Despite the large number of known Orphan diseases, it is estimated only about 5% of rare diseases have an approved treatment.
Over the last several years, we saw a major global effort in the clinical development of Orphan drugs. In fact, in 2018, the majority of new drugs approved by the FDA and EMA were Orphan medicines. Asian countries have been implementing legal frameworks similar to the Orphan Drug Act used in the US, encouraging clinical trials in many locations including South Korea, Taiwan, Singapore, and The Philippines.
These new legal frameworks in Asia set forth general criteria to accelerate the registration and approvals of drugs for the treatment of rare diseases and established new rules concerning health insurance coverage, expedited registration, and marketing exclusivity periods for biopharma companies.
Furthermore, the Action Plan on Rare Diseases launched in late 2018 by the Asia-Pacific Economic Cooperation is expected to accelerate supportive actions to develop public actions, disease registries, and patient access to clinical trials, and to reduce enrollment durations.
Providing Orphan diseases CRO services
Rare Disease clinical studies require a full-service CRO with expertise and experience in enrolling rare disease patients. This includes expertise in site selection and access to patient registries to accelerate patient enrolment, and global regulatory knowledge regarding the specific context of Rare Disease trials and Orphan diseases trials.
About 25% of drugs granted with an Orphan designation since 2015 were concentrated on 10 major indications. For most of these indications, clinical studies involving sites from Asia-Pacific outperformed the median patient enrollment duration of rare disease trials globally.
Novotech experience in Rare and Orphan diseases
Novotech has conducted over 20 trials including these rare disease indications:
- Alport Syndrome
- Alpha1antitrypsin deficiency
- Cystic Fibrosis
- Down Syndrome
- Duchenne Muscular Dystrophy (DMD)
- Fabry disease
- Friedreich's ataxia
- Gaucher's disease
- Macular Telangiectasia Type 2
- Growth Hormone Deficiency
- Muscular Dystrophy
- Peyronie's contracture
- Paroxysmal nocturnal hemoglobinuria (PNH)
- State of rare disease management in Southeast Asia. Shafie et al. Orphanet Journal of Rare Diseases (2016) 11:107. DOI 10.1186/s13023-016-0460
- Challenges in orphan drug development and regulatory policy in China. Cheng and Xie Orphanet Journal of Rare Diseases (2017) 12:13. DOI 10.1186/s13023-017-0568-6