The French Orphan

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This sounds like an intense and gripping series. I'd not read Michael Stolle's before but look forward to reading this series. Carol L Lucky at aol dot com. After all, as a teenage orphan living in a monastery school in Reims, all you have to worry about is dodging the unpleasant advances of a few unsavoury monks and looking forward to a life of penniless and celibate servitude in a religious order.

After a childhood and adolescence plagued by a constant longing to know who he really is, orphan Pierre has not the slightest idea that his questions are about to be answered.

But you know what they say — be careful what you wish for… Suddenly finding out who you are can bring with it not only happiness and fortune, but danger, friendship and the sort of swift education that the monastery could never have provided! The discovery of who Pierre really is affects not only Pierre and his friends, but has ramifications for the French nobility, the English crown, and most dangerous of all, the machinations of Cardinal Richelieu and his fierce ambition for the Church and for himself.

As healthcare organization and drug reimbursement by a national health insurance scheme are within the purview of each Member State, these four dimensions should be analyzed at national policy level. In this context, an assessment of the four dimensions determining effective access to orphan drugs after MA was conducted in France. All extractions were performed in August In previous studies, accessibility was measured either by market access [ 2 ] or by uninterrupted sales within a 1-year period [ 3 ], without specifically identifying the impact of reimbursement and healthcare organization on patient access to orphan drugs.

In this study, the assessment of effective access of French patients to orphan drugs is based on four factors: marketing, ease of supply, financial and time-based dimensions. In order to prevent delays in drug access, compassionate use of an orphan drug through the French temporary licensing system — temporary authorization for use, ATU is possible before the MA is granted, but was not taken into account in our study. Once the MA is obtained, drugs that previously received an ATU can benefit from a transitional provision so called post-ATU that continues to provide access after the MA until the ministerial decision on reimbursement and pricing has been made.

During the post-ATU period, access is granted both for inpatients and outpatients, but only from hospital pharmacies. Orphan drugs in the post-ATU period were included in our study. Number of assessed orphan drug—therapeutic indication pairs i.

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Among the 94 pairs with a published HAS opinion, only 4 had a negative opinion for reimbursement, based on an insufficient actual clinical benefit. An additional health economic assessment was required for 12 orphan drug—therapeutic indication pairs. Overall, The median period between the granting of the European MA and French access to the reimbursed drug market i. Compassionate use i. For the last two, pricing negotiation may be ongoing at the time of the study;. The use of orphan drug—therapeutic indication pairs was regulated: Prescriptions were restricted to some specialist doctors for Orphan drugs were mostly managed in hospitals: The retrocession mechanism provided outpatients with access to The broadest access i.

No financial contribution for orphan drugs was requested for inpatients. However, Orphan drug status has encouraged pharmaceutical investment and hence market access for treating rare and serious diseases [ 10 ]. In this manner, the European policy on orphan drugs has fulfilled its objective by making these diseases more attractive from a commercial point of view. However, the main purpose of this policy is to address highly unmet medical needs. Our results show that As we focused on registered orphan drugs, our study underestimates the real access to drugs for rare diseases in France.

First, drugs registered in rare diseases but that were not designated as orphan drugs were excluded. Then we also excluded drugs with an orphan designation for which the MA application was still in progress, even if they were available through the compassionate use provision i. ATU , which is known to accelerate drug access for patients [ 12 ]. Lastly, drugs already marketed for other conditions and available for rare diseases through the temporary recommendation for use RTU provision were not taken into account. While these drugs can be used for off-label indications, their use is accompanied by strict data collection requirements.

The final goal of this RTU provision is to expand the approved therapeutic indications. At the time of our study, Several factors can explain this difference: 1 our study is more recent and more exhaustive; 2 we studied orphan drug—therapeutic indication pairs because reimbursement is granted by therapeutic indication and we hypothesized that reimbursement has a big impact on marketing. A total of However, there is a bias between our theoretical approach and reality: when reimbursement is given for one therapeutic indication, the marketed drug can be used in another non-reimbursed indication based on medical argument for hospital delivery circuits or because validation of the indication is not always required for the community delivery circuit.

The negotiation phase for orphan drug prices could be shorter for two reasons. First, the high clinical added value ensures the price is consistent with European prices. So access to reimbursement and pricing did not appear to be slowed by the difficulties inherent to the scarcity of therapeutic indications in France. These prescribing and dispensing requirements promote the proper use of orphan drugs and careful patient monitoring, while helping regulate healthcare expenditures [ 15 ].

It can be noticed orphan drugs are four times more likely to be provided through retrocession than all the brand-name drugs marketed in France i. This restrictive distribution circuit allows special monitoring of prescriptions and deliveries [ 18 ]. Yet better safety should be balanced with restrictions of patients accessibility: according to statistics of the French chamber of pharmacists, there are 21, community pharmacies in France, and only hospital pharmacies [ 19 ].

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It should be noticed that At the end of this transitional period, some of them may be delivered by community pharmacies. For inpatients, funding was either universal in connection with Diagnosis related groups , or related to extra funding granted to hospitals for high-cost drugs in return for appropriate use, or specific to the post-ATU transitional provision.

For outpatients, when there was a theoretical excess to be paid by patients, the actual reimbursement rate could not be determined. According to our study, First because access to drug reimbursement is needed for marketing: a negative HAS opinion followed by a negative ministerial decision for reimbursement always result in no marketing of the orphan drug.

Hence, the first step for patient access is demonstrating a clinical benefit, which is a mandatory criteria for reimbursement in France. On the other hand, the severity of the disease and the lack of satisfactory therapeutic alternatives are elements in favor of orphan drugs. For the 4 orphan drug-therapeutic indications pair concerned in this study, the HAS justified the insufficient actual benefit by a level of evidence considered as too low [ 25 — 28 ].

Then, the second impact of health technology assessment on drug availability is time-based: our study was cross-sectional, but assessment and price negotiation delays must be considered. And lastly, as marketing is a function of business, strategic and commercial choices, pharmaceutical companies can decide not to market a drug if the negotiated prices are not acceptable to them [ 29 ]. In general, high levels of clinical added value contribute to better pricing.


Orphan drugs are more likely to obtain major or important clinical added value: this proportion is Moreover, payers value rarity in pricing decisions [ 31 ]. In our study, most orphan drug—therapeutic indication pairs available to outpatients were fully reimbursed since these orphan drugs were judged irreplaceable and particularly expensive treatments. In France, patients do not seem to be limited by the price of orphan drugs.

But the growing budgetary impact of orphan drugs is worrisome from a societal point of view [ 32 , 33 ]. Drugs claiming a major, important or moderate clinical added value i.

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We found that 12 orphan drug—therapeutic indication pairs were slated for a health economic assessment. As a reference, 21 and 23 drugs qualified for a health economic assessment in and , respectively, all drugs pooled. Major methodological limitations appeared in two efficiency opinions out of the three adopted for orphan drugs. A number of researches have suggested that the commonly used health technology assessment methods are poorly suited to rare diseases: it is well known that orphan drugs are usually cost-ineffective, yet restrictions in funding may not be in line with societal preferences [ 36 , 37 ].

Other studies aimed to establish societal preferences for funding drugs used to treat orphan diseases, but they have limitations and their results do not show a clear societal preference for prioritizing the treatment of rare diseases [ 38 , 39 ].

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Moreover, equity and ethical questions could influence decision makers [ 40 ]. Relationships between level of evidence, effect size, economic logic, ethics and equity are difficult to grasp [ 41 ]. France, like many countries, has implemented legislation to support the market access for orphan drugs [ 42 , 43 ]. However, regulation is essential to ensure the sustainability of orphan drug coverage [ 44 , 45 ], both at the European level and the Member State level. At the European level, facing the growing number of orphan drugs approved [ 10 ], the EMA has started publishing orphan maintenance assessment reports in January [ 47 ].

The objective is to assess whether a drug fulfils the criteria for orphan designation at the time of the MA to reconfirm the eligibility of the status. At the Member State level, criteria chosen for health technology assessments, combined with setting up of specific policies on orphan drugs, resulted in heterogeneity between European countries in patient accessibility to these drugs [ 48 ]. Several potential solutions exist to cover the gap between European drug market access and national patient access to licensed drugs. These include early contact between the pharmaceutical companies and the health technology assessment agencies and collaboration between the EMA and the national health technologies assessment agencies [ 49 , 50 ].

In conclusion, access to reimbursement and prices negotiation have a strong impact on effective patient access to orphan drugs in France. Prescribing restrictions and drug delivery systems influence the ease of patient supply for reimbursed orphan drugs. Patient access to orphan drugs remains incomplete and raises many questions. What is the collective interest of implementing incentives to develop and register these drugs if they are not accessible to all patients?

On one hand, this overview revealed difficulties in evaluating the reimbursement and pricing of orphan drugs. More transparency is needed to reduce the uncertainty for pharmaceutical companies about access to orphan drug reimbursement [ 52 ] to maintain their interest in rare and serious conditions. On the other hand, the orphan drug policy was marked by years without incentives followed by years with strong incentives.

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We believe the time has come to find a compromise in the form of reasonable and controlled incentives. European Union. European Organization for Rare Diseases. Marketing Authorization. Quality-Adjusted Life Year.

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Joanne Archambault, PhD for English editing assistance, and the anonymous reviewers for their comments and suggestions. MB analyzed and interpreted the data. All authors read and approved the final manuscript. The datasets analyzed for the current study are available from the corresponding author on reasonable request. Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations. Research Open Access. Patient access to orphan drugs in France. Orphanet Journal of Rare Diseases 14 Abstract Background Since incentives were introduced to promote orphan drugs in Europe, several dozens of drugs have been registered at the European level.

Results We identified 91 registered orphan drugs in Europe, corresponding to orphan drug—therapeutic indication pairs. Conclusions Patient access to orphan drugs is not universal in France.

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