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Table 1 Orphan drugs in France in August 2016

From: Patient access to orphan drugs in France

  Number Percent
Active MA and orphan designation
Number of drugs 91
Number of active designated orphan indications by drug   
 - 1 rare disease 76 / 91 83.5
 - 2 rare diseases 10 / 91 11.0
 - 3 rare diseases 3 / 91 3.3
 - 4 rare diseases 1 / 91 1.1
 - 6 rare diseases 1 / 91 1.1
Total number of orphan drug–therapeutic indication pairs 115
Assessment of orphan drug–therapeutic indication pairs (N = 115) by the HAS
Number of assessed orphan drug–therapeutic indication pairs (i.e. published HAS opinions) 94/115 81.7
Not found on the HAS website 18/115 15.7
Assessment is on-going 2/115 1.74
Withdrawn by pharmaceutical company 1/115 0.90
Actual clinical benefit of published HAS opinions
 - Important 72/94 76.6
 - Moderate 17/94 18.1
 - Mild 1/94 1.1
 - Insufficient (not accepted for reimbursement) 4/94 4.3
Clinical Added Value of published HAS opinions
 - Major 2/94 2.1
 - Important 16/94 17.0
 - Moderate 20/94 21.3
 - Minor 36/94 38.3
 - No clinical improvement 16/94 17.0
 - Not applicable (because of insufficient actual clinical benefit) 4/94 4.3
Eligibility for health economic assessment 12/115 -a
Adopted efficiency opinions 3/12 25.0
 - with major methodological criticisms expressed by the HAS 2/3 66.7
Published efficiency opinion 1/3 33.3
Marketing of registered orphan drug–therapeutic indication pairs
Number of marketed orphan drug–therapeutic indication pairs 90/115 78.3
 - through the post-ATU transitional provision 15/90 16.7
Number of non-marketed orphan drug–therapeutic indication pairs 25/115 21.7
Regulatory prescribing requirements of marketed orphan drug–therapeutic indication pairs (N = 90)
Reserved for hospital use 19 / 90 21.1
On hospital prescription 56 / 90 62.2
On initial hospital prescription 14 / 90 15.6
On specialist doctor’s prescription 60 / 90 66.7
Specific monitoring 55 /90 61.1
Drug delivery circuits for marketed orphan drug–therapeutic indication pairs (N = 90)
Hospital pharmacies for inpatients 90/90 100
 - through the post-ATU transitional provision 15/90 16.7
Community pharmacies 28/90 31.1
Hospital pharmacies for outpatients (retrocession) 40/90 44.4
 - through the post-ATU transitional provision 13/40 32.5
Funding / theoretical reimbursement ratesss
Inpatients (hospital) - -
 - universal reimbursement (in connection with Diagnosis Related Groups) 52/90 57.8
 - high-priced drug list 23/90 25.5
 - specific to the post-ATU transitional provision 15/90 16.7
Outpatients (hospital pharmacies) = retrocession - -
 - full reimbursement (100%) 33/40 including 13 post-ATU 82.5
 - reimbursement up to 65% 6/40 15.0
 - reimbursement up to 30%   2.5
1/40
Community pharmacies - -
 - full reimbursement (100%) 16/28 57.1
 - reimbursement up to 65% 9/28 32.1
 - reimbursement up to 30% 3/28 10.7
  1. aNot applicable because health economic evaluations were set up in France in October 2012 [53]