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Push is on to Expand FDA's Accelerated Approval Pathway

By Mari Serebrov

Washington Editor

Legislation to broaden the accelerated approval path is being gussied up for its Senate debut within the next few months.

The Transforming the Regulatory Environment to Accelerate Access to Treatments Act, or the TREAT Act, would expand accelerated approval to more drugs for unmet needs in serious or life-threatening diseases or conditions.

The Biotechnology Industry Organization (BIO), which is working with Sen. Kay Hagan (D-N.C.) on the bill, had originally proposed a separate "progressive approval" path that would give patients access to promising new therapies before final approval. The proposed path was seen as a way to get life-saving new drugs to a subset of patients, based on specific biomarkers, before the FDA determined the drug was safe and efficacious for all patients.

But widening the existing accelerated path, instead of creating a new path, would enable the FDA to implement the necessary changes quickly and more smoothly, Sara Radcliffe, BIO's executive vice president of health, said during a press briefing Monday.

While expanding the drugs that might be eligible for accelerated approval, the bill would not change safety standards, place additional regulatory strain on the FDA or permit undue influence on reimbursement decisions, BIO CEO Jim Greenwood said.

Hagan hopes to introduce the legislation early this year, according to her office.

Meanwhile, Greenwood said BIO is trying to reach a consensus with congressional members, the FDA and the Pharmaceutical Research and Manufacturers of America on how best to present the bill. For instance, should the legislation be considered along with the PDUFA reauthorization package that Congress will take up next month? (See BioWorld Today, Jan. 18, 2012.)

In addition to expanding accelerated approval, the TREAT Act includes many of the other FDA reforms BIO proposed last year as part of its five-year advocacy plan to reform the investment and regulatory environment for biotech innovation. (See BioWorld Today, June 27, 2011, and June 30, 2011.)

The proposed FDA reforms, based on advances in regulatory science, are aimed at making the agency more predictable. Its current unpredictability is often cited as a major obstacle to investment in small biotechs.

Part of that unpredictability stems from the FDA's focus on risk rather than a balance of risk to benefit, Greenwood said. While industry is seeing some improvement in agency approval times, the FDA still needs to take a more realistic approach to risk-benefit and work on its transparency, he added.

In the current economic climate, Greenwood predicted there will be a lot of pressure to cut federal health care spending over the next several years, given that other line items in the federal budget will be relatively flat. But cutting spending on drugs, hospitals and medical devices would only reduce quality of care and the incentives to innovate, Greenwood said.

"The real solution here is to have fewer sick people," he noted. Reaching that goal will require continuing reform at the FDA so its approval process is predictable and expeditious.

Removing unnecessary hurdles to approval would make innovative biotechs a more attractive investment target. With more financial security, biotechs could focus on drug R&D, which could lead to a significant reduction in health care expenses over time, Greenwood said.

Besides the TREAT Act, BIO is working with other congressional delegates on bipartisan legislation addressing capital formation issues, Greenwood said. At the top of its wish list is reauthorization of the Therapeutic Discovery Tax Credit, which was included in the 2010 Patient Protection and Affordable Care Act and which provided $1 billion to nearly 3,000 companies. (See BioWorld Today, Nov. 1, 2010, and Nov. 4, 2010.)

H.R. 1988 would extend the tax credit through fiscal 2017, providing $1 billion in tax credits and grants per fiscal year to small U.S. biotechs that show significant growth potential in new and cost-saving therapies. The bill has been in committee since being introduced in the House in May 2011. (See BioWorld Today, May 27, 2011.)

Although President Barack Obama voiced support for making the tax credit permanent in his 2012 budget proposal, BIO acknowledged that getting the bill passed with no cost offset could be challenging when Congress is focused on cutting the deficit. (See BioWorld Today, Feb. 16, 2011.)