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The Revolutionary Impact of Orphan Drugs on Rare Diseases

Assessing the impact of the Orphan Drug Act 40 years later.

For almost twenty years, orphan drugs have been one of the fastest-growing pieces of the pharmaceutical industry market, and it doesn’t look to slow down anytime soon.

Pediatrician Harry Shirkey

The term ‘orphan’ was first used in 1968 by pediatrician Harry Shirkey when he published his paper talking about a pediatric population of children with rare conditions that he felt didn’t have access to medication for their illnesses due to the infrequent use and small sales potential of the relevant drugs.

However, that changed in 1983 when then-President Ronald Regan signed the Orphan Drug Act into law. The Orphan Drug Act incentivized drug companies to allocate more funding and resources to the research, development, and access of therapeutical products to people with rare diseases. 

Different countries have different programs, and the way they designate the term ‘rare’ diseases is also different. For example, in the European Union, a ‘rare’ disease does not affect more than 5 out of 10,000 people. In the United States, a ‘rare’ disease is classified as one that affects less than 200,000 people out of the entire U.S. population.

The Implementation of the ODA and Founding of NORD

The Orphan Drug Act was signed into law primarily due to public awareness and advocacy surrounding the cause. After the ODA was signed into law, the National Organization for Rare Disorders (NORD) was formed to create a uniform voice for people with rare conditions.

The rare disease community today numbers close to 25 million Americans.

In 1989, the National Commission on Orphan Diseases published a report which was based on a NORD study that documented the continued need for a bigger, national approach to address delays in accurate diagnosis, lack of access to rare drug therapies, and the impact rare diseases have on patients and families. 

That report helped to speed up the creation of the National Institutes of Health (NIH) Office of Rare Diseases Research (ORDR).

The Future of Orphan Drugs

In 2022, orphan drug revenues totaled 156 billion U.S. dollars, and that number is expected to exceed 300 billion by 2028.

Assorted medications

Prior to the implementation of the Orphan Drug Act in 1983, only 38 drugs had been approved to treat rare diseases. Since then, over 7,000 rare diseases have been identified, and over 1,100 orphan indications for treatments have been granted FDA approval.

Almost 5% of rare diseases currently have an FDA-approved drug, and up to 15% of those have at least one drug that has been developed and shown promise in the disease’s treatment, diagnosis, or prevention.

Over the last forty years, we’ve seen tremendous change in the rare disease community, with many previously unknown diseases gaining therapeutic treatments, advocacy, and approved treatments. However, there is still a lot of room for improvement.

Of the over 7,000 rare diseases that have been identified, less than 10% have a treatment, and fewer have a known cure. Almost half of all rare diseases only affect children. We still have a long way to go to help the more than 25 million Americans currently affected by rare diseases.

Implications for Conditions Not Considered Rare

older man with a cane

Although some conditions are technically not in the rare category, such as Parkinson’s Disease, which only occurs in 1% of people aged over 60, work on rare conditions and breakthroughs in this category can benefit not only diseases such as Parkinson’s Disease but also flow back into rare diseases. 

Currently, Laxxon Medical is working on Levodopa, integrating its technology to improve significantly the quality of life of patients with Parkinson’s disease. 

By delaying the API release in Levodopa by approximately six hours, patients can experience the benefits of the medication for longer, with fewer gaps between medications. The gap between taking medication before sleep and waking up without the benefit of the medication in your system is a significant problem for many medications and treatments.

It’s this type of technology transfer that flows both ways. While work on a rare disease may only benefit a few, advancements in conditions on the fringe of rare classification can effectively flow back into the rare category.

Read the full article “The “Freezing of Gait” Effect From Levodopa Management of Parkinson Disease” to learn more about the potential benefits of Levodopa and the work that the team at Laxxon Medical are doing.

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Laxxon Medical is dedicated to engineering patented 3D pharmaceutical solutions that optimize products and benefit patients. Our goal is to establish SPID®-Technology as a manufacturing process with the individual and the pharmaceutical partner in mind.

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