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Effective engagement—from research to reimbursement—is key.
Patient advocacy groups (PAGs) are becoming increasingly visible across Asia. China, for example, has seen PAGs mushroom in recent years, ranging from umbrella organizations like the China Organization for Rare Disorders (CORD) to disease-specific PAGs such as HDCare for Huntington’s, Meier for spinal muscular atrophy, and Debra Butterfly Care for epidermolysis bullosa alike.
Japan, Korea, and southeast Asia have also seen vibrant development of PAGs, with some striving to spread beyond national boundaries. For example, Rainbow Across Borders (RAB Network) is an umbrella alliance active across Asia Pacific and beyond. Today, it has more than 40 affiliates in countries such as Thailand, Vietnam, Iran, the US, and the UK. It promotes collaboration and networking among these affiliates through various initiatives such as training, experience exchanges and disease registries across Asia and the rest of the world.
Asian PAGs are also identifying more areas to play their role. Some Asian PAGs started out as peer support and patient education organizations but have since evolved to fund research. For example, CORD set up a dedicated fund to support translational medicines and impact investments on mucolipidosis, epileptic encephalopathy, and other rare recessive diseases caused by Twinkle gene mutations among others. These conditions represent significant unmet needs and promising areas for research.
In addition, many PAGs proactively support patient recruitment for clinical trials and collect real-world data on the natural history of enrolled patients, which can be used to obtain regulatory approval for innovative therapies. For example, the Japan Patients Association (JPA) established J-RARE as a patient registry to support research and development of therapies for rare and intractable diseases. Based on the patient information collected by J-RARE, the JPA published the first edition of its guidelines for quality-of-life research of patients and patient organizations targeting rare and intractable diseases, which was intended to foster a more patient-centric approach to understanding diseases, drug development, and policymaking in Japan.
Most importantly, some PAGs are also playing increasingly significant roles in patient access and reimbursement policy. The Taiwan Foundation for Rare Disorders (TFRD) has been actively involved in several pieces of groundbreaking legislation related to rare diseases in Taiwan, such as the Rare Disease and Orphan Drug Act, medical care subsidy measures for rare diseases, and more. At the time of drug supply shortage for brittle bone disease in 2020, the TFRD petitioned for additional reimbursement of alternative therapies, provided financial support for self-paid therapies, and managed to convince relevant pharma manufacturers to address the shortage with more supplies.
Drug manufacturers should make PAG engagement an integral part of access in Asia. First of all, insights from PAGs can aid patient-centric drug development, as they can provide patient data for natural history studies, endpoint choice, trial design, and clinical outcome assessments. China’s Center for Drug Evaluation released guidelines on patient-centered clinical trial design, trial execution, and benefit–risk assessment in 2022, which advised that patient-reported outcomes and patient experience data been taken into considerations throughout the drug development process.
Secondly, PAGs could be a major driver of stakeholder engagement and policymaking, ranging from regulatory approval to market access and reimbursement. HDCare, for example, played a pivotal role for Huntington’s disease to land a much-coveted spot among the first 121 entries in the first batch of the Rare Diseases Catalog in China, and subsequently convinced the CDE to consider new therapies for priority review. When one did receive approval, HDCare obtained around 2,000 patient signatures for the petition to the public payer, and eventually secured nationwide reimbursement through its inclusion in the National Reimbursement Drug List.
Patient needs should be of the highest priority for all industry players, and effective engagement with patients and PAGs will increasingly be the key to define success going forward.
Duo Xu in Simon-Kucher’s China Life Sciences division contributed to this article.