Patient access to orphan drugs in France

Abstract

Background Since incentives were introduced to promote orphan drugs in Europe, several dozens of drugs have been registered at the European level. However, patient access on a national level remains very heterogeneous across Europe. This can be explained by healthcare organization and drug reimbursement, which are within the purview of each Member State. We studied access to orphan drugs in France from the patients’ point of view, including marketing but also ease of supply from patients’ perspective, financial and time-based dimensions. Results We identified 91 registered orphan drugs in Europe, corresponding to 115 orphan drug–therapeutic indication pairs. In France, 78.3% (90/115) of these pairs were marketed: 100% were available to inpatients and 75.6% were available to outpatients. The median period between granting of the European marketing authorization and publication of the reimbursement decision was 360 days. The broadest availability—through community pharmacies—was guaranteed in only 31.1% of cases. Prescriptions were mainly restricted either to hospital-based doctors or to specialists. Inpatients were not financially responsible for these prescriptions and 72% of the orphan drug–therapeutic indication pairs available to outpatients were fully covered by national health insurance in France. Conclusions Patient access to orphan drugs is not universal in France. Access to reimbursement has a strong impact on patients’ effective access to orphan drugs, which may be restricted by difficulties with assessing the clinical value of these drugs and with pricing issues. Prescribing restrictions and drug delivery systems influence the ease of patients’ supply for reimbursed orphan drugs for patients. Patients do not seem to be limited by financial issues, but the growing budgetary impact of orphan drugs is worrisome from a societal point of view.

An analysis of orphan medicine expenditure in Europe: is it sustainable?

“…This gap, which was about 1.4 percentage points in the study by Espin et al (2.9% list growth vs. 1.5% net growth) [18] is not captured in this analysis. It is true, however, that list prices are also used for non-OMP expenditure, but it seems that the level of (confidential) discounting and rebates is higher for medicines used in hospitals rather than dispensed in pharmacies, and OMPs are usually used in hospital settings [18, 20]. Therefore, observed total expenditure will be overestimated within this study, especially for more recent years.…”

“…Fourth, the analyses are based on expenditure at list prices, which overestimates the value of the market. However, to our knowledge there is no information available comparing the level of discounting between OMPs and non-OMPs, although confidential discounts have been shown to be greater for medicines used in a hospital setting [18, 20].…”

A systematic review of moral reasons on orphan drug reimbursement

“…Since 2010, several country-specific population surveys assessing the societal preference for rarity were published. In contrast to the previously mentioned reports, they mostly concluded that there was no societal preference for rarity alone [ 24 , 26 , 27 , 35 , 38 , 39 , 42 – 46 , 63 , 73 , 75 , 78 – 80 , 87 , 92 , 93 , 99 , 111 , 112 , 114 , 116 , 132 , 151 , 157 , 161 , 167 , 173 , 182 , 198 , 205 , 208 , 212 , 213 , 217 , 252 – 254 ], but did find general preferences for considering equity in healthcare resource allocation, including preferential reimbursement for severe diseases without available alternatives [ 24 , 44 , 63 , 80 , 116 , 167 , 182 , 205 , 212 , 213 , 254 ]. Similarly, it was shown that healthcare professionals were not generally prioritizing rarity [ 43 , 182 ].…”

Moving somatic gene editing to the clinic: routes to market access and reimbursement in Europe

“…Access granted through exemption at the individual level (permission for one patient) National reimbursement at the discretion of health insurance company [ 19 ] The unmet medical need for an individual patient and availability of the product in another country, possibly for another indication [ 19 ] Tailored access for the patient with unmet medical needs to treatments for very rare conditions Permission is only granted on a case-to-case basis for one patient (labor-intensive/not suitable for large groups), differences in national regulations, not necessarily reimbursed (in the Netherlands). A supplying party is responsible for safety and follow-up [ 19 ] Temporary authorization of use (French ATU) Yescarta [ 30 ] National medicines agency [ 31 ] nATU: Requested by a physician (permission for one patient); cATU: Requested by drug manufacturer (permission for a (sub-)group of patients) [ 22 ] National French Ministry of Health and French National Health Insurance [ 20 ] Urgent unmet medical need, presumed efficacy, and safety based on scientific data [ 31 ] Access before market approval for patients with unmet medical needs, complete reimbursement, strong financial incentives for commercial companies [ 20 ] Under construction, different maturity levels in different legislations, and potentially large budget impact for health care systems IRB Milasen, Fanconi [ 23 , 24 ] Local (national) research board Researchers, often in collaboration with clinical experts Local Research funding Local (national) criteria for safe and responsible research Fast Only small-scale application, not sustainable and equitable Compounding CDCA (not SGT) [ 32 ] Hospital pharmacy; eventually National Health Care Institute [ 33 ] Treating physician Local Reimbursement at the discretion of health insurance companies; eventually uptake in national health care package may occur Production for individuals or very small groups of patients Medicines can be produced at a lower price than a commercial product Tension in regulatory framework (lack of precedents) Hospital exemption – …”

“…In France, the ATU system encompasses aspects of both compassionate use and named-patient use, but with a different system of reimbursement prior to market approval [ 20 ]. During the ATU validity period, products are entirely reimbursed by the French National Health Insurance, potentially having a large budget impact on the French healthcare system [ 21 ].…”