Orphan drugs are the essential drugs that cure rare diseases, but they still lack proper treatment plans and financial initiatives to support further drug development. A large section of people in Europe and the United States have been impacted by orphan diseases, and there are approximately 5000 to 8000 orphan diseases that affect 50 million people in these regions. So, the new initiatives are taken into consideration for rare and orphan diseases drug development.
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What is drug development?
Drug development is the process of introducing a new drug to the market, followed by multiple stages. The drug development involves multidisciplinary stages, including drug discovery, preclinical research, clinical trials, and regulatory approval. The clinical development of rare and orphan diseases often faces risk and uncertainty.
Challenges faced for rare and orphan disease drug development
Almost 90% of human trials fail to get approval over registration of newly developed drugs for rare and orphan diseases.
- The significant challenges are faced by people due to lack of knowledge about the disease; most of them are unaware of the impact and criticality of rare and orphan diseases.
- Not only that, the longer duration of clinical development time is also a factor behind this rising concern over drug development for rare and orphan diseases.
- The research studies got stuck midway due to the difficulty in enrolling the patients for clinical tests. Only a small section of population gets affected by rare and orphan diseases, and most of them are unaware of the adversities of these diseases. As a result, R&D initiatives face major challenges.
Initiative for rare and orphan disease drug development
The efficient, innovative approach of drug development is necessary by considering the pharmacogenomic information during the clinical trial phase. The identification of patients having the adverse drug reaction is essential to get the optimal benefit-risk ratio. Pharmaceutical companies are encouraged to take initiative for drug development of rare and orphan diseases, and they are offered tax relief, grants, and waivers.
The FDA (Food and Drug Administration) has already approved 600 orphan drugs for the already recognized 800 orphan diseases till now. The European Medicines Agency (EMA) also approved 150 orphan drugs for treating orphan and rare diseases. Drug development programs for rare and orphan diseases are an absolute need because these diseases do not have suitable medical treatment procedures and require motivational drivers. Companies like Allucent are helping make these treatments better and easier for people around the world to get.
Frequently Asked Questions
Why does new drug development for rare and orphan diseases face challenges?
Drug development faces critical challenges mainly because of two reasons: either they do not have the efficacy or they have side effects on the human body.
What is a rare and orphan disease?
A rare disease is any disease that affects a small percentage of the population, and there are few available drugs and treatments. Orphan diseases are the neglected diseases that do not have specific drugs and treatments due to lack of knowledge and financial initiatives.
Who are the regulatory agencies of orphan drug approval?
The Food and Drug Administration (FDA), the European Medicines Agency (EMA), the Medicines and Healthcare Products Regulatory Agency, and the Korean Ministry of Food and Drug Safety (MFDS) are the main regulatory agencies of orphan drug development.
