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What is an Orphan Drug?

Orphan Drugs diagnose, prevent, and treat orphan diseases. An orphan disease is a rare disease or condition. Such a disease affects fewer than 200,000 people in the United States. Orphan diseases are often severe or deadly. As a matter of fact, the US Government passed a law called the Orphan Drug Act in 1983. This law promotes the making of safe, effective Orphan Drugs. It gives pharmaceutical companies certain financial benefits for their working on these drugs.

The FDA makes rare-disease research a priority. Thus, it supports the research and development of new treatments for rare diseases. Another key point is that having Orphan Drug status qualifies sponsors for incentives, including:

• Exemption from user fees
• Tax credits for qualified clinical trials
• Potential seven years of exclusive market rights after approval

Why are Orphan Drugs Important?

A recent MIT study observed that rare diseases are not that rare. These diseases affect a total of about 25 to 30 million Americans. More than half of those afflicted are children. Many of these diseases or conditions are deadly or life-limiting. Patients as well as caregivers in this group face many challenges. Lack of knowledge about their condition, even by doctors, is one of the biggest obstacles.

Most patients with a rare disease will never know anyone with the same condition. Thus, they and their families must cope alone. This isolation can be stressful for patients. Patients with rare diseases cannot stay on the sidelines. Science and medicine have made tremendous strides over the last century. It’s time for these patients to get a share of the pie. They have the same rights to treatment as any other patient.

Why Companies Need to Invest in Orphan Drug Market Research

The future is wide open for investment in Orphan Drug projects. Drugs are only available for about 5 percent of rare diseases. The benefits of investing in Orphan Drugs are manifold. Competition is almost non-existent. Another benefit is the long patent protection periods. The FDA gives these drugs seven years of exclusive rights from the approval date.

Orphan Drugs have become profitable. Hundreds of new rare-disease treatments have entered the market in recent times. Orphan Drugs often carry very high price tags. Why? Because of their rare nature and lack of competition. Another critical factor to consider is the approval time, which is faster. Swift approval is a huge advantage for getting Orphan Drug status. Companies are working with the FDA to bring these life-changing treatments to millions.

Belangrijkste functienamen

• Orphan Drug Research Assistant: This person processes regulatory documents and acts as an aide to researchers
• Patient Support Specialist: provides patients and caregivers with info about treatment options
• Clinical Trial Manager: This person performs and manages clinical studies. Moreover, they drive the process of developing drugs to treat those with rare conditions.

Belangrijkste succesfactoren

Developing drugs in the rare disease space can be successful when approached correctly. Big players across the industry must join forces for it to happen. They can then leverage the power of data to help drive potential treatment options. McKinsey has identified four strategic pillars of successful Orphan drug launches:

1. A commitment to the rare disease community
2. Use of state-of-the-art methods for pinpointing patients
3. Use of a tactical approach to patient access
4. Extensive support for patients and caregivers within the healthcare system.

About Orphan Drug Market Research

Orphan Drug Market Research calls for Qualitative Market Research. Drug companies must find out patients’ needs. They also need to generate ideas for improving products and expanding existing ones. These companies must further clarify the marketing mix. They must also understand how the drug will fit into clients’ lifestyles. Orphan Drugs also require

Quantitative Market Research. The most popular Quantitative method is Surveys. One-on-one Interviews are another popular method. You can use them to gauge the need for Orphan Drugs.

SIS International Research serves the Orphan Drug market. We provide Quantitative and Qualitative Market Research. We also offer UX Market Research and Competitive Analysis. Furthermore, we can work out your Go to Market Strategy with you. Another service provided by SIS is Market Entry Research. We also do Strategy Market Research and Market Sizing Research. We can also offer Focus Groups to aid in your Market Entry Research.