Mr. Chairman and members of the Committee, the National Organization for Rare Disorders (NORD) is the consumer organization that worked for passage of the Orphan Drug Act (ODA) of 1983, and we continue to closely monitor its implementation today. NORD represents approximately 25 million Americans with more than 6,000 rare “orphan diseases,” and we are very pleased to be here today. Thank you.
As you know, the Orphan Drug Act is one of the most successful pieces of health legislation ever enacted by Congress. Today, approximately 1,000 orphan drugs have been designated by the FDA, and over 200 of them have been approved for marketing in the United States. Recognizing the public health impact of the American law, other nations have implemented orphan drug statutes including Japan, Singapore, Australia, and the entire European Union.
It is extremely important that Congress not undermine the intent of the law, which is to encourage the commercial development of treatments for small populations of patients. We do not support H.R. 4242, the Orphan Drug Innovation Act, for many reasons, and we urge the committee not to approve the legislation for further consideration by Congress. Let me explain why we do not support H.R. 4242.
Early in the evolution of the American Orphan Drug Act, it became necessary to define the words “same” and “different”. In other words, as defined by FDAs carefully crafted regulations, if a manufacturer of a similar orphan drug can prove that their drug is chemically different or clinically superior, even though it contains the same active ingredients as the original orphan drug, the FDA will approve it for marketing.
There are several ways to prove that a drug is “clinically superior”: either by providing FDA with scientific data proving that it is either safer or more effective, or that it represents a “major contribution to patient care.” The latter is usually an obvious improvement, such as developing an oral version of an injectable drug -- so that patients no longer have to suffer painful injections -- or developing a long-acting version of a drug that must otherwise be taken several times each day, for example. Thus, the orphan drug regulation defining “same” and “different” not only promotes, but encourages development of new improved versions of marketed orphan drugs while PRESERVING the chief incentive of the ODA.
Since most orphan drugs have no competition because companies are generally not interested in investing the huge sums necessary for research and development of a drug that will have a very small market, the ODA offers an important incentive to encourage orphan drug innovation. Companies can receive seven years of exclusive marketing rights for both the innovator drug and the clinically superior follow-on drug.
Mr. Chairman, from time to time there are orphan drug “races” when more than one company is developing the same orphan drug for the same disease, and the law purposely creates a winner-take-all contest. This is the very core of the success of the ODA because it prevents competition for seven years, and ensures that a manufacturer of an FDA-approved orphan drug will recoup its investment and make a profit. But losers of the race sometimes ask Congress to change the law because they want an exception for their drug. Thankfully Congress has been wise enough not to allow this, knowing that tinkering with the Act could destroy it.
In this case, Congressman Thornberry’s bill aims to redefine FDA’s definition of “same” and “different”, and to codify it into law, based on the mistaken belief that people with rare diseases do not already have access to clinically superior orphan drugs. However, H.R. 4242 will NOT enhance patient choice because current regulations not only permit, but encourage competition when a therapeutic advantage can be scientifically proven.
We believe that H.R. 4242 would disincentivize companies to develop clinically superior orphan drugs and biologics, and it would allow companies to seek approval for clinically inferior products. Moreover, H.R. 4242 would reduce the exclusivity of orphan drugs and biologics that have demonstrated they are clinically superior, because it would limit exclusivity to the innovation that enabled a clinically superior product to reach the market.
There are no benefits to patients if H.R. 4242 becomes law. There are only benefits to companies that want to break the innovators exclusivity, but that exclusivity is the very backbone of the Orphan Drug Act which has since 1983 saved the lives of millions, and improved the quality of life for countless others.
The current regulations, which are based on sound scientific knowledge and common sense, were written to promote innovation and to allow consumers access to clinically superior orphan drugs. They are fair to consumers and fair to companies. The ODA is good public health policy and continues to be one of the most successful pieces of health legislation ever written.
We are profoundly grateful to Congress for enacting the Orphan Drug Act, and for preserving its integrity since 1983. If you write the Thornberry bill into stone, it will require an act of Congress to change it when new medical technologies emerge in the future. If you leave the orphan drug regulations alone, the FDA can easily fine tune the rules, if and when that becomes necessary.
Mr. Chairman and members of the Committee, “if it ain’t broke, don’t fix it.” We urge you NOT to enact H.R.4242.
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