Orphan m edicinal products Activities of the Committee for Orphan - - PowerPoint PPT Presentation

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Orphan m edicinal products Activities of the Committee for Orphan - - PowerPoint PPT Presentation

Mar 09, 2024 783 likes •1.03k views

Orphan m edicinal products Activities of the Committee for Orphan Medicinal Products (COMP) Presented by: Paolo Tomasi, European Medicines Agency Orphan designation Principles Designation criteria Incentives Procedure

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SLIDE 1

Orphan m edicinal products

Activities of the Committee for Orphan Medicinal Products (COMP)

Presented by: Paolo Tomasi, European Medicines Agency

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SLIDE 2

Orphan medicinal products - COMP 2

Orphan designation

  • Principles

– Designation criteria – Incentives – Procedure

  • Experience

– Designations – Authorizations – Conclusions

Orphans by Thomas Kennington

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Orphan medicinal products - COMP 3

W hy is there a EU legislation to stim ulate developm ent of drugs for rare diseases?

Ligneous conjunctivitis ~150 cases described

=

for

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Orphan medicinal products - COMP 4

W hy is there a EU legislation to stim ulate developm ent of drugs for rare diseases?

“Persons suffering from rare conditions should be entitled to the same quality of treatment as other patients” But… “ the pharmaceutical industry would be unwilling to develop the medicinal product under normal market conditions” As… “some conditions occur so infrequently that the cost of developing and bringing to the market a medicinal product (… ) would not be recovered by the expected sales”

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Orphan medicinal products - COMP 5

Orphan Regulations in the EU

Regulation ( EC) No 1 4 1 / 2 0 0 0 of the EP

  • n Orphan Medicinal Products ( 1 6 / 1 2 / 1 9 9 9 )
  • Criteria for designation
  • Committee (COMP)
  • Procedure
  • Incentives

Com m ission Regulation ( EC) 8 4 7 / 2 0 0 0 of 2 7 / 0 4 / 2 0 0 0

  • Criteria for designation
  • Similarity
  • Clinical superiority

Com m ission Regulation ( EC) 7 2 6 / 2 0 0 4 of 2 0 / 1 1 / 2 0 0 5

  • Mandatory centralised MA application for orphans
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SLIDE 6

Orphan medicinal products - COMP 6

COMP ( Com m ittee for Orphan Medicinal Products)

EMEA Com m ittee: 3 3 ( + 2 ) m em bers + chairperson

  • 1 member per Member State (N= 27)
  • 6 members nominated by the European Commission

– 3 patient representatives – 3 members proposed by EMA

  • 2 non-voting members (Iceland and Norway)

COMP tasks:

  • Opinions on designation
  • To advise Commission on establishment and development of a policy on
  • rphan medicinal products
  • To assist EU Commission in liaising internationally and with patient

support groups

  • To assist EU Commission on guidelines
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Orphan medicinal products - COMP 7

Orphan Medicines Section ( EMA)

I n the sector Hum an Medicines Special Areas

  • Head of Section: Jordi Llinares Garcia
  • 4 Scientific Administrators (physicians, pharmacists…

)

  • 3 Assistants

Tasks:

  • coordination of procedures for the designation of orphan medicinal

products:

– Preparation of Summary Reports on applications – Scientific and Administrative secretariat of COMP

  • Coordination of the review of orphan designation criteria at the time
  • f granting/ varying a marketing authorisation
  • coordination of the review of market exclusivity of authorised orphan

medicine products 5 years from the granting of the marketing authorisation

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Orphan medicinal products - COMP 8

Overview of orphan designation process

COMP MEETING I

Intent to file letter Application submission Validation Evaluation List of questions (oral explanation)

Opinion DAY 60 DAY 1 DAY 90 EU Decision

Publication of public summary of opinion (lay language) on EMA website

COMP MEETING II

“clockstop” (invalid applications)

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Orphan medicinal products - COMP 9

Orphan designation

  • For medicinal products for human use
  • Procedure is free of charge
  • Can be requested at any stage of development, but

before request for MA (even 1 day)

  • Sponsor can be either company or individual

– Established in the EEA (EU, Iceland, Lichtenstein, Norway)

  • European Commission Decision

gives access to incentives

  • Centralised procedure for MA compulsory
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Orphan medicinal products - COMP 10

Orphan designation: 3 criteria

NO YES 1: “Prevalence” criterion 2: “Seriousness” criterion Prevalence

(≤ 5 / 10,000)

Insufficient return on investment

(costs > expected revenues)

Life-threatening, seriously debilitating or serious and chronic Are there available “methods” for diagnosis / prevention / treatment? Significant benefit / current methods non satisfactory 3: “Significant Benefit” criterion Life-threatening or chronically debilitating

  • r

OK

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Orphan medicinal products - COMP 11

Prevalence of designated orphan conditions ( m ust be < 5 / 1 0 ,0 0 0 )

36% 53% 11% less than 1 in 10,000 between 1 and 3 in 10,000 more than 3 in 10,000

Data updated 21/ 09/ 2010

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Orphan medicinal products - COMP 12

I ncentives ( 1 / 3 )

Economic Economic / marketing: / marketing:

  • Fee reduction / exemption

– Extended incentives for SMEs (post authorisation)

  • Market exclusivity (10 years)

Product development: Product development:

  • Free protocol assistance (Scientific Advice

for Orphan Medicinal products)

Community marketing authorisation Community marketing authorisation National incentives (EC inventory) National incentives (EC inventory)

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Orphan medicinal products - COMP 13

I ncentives: econom ic/ m arketing ( 2 / 3 )

  • Fee reductions:

– 50% market authorisation application – 100% protocol assistance – 100% post authorisation fees

  • 10-year market exclusivity

– protection against:

  • similar products (structure/ mechanism of action
  • for the same indication

– Four possible derogations:

  • Sponsor’s consent
  • Lack of supply
  • Clinical superiority of another similar product
  • Review after 5 years at MS request
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Orphan medicinal products - COMP 14

I ncentives: product developm ent 3 / 3

Protocol assistance

  • Protocol assistance 

scientific advice for orphans

– Questions on quality-efficacy-safety – Questions on significant benefit – Company position required – SAWP provides answers – CHMP adopts answers – COMP involved if issues on benefit

(small) differences PA / SA

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Orphan medicinal products - COMP 15

Use of incentives fund

Use of EU special contribution for orphan medicines ( 2 0 0 9 )

Ma r k e ting a uthor isa tions 2 9 % Pr otocol a ssista nce 6 3 % I nspe ctions 4 % Post- a uthor isa tion pr oce dur e s 4 %

Data updated 21/ 09/ 2010

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Orphan medicinal products - COMP 16

Orphan designation is “com petitive”

Designation can be granted for the same orphan indication, to two or more sponsors, even for similar or identical products First sponsor with MA for an orphan indication

  • btains exclusivity (for the therapeutic indication)

Subsequent sponsor(s) for the same therapeutic indication

  • to break exclusivity:

Products similar? (CHMP) Yes No

Clinical superiority required Significant benefit still required as usual

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Orphan medicinal products - COMP 17

Orphan applications 2 0 0 0 -2 0 0 9

2000 2001 2002 2003 2004 2005 2006 2007 2008 2009

To Total tal

  • No. of

applications submitted 72 83 80 87 108 118 104 126 119 164

1061 1061

Positive Opinions 26 64 43 54 75 88 81 97 86 113

727 727

Commission Decisions 14 64 49 55 72 88 80 98 73 106

699 699

Negative Opinions 1 3 1 4 2 1 1 2

15 15

Withdrawals 6 27 30 41 22 30 20 19 31 23

249 249

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Orphan medicinal products - COMP 18

Designations by therapeutic area

immunology 9%

  • ncology

43% antiinfectious 4%

  • ther

12% musculoskeletal and nervous system 12% cardiovascular and respiratory 9% metabolism 11%

Data updated 21/ 09/ 2010

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Orphan medicinal products - COMP 19

Orphan conditions affecting children

Both adult AND paediatric 50% Paediatric ONLY 8% Adult ONLY 42%

Data updated 21/ 09/ 2010

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Orphan medicinal products - COMP 20

Orphan conditions affecting children

0% 20% 40% 60% 80% 100% 2000 2001 2002 2003 2004 2005 2006 2007 2008 2009 Medical conditions affecting adults only Medical conditions affecting both children and adults Medical conditions affecting children only

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Orphan medicinal products - COMP 21

Therapeutic areas of the 6 3 authorised

  • rphan m edicinal products

38% 38% 22% 22% 10% 10% 11% 11% 3% 3% 16% 16%

antineoplastic and immunomodulating agents metabolism blood musculoskeletal and nervous system cardiovascular

  • thers
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Orphan medicinal products - COMP 22

Conclusions

  • 90-day procedure
  • 3 legislation requirements

(rarity/ return, prevalence, no other treatments/ unsatisfactory/ benefit)

  • Designation is competitive
  • Orphan indication (at designation) is not

necessarily the same as therapeutic indication at MA

  • Significant benefit to be:

– Claimed at OD application stage, if authorised drugs exist (or satisfactory non-medicinal treatments); – Confirmed at MA with relevant data

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Orphan medicinal products - COMP 23

Thank you for your attention Questions?