Asia’s orphan drug market has always been far behind that of Europe or the US. General lack of understanding of rare diseases, insufficient funding mechanisms and options, absence of legislative frameworks have all contributed to this fact. Asia’s orphan industry can overcome the stagnant stage it is in by putting in place a sustainable access program through improved healthcare policies to protect the rare disease patients, and also to incentivize orphan drug developers.
With minimal movements and the complications in Asia’s orphan drugs industry, it’s difficult to pin-point industry experts or innovators. However, there are certainly the superheroes behind the scenes who’ve been pushing for more patient rights protection, healthcare policy revision, and public awareness of rare diseases in their individual state or country.
This book has selected 10 inspirational figures who will further encourage industry peers to push for greater access of orphan drugs in Asia, and better coverage of drugs pricing and health insurance.
We hope these ideas and innovations inspire your business in 2015.
Soo Min LEE
World Orphan Drug Congress Asia 2015
1. Ms. Serena Wu and Dr. Min-Chieh Tseng, Founders of Taiwan Foundation for Rare Disorders (TFRD)
Apart from the sizable number of orphan drugs accessible in Taiwan, the country also has a very proactive patient support group—Taiwan Foundation for Rare Disorders (TFRD). Like most patient groups, the Foundation was founded by two parents, Ms. Serena Wu and Dr. Min-Chieh Tseng who had loving children fighting rare diseases.
Strongly believing that only through a sustainable “well-established system” can the patients be under good medical care, the two have been the voice of rare disease patients in Taiwan since 1999. The Foundation is also heavily involved in research and has promoted the Rare Disease Control and Orphan Drug Act. A fund worth NT$ 2.2 (approximately USD 66.3 mil) was raised by the Foundation for lobbying to protect the rare disease patients’ rights.
Their determination gathered power from the families of those who suffered similar causes and stunned the entire society with attention. They fought simply for the survival and well being of their beloved ones. TFRD, established in 1999, has been an active force in advocating for rare disease patients in respect of medication, education, and employment, and will continue to give its best effort to light up the lives of many rare disease patients.
2. Durhane Wong-Rieger, Chair, Consumer Advocare Network; President and CEO, Canadian Organization for Rare Disorders; President and CEO, The Institute of Optimizing Health Outcomes
Durhane serves on many committees which concern both patient/healthcare policy advocacy, and health technology assessment. As president of the Canadian Hemophilia Society, Durhane advocated on behalf of victims of tainted blood and named to the Board of Directors of the Canadian Blood Services and the National Blood Safety Council. Dr. Wong-Rieger has served on numerous health policy advisory committees and panels, including Project Coordinator for the Policy Dialogues for the Commission on the Future of Healthcare in
Canada and consultant to the Ontario Premier’s Advisory Board on Organ Donation. She is a member of Health Canada’s Expert Advisory Committee on Vigilance of Health Products and Expert Advisory Panel on Special Access Programme. Durhane has been actively involved in the APAC and Lat-Am market with her contributions to improving the health policies for the regional rare disease community. She is also one of the key people behind the establishment of Asia-Pacific Alliance for Rare Disease Organizations (APARDO).
3. Hyun-Min Shin, Chairman, Korean Organization of Rare Diseases (KORD)
Korea has a pool of roughly 500,000 patients suffering from over 2,000 different types of rare disease. Though a rare disease legislation and strong reimbursement scheme is yet to be introduced, the country has been very proactive to gain more attention on rare disease in general, gathering financial support for R&D of orphan drugs, and establishment of patient groups or other relevant rare disease organizations.
A key figure behind the public’s interest and support for the rare disease community is Hyun Min who leads the national patient support group, KORD. With his strong involvement in the parliament and persuasion, the government has opened up to the rare disease community and has begun setting a sustainable system to assist the care givers, patients, and also an improved healthcare policy for easier access of orphan drugs.
4. Kevin Huang, President, Chinese Organization for Rare Disorders
A national patient support group of China, CORD represents over 45 individual patient groups. After working with China-Dolls Center for Rare Disorders, Kevin found CORD to support a larger patient pool. Today, he strives to promote cooperation amongst the patients, medical community, pharmas and government agencies. To overcome the biggest hurdle (the physicians’ lack of technical understanding of rare diseases), he strives to provide platforms for the public and medical community to understand the disease and exchange clinical and genetic information. He is heavily involved with rare disease policies, establishing international consortiums, and engages with the Rare Disease Branch of Beijing Medical Society.
5. Grace Wang, Director, Drug Evaluation Division, Center for Certification & Evaluation, Shanghai Food & Drug Administration
The Chinese New Drug Registration Regulation, introduced in 1999, had urged the regulatory review process to be speeded up shall the new drug have clinical therapeutic value for “life-threatening or difficult critical illness (such as AIDS, cancer, rare diseases, etc.), and was made as the first domestic application”. The regulation has been continuously improved, to allow special review and approval of drug registration for treatments of rare diseases. Since 2010, SFDA has approved the import of a handful of orphan drugs (some conditionally).
Though the local government has yet to devise a sound legislative framework or set a clear definition of “orphan drug” or “rare disease”, the regulatory improvements prove that China is on its way to providing better healthcare policies for the rare disease community.
Grace looks over the Shanghai FDA’s drug evaluation. Shanghai, amongst the municipalities of China, has taken proactive measures to accelerate the orphan drug policy reform. A group of physicians have initiated research programs, and have been working closely with the government. We hope that these efforts and Grace’s passion for the orphan drug community will lead to better access of orphan drugs in Shanghai and China.
6. Cynthia Magdaraog, President, Philippine Society for Orphan Disorders
PSOD was one of Genzyme’s 2013 Patient Advocacy Leadership (PAL) Awards winners. Despite the low level of awareness and knowledge of rare diseases and lack of resources for the orphan drug community, PSOD delivers their empathetic message in the most creative ways. Through their PAL project, the Society created an animated film to reach out to a larger audience via social media.
Cynthia first found her roles within the Society thanks to her son, Dickoy, who suffers from Pompe disease. Though she is very appreciative of the sponsored trial or access of drugs, she is also well aware of the high prices of orphan drugs, which most of her local patients cannot afford. Together with her son, who is also a patient-advocate himself, she continues to protect the rights of the rare disease patients in the Philippines, and provide a cooperative environment for orphan drug developers.
7. Shu-Ti Chiou, Director-General, Committee for the Review of Rare Disease and Orphan Drugs, Maternal and Child Division & Health Promotion Administration, Ministry of Health and Welfare, Taiwan
The Committee for the Review of Rare Disease and Orphan Drugs was established for a more thorough evaluation & review process of orphan drugs in Taiwan, as an effort by the government to support the orphan drug committee. Under the guidance of Dr. Chiou, the government has specifically classified more than 160 diseases as a “rare disease”, allowing more patients to enjoy National Health Insurance. Dr. Chiou is also involved in the Asia-Pacific Economic Cooperation (APEC) and Health Working Group (HWG) Symposium. The Symposium provides a regional platform for cooperation in orphan drugs clinical studies, and encourages the APAC region to exchange ideas on of orphan drug policies.
8. Beverley Yamamoto, Professor of Education, Gender and Sexuality Studies in the Department of Advanced Human Sciences, Graduate School of Human Sciences, Osaka University, Japan & President, Hereditary Angioedema International (HAEi), Japan
Beverley has recently become engaged in the field of patient advocacy in Japan, taking on the role of International Liaison for Japan Angioedema Association (JAEA) from November 2012 to August, 2013. In this role, she was a key player in organizing two large national patient meetings in Japan in the spring of 2013 in collaboration with the Hereditary Angioedema International (HAEi). Following on from this, Beverley worked to establish a dedicated HAE patient advocacy organization, the Hereditary Angioedema Association Japan (HAEj) as a Non-Profit Organization. Upon her election as President of HAEj in 2013, she has worked in a very short time to establish HAEj as strong voice for HAE patient advocacy in Japan and beyond. While only involved in the field for two short years, Beverley is already a highly reputable figure in patient advocacy within Japan’s medical and rare disease field.
9. Duraiswamy Navaneetham, Founder and Director, Foundation for Research on Rare Diseases and Disorders, Chennai, India
Foundation for Research on Rare Diseases and Disorders (fRRDD) was established in 2010. As a scientist, Duraiswamy’s main objective was to develop rare disease treatments that are accessible to patients, family members and experts in India. Given the geographic constrains of India, discovering and reaching out to each individual patients are an extreme challenge. The foundation’s current works are focused on identifying rare disease patients and developing registries and rare disease specific literatures. The foundation has also been establishing bio-specimen repository that in turn is expected to spawn basic rare diseases research in India and her neighbors