FDA appears just as nervous as the National Organization for Rare Disorders about what Congress would do if asked to tweak the Orphan Drug Act.

Still, rare disease advocates and drug sponsors continue to say changes are necessary and asked the patient community to increase its pressure for reforms.

CDER Director Janet Woodcock said the current political climate likely is especially volatile and could bring many unwanted changes that could hurt the celebrated law. Ultimately, it likely is better to leave the Orphan Drug Act alone, even if some minor improvements would help, she said.

“I’m afraid if this Congress blows something up, it might be the wrong object – your foot or something,” Woodcock said Oct. 11 during the U.S. Conference on Rare Diseases and Orphan Products sponsored by DIA and NORD.

“I’m not sure in this political environment … there’s enough rational deliberation that could result in the kind of legislation that we needed to change the legislative framework. I’m afraid people are in a blowing-up mood,” she said.

NORD Wants Administrative Changes

NORD already has articulated the same policy as Woodcock when it comes to the Orphan Drug Act. After saying early this year it was thinking about whether a legislative update was needed for the law, the group decided this summer that legislative changes would open the law to mischief and mayhem.

Instead, the group chose to push for changes within the existing framework to grow rare disease drug development (Also see "NORD Says Orphan Drug Act Is Fine As Is, Wants To Work For Improvements Within System" - Pink Sheet, 29 June, 2011.).

Whether or not changes will be proposed is up for debate, but if FDA and a large patient advocate like NORD oppose the idea, any proposal likely will not move forward.

The reauthorization of the Prescription Drug User Fee Act in 2012 will provide an opportunity to make some adjustments.

The commitment letter FDA negotiated with industry already includes several changes that will affect rare disease drug development, including adding more staff to the CDER and CBER rare disease programs to improve sponsor communication (Also see "PDUFA V Commitment Letter Front-Loads Guidance, Meeting Milestones" - Pink Sheet, 5 September, 2011.).

It also includes plans for public meetings to talk about endpoint selection for rare disease clinical trials and staff training on the review and approval of rare disease drugs (“Also see "PDUFA V Implementation: A Year-By-Year Breakdown" - Pink Sheet, 5 September, 2011.).

Congressional debate on the PDUFA reauthorization will kick into high gear next year, but in the meantime, NORD wants FDA to formally declare its flexibility on orphan drug reviews (Also see "Orphan Drug Reviews: Industry Wants FDA To Move Out Of Its Comfort Zone" - Pink Sheet, 5 July, 2010.).

To make its case, the association showed FDA has a history of allowing alternative trial designs and other development mechanisms (see related story, Also see "Orphan Products Mostly Approved With Regulatory Flexibility, NORD "Catalog" Finds" - Pink Sheet, 17 October, 2011.).

“Throw Away The Old Model,” Crowley Says

Woodcock’s position contrasted with that of John Crowley, chairman and CEO of Amicus Therapeutics, who called on the rare disease community to not be timid and “blow up” the old drug development model.

Crowley said during the conference that Congress will have to make changes, preferably in the next nine to 10 months, which will create a new, better system for drug development.

“We are really, I think, on the dawn of a new age in science and in medicine,” he said. “We have to completely throw away the old model of drug development to take risks, to take chances.”

Crowley said the existing development model is no longer working in part because of an aversion to risk by society. That has contributed to a drop in venture capital investment in the rare disease space, he said.

The pace of development also is inadequate. Crowley said it will take nearly 200 years to produce therapies for half of the existing rare diseases if the current development trends hold.

The ultimate goal should be “to change the risk-benefit ratio that FDA, based on its direction from Congress, and that broadly … people in this country are willing to accept, because otherwise it’s going to take a couple hundred years.”

“I think we all need to think about how we can create the right aggressive legislation to provide the opportunity for FDA to continue to advance its thinking and its culture and group leadership,” he said.

FDA, for its part, is challenging the idea that broad regulatory reform is needed, asking firms to identify specific bottlenecks that need to be widened (see related story, Also see "Regulatory Reform: FDA Pushes Back, Asks For Specific Problems To Fix" - Pink Sheet, 17 October, 2011.).

Crowley suggested potential changes FDA could employ, such as dropping p-value and statistical significance requirements for clinical trials in small populations.

Asking for a new model is in line with comments Crowley made earlier this year, when he asked for more creativity in coming up with potential Orphan Drug Act changes.

He said lengthening the exclusivity period will not attract investors, but a cheaper and faster approval mechanism would boost investor and sponsor interest (Also see "Orphan Drugs Need Easier, Speedier Approval, Not More Exclusivity, CEO Says" - Pink Sheet, 23 May, 2011.).

By Derrick Gingery

Woodcock Says Blowing Up Orphan Drug Act Not Advisable In Current Climate

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