CRISPR Therapeutics said CASGEVY launch momentum is building and multiple pipeline assets could generate data in the next 12 to 18 months. The company also highlighted zugo-cel progress in oncology and autoimmune disease.
India said its 2021 rare disease policy expanded Centres of Excellence from 8 to 15 and raised financial assistance to Rs 50 lakh. But rare disease treatment can cost up to Rs 1 crore or more annually.
India is expanding rare disease support under the 2021 policy, with aid up to Rs 50 lakh and 15 Centres of Excellence. Officials also called for local innovation as treatment costs can reach ₹16 crore.
The global API market is projected to grow to USD 198.39 billion by 2030 from USD 144.20 billion in 2025, at a 6.6% CAGR. Oncology, rare disease and diabetes pipelines are key growth drivers.
South Korea has approved fast-track drug listing for rare disease treatments, reducing the insurance coverage process from 240 days to 100 days. The reform includes post-listing effectiveness evaluations and flexible pricing agreements, aiming to improve patient access to innovative treatments amid ongoing budget concerns.
The artificial intelligence in drug discovery market is projected to grow from USD 3.25 billion in 2026 to USD 10.29 billion by 2031, representing a 25.94% CAGR. AI technologies are accelerating drug discovery timelines, reducing costs, and improving success rates through advanced analytics and machine learning. North America leads market adoption while Asia-Pacific emerges as a high-growth region driven by expanding biotechnology sectors.
China's newly revised Implementation Regulations of the Drug Administration Law will take effect on May 15, 2026, representing the first comprehensive revision in over two decades. The regulations strengthen the marketing authorization holder system, improve drug development processes, and introduce new provisions for clinical trials, data protection, and market exclusivity for pediatric and rare disease drugs.
The global orphan drugs market is projected to grow from $223.76 billion in 2023 to $486.51 billion by 2032, with North America leading at 38% market share. Over 500 orphan drugs are approved with more than 800 candidates in clinical trials, driven by regulatory incentives and major pharmaceutical company investments in rare disease therapies.
Global pharmaceutical R&D deal values surged 49% to $86.7 billion in 2025 as companies concentrate investments in AI-powered drug discovery platforms, with average deal sizes reaching a record $1.16 billion despite fewer total partnerships.
The FDA has established a "plausible mechanism pathway" to approve personalized genome editing and RNA-based therapies for rare and ultra-rare diseases without requiring large randomized controlled trials, streamlining access to individualized treatments.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07147465 |
Performance Indicators and Impact on the Care Pathway of Sequencing on the SeqOIA and AURAGEN (Seqogen) Platforms for Oncology Patients |
RECRUITING | |
| NCT05203250 |
Longitudinal Registry Including Patients Treated With Heavy Particles |
RECRUITING | |
| NCT02938793 |
Durvalumab in Combination With Tremelimumab in Subjects With Advanced Rare Solid Tumors |
TERMINATED | PHASE2 |
| NCT01440218 |
Idiopathic Diseases of Man |
ENROLLING_BY_INVITATION |
