The Czech health minister has urged the EU to jointly purchase medicines for rare diseases to lower the price of drugs and increase their accessibility for patients. According to The European Medicines Agency (EMA), there are an estimated 30 million people with rare diseases in the EU.
Although the FDA defines a rare disease as one that affects less than 200,000 people in the United States, it does not classify ultra-rare diseases. Europe also does not have a formal definition of ultra-rare diseases. For ultra-rare diseases, less is known about the prognosis of the disease and the number of patients is very small, making it difficult to develop therapeutics. This article proposes the necessary changes from a regulatory, clinical trial, and financial support perspective so that treatments for rare diseases can be well developed. It introduces proposals such as providing regulatory support by classifying ultra-rare diseases, providing treatment approval pathways for each group of rare diseases in common, or changing clinical trial methods.
FDA에서는 희소 질환을 미국 내 200,000명 이내로 발병되는 질환으로 정의하고 있지만 극 희소 질환을 구분하지 않습니다. 유럽도 극 희소 질환에 대한 정의가 없습니다. 극 희소 질환은 질환의 예후에 대해 알려진 바가 더욱 적고 환자 수도 극히 적어서 치료제 개발에 많은 어려움이 있습니다. 이 글은 극 희소 질환 치료제가 잘 개발될 수 있도록, 규제, 임상 시험, 재정 지원 관점에서 필요한 변화를 제시하고 있습니다. 극 희소 질환을 구분해 제도적 지원을 하거나, 공통점이 있는 희소 질환 군 단위로 치료제 승인 경로를 제공하거나, 임상 시험 방식에 변화를 주는 방안 등을 소개하고 있습니다. Original Article: Clinical Trials Arena.Drug development for ultra-rare diseases: What happens when N=1?
Rare disease diagnostics & research
This is an interview with Changwon Keum, CEO of 3billion. Based on 3billion's technological competitiveness, 3billion aims to achieve 1% of the total rare disease diagnosis market share and 100 billion won in annual sales within 5 years. In addition, the achievements of the AI-based drug development platform are introduced. Finally, after listing on the KOSDAQ market in the first half of next year, 3billion plans to focus on global commercialization.
Rare disease pharmaceutical industry trendsIonis Pharmaceutical and AstraZeneca announced that they had succeeded in Phase 3 clinical trial of co-developing transthyretin amyloidosis treatment(eplontersen). However, the…