In Europe, rare disease affects less than one in 2,000.
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“Thankfully for rare disease patients in Canada and around the world, many companies like those that are members of BIOTECanada are developing breakthrough medicines and therapies to address the unique medical needs associated with rare disorders”.
Minister Hoskins mentioned his commitment to establish a rare disease working group, which will include patient leaders and clinicians, surrounded by patients and caregivers during a luncheon hosted today by CORD at Queen’s Park. Thus, between 5,000 and 7,000 distinct rare diseases exist [1]. “Rare diseases have traditionally been overlooked and many patients do not receive adequate treatment or support”. The slogan for this year’s Rare Disease Day is “Join us in making the voice of rare diseases heard”.
We do not know the full extent of rare disease incidence and prevalence in Malaysia yet, but we can expect to have the same global trend of increasing rare disease incidences.
Michael Eygenraam, a patient living with atypical hemolytical uremic syndrome (aHUS), expressed similar hope, stating that “as rare disease patients, we have individual needs but we also share a common commitment to a common good”.
The IOF Working Group on Skeletal Rare Diseases recently published a comprehensive taxonomy of the known rare metabolic skeletal diseases.
Doctors examine a patient who is in a serious condition. Overall, almost half (47 percent) of all of the drugs approved in 2015 were orphan-designated drugs for rare diseases.
He stated that the urgency of the patients’ conditions inspires them to work harder and more efficiently in order to create medical breakthroughs in fields in which patients didn’t even expect them to. Submit photos to the Handprints Across America Gallery to show people acknowledging Rare Disease Day all across the United States in a variety of settings.
This is an example of the important role that smart public policy can play in supporting innovation and incentivizing the enormous amount of private investment that is required to get a new drug or therapy to patients.
There is also a lack of options (if any) and access to its medicines – hence the medicines that are used to treat a rare disease are also called orphan drugs. Learn how you can help support someone’s network of care by starting a conversation around how you could possibly help alleviate pressure.
In addition to the absence of an official definition for rare diseases in Malaysia, there is also no specific legislation of rare diseases and orphan drugs designation.
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Most rare diseases now don’t have treatment, and a lot of them have no research at all being done. All responsive applications will be reviewed and evaluated for scientific and technical merit by a panel of rare disease and natural history experts. With most focus and funding going to diseases that impact many people, rare disease research is considered to be a lower priority.
