The US Food and Drug Administration (FDA)’s newly-released Innovation Blueprint sets out the steps which the agency can take immediately to address concerns about biomedical innovation, which is slowing down despite record investments in R&D.
“America is at an important crossroads, where the science before us presents unprecedented opportunities to create new and better medical products and to promote better health for the public,” said FDA Commissioner Margaret Hamburg. “Our Innovation Blueprint highlights some of the initiatives FDA will be implementing to ensure that these opportunities are translated into safe and effective treatments that can help keep American patients and American industry healthy and strong,” she added.
The report’s proposals stem from a review of FDA’s current policies and practices, as well as months of meetings with major stakeholders nation-wide, and they focus on implementing the following major actions:
– rebuilding FDA’s small business outreach services;
– building the infrastructure to drive and support personalised medicine:
– creating a rapid drug development pathway for important targeted therapies;
– harnessing the potential of data-mining and information-sharing while protecting patient privacy;
– improving consistency and clarity in the medical device review process:
– training the next generation of innovators; and
– streamlining and reforming FDA regulations.
The document “represents a productive and important first step toward smart innovation-focused reform at FDA,” said Vicki Seyfert-Margolis, senior adviser for science innovation and policy for the FDA Commissioner’s Office. One of the things the agency had been told at the stakeholder meetings is that FDA needs to work better as one agency, rather than functioning as several semi-autonomous centres, she added.
The Pharmaceutical Research and Manufacturers of America (PhRMA) said it welcomed the “thoughtful and proactive” approach taken in the report, and pointed out that many of its themes are consistent with the provisions in the recently-negotiated Prescription Drug User Fee Act (PDUFA-V).
If implemented as published, PDUFA-V will “provide the FDA with much-needed resources and management tools to support patient safety and to promote innovation in the scientific ecosystem through increased transparency, predictability and efficiency in FDA’s science-based human drug review programme,” said PhRMA chief executive John J Castellani.
PDUFA-V will also establish an enhanced review model for New Molecular Entities (NMEs), which “targets completion of FDA assessments of efficacy and safety within the first review cycle and is intended to reduce the overall time for new medicines to become available to patients, while maintaining FDA’s gold standard of safety and efficacy. One aspect of this provision which should facilitate this objective is increased scientific communication between FDA and sponsors prior to and throughout the review process,” added Mr Castellani.
