There has been a tremendous amount of attention surrounding drug shortages in the U.S. over the past several months—and rightly so. Some reports coming out have projected that these shortages have already caused deaths. In addition to the Food and Drug Administration’s (FDA) recent guidance about drug shortages, a bipartisan group of Senators, including Senate Health, Education, Labor and Pensions (HELP)Committee Chair Tom Harkin (D-IA) and Ranking Member Mike Enzi (R-WY), recently released a draft bill that would address drug shortages.
Co-sponsors of the legislation also include Senators Alexander, Bennet, Blumenthal, Burr, Casey, Corker, Franken, Hagan, Isakson, Kirk, Klobuchar, Merkley, Mikulski, Murkowski, Murray, and Roberts release a bipartisan Prescription Drug Shortages Draft.
The drug shortage legislation would establish a Task Force to mitigate drug shortages and to prevent future drug shortages through enhanced interagency and intraagency coordination, communication, strategic planning and decision-making. The Task Force would be charged with developing a strategic plan and implementation plan to ensure that drug shortages are considered when FDA initiates a regulatory action that could precipitate a drug shortage or exacerbate an existing drug shortage.
FDA would also have to ensure that there is communication with all relevant stakeholders before any enforcement action or issuance of a warning letter to prevent drug shortages since the action or letter could cause, or exacerbate a shortage of the drug.
The proposed legislation would also impose a number of recordkeeping requirements on FDA and drug manufacturers, which would include a list of the known factors contributing to the drug shortages and a list of steps taken by FDA to prevent or mitigate such shortages. The list would also include the number of establishment inspections or reinspections FDA expedited for the purposes of a drug shortage. The records would also include the names of manufacturers who did not comply with the notification requirement (see FDA guidance).
The legislation also authorizes a study reviewing any findings that drug shortages have led market participants to stockpile affected drugs or sell them at significantly increased prices, the impact of such activities on Federal revenue, and any economic factors that have exacerbated a market for such actions.
New FDA Approval Pathways
The Drug Approval and Patient Access as currently drafted, represents a first step for Congress to reach a consensus policy proposal to accompany the FDA human medical product user fee legislation. As we have also noted, Congress will need to reauthorize several User Fee Acts (PDFUA and MDUFA), and approve several new ones (GDUFA and one for Biosimilars) by this fall. One provision in this bill would allow FDA to expedite the review of a supplement to a new drug application, a generic drug application, or a supplement to a generic drug application it it could “help mitigate or prevent such shortage or expedite an inspection or reinspection of an establishment that could help mitigate or prevent such drug shortage.”
The drug approval legislation would create a “Fast Track Product” for drugs or treatments of a “serious or life-threatening disease or condition” that demonstrates the potential to address unmet medical needs for such a disease or condition. In applying for approval, sponsors of drug applications would have to request such a designation. If designated as a “Fast Track Product,” FDA would be authorized to take steps to expedite the development and review of the application for approval of such product.
The legislation also provides for Accelerated Approval if FDA determines that the product “has an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit, or a clinical endpoint that can be measured earlier than irreversible morbidity or morality, that is reasonably likely to predict an effect on irreversible morbidity or mortality or other clinical benefit, taking into account the severity or rarity of the condition and the availability of alternative treatments.” Sponsors would still have to submit post-approval studies for verification and submit copies of all promotional materials. FDA would have the ability to withdraw approval if such studies are not submitted or do not provide adequate verification.
The Drug Approval and Patient Access bill would also allow FDA to designate a drug as a “Breakthrough Therapy” if the drug is intended, alone or in combination with 1 or more other drugs, to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on 1 or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.
Interestingly, this legislation would also require FDA to issue a guidance document that “describes the policy of FDA regarding the promotion, using the Internet (including social media), of medical products regulated by FDA.”
While there has been much scrutiny surrounding the involvement of “conflicted” experts on FDA Advisory committees, this legislation would authorize FDA to consult with external experts on topics including “rare diseases, targeted therapies, and genetic targeting of treatments.” The legislation recognizes that such consultation is necessary for promoting efficiency and informing the review by FDA. The provisions do not specifically state “industry,” but also do not prohibit such. Topics for consultation include:
- Rare diseases
- Severity of rare diseases
- The unmet medical need associated with rare diseases
- The willingness and ability of individuals with a rare disease to participate in clinical trials
- An assessment of the benefits and risks, including side effects, of current and investigational therapies;
- The design of clinical trials for rare disease populations and subpopulations, including regulatory and scientific policies affecting design of such trials; and
- Demographics and the clinical description of patient populations.
Other User Fee Act Proposed Legislation in the Senate
Additionally, a bipartisan working group of Senators Harkin, Enzi, Alexander, Bennet, Blumenthal, Burr, Casey, Franken, Hagan, Hatch, Isakson, Kirk, Mikulski, and Roberts release a bipartisan Incentivizing New Antibiotic Development Draft. A bipartisan working group of Senators Harkin, Enzi, Bennet, Burr, Grassley, and Whitehouse also released the Drug Supply Chain Integrity Draft. Chairman Harkin and Ranking Member Enzi also released a bipartisan Medical Device Policy Draft.
The Senators said they would release additional drafts in other policy areas in the upcoming weeks. The discussion drafts will be available on the HELP Committee website. In releasing the draft bill, the Senators solicited feedback on:
- The policy merits
- The potential unintended consequences, and
- The potential opportunities to improve the legislative language.
Comments on all of these drafts were due last week to the respective Senators and Committees, so it is likely that new drafts will be released shortly, given Congress’ strict timeline for reauthorization this fall. We will try to post relevant updates accordingly.