Updated: Jan 15
In France, the alarm is being raised: our collective consent to pay for the funding of therapeutic innovation to defeat rare diseases is in danger. Christophe Duguet, representing the AFM-Téléthon [the muscular dystrophy patient association], discusses the urgency of “rebuilding the means of organizing this consent” not because the existing social security system would be unsatisfactory, but rather out of concern for its sustainability (Deleuze et al., 2016).
Today, all patients who need it have access to orphan drugs, without paying a charge. The social security system set up after the Second World War in France is a solidarity-based system – everyone participates in the social protection of all (in the form of contributions and taxes) and everyone benefits from social protection. Social security protects people who reside permanently in France from risks related to diseases (i.e. illness, maternity, disability, and death), in addition to risks related to occupational diseases and accidents at work, family (childhood and youth), and old age (retirement and widowhood). This system of protection is intended to be both egalitarian and equitable.
France is one of the few countries to have implemented a policy of temporary authorization for use (ATU) of innovative drugs – including orphan drugs – in order to provide early access. The ATU, granted at the request of a pharmaceutical laboratory or the attending physician, makes them available to the patients prior to the granting of a marketing authorization; the ATU can subsequently be temporarily extended (post-ATU) between the time a marketing authorization is granted and the outcome of negotiations on the price and the rate of reimbursement of the drug by the social security system, in order to ensure continuity of patient treatment. Drugs with an ATU (or post-ATU) status are also 100% covered by social security.
In his three proposals to “rebuild the means to organize this collective consent to pay,” Duguet calls for defending the specificities of the French healthcare system (the policy of early access to innovative treatments in particular) with regard to the responsibilities that Europe is being asked to take on; we must, Duguet says, avoid European harmonization on the lowest common denominator. The two other proposals underline the importance that French rare disease patient organizations attach to evidence-based activism (Rabeharisoa, Moreira & Akrich, 2014): sustaining a solidarity protection system requires a better understanding of the issues at stake and an effort to clarify the choices being made. He advocates the design of new knowledge tools “allowing to answer essential questions, such as the economic impact of rare diseases”, mentioning for example the creation of an Economic Observatory of Orphan Drugs. By calling for transparency in the pricing of innovative drugs, he recommends the construction of a new pricing model, with a view to achieving “fair and controlled prices.”
The extremely high price of drugs for rare diseases, and what would make these prices acceptable, are issues of international debate. When the health economist Catherine Le Galès writes that the acceptance of these prices “cannot be justified on the grounds that only the rarity of the disease counts”, it is to better underline the “multiple concerns mixing moral principles, issues of social justice and efficiency of public spending” that these prices rise, as much as the fact that the marketing of drugs for rare diseases are the result of “the embedding of scientific, industrial, financial, political and economic reasons” (Le Galès, 2019). What holds the attention in the way Duguet approaches the question is this invitation to consider the sustainability of a solidarity-based system of social protection, which in France is the main payer, from the point of view of the practical and concrete ways in which solidarity is actually exercised. Or more precisely, in which solidarity will be exercised in the future if the hope of developing new treatments for a growing number of rare diseases becomes a reality. This raises a series of very interesting questions for SPIN (social pharmaceutical innovation), starting with what is counted and what counts from the point of view of patients, manufacturers, and the community, as well as the innovation process itself and the future of social protection.
This raises a series of very interesting questions for SPIN (social pharmaceutical innovation), starting with what is counted and what counts from the point of view of patients, manufacturers, and the community, as well as the innovation process itself and the future of social protection.
Deleuze Christian, Detournay Bruno, Duguet Christophe, Handweiler Valérie (2016). Table ronde 4 - Réalité et pérennité du modèle économique des maladies rares. Médecine / Sciences, 32 (hors série n°1): 29-33.
Le Galès Catherine (2019). Quel prix pour les médicaments orphelins ? Global Média Santé, 4 (62): 61 à 69
Rabeharisoa Vololona, Moreira Tiago & Akrich Madeleine (2014). Evidence-based activism: Patients’ organisations, users’ and activists’ groups in knowledge society, BioSocieties, 9(2): 111–28.
Written by: Florence Paterson
Photo: Gunnar Ries, Health care is expensive, 2009 (CC BY-NC-ND 2.0)