Contributors Participating in Society

We are committed to providing high-quality medicines to more patients with unmet medical needs through our efforts in research, development, and innovation, working with our supply chain partners to accelerate marketing authorization so we can satisfy the needs of patients requiring treatment. We also conduct cost analyses to demonstrate the cost effectiveness of Ropeg and to build a foundation for responsible pricing.

Commit to providing high-quality medicines to more patients with unmet medical needs

Access to Healthcare Programs

PharmaEssentia is committed to supporting disadvantaged groups and developing countries in advancing healthcare programs, thereby enhancing access to medicines. According to the World Health Organization’s definition, rare diseases are those affecting approximately 0.65-1% of the total population. Globally, there are estimated 7,000-8,000 identified rare diseases, accounting for about 10% of all human diseases. Patients with rare diseases, often hereditary in nature, represent one of the most vulnerable groups. Their small population size and limited public voice, coupled with the high cost and long timelines required for developing therapies and special nutritional products, have historically deterred manufacturers from investing in research, production, or introduction of treatments. Elderly rare disease patients face even greater challenges, as they are rarely prioritized in clinical attention, effectively becoming ‘the vulnerable among the vulnerable.’

Through the innovative technology of BESREMi®, which offers enhanced safety, longer-lasting efficacy, and elderly-friendly treatment, PharmaEssentia is dedicated to the research and development of orphan drug innovations, with a strong commitment to providing healthcare access and support programs to underserved populations worldwide.

Targets and Progress on Access to Medicines

• PharmaEssentia initiated special import programs in Colombia and Brazil to improve treatment opportunities for local rare disease patients. In April 2024, the Company successfully obtained special import approvals in both countries.
• PharmaEssentia has also pursued collaboration opportunities with foundations in China to improve drug accessibility for low-income rare disease patients. Implementation commenced in early 2025.

Measures to Enhance Drug Accessibility

• PharmaEssentia provides training for healthcare professionals in less-developed countries, including China, Colombia, and Brazil. Its initiatives also cover direct drug supply to designated local healthcare institutions and patients, the promotion of medical education programs on innovative therapies, and broad collaboration with local academic and research institutions to strengthen drug accessibility.

Promotion of Product Innovation

• PharmaEssentia has licensed its innovative drug technologies and patents to 20 countries, including Brazil, Colombia, Ecuador, Mexico, Peru, Egypt, Iran, Iraq, Morocco, Syria, Tunisia, Yemen, India, Armenia, Kyrgyzstan, Moldova, Tajikistan, Turkmenistan, Ukraine, and Uzbekistan (covering LMIC, MHDC, and HiHDI regions). This provides local medical communities with access to innovative therapies and supports their gradual adoption in clinical practice. In 2024, Brazil and Colombia granted special import approvals, giving patients timely access to these treatments.

Patient Assistance and Reimbursement

• PharmaEssentia actively advocates for national insurance coverage in countries where its products are marketed, to alleviate the financial burden on patients. The Company has successfully secured national insurance coverage in Taiwan and Japan and is working to secure reimbursement approvals for national insurance coverage in Korea and Brazil.

• Accelerating Drug Approval

  Ropeg obtained its first marketing authorization in 2019 and has since obtained marketing authorizations in more than 40 countries worldwide, including the US, Japan, Korea, Israel, China, Singapore, Malaysia, and many countries in Europe. We are currently working to obtain marketing authorizations in Brazil, Argentina, Columbia, Mexico, and Hong Kong. PharmaEssentia continues to expand the global market share of Ropeg by establishing subsidiaries and working with strategic partners to provide new drug options for more patients, reduce treatment burdens for rare diseases, and provide benefits for more patients.

  

• Product Delivery

  Product delivery is an important component of ensuring access to medicine. As different countries have differing drug packaging and labeling requirements, PharmaEssentia works with local stakeholders in the US, Korea, Japan, Singapore, Malaysia, and other countries; signed contract manufacturing agreements with local GMP-compliant pharmaceutical manufacturers; and signed delivery contracts with local GDP-compliant pharmaceutical delivery companies to ensure that our drug labels adhere to local requirements and to facilitate product delivery. We are also formulating relevant measures to prevent against counterfeit drugs.

• Focus on Patient Needs and Providing Comprehensive Support

  • US subsidiary: Patient Support SOURCE Program
  • Taiwan and Japan subsidiaries: Patient Support Programs

    PharmaEssentia’s subsidiaries in Taiwan and Japan promote “patient support programs” that provide patients with MPN health education information, medication guidance, and related advice and resources.

    

  Responsible pricing strategies to create medical values and cost-effectiveness

• Equitable Access to Medicine Strategies: Enhance Product Efficiency

  Providing innovative drugs with medical contributions that alleviate burdens on healthcare insurance systems and meet medical needs is a top-priority goal for PharmaEssentia. The medical value of BESREMi has been endorsed by European Leukemia Net (ELN) guidelines1 since 2021.

• Improving Affordability of New Drugs: Fair and Reasonable Pricing

  Ropeg is the first interferon approved for PV treatment by the US FDA which has been granted orphan drug status and has been listed as preferred treatment by US NCCN guidelines. Developing orphan drugs requires significant R&D investments and financial resources, and we have invested substantial amounts of resources to ensure that patients can obtain required medicines as soon as they are launched in accordance with access to medicine principles.

PharmaEssentia focuses on MPN issues and provides long term health education and healthcare resources to patients, HCPs, and medical experts to help patients and related personnel better understand disease courses while also helping patients obtain complete care and support. We provide health services for all stages of the patient journey, including disease awareness and screening, MPN diagnosis confirmation and treatment, and all-encompassing post-diagnosis patient care as part of comprehensive services.

1. Patients: Accompanying and caring for patients on the MPN journey

PharmaEssentia accompanies patients through all stages of their treatment and continues to enhance patient understanding of their diseases and treatments through patient associations and patient support programs. Our services include disease awareness and screening, diagnosis confirmation and treatment, and complete patient care cycles. We not only help patients obtain comprehensive medical and healthcare resources, but also share the latest information on MPN medications with medical personnel and healthcare professionals through regular academic seminars to make positive contributions to related communities and patients.

Measure Contribution to Health Outcome

The MPN Care Program is implemented globally, covering 100% of PharmaEssentia’s project portfolio and disease areas, and is continuously advanced on an annual basis. The following outlines the program’s initiatives and impact:

Note: Patient Assistance Programs include: United States – Source; Taiwan – iCare & MPN Care Association; China – Foundation; Japan – Foundation; Korea – NGO; Singapore – NGO; Malaysia – NGO.

Case Management and Community Empowerment

To help patient receive comprehensive care, PharmaEssentia has established patient support programs which actively support patient health education activities and case management, continued safety monitoring, reduction of patient financial burdens, and promotion of complete treatments. In Taiwan, we helped to establish the Taiwan Myeloproliferative Neoplasms Association (TMPNA), and also supported Patient Power programs and the MPN Research Foundation in the US.

Key achievements were as follows:

Local Capacity Building

PharmaEssentia supports developing countries identified by ATMI by enhancing local healthcare system capabilities, educating healthcare professionals and patients, and ultimately improving public access to essential medicines. The following outlines the relevant initiatives:

Patient Education: Enhancing patients’ awareness of rare diseases and addressing gaps in medical knowledge.

PharmaEssentia collaborates with patient organizations in China to regularly publish educational articles on patients’ social media platforms and invite disease-area experts to provide ongoing patient education through activities such as online sessions and offline free clinics.
Through systematic patient education (e.g., guidance on medication intervals, management of common side effects, and monitoring instructions), patients are supported in recognizing early symptoms and encouraged to seek timely treatment, thereby reducing the risk of severe complications and hospitalization costs. In addition, patient education enhances PV patients’ self-management and treatment adherence, which in turn strengthens the real-world reputation of Ropeginterferon and expands its market penetration in China, further increasing the willingness of hospitals and the national basic medical insurance system to adopt the therapy.

Implementation Frequency

1. Patient Education Meetings: Held once a month, from July 2024 to June 2025, reaching a total of 53 participants.
2. CSCO Hematology & Oncology Symposium: Held once a year, from July 2024 to June 2025, reaching approximately 500 participants
3. CASH China Hematology Development Conference: Held once a year, from July 2024 to June 2025, reaching approximately 500 participants.
4. Educational Articles on TongXinJiaYuan Platform: 15 patient education science-based articles published from July 2024 to June 2025, reaching a total of 27,491 participants.

Provider Education: Enhancing awareness of hematologic diseases in developing countries, advancing medical development, and fostering academic exchange in MPN-related research.

PharmaEssentia organized a symposium in Beijing, China, to support physicians in mastering key aspects of Ropeginterferon therapy, including dose adjustment, biomarker monitoring, and side-effect management, thereby facilitating the integration of the new drug into routine clinical practice and supporting the education of standardized treatment pathways. Given that treatment strategies and clinical decisions may vary across specialties, the symposium provided healthcare professionals with a unified and efficient understanding of symptom management and comorbidity care across the early, middle, and advanced stages of disease. This initiative also promotes cross-hospital, cross-regional, and cross-disciplinary collaboration in clinical practice.

Implementation Frequency

1. Patient Education Meetings: Held once a month, from July 2024 to June 2025, reaching a total of 53 participants.
2. CSCO Hematology & Oncology Symposium: Held once a year, from July 2024 to June 2025, reaching approximately 500 participants.
3. CASH China Hematology Development Conference: Held once a year, from July 2024 to June 2025, reaching approximately 500 participants.
4. Educational Articles on TongXinJiaYuan Platform: 15 patient education science-based articles published from July 2024 to June 2025, reaching a total of 27,491 participants.

Pharmacovigilance: Continuously monitoring and reporting drug safety information to safeguard patient rights and well-being.

In 2024, PharmaEssentia’s Beijing subsidiary in China established and implemented a comprehensive Pharmacovigilance (PV) program, covering areas such as training systems, continuous monitoring, and process review. This initiative enhances patient safety, ensures regulatory compliance, and strengthens market trust. PV training and SOP procedures support the timely provision of complete and consistent safety data and supporting documentation, thereby improving the transparency and credibility of safety monitoring data and further enhancing the confidence of healthcare professionals and patients in the Company’s products.

Implementation Frequency

1. Provide timely training for new employees.
2. Conduct annual training sessions for all employees.
3. Supplier Management: In 2024, the PV CRO (Pharmaron) report-handling team and call center team will circulate weekly/monthly project updates and submission status.
   Literature research is conducted biweekly.
4. Review PV SOPs once every two years.

Supply Chain Management: Strengthening cross-regional collaboration to establish a global standardized supply chain system, leveraging global experience to inform the China market and deepen local competitive advantages.

PharmaEssentia centers its supply chain and Pharmacovigilance (PV) management system on compliance with regulatory requirements and guidelines across different jurisdictions, aiming to establish a standardized model that enables synchronized operations across multiple countries. This model encompasses end-to-end temperature-controlled monitoring, execution of global SOPs, and cross-border training mechanisms to ensure consistent product quality and safety under diverse regulatory environments. The global supply chain management framework covers not only major markets such as the United States, but also extends to developing countries, combining centralized and localized supply models to flexibly allocate resources across markets. Cold-chain logistics are adopted to guarantee safe and stable drug transportation. The standardized global supply chain and PV mechanisms also serve as a valuable reference for the China market, ensuring alignment of local supply chain and safety monitoring standards with global practices, thereby enhancing compliance efficiency and strengthening clinical trust in China.

Accessibility & Transparency of Research

• PharmaEssentia strengthens its commitment to clinical research transparency in accordance with the PhRMA/EFPIA Principles for Responsible Clinical Trial Data Sharing. Since 2009, the Company has established a data request and sharing process for approved clinical trials (Phases I–IV). This process allows external researchers, upon signing a Confidentiality Agreement and receiving approval, to request access to the following: study protocols, Data comprising anonymized patient-level data and results from statistical analyses, and Clinical Study Reports (CSRs).

• The Company also actively shares research and development findings with the medical community through participation in domestic and international medical conferences and publications in academic journals.

✽ Data will not be provided in cases where there is a potential risk of patient re-identification, when restricted by regulations, local requirements, or data licensing conditions, or if the study is still under regulatory review or the publication process.

In line with its commitment to patient privacy, regulatory compliance, and integrity, the Company shares clinical research data to promote public health advancement and ensure the continued progress of biomedical research.

• Center for Drug Evaluation, Foundation for Medicinal Product Evaluation

2. Medical and professional personnel: Clinical trials and healthcare knowledge exchanges

In terms of diagnosis confirmation and treatment, PharmaEssentia has hosted many disease education events in markets around the world and has published various research and development achievements in medical journals. We support physicians from Taiwan in attending international hematology meetings to promote international exchanges of medical knowledge and to expand and enhance public health and medical health achievements.

Key achievements were as follows:

PharmaEssentia is focused on patients, the elderly, schoolchildren, women, and other minorities in society. Apart from the aforementioned access to MPN medicines and healthcare empowerment, we also use our manpower and financial resources to provide long-term support for many charity activities such as health promotion in rural areas, biodiversity education for schoolchildren, and sponsorships for local classical music activities. Additionally, our Japanese subsidiary also donated to local disaster relief funds following the Noto Peninsula earthquake, and we continue to accumulate social influence through long term issues and emergency incidents.

Seniors:

Health promotion program for rural areas to eliminate health inequality

We extended our core expertise into health and long term care issues, collaborating with the NPO Digital Humanitarian Association, which has long focused on telehealth issues, to gather physicians, nutritionists, nurses, physical therapists, and sports coaches at community long term care sites in Taitung lacking medical resources. We also organized monthly online health education courses to provide health promotion knowledge and services to elderly people. Additionally, health promotion managers provide online consultations and work with local caregivers and family members to understand the needs of seniors so customized courses can be organized. PharmaEssentia fully funded projects at three long term care sites in Taitung from January to December 2024 to help seniors prevent and delay disability and chronic diseases. We also responded to digital gaps and discrepancies in healthcare resources in rural areas of Taiwan by making investments to enhance the availability of health and healthcare resources in these areas, ensuring they were not limited by geographical factors or existing resources.

PharmaEssentia achievements on remote health promotion and charity projects for seniors in rural areas in 2024

Children:

Support in-depth educational experiences and cultivate biodiversity awareness

PharmaEssentia worked with the NPO Jane Goodall Institute to help elementary school students cultivate an interest in plants, the environment, and animals, aiming to cultivate green youth leaders and encourage the next generation of citizens to continue taking action to protect the Earth and their everyday environments.

PharmaEssentia 2024 Biodiversity Hope Box project achievements

Women:

US subsidiary participate in Rosie’s Place volunteer activities

Rosie’s Place is a community center in Boston, USA, which helps local women who are impoverished or homeless by providing emergency shelter, meals, food items, English for Speakers of Other Languages (ESOL) courses, legal aid, healthcare, one-on-one support, housing, and job seeking services, as well as community outreach. Our US subsidiary supported Rosie’s Place and participated in volunteer activities. Our colleagues from the Boston PIRC and US subsidiary teams helped to prepare meals and clean up for events held in August and November 2024, interacting with Rosie’s Place and local communities with supportive actions and care.

Local music group:

Sponsored OneSong Orchestra New Year Charity Concert

PharmaEssentia has sponsored the OneSong Orchestra New Year Concert, which focuses on local and classical music, for 6 consecutive years to support local music groups and cultural arts developments. We invited 32 PharmaEssentia employees, employee family members, and stakeholders to attend the concert to promote public understanding of localized classical music.