Does the US Need Another Expedited Drug Development Pathway?
The FDA’s meeting to discuss a proposed new pathway aimed at expediting the development of “medicines intended to treat serious or life-threatening conditions with unmet medical needs”, met with a mixed response on February 4th. The proposal was prompted by a report last fall from the US President’s Council of Advisors on Science and Technology(PCAST), which said a “special medical use”(SMU) pathway could improve drug evaluation in the US.
The Public Weighs In
Participants weighing in on the issue included representatives from Cubist Pharmaceuticals, Trius Therapeutics, Biotechnology Industry Organization, The National Research Center for Women & Families, and Antibiotics Working Group (AWG).
Is There a Need for an Alternate Drug Development Pathway?
Creating an alternative approval pathway could "greatly enhance" the prospects for successful clinical development of novel antimicrobials against the ESKAPE pathogens and would complement the GAIN Act's incentives (Generating Antibiotic Incentives Now (GAIN) Act).
However, there are already six other expedited drug development pathways in place and 50% of the medicines approved in 2009 and 2011 were through a priority pathway. Additionally, various other proposals from the anti-infective development community and the Infectious Diseases Society of America (IDSA) are already on the table, with an assortment of names and acronyms, such as "special population limited medical use" (SPLMU) and "limited population antibacterial drugs" (LPAD).
Some Concerns Brought to Light
There were a few concerns expressed during the discussion, including:
- Could another expedited drug development pathway create potential congressional intervention resulting in added demands on the FDA?
- If the FDA pursues its new alternative pathway, could the expedited approval pathways in the Food and Drug Administration Safety and Innovation Act, such as the breakthrough therapies designation, get short shrift?
Suggestions to Enhance Potential Advantages:
Suggestions made for enhancing the potential advantages of the proposal included:
- Adopt a tiered regulatory framework for the new alternative regulatory pathway, which would allow either disease- or pathogen-based label indications, along with labeling that encourages the most appropriate use of the new medicines commensurate with the approved indications.
- Expand the new initiative to include later stage products, and include indications for which there currently is no guidance, such as prosthetic bone and joint infections, osteomyelitis and bacteremia.
- For antibacterials, the FDA's definition of unmet medical need should not only focus on current needs, but also future needs.
- Balance the dual priorities of expediting clinical development through smaller and more targeted studies and use of authorities that promote responsible prescribing for specific sub-populations through appropriate labeling and restrictions of use.
- Provide clarity about mechanisms or processes to expedite the clinical development of limited use products if it is to include postmarket restrictions of use.
- Make any SMU designation available early in drug development so that manufacturer can design appropriate clinical studies for use under the pathway, for example by conducting trials based on only the most severe manifestation of the disease without having to progress through more moderate disease populations first.
Is there a need for yet another expedited drug development pathway? As mentioned in our prior post, a streamlined clinical trial process for drug development could have significant strategic planning and competitive intelligence implications for pharmaceutical and biotech firms. Please share your thoughts.