CBI Scene Blog

An Analysis on Effectiveness Evidence in FDA's Approval of Orphan Drugs

Posted by Stephanie V on Jun 20, 2014 2:08:00 PM

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The FDA has a commitment to getting products to treat orphan diseases on the market as quickly as possible. Some of the people who have these diseases have no o

ther options for treatment, and a new drug could offer them their only hope of survival.

Is the Current Process Working in Order to Get Orphan Products on the Market Quickly?

In a word, yes. While the FDA still uses its same safety and testing requirements on orphan drugs that it uses on all other drugs that come before it for approval, it does have some leeway in the approval process.

Some orphan diseases only have a few hundred or a few thousand people suffering from them. These are not large enough numbers for a traditional FDA study cycle, which typically tests thousands of people in multiple trials.

Only Small Numbers of People Are Required to Test Orphan Drugs, Because Only Small Numbers are Typically Available

With orphan products, the drugs are often tested on only a small number of people, because there aren't enough people with the condition to do a traditional clinical trial. Also, some therapies may be approved after only one trial, due to the critical nature of getting a treatment to market for the few people who have a particular orphan disease.

The process of getting these orphan drugs to market quickly is working. However, the FDA has not made it widely known public knowledge or official policy that there is leeway in the approval process of such drugs. It is written into its 1983 orphan drug guidelines, but very little else has been said about the orphan drug approval process since then.

What Major Obstacles Does the FDA Face that Hinders These Types of Unique Therapies Getting on the Market Quickly?

The FDA actually does a really good job with getting orphan drugs onto the market quickly. The FDA realizes there is a need for such therapies, and has a long list of approved drugs for conditions that may not even affect 100 people worldwide in some instances. If the FDA faces any hindrances in getting these drugs to market quickly, those obstacles lie in two areas:

  • Locating sufferers of very rare diseases and convincing them to be brave pioneers in the testing of new therapies that may have had no human trials yet.
  • Lack of knowledge of some of the rarest orphan diseases. A therapy cannot be developed unless there is a knowledge that the disease exists, and a demand among the small community of sufferers for something to be done to help treat them. Sufferers of orphan diseases and their families oftentimes have to truly be their own advocates in getting treatments developed and on the market.

How Can the FDA, Pharma, and Healthcare Organizations Better Leverage Partnerships in Order to Get Rare Disease Products/Therapies on the Market Faster?

The federal government already provides incentives to the FDA and pharmaceutical companies for developing drugs for orphan diseases and for fast-tracking their approval. Grant money is even provided to healthcare organizations and pharmaceutical companies to do the necessary research to develop treatments for rare diseases. If there are ways in which this process can be improved, it lies in:

  • Identification of more rare diseases that may have not received a lot or (or any) attention by pharmaceutical companies.
  • Better outreach to patients of orphan diseases and their families to determine their wants and needs in terms of the development of treatments. Better patient outreach can lead to better patient advocacy in getting new orphan drugs approved.
  • More effort placed on healthcare organizations to identify sufferers of orphan diseases and invite them to participate in clinical trials of new drugs.


While the FDA has a process for fast-tracking orphan drugs to market without sacrificing its quality or safety standards in the approval of such drugs, some pharmaceutical companies are not aware of this, because the fast-track program is not well-publicized. Further, some populations of orphan disease sufferers are so small that it is only possible to test a handful before moving a drug into the FDA approval process. With better communication between the FDA, pharmaceutical companies, and healthcare organizations, more people with orphan diseases can be identified, more can participate in clinical trials,and more pharmaceutical companies will work on developing these drugs, knowing they can be fast-tracked to approval through the FDA's leeway in approval guidelines for this class of drugs.

To learn more about how the FDA, pharma, and healthcare organizations can work more closer to bring speed to therapy for rare diseases, join us at NORD's 2014 Rare Disease and Orphan Products, October 21-22, 2014.

Click on the button below to learn more about this industry-leading event!

NORD 2014

Topics: Patient Access