What about treatments for major diseases?
At the same time, regulatory officials worry about declining investment in new therapies for broader conditions, such as diabetes
and cardiovascular diseases affecting millions. Jenkins urged another look at "me-too" drugs. "Having more choice is good,"
he says, noting the first-in-class drug is not always the best.
Can the industry regain its credibility and public trust ?
Can new partnerships fill gaps in biomedical research funding?
The budget squeeze at FDA and the National Institutes of Health, along with declining drug company revenues, are spurring
more collaboration across the biomedical research enterprise. Three leading cancer research centers recently formed a joint
venture to investigate innovative pathways for developing cancer treatments. Major manufacturers are negotiating research
agreements with biotech firms, instead of just buying them. Pre-competitive partnerships are proliferating, tackling data
sharing and patent issues in the process.
Can clinical trials become more efficient?
High R&D costs underlie the hefty price tags on many new drugs, emphasizing the importance of streamlining research operations
and testing requirements. Multiple partnerships and collaborative initiatives are working to establish standards, harmonize
electronic data systems and utilize new models for protocol design. Yet concerns about patient privacy and safety complicate
the process. Debate will continue over these issues, plus appropriate use of biomarkers and endpoints in clinical trials
and the role of more innovative analytical methods and study designs.
Will drug shortages persist?
Patients and health providers become alarmed when they can't obtain needed medicines, and when alternate supplies from compounders
turn out to be contaminated and lethal. FDA is moving quickly to establish policies and procedures authorized by new legislation
to detect and prevent critical shortages and to regulate large-scale compounders of sterile injectibles. However, the long-term
solution, say agency officials, is for manufacturers to invest more in systems to ensure consistent production of quality
products, and FDA wants to see more action in this area.
How will social media transform pharma promotion?
Personalized medicine means more targeted marketing, along with less spending on traditional advertising. Reliance on legions
of sales reps continues to decline as doctors and hospitals close their doors. Yet, FDA regulations limit marketer use of
the Internet and electronic messaging systems to discuss their products. Pharma companies want to utilize social media to
better educate patients about proper compliance and prescribing. FDA oversight of these strategies is critically needed.
Will FDA get sufficient resources?
Jenkins and FDA commissioner Margaret Hamburg also emphasized at the FDA/CMS summit the need for more resources to support
the agency's ever-expanding responsibilities. While praising Congress for providing added authorities that will help FDA fulfill
its mission, Hamburg urged the legislators to approve the "kind of real resources — not just new mandates and authorities"—
needed to meet greater challenges. Jenkins expressed frustration that budget cuts and sequestration have siphoned off resources,
including a portion of already-paid user fees. This development, together with the steady rise in fees supporting drug regulation,
may prompt a broader re-examination of the user fee program in coming months.
Jill Wechsler is Pharm Exec's Washington correspondent. She can be reached at email@example.com