Why Investing in Orphan Drugs is Crucial?

Dear reader, it is crucial to understand what orphan drugs are before delving into the question posed in the title of this article. These drugs are specifically designed to treat rare diseases with limited information and high associated development costs. Cystic fibrosis and Angelman syndrome are a few examples of rare diseases, with an incidence rate of 1 in 15,000 people.

According to the US Food and Drug Administration (FDA), an orphan drug is a medication or biological product that is developed to cure a rare disease or condition. Such diseases or conditions are defined as affecting less than 200,000 people in the United States. Since orphan drugs are limited to a small number of patients, pharmaceutical companies may not have significant financial incentives to research and develop them.

On the other hand, according to the European Medicines Agency (EMA), orphan drugs are drugs that aim to treat rare diseases or chronic diseases that are serious, life-threatening, or affect less than 10,000 individuals in the European Union.

According to the Saudi Food and Drug Authority (SFDA), a rare disease is defined as any condition that affects only one out of every 2,000 individuals.

There are approximately 8,000 rare diseases worldwide, with symptoms typically manifesting during infancy and childhood. Genetic disorders are the primary cause of these conditions, which can be fatal, with mortality rates reaching up to 50%.

The percentage of approved drugs to treat these diseases ranges from 5%.

Like other drugs, orphan drugs undergo the same regulatory development process, and pharmaceutical companies can seek orphan drug designation. Various countries provide financial benefits for registering these drugs, such as tax exemptions for clinical research expenses and the possibility of up to 7 years of additional exclusivity in the market if the drug is approved.

Dear reader, let us ponder on today’s question, "Why should we invest in orphan drugs?" There are multiple compelling reasons to contemplate investing in such drugs.

1. Unmet medical need: Investing in orphan drugs can help meet this need and provide new novel treatment options for patients with these rare conditions.

2. Regulatory incentives: Regulatory agencies offer various incentives to encourage investment in orphan drugs. These incentives help offset the expenses of developing drugs for rare diseases and make it financially feasible for investors to participate in this sector.

3. Stimulating market potential: Even though rare diseases only impact a small portion of the population, the overall number of individuals affected by these diseases is significant and is expected to grow as more rare diseases are identified and diagnostic methods improve. As a result, there is a substantial demand for orphan drugs in the marketplace.

4. Social responsibility: Investing in orphan drugs can be considered a responsible social investment, as it can help build trust and confidence with patients and healthcare providers.

5. Scientific innovation: When it comes to developing drugs for rare diseases, innovative drug discovery and development approaches are often necessary. This can result in scientific breakthroughs that have implications beyond just treating the specific targeted disease. For instance, drugs created for rare genetic diseases can help uncover the underlying reasons for these conditions, leading to new findings related to more common diseases.

Before investing in this field, it is crucial to evaluate the potential risks and challenges involved, such as the high cost and complexity of drug development, regulatory approval hurdles, pricing, market accessibility, and intense competition. Taking these factors into consideration can help make informed investment decisions.

Finally, the development of orphan drugs has become an important area for the pharmaceutical industry in recent years, as pharmaceutical companies are looking for innovative ways to treat these rare diseases.