Table 1 Orphan drugs in France in August 2016
| Â | Number | Percent |
|---|---|---|
Active MA and orphan designation | ||
Number of drugs | 91 | – |
Number of active designated orphan indications by drug | Â | Â |
 - 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Withdrawn by pharmaceutical company | 1/115 | 0.90 |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 |