …. “
• This has caused concern
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Commission response
Developing a guideline on sufficient profitability:
• Orphan pricing study
• Working Group of the transparency committee
• Consultation
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Orphan pricing study - findings
• Variability of Manufacturer’s Price Before Tax is low (mean 122% of minimum price)
• Public Price including Taxes is the main explanation for the variability in prices (ratio 1.7) due to: Tax, Distribution costs, Profit for retail pharmacies, Hospital pharmacy profit
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Orphan pricing study - findingsPrice comparators
c.f. non orphan indication of same medicine – not possible
c.f. US price – very similar – ratio 1.06 (8 products)
c.f. surgical treatments – 2-3 times cheaper
c.f. non-designated ‘orphan’ drugs in same indication - very variable
c.f. ‘expensive non-orphans’ – x10
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Orphan pricing study - findings
Average cost per patient per year
6,000 – 300,000 Euros
Correlates with prevalence but not ‘innovation’ or ‘medical benefit’
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Orphan pricing study - findings
Industry revenues
Potential: 100 million – 1.5 billion Euros worldwide
c.f. world top ten blockbusters: 2 – 7 billion Euros worldwide
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Orphan pricing study - findings
Calculating sufficient profitability:
• Equation / system for measuring profitability:
–will not be easy
–various possible methods:• revenues only,
• complete profit calculation etc
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Looking foreword….
• Regulation on SME
• Regulation on paediatrics
• Regulation on conditional marketing authorisations
• “2005 Report” of the orphan regulation
• (Public Health programmes)
• (7th Framework Program)
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Regulation on SMEsIF SME + Orphan status :
=>100% ‘reduction’ on :Scientific advice/protocol assistanceScientific services
+ fee deferrals:
Post-authorisation
Marketing Authorisation Application+Inspections (pre-authorisation)
Granting of theMarketing Authorisation
Payment deferred until the end of the marketing authorisationprocedure
TIME
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Regulation on paediatrics
• Requirement for studies in children or waiver or deferral
• Reward for orphan medicines 2-years additional market exclusivity (10+2)
In force by end of 2006 ?
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Regulation on conditional marketing authorisations
• lays down the conditions and procedure for granting a conditional MA
• orphan medicines within scope
• “public health interest”….”unmet medical need”
• MA with specific obligations and annual renewals
In force before November 2005
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“2005 Report” of orphan regulation
Article 10 states:
• “Before 22 January 2006, the Commission shall publish a general report…experience acquired…public health benefits..”
• Plan:– Currently collecting information / data– Public consultation
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Conclusions
• Much has been achieved in the past 10-years – orphan regulation central to this
• DG Enterprise and Industry is committed to: – the development of orphan medicines– the orphan regulation– supporting SMEs– promoting public health