NEWS CORNER

Care About Rare: ODA in Danger?

Guest blog post by Connie Selinsky

On August 16th, 2018 a drug called WVE-210201 was granted orphan drug and rare pediatric disease designations by the FDA.

What Makes A Disease Rare?

In the US, a rare disease was defined in 1983 by Congress in the Orphan Drug Act (ODA) as a disease that affects less than 200,000 people (4). There are currently over 7,000 known rare diseases that affect approximately 1 in 10 Americans (2). Ten percent of the population may not seem as though it were a large number but with America’s population reaching just over 300,000,000 (7), this means that there are roughly 30 million people with a rare disease and 30 million families with a loved one facing a rare disease.

How Do Genetics And Rare Diseases Relate?

Eighty percent of diseases that are considered to be rare are also diseases that have genetic components for disease nature (2).

What Role Does The ODA Play In Rare Diseases?

The term “orphan drug” is used to describe drugs used for treatment and management of rare diseases. The act was created for the purpose of increasing the development of orphan drugs by creating incentives for pharmaceutical companies to produce these drugs where they otherwise might not due to the profit generated from smaller populations of affected individuals (1). There is tax credit given to companies researching orphan drugs of 50% of qualifying costs related to the orphan drugs being studied. The impact of the ODA has been substantial where it has helped the rare disease community by increasing the FDA approved drugs for rare diseases to increase from just 34 to over 500 drugs (6).

ODA In Danger?

Progress is occuring in the rare disease community every day; on August 16th, 2018 a drug called WVE-210201 was granted orphan drug and rare pediatric disease designations by the FDA (5). This approval was granted for the treatment of Duchenne Muscular Dystrophy (DMD), an X linked genetic disease where boys as young as three begin to have muscle weakness and degeneration due to a lack of protein called dystrophin. Those with DMD have a lack of dystrophin because their genetic code has a mutation which results in dystrophin being made incorrectly or not at all. As DMD progresses, affected individuals will require orthosis to support the legs, wheelchairs, and standing frames. Additionally, there is potential for cardiac and respiratory problems to occur.

Currently, corticosteroids are being used to slow the progression of DMD, but have potential for life-threatening side effects (3). WVE-210201is being studied for its potential to restore the production of dystrophin (8). The potential of this drug can be life-changing for patients and research will be able to progress faster with help from the benefits of ODA.

While there are benefits of ODA, within the past year alone there has been conversation around repealing the ODA; to which the response from many rare disease advocates rallied around the need for the Act. The progress shown from its existence should be enough to justify its value in healthcare, but the problem is that there are many misconceptions about the ODA.

Some fear that pharmaceutical companies are abusing the act because seven out of ten major selling drugs are used for multiple conditions including non-orphan diseases but have also been approved to be used for rare diseases. It is important to note that the majority of those sales fall under the non-orphan disease category (4). This means all of the tax credit attributed to the drug can only be applied in costs associated with clinical trials used to study a rare disease so that the tax credit can only be applied in its intended manner.

Other concerns stem from rising healthcare costs where the misconception is that orphan drugs are contributing to this rise. Orphan drug costs are typically less than 10% of the total drug sales in the United States so they are not a major contributor to rising healthcare costs (4). It is also possible insurance companies may not cover costs of drugs that are not FDA approved for treatment and management of a rare disease. Families who could benefit from these drugs are then not able to receive adequate help.

When research is stalled, it takes longer for the FDA to approve drugs – which heavily impacts the rare disease community. For this reason, maintaining the ODA is critical to the rare disease community.

Knowledge Is Power

Being aware of the falsehoods associated with ODA, orphan drugs, and rare diseases is incredibly important to helping promote research and growth of knowledge for the rare disease community. Knowledge is critical for those doing the research to make orphan drugs and understand and treat rare diseases so that progress is actively being made. It is also critical for the general public to be informed for voting and being supportive to those who live with or love someone with rare diseases.

Rare diseases may be rare to the full population, but to a single person affected, it is a major part of their life. Empowering those with rare diseases is the best thing to do for this community. Just because a disease affects a smaller subset of the population does not mean that a smaller proportion of people need to care.

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