Rare Disease Patient Groups Emerging as Key Stakeholders in Asia

SINGAPORE – Across much of Asia, and in China in particular, the responsibility of fighting for the rights of patients with a rare disease mostly falls on the shoulders of the patients themselves. By talking with the government and industry, patient groups are pushing for more effective drug development and orphan drug discovery policies. Unfortunately, once disease conditions and cultural elements are taken into account, those groups may have significant challenges ahead.

"Patient organizations are relatively new in China; the very first was established in 2000," said Rachel Yang, director of international affairs at the Chinese Organization of Rare Disorders (CORD), during the BioPharma Asia Convention in Singapore last March. "Right now, we have over 60 rare disease patient organizations in total, with 10 new ones each year."

"The majority of these organizations are very new and very much grass-roots, poorly funded and with no dedicated resources, but we also have some really good ones like CORD," said Yang.

Drug developers can benefit from access to identified groups of patients that can provide a pool of people to register for clinical trials, as an example. That is particularly important for rare diseases, where a lack of access to patients can be a significant hurdle in getting a drug through the development process, into the clinic and onto the market.

Western biopharma companies often have well-established relationships with patient groups in their focus area and provide significant levels of funding. Most of the rare disease patient groups in Japan and China, however, receive much less support. Few get any financial support from government and most of their income is from individual memberships and, to some degree, funds from industry or other organizations.

"Individual-specific disease organizations have been told that they can apply for some government funding," said Beverley Yamamoto, a professor at the Graduate School of Human Sciences at Osaka University and president of Hereditary Angioedema Japan. "The specified disease program does provide support to patient organizations, but the application process is very arduous."

In China, in particular, the government is suspicious of organized groups that may be considered to be social activists. But the environment is starting to change. "Due to the work of some advocates in this area, the government is starting to see that these organizations are not working against them," said CORD's Yang. "So we have been pretty successful in working with government agencies like hospitals and research institutes.

"The government is really an abstract concept [for us], but we have received quite a bit of support from these institutes and the people working there," Yang added.

CORD, a Mainland Chinese umbrella organization founded in 2013, has played a significant role in helping many other organizations upgrade their operations and keep on top of developments abroad. While some patient organizations do little more than provide social networks, groups like CORD aim to dig deeper while fighting for patient rights, greater access to treatment and more drug development.

"We let the voice of the patients be heard by working with the industry and regulatory agencies," said Yang. "And we have made quite significant progress in the last few years."

The upgraded models employed by some of these organizations are already paying off with real progress. The CFDA has put out some mandates related to rare diseases and orphan drugs. During a reform of drug registration policies last year, the CFDA added orphan drugs to the fast lane registration category. "We have also been working with provincial regulatory agencies because, in China, medical care runs on a provincial level," said Yang. "We're seeing more and more diseases being covered by social medical insurance in places such as Qingdao, Shanghai and Zhejiang and at high rates like 80 percent.

"A lot has yet to be done, but we remain positive."

Getting Patients Involved

Although patient organizations can help patients with treatment access and better policies, there's an anxiety hurdle of being exposed to the public in places where stigma associated with the disease remains quite high. As a result, patients may be reluctant to participate in organizational activities.

"In the Japan context, it's a huge risk being visible [to the public as a rare disease patient] because of discrimination, particularly when they have any kinds of blood disorder," said Yamamoto. "When I decided to set up a patient organization on hereditary angioedema [HAE], I was told by a lot of people that it wasn't going to happen, that no one is going to risk coming forward."

Blood disorder patients are especially vulnerable to the discrimination issue. A patient in Yamamoto's group was let go by his company immediately after discovering he had HAE.

"You have to create a space where the patients can feel safe," said Yamamoto. "Now we have patients that are willing to put their faces on the website but there are still huge barriers."

Yamamoto's organization adopts a method by which it tries first to get the patients to come to a meeting where they can get information; later efforts to keep them in the group include covering their travel expenses.

"I think culture is the issue, but most of the patients have to come out in the end out of necessity. They need help," said Yang. "But it really depends on the kind of disease. For example, with ones that are considered sexually related, it really takes tremendous courage to come out in those cases."

But rare disease groups in China are starting to see pioneer patients that have realized there's nothing wrong with being in a rare disease patient organization. Some of those patients are the founders of their patient groups.

"Of course the efforts you make working on providing more tangible benefits to the patients and the incentive they enjoy goes hand-in-hand," said Yang.