Roundtable discussion with patient group representatives of hereditary angioedema (HAE)

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<Introduction>

Deloitte is committed to helping patients with any diseases proactively manage their wellbeing, with the belief that they should take ownership in their treatment paths and receive timely treatment and medicine, working along with healthcare professionals (HCPs).　To this end, Deloitte’s Life Science Healthcare (LS&HC) has been advocating patient-centric activities in partnership with various stakeholders.

One case of these efforts is support of patient advocacy of hereditary angioedema (HAE). Due to the rarity of the disease, with only one in fifty thousand likely to be diagnosed^1),2), HAE is not widely known. The difficult-to-diagnose nature of the disease forces many patients to suffer for long periods of time without proper treatment.

In 2021, with the aim of improving the diagnosis rate, Deloitte had established a consortium “DISCOVERY” in partnership with stakeholders from the pharmaceutical sector. The consortium is designed to establish an ecosystem of different expertise among HCPs, academia, patient groups, and private companies in such a way that patients are diagnosed promptly and correctly.

In prior to HAE DAY (May 15th of every year), patient group collaboration team of Deloitte LS&HC unit held a roundtable discussion, inviting representatives of HAE patient groups from different counties in Asia. What is the situation and challenges surrounding HAE patients in different countries and how do they see the possibility of collaboration across the region?

<Discussion>

Tomoko Osawa
Thank you for joining us today. We have participants from Japan, Indonesia, and Thailand. Will you briefly introduce yourself?

Makiko Matsuyama
My name is Makiko Matsuyama. In July 2023, I was appointed President of HAEJ, an HAE patient group in Japan. Previously, I served the group as one of the representatives for over 10 years.

Established in 2014 as one of the national member organizations of HAE International, HAEJ currently has more than 100 members, including patients, their families, and HCPs. We offer opportunities to our members to get to know each other and obtain the latest medical information, while advocating for studies on HAE and facilitating approval and introduction of treatment drugs in Japan.

Rudy Purwono
I am Rudy Purwono from Indonesia. I am an HAE patient myself, diagnosed in 2015. In my case, the attacks are mostly on my stomach, but sometimes I have swelling on my hands and feet. I am a national contact for the Indonesian branch of HAE International, but advocacy activities in my country are still in the early stage.

Suchitta Kengtanyagarn
I am Suchitta Kengtanyagarn, a representative of the patient group in Thailand. I am not a patient, but a caregiver. In fact, my mother was the first case that was diagnosed as HAE. My brother was also diagnosed as HAE five years ago. It has only been within the last five or six years that we truly understand HAE. It was only two years ago when we finally had a lab in the country that can conduct tests for C1 inhibitor for potential HAE patients.

Tomoko Osawa
From the Patient Group Collaboration Team of Deloitte’s LS&HC, we have myself, Tomoko Osawa, Senior Consultant, Mio Tsuzuki, also Senior Consultant, and Yasuaki Imai, Consultant.

To begin with, Rudy and Suchitta, will you share with us the situation and challenges facing HAE patients in Indonesia and Thailand respectively?

Rudy Purwono
The biggest challenges for HAE patients in Indonesia are the lack of access to doctors who can properly diagnose the disease and access to medicine.

Talking to doctors is very difficult and getting drugs　for long-term prophylaxis or attacks is almost impossible. I am a patient of a doctor in Hong Kong and purchase drugs from Singapore and Hong Kong. On top of this, the medicine is very expensive and not covered by the public health insurance scheme in Indonesia.

Suchitta Kengtanyagarn
HAE patients in Thailand face two challenges – one is the access to medicine, like the case in Indonesia, and the other is the lack of patient data to improve the healthcare system.

In terms of access to medicine, there are some HAE medicines available in Thailand. However, the drugs are not affordable unless you are covered by the health insurance scheme for government workers. Among the three different healthcare systems in Thailand, around 90% of HAE patients are in a universal healthcare scheme which does not cover HAE drugs yet. The medicine is not affordable either - it costs almost three-month’s worth of a Thailand’s average income. I am hoping that HAE medicine will be provided by every type of health insurance scheme soon.

The other challenge is about getting HAE patient data. Medical institutions do not disclose data on the number of patients, diagnosis rates, or treatment status, and there is no collaboration between hospitals and healthcare organizations. Meanwhile, the health care regulators need such data to make decisions on whether to provide HAE medicines for each healthcare scheme.

We still need to keep advocating among multiple organizations to ensure that HAE medicines are covered in every healthcare scheme. As we always say, “everyone must have lifesaving medicine.”

Tomoko Osawa
What are the goals of the patient group and what are you focused on?

Suchitta Kengtanyagarn
We established the HAE patient group in Thailand about five years ago and have become more active over the last two years. Presently, we are a self-help group of volunteers, offering mental support and non-medical consultation to HAE patients. Some patients do not know anything about the disease even when diagnosed, while others have difficulties finding doctors who can diagnose their symptoms. We provide information on hospitals and doctors, offer mental support, and give advice on how to take care of themselves.

We also organize events for patients. In fact, we have one event planned in May, in line with the global HAE Day which is May 16 every year. This year, we are planning an offline event, inviting not only patients, but also HCPs and regulators to share knowledge through seminars.

In addition, we are actively participating in other patient advocacy events in Thailand. Especially, as we collaborate with the Thai Rare Diseases Foundation, we take part in their events with the aim of raising awareness of HAE among HCPs and regulators. We do whatever it takes to let everybody know that there are these patients who need medicine to stay alive.

Makiko Matsuyama
How many people are involved as members of your advocacy group in Thailand?

Suchitta Kengtanyagarn
We have 20 to 30 members, including roughly 15 patients.

The problem is that some patients do not know about the patient group and others do not see the necessity of such a group. In addition, because this is a genetic disease which affects your personal and your family’s life, I believe quite a few patients are reluctant to register with us for fear of disclosing that they are HAE patients.

Makiko Matsuyama
I totally understand the situation and how patients feel about others finding out about the disease. In Japan, too, genetic diseases are sometimes regarded as something like a “family stigma” especially in rural areas. I see a similar sentiment across Asian countries.

Mio Tsuzaki
We believe that the stigma surrounding genetic diseases makes it even more challenging for rare disease patient groups to advocate for their unmet needs. Patients should take ownership of their own well-being and treatment paths.

Therefore, we aim to encourage patient groups to collaborate with other patient groups and various healthcare industry stakeholders globally. Our aim is to make a meaningful impact on society, promoting a more patient-centered world.

Tomoko Osawa
Rudy, how do you see the situation surrounding the HAE patient group in Indonesia?

Rudy Purwono
Unfortunately, we do not have much interaction among patients - the patient group members are only me and my family members. I would like to find other members and socialize with other patients. I almost want to go to Thailand for the HAE Day event to talk with the members!

As I said before, we do not have HAE doctors and have difficulty in accessing drugs. And doctors are not at all accessible – we have a hard time talking to them to raise awareness of the disease. Especially senior doctors do not want to listen to us. In fact, when I had an intestinal problem, the doctor diagnosed it as cancer, not HAE, and cut off my intestine. Thus, we need to take a different approach if we want to raise awareness of HAE among HCPs. We are now trying to start with medical school students. We hope that the situation will improve in the future once these students gain a knowledge and awareness of HAE.

Suchitta Kengtanyagarn
Five years ago, our situation in Thailand was exactly the same as where Rudy stands now in Indonesia. Some of my family members died from airway swelling or unnecessary surgery, because they don’t know they had HAE. The situation has been improving, however, partly thanks to very understanding doctors and partly due to our advocacy.

In particular, the HAE International’s APAC Conference in March 2023 was a turning point for us – we saw some significant changes between medical professionals and patients. The conference was organized by HAE International (HAEi). As a strong advocate, HAEi has been supporting our patient group and proactively talking to the doctors who collaborate with healthcare regulators in the country. We are starting to gain support from The Allergy, Asthma, and Immunology Association of Thailand.

They take the initiative to collaborate with medical schools and hospitals to enhance academic support and HAE research. This collaboration enables authorized government organizations to begin the process of approving HAE drugs for the purpose of all healthcare schemes. Also, now our HAE patient group and the allergy doctors are working together as partners to help the HAE community attain a better life.

Rudy, I know you're currently facing challenges on your own in Indonesia, but I assure you that positive changes will come as you keep collaborate with partner like HAE International and patient groups in other Asian countries.

Makiko Matsuyama
I agree with you, Suchitta, that HAE International’s conference in Asia was a turning point. Those conferences were traditionally organized by US or Europe and stakeholders from Asia found it difficult to take proactive roles and speak up.

The situation in Japan, though still with challenges, may be better than the rest of Asia. We do have on-demand treatment that stops attacks and long-term prophylactic treatment. In a way, patients here have some options. Hearing your stories, however, you do not have many options. In Indonesia, even access to basic medicine is difficult. We have to do something, especially through collaboration among patient groups in Asian countries.

Rudy, you said that you would like to participate in the HAE Day event in Thailand – I suggest you really do it! We will have to seek out more opportunities to frequently meet each other among patient groups in Asia. We need to get to know each other and expand our collaborations.

Another idea would be to have the next HAE International’s APAC conference in Indonesia. Rudy mentioned earlier that senior doctors in Indonesia are not open to learning about HAE, but even they cannot ignore the authority of an international conference. We can make them listen.

Mio Tsuzaki
I believe that involving HCPs would be a key factor for successfully improving the access to treatment. As Suchitta mentioned earlier, having one Key Opinion Leader can change the situation. As HAEJ seems to be building a good relationship with specialized doctors, we can share our knowledge and experiences in advocating for better access to medication as well as raising awareness among HCPs.

Rudy Purwono
In addition to grown-up patients, I am also concerned about our children’s generation. This is a genetic disease, affecting a patient’s entire life events. Children born with the disease, especially those who are unable to access medicine, are in a very vulnerable position. With no fundamental treatment like genetic treatment available, will their future partners accept their disease?

We need a program to support patients while they are still at a young age. We need to assure them that they do not need to suffer alone and that they can lead a positive life even with HAE.

Makiko Matsuyama
I agree with you. At HAEJ, we are planning a children’s camp, youngster camp, this or next summer. We understand that patient events tend to focus more on grown-ups and that children do not have opportunities to get to know each other and talk about their situation among themselves. So, the camp is designed not just for skills and knowledge but for making friends, understanding that they do not need to feel alone. We would like to build a confidence among young patients so that they can proactively manage their own health.

Suchitta Kengtanyagarn
As a child of an HAE patient, I frequently saw my mother almost dying from pain due to HAE attacks. I can relate with what you said, Rudy. While first-line HAE treatments are now available in Thailand, they are unaffordable and not covered by any insurance or healthcare schemes. As a result, we resort to using indirect medications, which come with side effects that can seriously affect children.

Tomoko Osawa
Deloitte has been proactively involved in supporting HAE patients, for example, supporting the establishment of a consortium called, “DISCOVERY,” that aims to improve the diagnosis rate in Japan. Please share with us the overview of the initiative.

Yasuaki Imai
We have been promoting HAE education to HCPs and accelerating doctor-to-doctor consultation. We also established a website for potential patients and their families so that they can understand treatment options, patient history and which hospital to visit for diagnosis and treatment. In addition, we have a moonshot program for AI diagnosis, trying to build an algorithm to identify potential HAE patients. We are currently negotiating with several hospitals to apply the algorithm. We are very committed to helping patients who are still suffering from HAE attacks.

We may be able to propose to support collaboration across the Asian region, like you have been discussing.

Mio Tsuzaki
One idea would be for Deloitte to support HAE groups in Asia to organize an HAE international conference or seminar in Indonesia where we can invite patients, families, HCPs, and anyone interested in the disease or patient group activities. This would be a good start for raising the awareness of HAE in Indonesia.

Makiko Matsuyama
We need to advocate from a more positive side – not just seeing HAE as a negative genetic disease. We can work together to make sure patients, their families, and HCPs understand that there are options, and we can access prophylactic treatments.

HAEJ is a relatively large patient group compared to other groups in Asian countries. We can share our activities and campaigns across the region and connect you with experts in the region.

Suchitta Kengtanyagarn
For successful collaboration, we need to first answer some questions - how can we encourage people to join the advocacy group? How can we gain their trust and raise awareness of HAE? How can we improve the well-being of the patients? It is when many more people take part in patient groups, that regional collaboration becomes fruitful.

Yasuaki Imai
While HAE and its patient groups are not necessarily positively recognized in Japan and Asian countries, the same disease is regarded as a unique attribute of individuals in the US and Europe. We should continue our work to change the image of patient groups and win more members. We need to continue our efforts to raise the general awareness of HAE and improve the treatment environment. Deloitte is committed to being a catalyst of change, supporting HAE patient groups in collaboration with healthcare stakeholders across countries.

Reference:
1) Zuraw BL. N Engl J M ed 359 :1027-1036, 2008.
2) Lang DM, et al . Ann Allergy Asthma Immunol 109 (6) :39 5-402, 2012.