In recent months, the presidential election confirmed the status quo in the White House and on Capitol Hill, the Supreme Court
retained health reform, and Congress reauthorized user fees and important new authorities for the Food and Drug Administration.
So one might expect 2013 to be less strenuous for the biopharmaceutical community. But as the Obama administration tackles
the monumental task of implementing new health programs authorized by the Affordable Care Act, pharma faces the daunting assignment
of making sure that these policies broaden patient access to prescription drugs without imposing price controls.
Moreover, pressure to fill depleted new drug pipelines, challenges in dealing with a diverse global pharmaceutical supply
chain, and opposition to high drug prices will keep pharmaceutical executives fully engaged in the coming months. Add to the
mix the legal debate over the constitutionality of FDA curbs on off-label communications (see sidebar), continuing shortages
of critical drugs, and alarms over deaths from questionable drug compounding activities, and the road ahead looks pretty rocky.
It's not clear whether FDA commissioner Margaret Hamburg will remain on the job for just one more year or longer, creating
uncertainty about the agency's future directions.
Key Issues Before the Courts
Casting a cloud on all these situations is the imperative to reduce the federal deficit by spending less and collecting more
taxes. Pharma companies have a stake in corporate tax reform efforts related to foreign-source income, as well as tax incentives
for R&D. Federal budget cuts threaten Medicare and Medicaid payments for drugs and adequate funding for FDA, the National
Institutes of Health, and other programs involved in biomedical research and pharmaceutical utilization.
New focus at FDA
This is a "transformational time" in the nation's healthcare system, raising expectations about medical product effectiveness
and demands for more effective oversight despite limited resources. FDA is responding by working overtime to implement the
multiple requirements of the FDA Safety & Innovation Act (FDASIA), reported Douglas Throckmorton, deputy director of the Center
for Drug Evaluation and Research (CDER), at the FDA/CMS Summit sponsored by Elsevier Business Intelligence. There are new
user fees for generic drugs designed to speed up approvals and ensure timely plant inspections. A clear pathway for approving
new biosimilars is emerging, spurring several INDs, but so far no product applications.
A main focus of FDASIA is to encourage development of new antibiotics, orphan drugs, and "breakthrough" therapies for critical
diseases. CDER is drafting guidance on its expedited approval pathways—fast track, breakthrough, priority review, and accelerated
approval—to clarify the opportunities and requirements created by these options, noted CDER Office of New Drugs (OND) director
John Jenkins. He pointed to the rise in commercial INDs in recent months as a factor boosting FDA approvals of new molecular
entities for 2012, including several breakthrough therapies. OND's more transparent and managed review process aims to meet
user fee goals and achieve more first review cycle approvals, particularly for priority applications. And liaison staffers
at CDER will offer assistance to firms navigating the FDA review system, which should be particularly helpful for the growing
number of "emerging sponsors" filing their first new drug applications.
CDER will continue to address postmarketing safety issues, as seen in its expanding Sentinel and mini-Sentinel systems that
provide rapid access to patient data to answer questions about drug adverse events. The agency is fine-tuning its REMS (risk
evaluation and mitigation strategies) program, responding to demands from providers for integration of REMS into healthcare
systems and pharmacy operations. A number of high-profile public health issues, namely drug shortages, opioid abuse, and drug
compounding, are top concerns for FDA, and FDASIA provides added authority to require early notification of looming shortages
and to halt violative drug imports and counterfeits.
To better handle these changes CDER director Janet Woodcock recently made the Office of Generic Drugs an independent "super"
office. A new Office of Pharmaceutical Quality (OPQ) will tackle drug manufacturing quality issues and supply chain challenges
and better coordinate field plant inspections. More details on CDER reorganization should emerge over the next six months.