ASCRS supports efforts to ensure the safety and sterility of compounded drugs. The recently enacted H.R. 3204, the Drug Quality and Security Act (P.L. 113-54), seeks to improve safety of compounded drugs. H.R. 3204 represents a compromise between the Health, Education, Labor, and Pensions (HELP) and House Energy and Commerce Committees. The bill creates a new category of pharmacies—outsourcing facilities—that would voluntarily submit to stricter FDA oversight.
The bill’s sponsors intended for traditional compounding for patient-specific prescriptions and limited office use to continue to operate under current law, however, the medical community continues to have concerns with the affect this new law could have.
Most importantly for ophthalmology, the bill does not include repackaging in the compounding definition and leaves it to the FDA to determine how repackaged drugs, such as Avastin, would be regulated.
FDA has now released draft guidance for 503A traditional pharmacies and 503B outsourcing facilities, which states that FDA will allow both traditional pharmacies and outsourcing facilities to repackage biologics, including Avastin for ophthalmic use.
However, ASCRS and other ophthalmology stakeholder groups have concerns with the Beyond Use Dates (BUD) that the draft guidance lays out. The draft guidance provided for a BUD of four hours or equal to the time within which the opened product is to be used as specified on the approved labeling, whichever is shorter, for traditional compounders. The BUD can be extended to 24 hours if microbial challenge studies are performed. The same BUDs apply to outsourcing facilities, but can be extended to five days if the outsourcing facility conducts adequate compatibility studies on the container closure system (the syringe) to ensure product integrity.
In ASCRS’ discussions with providers and compounding stakeholders, the conclusion has been that the BUD timeframe is extremely short. Specifically, there are concerns that the 5-day expiration date will severely limit the use of Avastin since it would not be enough time to conduct the necessary sterility testing. ASCRS is now working in a coalition with other ophthalmology groups to notify the FDA of the issues surrounding these short BUD timeframes and asking these to be extended in the final guidance document. ASCRS has also submitted comments on the draft guidance recommending longer BUD timeframes.
Following ASCRS advocacy, Reps. Morgan Griffith (R-VA), Raul Ruiz, MD (D-CA), Phil Roe, MD (R-TN), and Paul Tonko (D-NY) spearheaded a letter to the FDA, which was co-signed by 29 other House members, asking them to modify its “one-size-fits-all” policy for beyond use dates (BUDs) and develop policies appropriate for each drug. ASCRS and others in the ophthalmic community are concerned the draft guidance would limit access to repackaged biologics, such as Avastin, because the BUDs are too short to ensure the necessary sterility testing for the drug. A similar Senate letter led by Sens. Rand Paul, MD (R-KY), and Sherrod Brown (D-OH) is expected soon.
21st Century Cures- Accelerating Access to Drugs and Devices
ASCRS has long supported efforts to improve access to new drugs and devices, maintaining that the current Food and Drug Administration (FDA) approval process is outdated and overly onerous. Over the last several years, ASCRS has been actively involved in accelerating access to new drugs and devices and increasing focus on improving regulatory predictability necessary for innovation.
ASCRS continues to urge Congress to be vigilant about any measures that would inappropriately increase the regulatory burden for medical device innovation, hurt America’s competitive advantage, and delay or deny appropriate care for patients. Therefore, we continue to encourage the passage of much-needed reforms that will address the delays in the approval of new devices and drugs.
21st Century Cures Initiative
In 2014, the House Energy and Commerce (E&C) Committee created a new, bipartisan initiative—21st Century Cures—aimed at identifying ways that Congress might bolster American medical innovation—including speeding access to new drugs and devices. The initiative, led by full committee Chairman Fred Upton (R-MI) and Rep. Diana DeGette (D-CO), is a comprehensive effort to investigate the current system of federally-supported research, drug development, clinical trials, available capital and patient experience to determine where Congress can most effectively make legislative changes or direct funding. Throughout 2014, the committee held a series of roundtables and hearings exploring different aspects of the issue and met with stakeholder groups—including ASCRS—to provide input.
In early 2015, Chairman Upton released a discussion draft that outlined several policy initiatives including: developing a process for qualifying and using surrogate endpoints in clinical trials to shorten the approval process; modernizing off-label communication regulations; developing a Medical Product Innovation Advisory Commission similar to the Medicare Payment Advisory Commission (MedPAC); improving clinical trial data sharing through registries; and the Advancing Innovation in Medicine (AIM) Act. In addition, Senate Health, Education, Labor and Pensions (HELP) Committee Chairman Lamar Alexander (R-TN) and Ranking Member Patty Murray (D-WA) have begun a complementary initiative in the Senate. ASCRS joined with the Alliance of Specialty Medicine to comment and provide input on the Energy and Commerce Committee’s discussion draft and provided feedback to the HELP committee on their initial exploratory questions on the issue.
In May 2015, the Energy and Commerce Committe passed H.R. 6, the 21st Century Cures Act. ASCRS provided comments supporting several provisions of the bill including enhancing combined products review, expanding sources of valid scientific evidence to include peer-reviewed journals and registry data, revisions to the advisory committee process and creation of a Medicare pharmaceutical and technology ombudsman. ASCRS recommended revisions to provisions relating to off-lable use, and expressed caution with some EHR interoperability proposals.