11 August 2017
Luxembourg makes steps towards a national plan on rare diseases
In Luxembourg, it has been estimated that 30,000 people are affected by a rare disease. Every rare disease is unique, however, most patients face the same difficulties: lack of diagnosis, limited knowledge of rare diseases, few available therapies, inadequate care, and expensive treatments.
On 16 June, the first information meeting took place at the Health Ministry to show the progress on the rare diseases national plan in Luxembourg. According to a press release from Luxembourg Health Ministry, "The national plan on rare diseases will follow aimed to provide a fair access to diagnosis, and to give access to appropriate care, as well as medical care, psychosocial care, access to health insurance, but also the possibility to satisfy the needs and make progress towards the professional and personal self-fulfilment". This plan will be carried out in collaboration with national and international organisations. To elaborate the plan, a specific committee was established last February, chaired by Dr Jos Even, Vice-President of the Luxembourg’s Association for people suffering from neuromuscular and rare diseases (ALAN).
The plan will be structured around 4 major axes and span over 5 years (2018-2022):
- Care system and patients’ care for those suffering from rare diseases;
- Creation of an information website;
- Codification and registration of rare disease patients as well as research on rare diseases;
- Social services.
The plan will also reflect the EUROPLAN (2012-2015) recommendations. EUROPLAN was a European project that supported the development of national plans on rare diseases, as recommended by the 2009 European Council Recommendation in the field of rare diseases. This plan shall be written and submitted to the Health Ministry Officer by the end of 2017.
Spotlight On
Establishing rarity in the context of orphan medicinal product designation in the European Union
In this study published in Drug Discovery Today, concerns about orphan medicinal product designation framework have been raised related to a prerequisite for applications based on rarity. Authors have identified three important factors relating to prevalence calculations when making an application for orphan drug designation in the European Union. The authors have illustrated this study with reference to recent applications to the European Medicines Agency (EMA), and as such can act as a useful guide for making a successful submission.
The three major issues that have been highlighted are:
- The discernment between diagnosed and undiagnosed cases;
- The duration of the disease;
- The need for a more accurate conclusion.
They also acknowledge that the Committee for Orphan Medical Products within the EMA purses independent deliberation as well as assessing prevalence indicators.
In this article, authors have expressed their personal views and may not be quoted on behalf of or reflecting the position of the European Medicines Agency or working parties/committees.
Orphanet News
List of rare diseases and synonyms in alphabetical order
The new report of rare diseases and synonyms in alphabetical order is now available online.
Prevalence and incidence of rare diseases: Bibliographic data N°1 & 2
The new reports on prevalence and incidence of rare diseases from June 2017 are now available online.