The International Association of Relatives and Affected of Lipodystrophies AELIP, has presented a motion addressed to the councilor of health of the municipality of Totana urging the Ministry of Health to ensure the supply and access in equity to metreleptin by the National Health System.
Lipodystrophy is an ultra-rare and chronic disease, composed of a set of congenital or acquired disorders associated with the loss of adipose tissue.
A classification can be made according to its etiology, being congenital (genetic) or acquired, and according to its anatomical location, being able to be generalized, partial or localized.
Lipodystrophy is an ultra-rare disease with a worldwide prevalence of 0.2-1.0 cases / million for the generalized form and 1.7-2.8 cases / million for the partial form (Chiquette 2017).
In the only Spanish Reference Center for Lipodystrophies, 99 patients with lipodystrophy have been detected, with 19 patients with generalized lipodystrophy and 80 patients with partial lipodystrophy (Data Dr. Araujo-Vilar 2018).
The lack of subcutaneous adipose tissue causes a decrease in the level of leptin in the body and the ability to store lipids, so they accumulate abnormally in other organs.
Lack of leptin causes the early development of serious metabolic disorders such as insulin resistance (diabetes), hypertriglyceridemia, hepatic steatosis, pancreatitis, renal and cardiovascular disorders, etc.
that lead to a deteriorated quality of life and an early death (Brown 2016, Araujo-Vilar 2018).
The average age of death in lipodystrophic patients is lower than the general population, being 12 years for patients with generalized congenital lipodystrophy, 32 years for generalized acquired lipodystrophy, 28 years for partial family lipodystrophy and 23 for partial acquired lipodystrophy (Gupta 2017 ).
The therapeutic approach of lipodystrophies has a double aspect, on the one hand, the prevention and treatment of associated metabolic and cardiovascular disorders, and on the other, the cosmetic treatment produced by the loss and / or accumulation of adipose tissue.
Metreleptin (Myalepta) is an orphan drug.
It is the first and only authorized drug in Europe for the treatment of lipodystrophies since July 2018.
Metreleptin is a recombinant human leptin analogue and is indicated, together with diet, as a restorative treatment to treat complications derived from a leptin deficit in patients with:
- Acquired generalized lipodystrophy (Lawrence syndrome) or generalized congenital lipodystrophy (Beradinelli-Seip syndrome), confirmed in adults and children ≥ 2 years.
- Family partial lipodystrophy or partial acquired lipodystrophy (Barraquer Simons syndrome) confirmed in adults and children aged ≥ 12 years for whom the usual treatments have not achieved adequate metabolic control.
The Spanish Federation of Rare Diseases (ERDF) has urgently demanded a response to the serious situation suffered by many people with rare diseases regarding equity access to Orphan Medicines (MMHH).
These medications represent a unique opportunity for patients who do not usually have any therapeutic alternative or whose classic treatment might be of little use (FEDER News Portal: https://enfermedades-raras.org/index.php?option=com_content&view=article&id=2791&Itemid = 999)
From FEDER, it was denounced that at present there are significant delays in the access of those affected to MMHH due to the lack of specific and additional financing for the hospitals that must start each treatment and bear the high cost of these medicines.
The public funding model for orphan drugs currently in force in Spain is a decentralized management model where authorization decisions correspond to the Spanish Agency for Medicines and Health Products (AEMPS) and financing decisions fall on the Ministry of Health , Social Services and Equality (MSSSI) (more specifically, about the Pharmacy department).
After this, the price is set by the Interministerial Commission for Drug Prices (CIPM) and the orphan drug is made available to the health services of the different autonomous communities, assuming responsibility for its management and rational use (Portal of FEDER News: https://enfermedades-raras.org/index.php/actualidad/3717-feder-participara-en-un-congreso-de-medicamentos-huerfanos-2)
According to the Spanish Association of Laboratories for Orphan and Ultra-Orphan Medicines (AELMHU), only 54% of MMHH with a marketing authorization from the European Union, are finally marketed in Spain, that is, 58 of 108 (News Portal: https: //aelmhu.es/index.php/actividades/eventos-aelmhu/item/909-aelmhu-presenta-los-datos-de-acceso-a-los-medicamentos-huerfanos-en-espana).
To this is added that the time from the allocation of the National Code to the commercialization was 13.4 months, during the period 2002-2017 (Mestre-Ferrándiz 2019).
And according to AELMHU data, the time elapsed from the assignment of the National Code to the effective commercialization has worsened by 3.8 months more (average) and 3.5 months more (median), compared to the 2018 data (Portal de News: https://aelmhu.es/index.php/actividades/eventos-aelmhu/item/909-aelmhu-presenta-los-datos-de-acceso-a-los-medicamentos-huerfanos-en-espana).
Therefore, it is urged that access to this orphan medicinal product be guaranteed under conditions of fairness for an ultra rare disease such as this.
Through this petition, AELIP requests: Urge the Ministry of Health to expedite the necessary procedures to guarantee the provision by the National Health System of the only treatment authorized in Europe for the treatment of lipodystrophy, metreleptin, guaranteeing access in equity and equality to all patients who need it in the different autonomous communities of Spain.
