Senate's User Fee Bill Largely Steers Clear Of Controversy
Executive Summary
HELP Committee manager’s amendment lacks provisions on such hot-button issues as off-label drug promotion, REMS abuses and right-to-try legislation.
It appears as if Democrat and Republican lawmakers wanted to avoid any politically charged add-ons with the upcoming US FDA user fee program reauthorizations, as several topics that have received congressional attention were left out of the Senate Health, Education, Labor and Pensions (HELP) Committee’s manager's package.
The manager's amendment – released May 8 in advance of the committee's May 10 markup of the user fee bill – did not include legislative additions related to such hot-button issues as off-label drug promotion, abuse of risk evaluation and mitigation strategies (REMS), right-to-try legislation, and allowance of drug importation, suggesting the lack of a bipartisan consensus on these issues.
Senators still could bring up these topics as individual amendments at the committee markup, but they would likely be undesired and fail to gain much traction.
Lawmakers at previous Senate and House user fee hearings have stressed the bipartisan nature of the agreements, which must pass Congress and be signed into law by President Donald Trump to keep FDA’s prescription and generic drug, biosimilar and medical device review programs running smoothly.
Any major amendments or new provisions introduced at the last minute could inject unwanted complications into debate over the must-pass measure, potentially impacting its ability to move through Congress smoothly and quickly.
Measures aimed at addressing the abuse of REMS to keep generics off the market have received bipartisan support in the past, albeit with more attention from Democrats. (Also see "FDA User Fee Hearing Hijacked By US Health Care Reform Arguments" - Pink Sheet, 21 Mar, 2017.) FDA Center for Drug Research and Evaluation Director Janet Woodcock has testified that REMS abuse remains problematic, but the issue did not make it into the user fee bill, suggesting a lack of a consensus on a solution. (Also see "REMS Reform Seems Distant Goal For Generics After Limited Support At US House Hearing" - Pink Sheet, 2 Mar, 2017.)
The topic looks as if will continue to be debated outside of the user fee reauthorization with the reintroduction of the Creating and Restoring Equal Access to Equivalent Samples (CREATES) Act (S 974) and the Fair Access for Safe and Timely (FAST) Generics Act (HR 2051). (Also see "REMS Could Block ANDAs With 'Legitimate Business Justification' In Senate Bill" - Pink Sheet, 30 Apr, 2017.)
Drug importation advocates already faced one political rebuke this year, when the Senate voted 52-46 against an amendment from Sen. Amy Klobuchar, D-MN, proposing to establish a deficit-neutral reserve fund to lower prescription drug prices by importing drugs from Canada.
Right-to-try legislation also has faced resistance, not only from FDA and industry but also influential patient advocacy groups. (Also see "'Right To Try' And PDUFA VI: Patient Advocates, Industry Fight Senate Add-On" - Pink Sheet, 2 May, 2017.)
However, the manager’s package does include new measures aimed at earlier planning and completion of pediatric studies under the Pediatric Research Equity Act and Best Pharmaceuticals for Children Act. (See sidebar for story.)
Below is a chart of current bills that have not yet found their way into the user fee reauthorization package but still could be the focus of congressional discussions inside or outside of the FDA bill’s confines.
PDUFA Reauthorization: What Could Have Been Or What Might Still Be? | |
Bill | Purpose |
Clinical Trials | |
Enhanced Clinical Trial Design Act (S 1048) | Expands clinical trial eligibility to include high-risk patients; Improves institutional review board assessment of individual expanded access protocols; Allows sponsors to make expanded access policies available during Phase I for drugs going through the breakthrough therapy, fast track or regenerative advanced therapy pathways |
Drug Access | |
Improving Access to Affordable Prescription Drugs Act (HR 1776, S 771) | Allows Medicare to negotiate drug prices; Allows drug importation; Makes changes in awards of 5-year new chemical entity and 3-year Waxman/Hatch exclusivity; Reduces exclusivity term for novel biologics from 12 to 7 years; Makes pay-for-delay agreements illegal and allows FDA to take away 180-day generic exclusivity from ANDA sponsors that enter into such arrangements |
Drug Promotion | |
Pharmaceutical Information Exchange Act (HR 2026) | Clarifies scope of permitted health care economic and scientific information communications between biopharma manufacturers and population health decision-makers to encompass competent and reliable scientific evidence related to an investigational new drug or an investigational use of an approved drug |
Generics | |
Creating and Restoring Equal Access to Equivalent Samples (CREATES) Act (S.974, H.R.2212) Fair Access for Safe and Timely (FAST) Generics Act (H.R.2051) | Seeks to prohibit brand companies from using Risk Evaluation and Mitigation Strategies and other restricted distribution programs to deny generic companies access to samples for bioequivalence testing; Establishes a legal remedy for generic companies that have been unable to obtain product samples |
Lower Drug Costs Through Competition Act (H.R.749) Increasing Competition in Pharmaceuticals Act (S.297) | Creates a180-day (House) or 150-day (Senate) priority review pathway for certain ANDAs; Establishes priority review voucher program for generics; Requires new essential clinical investigations for tropical disease NDAs to be eligible for a priority review voucher |
Importation | |
Affordable and Safe Prescription Drug Importation Act (HR 1245, S 469) | Instructs the secretary of Health and Human Services to issue regulations allowing importation of qualifying prescription drugs from licensed Canadian sellers; Authorizes HHS secretary to permit importation from countries in the Organization for Economic Co-operation and Development with standards for drug approval and sale comparable to those in US |
Opioids | |
Abuse-Deterrent Opioids Plan for Tomorrow (HR 2025) | Prevents 3-year Waxman/Hatch exclusivity for abuse-deterrent labeling claims from blocking similar claims for another product if the latter has “meaningful technological differences”; Requires FDA to issue guidance on the award and scope of 3-year exclusivity for abuse-deterrent formulations |
Pediatrics | |
Research to Accelerate Cures and Equity (RACE) for Children (S 456, HR 1231) | Requires pediatric studies based upon a cancer drug’s molecular target; Eliminates Pediatric Research Equity Act study exemption for drugs approved in rare adult cancers |
Rare Disease | |
Orphan Product Extensions Now Accelerating Cures and Treatments Act (OPEN Act) (HR 1223) | Provides additional six months of marketing exclusivity on currently approved drugs that are subsequently studied for, and approved in, rare diseases |
Right To Try | |
Trickett Wendler Right to Try Act of 2017 (S 204) Right To Try Act (HR 878) | Directs FDA not to interfere with “right to try” state laws and allows terminally ill patients to access unapproved treatments once they have cleared early-stage safety studies; Prevents FDA from using data reported during an expanded access program to delay or adversely impact the review or approval of the investigational drug |