Launch of the 2023 Campaign on Rare Diseases

31 January 2023

Press Release
31 March 2023

Launch of the 2023 Campaign on Rare Diseases

An 18-year-old Maltese man with a rare condition in his eyes (LHON disease) has been accepted to take part in a clinical trial with 9 other patients in a hospital in Hubei Province in China.
This could be done because of an agreement that the National Alliance for the Support of Rare Diseases signed with the Chinese authorities with the help of the Chinese Embassy in Malta.
This was announced by Mrs. Michelle Muscat, President of the National Alliance for the Support of Rare Diseases, at the launch of the Annual Campaign for Rare Disease Awareness for this year, in the Parliament building.

Mrs. Muscat said that while no one should be filled with too much hope, this step is offering a glimpse of hope where there was no hope. This clinical trial includes the administration of a drug whose market price is approximately $850,000. The medicine will be given free of charge by the Chinese authorities to the Maltese patient through the agreement signed by the National Alliance for the Support of Rare Diseases.

Through this same agreement, therapy will be provided to patients who are members of the Alliance at the Mediterranean Centre for Traditional Chinese Medicine in Kordin, with a group of 24 people starting in the coming weeks.

Mrs. Muscat said that this year’s Rare Disease Awareness campaign will focus mostly on schools to convey information about these conditions and show the value of careers in medicine, science and research. To do this, the first book on rare diseases aimed at primary school students will also be launched.

The Alliance, Mrs. Muscat continued, is pleased to note that in recent months the Government has started to take action and hopes that the NGO is invited in discussions on a National strategy on rare diseases. The Alliance looks forward to sitting around a table with the Government to discuss all this.
“There is a need for a strategy at both Maltese and European level, which serves to ensure that patients have access to the best treatment, care and research. At the same time, there is still a need for more awareness among those who make decisions about the importance of a holistic attitude, which can be measured according to the Sustainable Development Goals of the United Nations”, emphasised Mrs. Muscat.

The speaker of the House Dr. Anglu Farrugia, Prof. Alex Felice as well as Dr. Robert Xuereb addressed the press conference.

FACTS

In Malta, it is estimated that there are 27,000 individuals living with a rare disease.
The National Alliance for Rare Diseases Support Malta is a member of EURORDIS, of Rare Diseases International and a member of the NGO Committee on Rare Diseases at the Economic and Social Council (ECOSOC) of the United Nations.

STATISTICS as at 2023

The Malta National Register for Rare Diseases lists approx 6k patients suffering from a rare disease. However, it is estimated that there are approx 27,000 people suffering from a rare disease/condition.

EU statistics

•30,000,000 EU CITIZENS LIVING WITH A RARE DISEASE
•6,000 DIAGNOSED RARE DISEASES
•1 in 2,000 GET A RARE Disease

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Stqarrija ghall-istampa

Tnedija tal-Kampanja Annwali għal Kuxjenza dwar il-Mard Rari għall-2023.

Żagħżugħ Malti ta’ 18 il-sena b’kundizzjoni rari f’għajnejh ġie aċċettat biex jieħu sehem fi clinical trial ma’ 9 pazjenti oħra fi sptar fil-Provinċja ta’ Hubei fic-Ċina. Dan seta jsir riżultat ta’ ftehim li l-Alleanza Nazzjonali għas-Sapport tal-Mard Rari ffirmat mal-awtoritajiet Ċiniżi bl-għajnuna tal-Ambaxxata Ċiniza f’Malta.

Dan tħabbar mis-sinjura Michelle Muscat, President u Fundatrici tal-Alleanza Nazzjonali għas-Sapport tal-Mard Rari, fit-tnedija tal-Kampanja Annwali għal Kuxjenza dwar il-Mard Rari għal din is-sena, fil-bini tal-Parlament.

Is-sinjura Muscat qalet li fil-waqt li ħadd m’għandu jimtela b’tama żejda, dan il-pass qed joffri xaqq ta’ tama fejn ma kienx hemm tama. Dan il-clinical trial jinkludi l-għoti ta’ mediċina li l-prezz tas-suq tagħha huwa ta’ madwar $850,000. Il-mediċina se tingħata b’xejn mill-awtoritajiet Ċiniżi lill-pazjent Malti permezz tal-ftehim li ffirmat l-Alleanza Nazzjonali għas-Sapport tal-Mard Rari.

Permezz ta’ dan l-istess ftehim se tkun qed tigi pprovduta terapija lil pazjenti membri tal-Allenaza fiċ-Ċentru Mediterranju għall-Mediċina Tradizzjonali Ċiniza f’Kordin, bi grupp ta’ 24 persuna jibdew minn dan ix-xahar.

Is-sinjura Muscat qalet li l-kampanja għal Kuxjenza dwar il-Mard Rari ta’ din is-sena se tiffoka l-aktar fuq l-iskejjel biex titwassal informazzjoni dwar dawn il-kundizzjonijiet u jintwera s-siwi ta’ karrieri fil-mediċina, xjenza u riċerka. Biex isir dan se jkun qed jitnieda wkoll l-ewwel ktieb dwar mard rari mmirat lejn studenti tal-iskejjel primarji. Se jibda wkoll xoghol fost studenti universitarji fil-Fakulta tal-Medicina.

L-Alleanza, komplit is-sinjura Muscat, tieħu pjaċir tinnota li fl-aħħar xhur il-Gvern qisu beda jieħu azzjoni u qed juri interess biex jiddiskuti strateġija Nazzjonali dwar il-mard rari. L-Allenza tħares ‘il quddiem li tkun mistiedna tpoġġi madwar mejda mal-Gvern biex ikun diskuss dan kollu.

‘Hemm bżonn strateġija kemm fuq livell Malti kif ukoll fuq livell Ewropew, li sservi biex il-pazjenti jkollhom aċċess għall-aħjar trattament, kura u riċerka. Fl-istess hin għad hemm bżonn ta’ aktar għarfien fost min jieħu deċiżjonijiet dwar l-importanza ta’ attitudni ħolistika, li tista’ tkun imkejjla skont is-Sustainable Development Goals tal-Ġnus Magħquda’, sahqet is-sinjura Muscat.

Il-konferenza giet indirizzatha mill-iSpeaker tal-Kamra Dr. Angelo Farrugia fost ohrajn.

FATTI

F’Malta, huwa stmat li hawn 27,000 individwu li jgħix b’marda rari. L-Alleanza Nazzjonali għall-Appoġġ għall- Mard Rari Malta hija membru tal-EURORDIS, ta’ Rare Diseases International u membru tal-Kumitat tal-NGOs dwar il-Mard Rari fil-Kunsill Ekonomiku u Soċjali (ECOSOC) tan-Nazzjonijiet Uniti.

STATISTIKA fl-2023

Ir-Reġistru Nazzjonali Malti għall-Mard Rari jelenka madwar 6.000 pazjenti li jbatu minn marda rari. Madankollu, huwa stmat li hemm madwar 27,000 persuna li jbatu minn marda/kundizzjoni rari.