What are rare diseases?

Rare diseases are defined as those diseases that very few Americans suffer from. Most patients with these diseases have these diseases for their entire lifetime. With the passage of this bill, the federal government gave pharmaceutical companies major impetuses for the development of orphan drugs. Prominent among these was the extended period for which a pharmaceutical company could hold rights over its product.

Since the promulgation of this Act, the number of orphan drugs that have come into the US market has exploded. From less than a dozen in the whole decade preceding the passage of the Act, the number grew to 400 in the subsequent three decades, registering an impressive growth percentage of over 1300.

The OOPD and its programs

The Act commenced with the formation of the Office of Orphan Products Development (OOPD) at the FDA. The OOPD was given the responsibility of ensuring that incentives were handed out to pharmaceutical organizations that came out with orphan drugs. These have been done through its two programs –the Orphan Drug Designation Program and the Orphan Products Grants Program. Among the products these two agencies identify for incentives are biologics such as vaccines or blood products, drugs, medical foods and medical devices that are used to treat rare diseases.

Notable achievements

The OOPD, with its two programs has achieved significantly in the years since it came into existence. While the Orphan Drug Designation Program, which helps classify drugs as orphan drugs, has classified over 2500 drugs as classified drugs; the Orphan Products Grants Program has overseen the authorization of grants of close to $300 million toward clinical studies.


In continuity of its programs marking three decades of its existence; the OOPD has achieved significantly by embarking upon many social outreach programs. Some of these can be listed under the following:

1. The program has helped create greater awareness among the lay public about the existence and nature of rare diseases. Its collaborative programs have roped in patient advocates, with whose help the OOPD has brought about heightened awareness nationally about the challenges faced by Americans who suffer from rare diseases. These groups have helped the public and government and NGO’s support the families of people with rare diseases. These groups have also motivated organizations to delve deep in their research into their diseases. These advocates have also brought lawmakers into the direct contact with patients of rare diseases. This has played a major role in the enactment of the law.

2. The OOPD has also brought the research community into close contact with patients. This has helped to build resources for these programs while enabling a greater sense of interaction between industry and researchers. As a result, today, the industry, comprising the entire set of stakeholders such as biotech and pharmaceutical companies and investors, has collaborated to develop products for rare diseases.

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