On August 23, Rare Disease and Orphan Drugs Journal (RDODJ) was delighted to have a special interview with Director of the Chinese Organization for Rare Disorders (CORD), Kevin Huang.
The following are the details of the interview:
RDODJ: We know that you are the founder of the CORD. You have been promoting the improvement of medical insurance for rare disease patients in China and have achieved excellent results. What inspired you to engage in charity for rare diseases? What beliefs have sustained you so far?
Mr. Huang: I have been engaged in rare disease work since 2008, at first because I like to participate in public welfare activities and pay attention to the latest developments in some specific areas. But then I noticed that no more people in China pay attention to the rare disease group. I am a part of the rare disease group with Pseudoachondroplasia (PSACH). Therefore, I feel real empathy for patients with rare diseases who need understanding and respect, as well as equal rights in health care, education, employment, etc. This inspires me to pursue a career in rare diseases. Later I met more and more rare disease patients who needed attention and help. This started me thinking about how to help them and what the solution is for dealing with rare diseases. Then, I began to sort out the relevant information around the world and finally decided to establish a patient organization covering all rare diseases. Some ordinary people may give up on a particular research project, research field, or disease for some reason, but our rare disease patients never give up. That's why I've been sticking with it until now.
RDODJ: What do you think are the most pressing issues encountered by patients with rare diseases in China?
Mr. Huang: I think the diagnosis of rare diseases and the availability of medicines are urgent problems that need to be solved. First, due to the country’s massive population, wide distribution of patient groups, complicated disease profiles and imbalance of medical resources in China, many patients cannot achieve a timely diagnosis. Second, there is no effective clinical drug use. More than 90% of rare diseases worldwide still don't have effective therapies, and 60% of the world's orphan drugs are currently unavailable in China, which is another hardship for Chinese patients. Third, even if these orphan drugs are imported, their sky-high prices are unaffordable for the patient's family. Furthermore, other difficulties include social integration, education, employment, etc.
RDODJ: How do you promote research and development of orphandrugs in China?
Mr. Huang: We have to improve drug accessibility and affordability. First, we have been working with many stakeholders to perfect the relevant national regulatory policies and support foreign drugmakers applying for market approval. Now we have witnessed some progress in this endeavor. Second, we need to promote the inclusion of orphan drugs in the medical insurance program for significant reimbursement to ease the financial burden on patients. In the past, we invested less in drug research and development, so this year, we have set up the HOPE FOR RARE FOUNDATION to promote rare disease research and translational medicine, which will fund more research and translational projects in the future.
RDODJ: These past years have seen tremendous developments in national policies, medical solutions and social awareness of rare diseases in China, but many countries, such as the United States and other European countries, have paid attention to rare diseases much earlier. What are the current gaps between China and these countries?
Mr. Huang: The main gaps lie in three aspects. First, the basic research in China is relatively weak, while the basic research and clinical trials in the United States and other European countries are under active development. Second, these countries have a relatively complete set of policies on medication, security, scientific research, and payment, while our country has been catching up and updating policies. Also, China has not effectively solved the payment problems for rare diseases at present. Thus, the development of marketization and industrialization is severely restricted, which may discourage pharmaceutical manufacturers.
RDODJ: As the organizer of the China Rare Disease Summit Forum, what are the highlights of the 11th conference compared with previous ones?
Mr. Huang: At present, the Rare Disease Summit Forum is the largest and most influential comprehensive forum in the field of rare diseases in China. It has played a very active role inpromoting the development of the field, the improvement of policies, and the communication of various stakeholders. Small communities of rare disease patients need government support. Whether they are affected by the COVID-19 pandemic is a relatively big challenge at present. The 11th conference will focus on the policy of payment, exploring more about how orphan drugs are effectively paid for in China and how patients can get affordable medication.
RDODJ: RDODJ aims to provide a forum for scientific studies and discussion covering the important regulatory, socio-economic and human science issues related to rare diseases and orphan drugs. As a seniorrare disease practitioner, do you think there are some new forms of collaboration that can better facilitate academic research on rare diseases and meet the needs of patients?
Mr. Huang: Rare diseases and rare disease patients need more platforms, whether academic or patient service platforms. I am delighted with the launch of the new journal RDODJ. This means that more and more academic publishing companies have paid attention to the field of rare diseases. As a platform that has been growing for nearly ten years, CORD is very willing to have the opportunity to cooperate with RDODJ in the future, especially in academic forums and conferences. It will make sense to combine patient needs with medical research through constant communication. Therefore, we can co-host some academic conferences to help both sides combine academic research with patient reality to promote progress in this field. Looking forward to more collaborations with RDODJ in the future.
Kevin Huang is the founder of CORD, the largest social organization for rare diseases in China. He has been committed to promoting social and public understanding and respect for patients with rare diseases, eliminating discrimination against them, and safeguarding the rights and interests of the group in medical care, education, employment, etc.
It is a great honor to have this opportunity to talk with Mr. Huang about the current situation of rare diseases in China. In the future, RDODJ will continue to pay attention to the research progress of rare diseases in China and promote international academic exchanges in this field.
Respectfully Submitted by the Editorial Office
Rare Disease and Orphan Drugs Journal