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Compassionate use programs and the
European regulatory system
Filip Josephson M.D., Ph.D.
Clinical Assessor
The European Medicines Agency (EMA) and the
Committe for Medicinal Products for Human Use
(CHMP)
The European Medicines Agency is a decentralised agency of the EU, located in
London, and is responsible for the scientific evaluation of medicines developed by
pharmaceutical companies for use in the EU.
The CHMP is the ultimate scientific decision-making assembly at the EMA. It consists
of representatives from each EU national agency
The decisions of the CHMP are based on assessments and recommendations
generated by national agency assessors
The responsibilities of EMA/CHMP and that of
the national agencies
• Decisions concerning the approval, conditions for approval,
and post-approval follow-up of new antivirals are taken by the
CHMP. CHMP decisions pertain to the entire EU
• Decisions on clinical trial applications are taken by the
national agencies alone (or cooperatively through VHP
procedures) and pertain to the area of jurisdiction of each
national authority
• Decisions on the approval and implementation of
compassionate use programs (and other forms of ”early
access” such as availability on a named-patient basis) are
principally the responsibility of national agencies in
accordance with national legislation.
Forms of early access (treatment prior to drug
approval
• Clinical trials
• Expanded access programs (understood as clinical trials
with primarily a safety endpoint)
• Compassionate use programs
• Named patient basis
Compassionate use programs
• Compassionate use programmes can only be put in
place for medicines that are expected to help patients
with life-threatening, long-lasting or seriously disabling
illnesses who currently cannot be treated satisfactorily
with authorised medicines, or who have a disease for
which no medicine has yet been authorised.
• The compassionate use route may be a way for patients
who cannot enrol in an ongoing clinical trial to obtain
treatment with a potentially life-saving medicine.
General limitations on knowledge when
deciding on compassionate use
• At this stage in the development of the medicine, what is
known of the medicine’s safety may be limited.
Generally, toxicology studies will have been completed
and analysed, and early pharmacokinetics studies will
have been completed.
• There may still be uncertainties about the best way to
give the medicine to patients, such as the exact dose to
use, and the dose frequency, and the medicine’s safety
profile is not yet fully established.
The role of the EMA concerning
compassionate use (article 83)
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The objectives of article 83
• To facilitate and improve the access of patients in the
European Union to compassionate use programmes
• To favour a common approach regarding the conditions
of use, the conditions for distribution and the patients
targeted for the compassionate use of unauthorised new
medicinal products,
• To increase transparency between member states in
terms of treatment availability.
Compassionate use by EC article 83
Medicinal use to be made available to:
patients with a chronically or seriously debilitating disease or a lifethreatening disease, and who cannot be treated satisfactorily by an
authorised medicinal product in the European Union. The medicinal
product is either the subject of a marketing application or is undergoing
clinical trials, and would be approved through the ”centralised
procedure” (applicable for all new antiviral substances).
The compassionate use program is intended for ”a group of patients”
(as opposed to Named Patient Basis). “Group of patients” can be
interpreted as any set (i.e. more than one) of individual patients that
would benefit from a treatment for a specific condition.
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Article 83 versus member state authority
•
The CHMP may adopt an opinion on the conditions for
use, distribution and patients targeted
•
CHMP opinions are not binding on MS; however, ”MS
shall take account of any available opinions”
•
The recommendation complements national legislation,
which it does not replace
•
The recommendations are optional, and are only
implemented by the MS that wish to use them for their
patients
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Request for a CHMP opinion according to
article 83
•
When a MS envisages the need to make a medicinal
product available for compassionate use, the national
agency of that MS should notify the EMA
•
Companies should not directly contact the EMA for
CHMP opinion
•
Companies may, at their own initiative, inform the EMA
of compassionate use in MS(s)
•
Article 83 has hitherto only been implemented for i.v.
oseltamivir and i.v. Zanimivir (for treatment of influenza
in severely ill patients)
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Principal requirement for compassionate use
•
Access to documentation about the drug
•
Access to the drug
European regulators have no legal provision to force a
company to provide early access through compassionate
use
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The European Medicines
Agency
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