David Pittman
www.fdanews.com

The Senate has released its draft of a bill on accelerated approval changes, largely mirroring Sen. Kay Hagan's (D-N.C.) TREAT Act and giving a strong indication of what could come in a final FDA user fee bill.

The Transforming the Regulatory Environment to Accelerate Access to Treatments Act, or TREAT Act (S. 2113), mostly clarifies the statute on the FDA's current accelerated approval process but - unlike before - would allow sponsors to request the designation themselves. Such requests would be approved or denied within 60 days. The draft would also give the FDA greater flexibility to approve a drug using surrogate endpoints.

The draft bill, the latest of several floated by the Senate Health, Education, Labor, & Pensions (HELP) Committee, also includes a definition for "breakthrough therapy," a type of innovative drug currently bestowed fast-track status for showing a substantial improvement over existing therapies.

CDER Director Janet Woodcock has told lawmakers that even those within the FDA could use clarification on an accelerated approval definition. But the FDA has "concerns" with such legislation, as it doesn't want standards of safety or efficacy for accelerated approval lowered, she added (DID, March 9).

The draft bill mandates that within one year the FDA issue draft guidance on the accelerated approval and fast-track processes. Woodcock said the FDA is already writing guidance clarifying surrogate and clinical endpoints used in the current accelerated approval process.

Supporters of the bill said a lack of clarity on the process has led to some drug classes not realizing the same benefits that HIV and cancer therapies have gotten from the accelerated approval pathway. While the process was initially developed with HIV drugs in mind, greater flexibility would allow more use of the pathway for other products.

"I think it became pretty apparent to folks on the Hill, or in FDA or even in industry, that ... there were several different interpretations," Jeff Allen, executive director of the Friends of Cancer Research told DID.

The accelerated approval changes largely come from a push by the Biotechnology Industry Organization (BIO). But the group has backed off from its original proposal of creating a progressive approach, where a product would be approved for a narrow population and the indication gradually expanded over time as efficacy and safety are shown in additional trials (DID, June 30, 2011).

The TREAT Act encourages the agency to use surrogate endpoints to speed drug development and review. Such a process, used since the early 1990s, isn't written into law now (DID, Feb. 17).

BIO has praised the TREAT Act and companion legislation in the House, the Faster Access to Specialized Treatments (FAST) Act, H.R. 4132. Meanwhile, PhRMA, which hasn't taken an official position, is supportive of the accelerated approval language in the TREAT Act and the discussion draft.

April Markup Still Expected

Meanwhile, the Senate is still on track to markup an omnibus user fee bill by late April with hopes to have a package signed into law by July, an aide told DID Thursday.

The HELP Committee has worked feverishly to release and field feedback on several riders to user fee legislation, including those on drug shortages, antibiotic development incentives and supply chain security (DID, April 2).

The GOP-led House unveiled its draft last month with its accelerated approval section largely mirroring Hagan's TREAT Act as well (DID, March 16).

Because language in the rider appears in the chambers' draft discussion, Allen says it's likely to be included in a final bill.

View the Senate discussion draft on accelerated approval at www.fdanews.com/ext/files/04-06-12-draft.pdf.