Comparing Orphan and Non-Orphan Clinical Trials « Rare Disease Blogs.

Comparing Orphan and Non-Orphan Clinical Trials « Rare Disease Blogs.: "Comparing Orphan and Non-Orphan Clinical Trials

A recent article published in the Journal of the American Medical Association (JAMA) discussed a comparison of clinical trials for orphan vs. non-orphan oncology drugs. The article questioned whether safety and efficacy standards were as stringent in the orphan trials as in the non-orphan trials. A news story circulated after the article was published went considerably beyond the original article in its implications, and NORD felt it important to respond.

This is an important topic for everyone in the rare disease community. NORD President and CEO Peter L. Saltonstall submitted the following comments as a Letter to the Editor of JAMA:

To The Editor, Journal of the American Medical Association:

The article “Characteristics of Clinical Trials to Support Approval of Orphan vs Nonorphan Drugs for Cancer” by Kesselheim et al (JAMA. 2011;305(22):2320-2326. doi: 10.1001/jama.2011.769) presents the predictable finding that pivotal trials for orphan cancer drugs often have fewer patients and use different trial designs than trials for drugs for more prevalent cancers. Rare diseases by definition have small patient populations; trials in such populations will commonly have fewer patients than those for more prevalent diseases. Recruitment of participants for such studies is challenging, and trials of new agents for orphan diseases may, of necessity, be non-randomized and open label.

The article asserts that “safety and efficacy questions have emerged about some of these agents” and offers examples of one orphan drug being removed from the market for safety reasons in the U.S., and two orphan drugs not being reimbursed in the U.K., largely due to high cost. However, we are not aware of a higher rate of safety and efficacy questions for orphan products than for drugs for more common diseases, and the article offers no support for questioning the safety or effectiveness record of orphan drugs.

The rare disease patient community is thankful that the Food and Drug Administration (FDA) and other regulatory agencies recognize the special challenges posed in testing orphan drugs. FDA has shown flexibility in accepting protocols for pivotal trials of orphan drugs, while maintaining its traditionally high standard that all drugs must be proven safe and effective for their intended populations. Furthermore, the law places responsibility on manufacturers to report signals of risk associated with all approved drugs; manufacturers of orphan drugs are required to abide by that requirement just like manufacturers of other drugs.

There are still more than 6,000 rare diseases with no approved medications at all. These diseases affect as many as 30 million Americans. Most of these diseases are chronic, life-threatening or lifespan-reducing. More than half of the patients are infants or children. There is a continuing and critical imperative for innovation to find treatments for patients with rare diseases, while assuring that these treatments are effective and safe for the target populations.

We who advocate for patients with rare diseases believe that more resources are needed to support innovative research. The medical and regulatory communities understand the special challenges inherent in developing treatments for rare diseases. They must also recognize the need for flexibility in clinical trial design and the fact that patients with such disorders are willing to accept reasonable risk in return for hope of effective treatment.

Sincerely,

Peter L. Saltonstall

President and CEO, National Organization for Rare Disorders (NORD)

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