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CMIC Resources

Navigating Rare Disease Research: CMIC Group’s Commitment to Breakthroughs and Sponsor Guidance

Rare Disease Day, observed on the last day of February each year, brings attention to the unique challenges faced by individuals dealing with rare diseases. As a global Contract Research Organization (CRO), CMIC Group recognizes the pivotal role it plays in advancing the understanding and treatment of rare diseases. This blog post explores the significance of rare disease clinical research, current statistics, industry struggles, the vital role of patient advocacy and also provides essential guidance for sponsors navigating the complexities of rare disease research.


Why Rare Disease Clinical Research Matters:

Rare Disease Day Logo

Rare diseases, often affecting a small percentage of the population, require comprehensive clinical research due to a lack of understanding, misdiagnosis, and limited treatment options. The urgency to develop effective therapies for these conditions cannot be overstated. Orphan drugs, specialized pharmaceutical agents developed for rare medical conditions, play a pivotal role in addressing the unique challenges of rare diseases by providing targeted treatments tailored to specific conditions, thereby contributing to the advancement of therapeutic options in this underserved field.

Historically, rare diseases and orphan drugs were overlooked and pharma companies were not likely to put priority on orphan drug development due to limited patient populations.  However, advancements in genetic research, technology, and increased awareness have prompted a shift, with governments and regulatory bodies worldwide incentivizing rare disease research and fostering collaboration.

The prevalence of rare diseases is staggering, affecting over 300 million people globally. The clinical research industry faces challenges such as limited patient pools, difficulty recruiting participants, and the complexity of diagnosis and trial design. Standardized endpoints and regulatory hurdles further hinder progress in rare disease clinical trials.


Guidance for Navigating Rare Disease Research:

Early Engagement with Patient Advocacy Groups: Patient advocacy groups play a crucial role in rare disease research, advocating for increased research funding, improved access to therapies, and a better understanding of unique challenges. Actively collaborating with patient advocacy organizations to ensure the patient perspective is integrated into every stage of the clinical research process is highly suggested.

Flexible Trial Designs: Rare diseases often have diverse manifestations, making it essential to design trials with flexibility. Consider adaptive trial designs and endpoints that can accommodate variations in patient responses.

Global Collaboration: Collaborate with international research centers to access larger patient populations. The rarity of the diseases means that patients might be dispersed globally, requiring a broad approach to recruitment. Connecting with companies such as the National Organization for Rare Disorders (NORD) can help with reaching the right patient groups, grants, and provide resources for the disease.

Regulatory Expertise: Stay abreast of regulatory incentives and designations for rare diseases. Engage with regulatory authorities early to navigate the specific requirements and streamline the approval process. To help make this process smoother, partner with companies like OrphanPacific, a pharmaceutical company within CMIC Group. With expertise in the orphan drug market in Japan, OrphanPacific can guide you through regulatory affairs, supply chain management, and more as you bring your rare disease therapies to the Japanese market.

Patient-Centric Approach: Prioritize the patient experience by designing protocols that minimize the burden on participants. Consider remote monitoring options and leverage technology for data collection to enhance patient engagement.


CMIC Group’s Commitment to Rare Disease Drug Development


Rare Disease Day image of a woman with cancer

As a global CRO, CMIC Group is committed to overcoming the challenges associated with rare disease research. With end-to-end solutions, including Japan Market Entry support, and a patient-centric approach, we streamline clinical trial processes to accelerate the development of therapies for rare diseases. CMIC Group has a subsidiary company OrphanPacific, who is a Japanese pharmaceutical company dedicated to providing orphan drugs to patients across Japan, and is able to provide solutions for foreign pharmaceutical companies and biotechs to enter the Japanese market. For your orphan drug development and marketing in Japan, consider OrphanPacific as your partner. You can learn more about OrphanPacific on their website.

To further support sponsors and industry professionals in navigating rare disease research, CMIC Group invites you to view our recent webinar, providing insights on patients with rare diseases and how to navigate the rare disease market in Japan. For more details, click here.

Rare diseases demand a unique and collaborative approach from sponsors, researchers, and patient advocates alike. CMIC Group stands at the forefront of rare disease research, providing end-to-end solutions and guidance to sponsors. Join us in our commitment to advancing rare disease cures, and mark your calendars for our upcoming webinar – together, we can navigate the complexities of rare disease research and make a lasting impact on the lives of those affected by these conditions.



National Library of Medicine https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9632971/#:~:text=Although%20individually%20rare%2C%20the%20collective,million%20people%20worldwide%20(10).


National Organization for Rare Disorders (NORD)




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