Orphan Drugs: The Need for More Research and Development

By Potvin, Fern R. | The Exceptional Parent, August 1998 | Go to article overview
Save to active project

Orphan Drugs: The Need for More Research and Development


Potvin, Fern R., The Exceptional Parent


Imagine that you are the manager of a major league baseball team, about to start the first game of the World Series. As your team prepares to take the field, you walk to home plate to meet with the opposing manager and the umpires to go over the ground rules for the game. Then the umpire tells you about some rule changes that will apply only to your team:

* Your team will be allowed only two outs per inning, instead of three.

* Each of your players will get only two strikes per out, instead of three. The other team can play by the established rules of the game, but your team must play by these new rules. As you shake your head in bewilderment the thought hits you: the ground rules aren't the same, the playing field isn't level, and it's just not fair!

That is precisely the situation for companies developing drugs based on naturally occurring compounds which are also sold as nutritional supplements. Such companies are being forced to compete in an environment where they are hard-pressed to recover their investment of time and money, much less make a profit that could fund additional research. Many may choose instead to focus their efforts on developing new compounds. It is a circumstance that must be addressed and we believe that you, the readers of Exceptional Parent, because of your strength as advocates for your families and other loved ones, are uniquely positioned to help correct this inequitable situation.

For the 20 million people with rare disorders, and millions of others with other debilitating illnesses, this is not a matter of economics or bottom-line profits. It is about reducing or even eliminating suffering and improving the quality of life. We believe that companies must be provided with rules for development and marketing of new drugs that apply equally to everyone, and sufficient incentives to justify investment in, and creation of, new, safe, and effective products.

It was not until after Congress passed the Orphan Drug Act in 1983 that many new treatments and therapies were developed for a variety of rare disorders. Congress wrote the law to give drug developers total market exclusivity for seven years as both incentive and reward for investing the enormous amounts of time and money it takes to develop new products with limited lifetime sales potential, seek approval for them from the Food and Drug Administration (FDA), and bring them to market. Without such exclusivity, pharmaceutical companies would have no way of recovering their investment in drugs for rare disorders. (See the article in the July issue of Exceptional Parent for a more complete explanation of the Orphan Drug Act.) But the incentives of the Orphan Drug Act alone cannot eliminate all the barriers restricting research and development of treatments based on naturally-occurring compounds and other non-patentable entities.

Supplements and regulation

Although the need for vitamins, minerals, and other trace elements in the human diet has been recognized anecdotally for generations, it has been only about a century since vitamin A, the first vitamin to be isolated, was identified. Since that time, advances in microscopy, human biology, and nutrition have fostered additional identification of and research into nutrients.

Within the last 35 years or so, the exponential growth of scientific knowledge from advances in cell physiology, genetics, and microbiology has enabled researchers not only to identify nutrients but also to learn how they work within the cell. Concurrently, other researchers have been studying cell metabolism, sub-cellular particles, and the underlying systems of human life. By understanding how nutrients act in the body, scientists can investigate the properties that might serve as treatments for a variety of disorders. Researchers have learned, for example, that the symptoms of some abnormal metabolic conditions can be alleviated by naturally-occurring compounds.

The rest of this article is only available to active members of Questia

Sign up now for a free, 1-day trial and receive full access to:

  • Questia's entire collection
  • Automatic bibliography creation
  • More helpful research tools like notes, citations, and highlights
  • Ad-free environment

Already a member? Log in now.

Notes for this article

Add a new note
If you are trying to select text to create highlights or citations, remember that you must now click or tap on the first word, and then click or tap on the last word.
Loading One moment ...
Project items
Notes
Cite this article

Cited article

Style
Citations are available only to our active members.
Sign up now to cite pages or passages in MLA, APA and Chicago citation styles.

Cited article

Orphan Drugs: The Need for More Research and Development
Settings

Settings

Typeface
Text size Smaller Larger
Search within

Search within this article

Look up

Look up a word

  • Dictionary
  • Thesaurus
Please submit a word or phrase above.
Print this page

Print this page

Why can't I print more than one page at a time?

While we understand printed pages are helpful to our users, this limitation is necessary to help protect our publishers' copyrighted material and prevent its unlawful distribution. We are sorry for any inconvenience.
Full screen

matching results for page

Cited passage

Style
Citations are available only to our active members.
Sign up now to cite pages or passages in MLA, APA and Chicago citation styles.

Cited passage

Welcome to the new Questia Reader

The Questia Reader has been updated to provide you with an even better online reading experience.  It is now 100% Responsive, which means you can read our books and articles on any sized device you wish.  All of your favorite tools like notes, highlights, and citations are still here, but the way you select text has been updated to be easier to use, especially on touchscreen devices.  Here's how:

1. Click or tap the first word you want to select.
2. Click or tap the last word you want to select.

OK, got it!

Thanks for trying Questia!

Please continue trying out our research tools, but please note, full functionality is available only to our active members.

Your work will be lost once you leave this Web page.

For full access in an ad-free environment, sign up now for a FREE, 1-day trial.

Already a member? Log in now.

Are you sure you want to delete this highlight?