Orphan Drugs: The Need for More Research and Development

Article excerpt

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. …