Orphan Drug Incentives Would Go Further Than Regulatory Reform - Reardan

Eleos VP-Regulatory Affairs Dayton Reardan suggested that instead of focusing on problems with FDA's review process for rare disease drugs, government should create more development incentives in order to bring more drugs for rare diseases to market.

In fact, Reardan, who has been involved in several orphan drug registrations, said agency staff is flexible in its review of orphan drug applications.

"I think within the division most people are flexible and receptive to most approaches in the clinical trial arena," Reardan said in an interview.

That was contrary to nearly all the presenters at an FDA public hearing June 29 and 30 on the treatment of rare diseases. Most said the agency was inflexible when considering study designs and other characteristics of orphan drug applications (see ¹ (Also see "Orphan Drug Reviews: Industry Wants FDA To Move Out Of Its Comfort Zone" - Pink Sheet, 5 Jul, 2010.)).

Reardan said providing more intellectual property protection will go a long way toward bringing more companies to the sector. He did agree with other speakers when he said additional regulation will not help unless the registration path is less risky or substantially clarified.

Longer Patents and Exclusivity Needed

Market exclusivity under the Orphan Drug Act is seven years. Reardan said the number should increase to 10 to 12 years not only to align the U.S. standard with Europe, but also to give companies more time to recover development costs.

"The recent legislation around biosimilars is talking about 12 years, so why are orphan drugs languishing at seven?" he said during the meeting.

The length of orphan drug exclusivity relative to biologic exclusivity was a key concern for the National Organization for Rare Disorders, but it was unable to get the extra two-year "delta" it had been seeking (² ).

Short-term prospects for any legislative changes to address the issue appear dim, but Lundbeck VP- Medical Affairs Mark Weinberg also said the U.S. should consider adding to its process a European policy of adding an evaluation to the endpoint after 10 years of exclusivity to determine if it should remain intact.

Lengthening the patent term for orphan drugs would bring more development, Reardan said. Drugs receive 20 years of protection from filing, but he said real protection is more like seven to 10 years.

Patent term restoration legislation adds an additional five years, but the law could be improved, Reardan said.

"It would be great if patents, their term, could start when the drug is registered," he said.

Reardan, along with the Biotechnology Industry Organization, also called for increased funding for the Orphan Drug Grant Program.

The program has not been appropriated all the money that has been authorized for it, Reardan said. It also has not received funding increases equal with inflation, BIO said in a written statement.

In May, FDA offered orphan drug status and the grant program, as well as fast-track reviews, as incentives to spur development of a new coral snake antivenin. Legacy supplies of the vaccine are running out and there is no alternative approved by the agency (³ 'The Pink Sheet,' May 24, 2010).

A national program providing tax credits for orphan product angel investors also would entice more money to enter than just relying on individual state programs, Reardan said.

"I think all of us know, and not to be crass, but it is all about money," he said. "If there's no money, there's no research, there's no development."

- Derrick Gingery ( ⁴ [email protected] )