Download Summary of regulatory concepts and legislation, and the role of

European Patients’ Academy
on Therapeutic Innovation
http://www.eupati.eu
Fact Sheet: Summary of regulatory concepts and legislation, and the role of patient
organisations
Type of
products
Definition of the product
Medicines for the treatment of
rare diseases (life-threatening
or chronically debilitating
conditions that affect no more
than 5 in 10,000 people in EU)
Orphan
medicines
Key aspects of regulatory
pathway for Marketing
Authorisation (MA)
application and approval
Dedicated scientific
committees
Specific role of patient
organisations
European Medicines Agency
(EMA) centralised procedure:
mandatory
COMP (EMA – Committee for
Orphan Medicinal Products):
assesses the Orphan
Designation application
Members of the COMP
Application for Orphan
Designation as a pre-requisite
before MA application
Incentives from EMA:
scientific & technical support;
fees reduction for small &
medium enterprises (SMEs);
extension of market exclusivity
1
European Patients’ Academy
on Therapeutic Innovation
http://www.eupati.eu
Advancedtherapy
medicinal
products
(ATMPs)
Complex medicines made
from genes and cells or
tissues (different from
conventional medicines made
from chemicals or proteins)
They propose novel
approaches for treating
diseases and injuries (and not
only genetic diseases) and
are from the most advanced
scientific researches
Medicines for children from
birth to less than 18 years
Paediatrics
medicines
EMA centralised procedure:
mandatory
CAT (EMA -Committee for
Advanced Therapies): assess
the MA application
Members of the CAT
PDCO (EMA – Paediatric
Committee): assess the PIP
and issue PIP waiver
Members of the CAT
Beyond quality, safety, and
efficacy, assessment may also
consider criteria due to the
nature of the product:
i.e., environmental risk
Full traceability is required
Incentives from EMA: fees
reduction
EMA centralised procedure
Submission of a proposal of
paediatric investigation plan
(PIP) as pre-requisite before
application at EMA level
(mandatory for all new
medicinal products)
Application of MA must
contain results and
information required in the PIP
– except when PIP has been
waived when a product targets
diseases not affecting children
2
European Patients’ Academy
on Therapeutic Innovation
http://www.eupati.eu
Incentives from EMA :
scientific consultation &
extension of patent exclusivity
Medicines developed to be
the same as a medicine
already authorised ‘the
reference medicine’
Generic
medicines
Biosimilar
medicines
Contain the same active
substances and used to treat
the same diseases at the
same dose
EMA centralised procedure
(under conditions) or
national and other procedures
No specific committee
Application dossier: based on
the reference medicine data
and on the bioequivalence
data of the generic compared
to the reference
But may differ on inactive
ingredients, name,
appearance, taste
MA only granted after the
period of exclusivity of the
reference medicine has
expired
Medicines that are developed
to be similar to biological
medicines already authorised
(biological medicines are
complex medicines made or
derived from a biological
source/living cells and may
contain blood and blood
components, gene therapies,
tissues, and proteins.
EMA centralised procedure or
national/ other procedures
MA only granted after the
period of exclusivity of the
biological medicine (as
reference) has expired
No specific committee but
BMWP (EMA – Working Party
on Biosimilar Medicinal
Products): provide
recommendations to the
CHMP and liaise with
interesting parties
Part of interested parties in
liaison with BMWP
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