Compassionate Use Study Added To House Approps Orders
This article was originally published in The Pink Sheet Daily
Executive Summary
House Appropriations Committee wants to better understand issues with denied expanded access requests and requests Government Accountability Office study of FDA efforts in funding bill.
FDA’s compassionate use policies would come under closer scrutiny in a provision added to the agency’s fiscal year 2015 House appropriations bill, which could inform efforts to pass legislation expanding the program.
But it also appears that FDA’s appropriations could be caught in a partisan fight over waivers for school meal program standards, which may drag out the bill’s consideration on the House floor.
The House Appropriations Committee voted 31-18 May 29 to report the FY 2015 Agriculture, Rural Development, FDA and Related Agencies appropriations bill to the House floor.
Similar to the Senate’s consideration of its bill, much of the House committee mark-up was dominated by agriculture-related issues.
FDA received some attention, however. One of the two additions approved to the committee report indicated concern about “a lack of useful data regarding the number of expanded access … requests made on behalf of patients that are denied by sponsors of investigational products.”
To help committee members understand the scope of the issue, they requested that the Government Accountability Office review how the agency works with stakeholders “to accelerate the approval of innovative, safe and effective medicines and how FDA takes into account safety and efficacy data from expanded access programs,” according to the language added as part of the manager’s package.
The report lengthens an already significant list of FDA-related items included in the draft committee report and appropriations bill, including a new economic analysis of the agency’s proposed generic drug labeling rule and detailed post-marketing surveillance plans for Zogenix Inc.’s Zohydro ER (hydrocodone) (Also see "FDA Directives From House Range From Antibiotics To Zohydro" - Pink Sheet, 28 May, 2014.).
Expanded access, also called compassionate use, allows patients with serious or immediately life-threatening diseases and no comparable alternative therapy access to an investigational drug outside of a clinical trial.
FDA receives many expanded access requests each year and has said it has a liberal policy about it (Also see "Expanded Access Theme Is One Sour Note For Biopharma In “21st Century Cure” Roundtable" - Pink Sheet, 15 May, 2014.).
The agency approves near all the requests it receives, a performance that has failed to mollify critics (Also see "Expanded Access INDs: FDA’s Near-Perfect Approval Rate Underscores Problems, Advocates Say" - Pink Sheet, 31 Mar, 2014.).
Regardless of the source of the denials, some patients initially refused access to experimental drugs have been able to grab media attention and reverse the decisions. A recent example involved Chimerix Inc. and its antiviral drug brincidofovir.
A critically ill 7-year-old boy wanted expanded access to the drug, but was denied. After the public outcry, FDA and the company agreed on a 20-patient open-label trial of the drug that would include the boy (Also see "Chimerix And FDA’s Compassionate-Use Solution" - Pink Sheet, 26 Mar, 2014.).
The new report language may be in part a response to such headlines.
The attention also may generate momentum for pending legislation that could make it easier for patients to use the expanded-access program.
Rep. Morgan Griffith, R-Va., has introduced a bill, H.R. 4475, which would declare that the Food, Drug and Cosmetic Act and Public Health Service Act cannot restrict FDA from preventing or restricting the manufacture, importation, distribution or sale of investigational drugs or devices for terminally ill patients.
The bill has not received any action yet, but could become part of legislation expected from the House Energy and Commerce Committee’s 21st Century Cures Initiative (Also see "Congress Dives Into Biomedical Innovation: Will It Drown In A Deep Sea Of Competing Needs?" - Pink Sheet, 12 May, 2014.).
Ensure Compliance With Scientific Integrity Policy, Committee Says
FDA also would have to ensure it is following a White House policy to ensure the best scientific experts are engaged to help make decisions, although there were concerns about the implications of the language on the agency.
Rep. Tom Cole, R-Okla., proposed adding report language directing Commissioner Margaret Hamburg to ensure all agency centers comply with FDA’s scientific integrity policy and use it “to guide their policy and regulatory decisions.”
A presidential memorandum directed federal agencies to ensure scientific integrity in decision making. FDA adopted its policy in 2012 (Also see "FDA Develops Scientific Integrity Guide For Staff" - Medtech Insight, 16 Jan, 2012.).
The language was inadvertently left out of the original draft committee report. Cole said there was no hidden agenda and that he wanted to make sure FDA found the best experts to inform scientific decisions.
Rep. Sam Farr, D-Calif., questioned the broad approach of the amendment and worried about its unintended consequences. He asked Republicans to work with him to tweak the language.
There also was a concern the change could impact the biologics sector and that it could require disclosure of proprietary information.
FDA likely would argue that it already uses the best science available to help make decisions. But the agency also has had its judgments questioned.
Department of Health and Human Services Secretary Kathleen Sebelius overruled FDA’s decision to allow Teva Pharmaceutical Industries Ltd.’s Plan B One-Step to be sold over-the-counter without restrictions (Also see "Legal Experts Debate FDA Independence After Plan B Fallout" - Pink Sheet, 6 Nov, 2013.).
Several members of Congress also have repeatedly questioned FDA’s decision to approve Zohydro without abuse-deterrent properties (Also see "Abuse-Deterrent Testimony: Hamburg Defends Opioid Policy On The Hill" - Pink Sheet, 14 Mar, 2014.).
School Meal Waiver Could Muddy Floor Action
In the midst of the new FDA mandates is the possibility of a bumpy ride for the appropriations bill once it reaches the House floor.
The legislation includes a provision allowing school districts to receive a temporary waiver from compliance with nutrition standards for school breakfast and lunch programs, if the districts can verify a net loss from food service operations.
Democrats strongly opposed the measure, along with First Lady Michelle Obama, who published an editorial
in The New York Times May 28 advocating against the change.
Democrats indicated they were otherwise mostly pleased with the bill’s contents (Also see "FDA’s Funding Bridle: House Demands Opioid Guidance, Limits Compounding Boost" - Pink Sheet, 20 May, 2014.).
Farr proposed an amendment striking the language. Just before it was defeated, he warned that leaving the waiver section in the bill would force Democrats to oppose it. He also said it would result in several procedural moves once it reached the House floor.
Republicans should be able to pass the bill regardless, but the issue could create problems during the conference committee with the Democratically-controlled Senate, which already disagrees with the House on how funds should be allocated to some FDA centers.
The Senate Appropriations Committee moved its Agriculture, Rural Development, FDA and Related Agencies appropriations bill to the floor May 22 (Also see "FDA Opioid Policy: Senate Takes A Speak Softly Approach" - Pink Sheet, 22 May, 2014.).