Challenges and Recommendations
Challenges faced by Malaysia Rare Disease communities and Recommendations are authored by
Prof Thong Meow Keong and Dr. Azlina Ahmad-Annuar.
This content published as
Brief IDEAS No 10 –National Policy on Rare Diseases Living with Dignity: In Search of Solutions for Rare Diseases.
Prof Thong Meow Keong
Dr. Azlina Ahmad Annuar
(BSc Neuroscience – University College London; PhD Neurogenetics -Imperial College London) is a senior lecturer at the Department of Biomedical Science, Faculty of Medicine, University of Malaya. Her research interests are on the neurosciences and the genetics of neurological diseases, some of which fall within the rare disease category. She runs a number of community-based activities like the annual Brain Awareness Week, and the Jeans for Genes Malaysia campaign which works closely with patient support groups to help raise awareness about genetic disorders.
References
Cremers, Serge, and Jeffrey K. Aronson. 2017. “Drugs for Rare Disorders.” British Journal of Clinical Pharmacology 83, no. 8. https://doi.org/10.1111/bcp.13331
EURORDIS. 2009. “What is a Rare Disease?” Accessed April 8, 2018. http://www.eurordis.org/content/what-ra-re-disease. Accessed April 8th 2018.
Gammie,Todd, ChristineY. Lu, and Zaheer Ud-Din Babar.“Access to Orphan Drugs:A Comprehensive Re- view of Legislations, Regulations and Policies in 35 Countries.” PLoS ONE 10. https://doi.org/10.1371/journa.pone.0140002
Hunter, Nina L., Gayatri R. Rao and Rachel E. Sherman. 2017.“Flexibility in the FDA Approach to Orphan Drug Development.” Nat. Rev. Drug Discov. 16, 737-738. https://doi.org/10.1038/nrd.2017.151
Malaysia Rare Disorders Society, and Malaysian Lysosomal Diseases Association and Malaysian Metabolic Soci- ety.“Conference Report on Developing Strategies for a National Rare Disease Plan.” Second Malaysia Confer- ence on Rare Disorders. October 25-26, 2013.
Ministry of Health Malaysia. 2016.“National Strategic Plan for Non-Communicable Disease (NSP-NCD) 2016- 2015.” http://www.moh.gov.my/english.php/pages/view/698
CremeMinistry of Health Malaysia. 2017.“HealthTechnology Assessment: Enzyme ReplacementTherapy for Metabolic Diseases.” http://www.moh.gov.my/update2017/1466.pdf
NSTTeam. 2017.“RM27 Billion to Further Enhance Quality of Health Services.”The New StraitsTimes, 27th Oct 2017. https://www.nst.com.my/news/nation/2017/10/295840/rm27-billion-further-enhance-quali- ty-health-services.
Pharmaceutical Research and Manufacturers of America (PhRMA). 2015. “2005 – 2015: A Decade of Innovation in Rare Diseases.” https://www.phrma.org/report/2005-2015-a-decade-of-innovation-in-rare-diseases
Right Diagnosis from Healthgrades. 2015.“Statistics by Country for Rare Diseases.”Accessed 23 September 2018. http://www.rightdiagnosis.com/r/rare_diseases/stats-country.htm.
Shafie,Arsul Akmal,Azuwana Supian, andThong Meow Keong.“Taking Stock of Rare Disease.”The Star, March 1, 2017. https://www.thestar.com.my/opinion/letters/2018/03/01/taking-stock-of-rare-disease/