Chari: Marco Taglietti, M.D.
Globally, an estimated 1.5 million people die every year from invasive fungal infections. From being relatively uncommon during the earlier part of the 20th Century when the world was plagued with bacterial epidemics, fungi have evolved as a major public health problem with mortality rates often exceeding 50%. The increasing number of life-threatening, hard-to-treat fungal infections has driven global demand for new, safe and effective treatments. Available antifungal agents can be difficult to use, are often poorly tolerated, or have become increasingly ineffective due to the rise of drug resistant strains. Despite the pressing medical need, it has been over 15 years since the approval of an antifungal therapy with a novel mechanism of action. Innovative antifungals for treatment of invasive fungal infections and next generation diagnostics are imperative to expand the antimicrobial armamentarium and effectively combat AMR.
The AWG Antifungal Subcommittee focuses on the following priorities:
- Education on fungal infections and urgent needs in the fungal space
- Advocacy for inclusion of antifungals in priority antimicrobial legislation
- Outreach to government agencies to ensure antifungals are included in scope of various efforts/programs
Chair: René Russo, PharmD, BCPS
According to The Pew Charitable Trusts in December 2017, “because the conventional antibiotics pipeline remains so thin, finding new approaches, using nontraditional products to treat or prevent bacterial infections, is critical.” Biological products, such as antibacterial monoclonal antibodies (mAbs) and lysins, can provide new treatment options for patients with resistant infections. In some cases, biologics that treat resistant bacteria could provide options that are superior to current broad-spectrum antibiotics generally used in therapy. Biologics have the potential to create new ways of preventing and treating serious infections to improve individual outcomes and are crucial components to combatting the rise of AMR. Some of these products can prevent infections, others can replace traditional antimicrobials as treatment, and others make traditional products more effective at fighting infections – all of which slow the development of resistance, and extend the utility of the rest our armamentarium.
The AWG Biologics Subcommittee focuses on the following priorities:
- Expansion of Qualified Infectious Disease Product (QIDP) eligibility criteria to include non-vaccine therapeutic biological products – fast track designation/priority review, without 5 years extended exclusivity
- Advocacy for inclusion of biological products in priority antimicrobial legislation
Chemistry, Manufacturing and Controls (CMC) Subcommittee
Chair: Randy Brenner; AWG Sponsor: Evan Loh, M.D.
FDA Chemistry, Manufacturing and Controls (CMC) requirements can present significant hurdles for small, innovative companies developing new antimicrobials. While CMC processes are in place to ensure drug quality meets appropriate standards, is consistent, and is appropriately described on the label, rigid CMC processes regarding registration batch requirements and starting materials can be a substantial burden for small infectious disease companies with limited budgets and research and development (R&D) capacity. R&D of antimicrobials poses unique scientific, regulatory and economic challenges, often making R&D far riskier than for other types of drugs. Investment in the space has diminished, due to the largely to cost and uncertainty of the development process, leaving the critical innovation domain of discovering new antimicrobials to a few small biotech companies. Antimicrobials also provide less financial reward for companies since they are used for a short duration due to the acute nature of most infections, and are encouraged to be held in reserve to protect against the development of resistance. CMC requirements incur substantial costs, and are often done prior to pivotal data. AWG seeks to explore opportunities where the FDA could provide flexibility of relief in some areas of CMC, without introducing too much risk around patient safety or drug shortages.
The AWG CMC Subcommittee focuses on the following priorities:
- Engagement with key stakeholders, including the FDA, regarding specific regulatory challenges for small to mid-size antimicrobial companies related to CMC requirements – including changes to registration batch requirements and increased communication regarding starting materials
- Outreach to government to discuss potential for regulatory flexibility for companies with low patient numbers or slow uptake
Payment Reform and Reimbursement Subcommittee
Chair: Steve Sandor
A critical element in the battle against AMR is reducing inappropriate use of antimicrobials. The Centers for Disease Control and Prevention (CDC) estimates that 20% – 50% of all antibiotics prescribed in acute care hospitals are either unnecessary or inappropriate. It is more critical than ever that optimal antimicrobial use be required to limit the unintended adverse events of inappropriate prescribing.
AWG has been engaged in efforts to help the U.S. Department of Health and Human Services recognize the unmet need for new agents in the fight against antimicrobial resistant pathogens. This effort is balanced by the costs/benefits of these new drugs and the requisite to provide adequate reimbursement for innovation that will save lives. When reimbursement is fixed under the DRG regardless of the administered drug, hospitals are reluctant to use higher cost agents, and therefore impose barriers to discourage use. In many instances the new agents have the potential to be clinically more appropriate than older, less effective drugs.
The AWG Payment Reform and Reimbursement Subcommittee focuses on the following priorities:
- Educate HHS and CMS on the importance of timely and effective access to new antimicrobials, with attention to the overall value these new agents provide to the healthcare system
- Engagement with stakeholders to encourage medication management standards/guidelines that support appropriate antimicrobial use and drive the adoption of those standards, all of which can improve the quality of care and clinical outcomes provided to patients
- Seek opportunities to demonstrate decreased hospital stay, reduction of readmissions and overall improved patient outcomes, without increasing cost of care, through appropriate use of new, innovative, antimicrobials
- Explore how AWG can work with CMS to improve the coding process that currently exists on outpatient administered products