4 Historical Orphan Drug Development Barriers, Have We Broken Through?

Both understanding the role of patient advocacy groups in building better clinical trials and improving the research process mean first taking a look at the history of orphan drug development in the United States and Europe.

An “orphan drug” is a drug that treats a condition that fewer than 200,000 people have. Because rare diseases affect so few people, there have historically been some key barriers to drug development in this field:

  1. Limited funding. Orphan drug research is extremely costly, and the market for these products is small, making it a difficult investment for drug companies.
  2. Lack of research interest. Because of the lack of financial incentives and other market realities, researchers have shown limited interest in investigating treatments for rare diseases.
  3. Lack of patient advocacy groups and limited involvement in research. Though this has changed today, for many years, very few influential patient advocacy groups existed, and, therefore, patient voices did not play a major role in drug development.
  4. High cost of development; low return on investment. Prior to the passage of the Orphan Drug Act in 1983, companies had little economic incentive to research and produce orphan drugs.

As evidence of these challenges, only 34 new orphan drugs were introduced to the market between 1973 and 1983. Two major shifts occurred in the 1980s that began to transform the status of orphan drug research in the United States and Europe.

First, the Orphan Drug Act was passed in 1983 with the goal of giving pharmaceutical companies greater incentives to invest in research around rare diseases. Specifically, the law guaranteed market exclusivity for a period of seven years, offered reduced regulatory fees, and provided tax incentives to companies that developed orphan drugs. This led to a drastic increase in the number of applications for orphan drug approvals from 1983 to today.

The second major factor in changing the role of patient advocacy groups in drug development was the HIV/AIDS epidemic of the 1980s. For the first time, an active, outspoken and influential patient advocacy group worked directly with the FDA to create avenues for greater investment in research for a particular therapeutic area, signaling the growing importance of patient voices in the drug development process. Out of the activism of that period came the Patient Representative Program, begun by the FDA to facilitate more dialogue between patients, researchers and industry figures.

A more recent turning point in the relationship between patient advocacy and drug development was the 2012 passage of the Food and Drug Administration Safety and Innovation Act (FDASIA) legislation. The key components of this initiative are summarized by the FDA:

  • Giving the authority to collect user fees from industry to fund reviews of innovator drugs, medical devices, generic drugs and biosimilar biological products
  • Promoting innovation to speed patient access to safe and effective products
  • Increasing stakeholder involvement in FDA processes
  • Enhancing the safety of the drug supply chain

The key goal of this legislation is to increase patient engagement and involvement with the drug development process, and specifically, to incorporate patient perspectives and insights into drug development from the very beginning. This signals a radical transformation, but work remains to be done.

To learn more about the powerful and evolving influence of patient advocacy on drug development, watch the webinar.