five years with the orphan drug directive in the eu achievements by the comp five years with the...
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Five years with the Orphan Drug Directive in the EUFive years with the Orphan Drug Directive in the EU
Achievements by the COMPAchievements by the COMP
Josep Torrent-FarnellAutonomous University of Barcelona
Chairman COMP/EMEA, London
Grand Hôtel, Stockholm, SwedenGrand Hôtel, Stockholm, Sweden
14th, 15th and 16th February 2005
International Conference on Rare Diseases and Orphan Drugs
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CONTENTSCONTENTS
1. Overview of the COMP tasks
2. Snapshot of the COMP achievements and
challenges
3. The COMP/EMEA ad-hoc Group on benefits
of the EU Orphan Legislation
4. Conclusions
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1)Designation-related activities: the EMEA OMP Policy continuity
2)Non designation-related activities: public-health oriented
* All COMP activities are fully and closely supported by the EMEA (Sector of OMP and Scientific Advice): legal, regulatory, procedural, administrative and specific scientific support
Overview of the COMP main tasks*Overview of the COMP main tasks*
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What does Orphan Designation implies?
– Scientific-based/product-oriented and
disease-management assessment.
– Provides different incentives to facilitate
drug research and Market Exclusivity
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……Conditions for achieving orphan drug Conditions for achieving orphan drug status…status…
• The sponsor’s hypothesis should be biologically plausible
• The indication should be a genuine one (i.e. valid condition) and not a ‘manufactured’ one by sub-setting a common condition (i.e. artificial subset or “salami-slicing”)
• The prevalence limit should not exceed 5 per 10,000 (227,000 patients affected in the EU)
• Significant Benefit Criterion (“added value”)
• Alternatively an economic criterion can be considered
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Orphan Medicinal Products
Main EU Incentives• Ten years exclusivity from the date of
marketing authorisation• Protocol assistance from the EMEA• Direct access to Centralised Procedure• Fees reduction for centralised applications• Priority access to EU research programs
National Incentives• Inventory published on Commission Web-site
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COMPCOMP Orphan Designation Orphan ConditionOrphan Condition
SWAGSWAG Development phase
CHMPCHMP MA application
(designated as orphan)
Therapeutic indication
COMPCOMP Significant Benefit
(69%)
To be kept as orphan
10 years Market Exclusivity
EMEA/OMP Policy EMEA/OMP Policy ContinuityContinuity
(Article 8, 141/2000)
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1)To advise the Commission: EU Policy on OMP
2)To assist the Commission:
a) In liaising internationallyb) In liaising with patients support groupsc) In preparing guidelines
* The COMP/EMEA 3-year report (1st Mandate, have addressed this issue)
Public-Health COMP related activitiesPublic-Health COMP related activities
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Applications submitted
440
Positive COMP opinions
297
Commission decisions
259
Final negative COMP decisions 6
Applications withdrawn
128
Turning Hopes into Reality: Turning Hopes into Reality: DesignationDesignation
COMP achievements April 2000-February 2005COMP achievements April 2000-February 2005
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Overall (April 2000 – February 2005): 31%
2000: 19%
2001: 31%
2002: 43%
2003: 44%**
2004: 23%
*Including negative COMP opinions** Reinforcing the pre-submission EMEA meetings + COMP Guidelines
Withdrawals failure rate*Withdrawals failure rate*
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PROFILLING THE COMP OPINIONS -I-
By ATC CodeBy ATC Code::• 36% Oncology• 11% Metabolism• 11% Immunology• 7% Musculoskeletal and CNS
By Product typeBy Product type:• Innovative / “old” substances ratio: 60%• Biotech-based products: 33%• Biological / plant-based products: 6%
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PROFILLING THE COMP OPINIONS -II-
By prevalenceBy prevalence:• < 1 / 10 000 = 61%• 1-3 /10 000 = 31%• 3-5 / 10 000 = 8%
By population type:By population type:• Children and adults 56.3%• Children only 9.4%• Adults only 34.4%
By company size: By company size: SME 85%
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18 Authorised Orphan Medicinal Products
2 CHMP positive opinion in decision-making
3 Extension-line of new indications granted
9 Centralised application on-going (review
phase)
4 Applications submitted MR*
67 Protocol assistance**
*Centralised review Mandatory as November 2005** Provided by SAWG with COMP contribution
Turning Hopes into reality: MATurning Hopes into reality: MA
CHMP Achievements: up to February 2005CHMP Achievements: up to February 2005
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Challenges on Orphan Drug Clinical Development
• Conventional methodological designs need to be adjusted and applied in a flexible manner
• Alternative methodological approaches and patient-saving designs should be encouraged
• Compassionate and expanded access programmes should not undermined the conduct of well-designed studies
• Investigation phase goes beyond the MA: conditional / under exceptional circumstances approval, thus early PhV planning becomes crucial
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1) Regular interactions with OOPD/FDA
2) More input to funding community projects:
DG Research and DG SANCO
3) Gathering information from national
initiatives
4) Contributing to make RD more “visible” and
“known” by society and health professionals
by catalyzing discussions among relevant
stakeholders
Public Health related COMP/EMEA Public Health related COMP/EMEA achievementsachievements
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2nd Workshop for Learned Societies and
Academia: EMEA, 4TH March 2005
Contribution to the EUROPEAN CONFERENCE
FOR RARE DISEASES (“From difficulties to solutions for
the rare disease community”), organized by Eurordis,
under de Luxembourg presidency of the EU:
21-22 June 2005
Increasing the Awareness on RDIncreasing the Awareness on RD
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COMP adoption – May 2005
Transmission to the Commission – June 2005
Commission shall publish a general report on
the experience gained + public health benefits
obtained by 22 January 2006 (art. 10;
141/2000)
EMEA/COMP ad-hoc group for the review EMEA/COMP ad-hoc group for the review of the Orphan Legislation (horizon 2006)of the Orphan Legislation (horizon 2006)
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EU Regulation Benefits of Orphan EU Regulation Benefits of Orphan MedicinesMedicines
1. Patients
2. Academic and professional development
3. Pharmaceutical Industry
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PatientsPatientsIncrease of society awareness and advocacy
– EURORDIS– National organization– Patients associations– Affected families
More authorised medicines and drugs on the investigational track.
More objective information on new medicines is available.
The role of patients groups as “non-medical experts” will be increased
Clinical Trials Public Rare Diseases Database is ongoing.
Availability and true access to OMP is what really matters
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Academic Environment and Academic Environment and Professional DevelopmentProfessional Development
• More research activities founded by EU/National grants.
• Translation from basic research to clinical (e.g. emerging therapies derived from Human Genome knowledge).
• Pan-European network of centres of excellence.
• Contributing to expand the rare diseases paradigm.
• Empowering patients in the co-decision interventions.
• Clinical Trials Public Rare Diseases Database is ongoing.
More public founds for research and continuing activities are still a priority
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Pharmaceutical IndustryPharmaceutical Industry
• Incentives to develop medicines for a small segment of market.
• Positive impact on SME’s.
• Increasing use of EMEA Protocol assistance.
• Guidance for planning and conducting research on rare diseases by the EMEA’ scientific committees has been released for public consultation.
Establishing the fair price of Orphan Medicines a pending issue for ensuring long-term sustainability by national competent authorities
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The experience gained so far …The experience gained so far …
• RD/OMP is a high priority in the EU Public Health
• The current EU OMP legislation has demonstrate clear benefits for all stake holders, paving the road to fostering biomedical research in this field
• Health professionals and voiced patients play an irreplaceable role
• The EMEA together with its scientific committees gives the appropriated forum to maximize the overall orphan drug research
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In ConclusionIn Conclusion
COMP Members and EMEA Staff are ready to interact openly with all sponsors in order to improve the overall outcome of the Orphan Legislation for the sake of patients and Public Health Interested.
EMEA/COMP is working together with interested parties to make proposals and give advice to Commission on the next revision of the EU OMP legislation frame.