Latest Drugwonks' Blog
That makes Secretary Clinton the fourth person who is running or almost ran for president this year to call for a national assault on disease. (Ted Cruz, Mike Huckabee and Joe Biden are the others. And yes, I am sure if Donald Trump joins them it will be to announce something really, really spectacular.)
That's on the heels of a "$2 billion funding boost for NIH and a $133 million increase for FDA – levels near what the bipartisan H.R 6, the 21st Century Cures Act, sought for Fiscal Year 2016. Energy and Commerce Committee Chairman Fred Upton (R-MI) commented, “This research boost is an important milestone - we’ve made great progress, but more work remains as we seek to build upon this momentum to deliver cures now. As the Senate continues to do its work, we will continue to seek out every legislative opportunity to improve the future for patients and cures.” The momentum continues as The Hill reports, on the heels of last week’s milestone, Senate Committee on Health, Education, Labor, and Pensions Chairman Lamar Alexander (R-TN) reiterated that the Senate’s cures bill is a priority for the New Year."
And that's not all. In addition to Senators Roger Wicker and Angus King's EUREKA Act (which would authorize the Director of the National Institute of Health to work with other federal agencies to establish prize challenges to reach research milestones) there have been several other legislative proposals to encourage and reward initiatives that cure expensive and difficult to treat diseaes.
There can and will be aspects of all these proposals that can be picked apart. But as my colleague and cure strategist Jim Pinkerton points out: "history tells us that when the country commits to a goal, big things tend to happen."
Things are heating up at the CDC.
The agency’s lack of transparency regarding their development of opioid prescribing guidelines has drawn the attention of the House Committee on Oversight and Government Reform.
In a letter to CDC Director, Dr. Tom Frieden both the Chair, Representative Jason Chaffetz (R, UT) and Ranking Member, Representative Elijah Cummings (D, MD) share their displeasure at the secretive nature of the guideline development process. Per the Committee, “We have questions about why a Core Expert Group established by CDC to assist with drafting these guidelines is not considered an advisory committee under the Federal Advisory Committee Act (FACA).”
Those questions and an aggressive documents request are included in the letter which can be found here. And the Committee wants them by January 8th. Looks like there will be some people called back from their Christmas vacation at the CDC.
Perhaps the best way to address the CDC’s fairy tale process is to quote from author of Peter Pan:
“Those who bring sunshine into the lives of others cannot keep it from themselves.”
James BarriePresident Obama Signs Off on Omnibus Bill Upping FDA’s Funding
President Barack Obama signed into law on Friday the fiscal 2016 omnibus spending bill, which contains $4.68 billion in total funding for the FDA.
Congress had wasted no time approving the measure Friday morning, as it sailed through both chambers by wide margins.
The bill – which includes more than $2.7 billion in discretionary funding, a $132 million increase from the previous year – passed the House and the Senate by margins of 316 to 113 and 65 to 33, respectively.
Industry insiders have touted the bill as a positive for the FDA. “[Congress] is beginning to recognize that the FDA, to do its job, needs appropriate funding to be an innovator,” Peter Pitts, president and founder of the Center for Medicine in the Public Interest and a former FDA associate commissioner, tells DID.
Ladd Wiley and Steven Grossman, executive director and deputy executive director, respectively, of the Alliance for a Stronger FDA, describe the funding in a prepared statement as a “victory.” They add that the distribution of funds appears to largely mirror the priorities of the administration’s request.
New technologies that support postmarketing patient communication and safety surveillance can produce a mountain of data. But are these new tools creating value or just more noise?
YourEncore recently convened a panel of experts to discuss this topic after the 2015 Regulatory Affairs Professional Society (RAPS) Convergence conference in Baltimore, Maryland. To watch the panel discussion, click here.
YourEncore Executive Partners, Peter J. Pitts, former FDA Associate Commissioner, and Dr. Don Therasse, former VP of Global Patient Safety and Global Medical Affairs at Eli Lilly & Co., shed some light on the topic.
Peter Pitts: There is no such thing as a risk-free product. From a regulatory perspective, when you’re looking at outcomes, we’re learning the difference between efficacy in a clinical trial and effectiveness in the real world and how to capture and compare the data sets.
The general consensus is that MedWatch captures around 10 percent of adverse events. That’s probably high. But even if it’s spot on, it’s remarkably and dangerously low. How are we capturing and reporting substandard pharmaceutical events when a drug doesn’t necessarily have an adverse event but just isn’t working? API problems, excipient problems, manufacturing problems—from a lexicon perspective, unless you can put a name to it and put systems in place, it’s anecdotal. If we want to bring drugs to market faster with truncated clinical trials, we’re going to need a much more robust post-marketing proposition.
There are a lot of ways to collect data. Social media is one that has caused problems. The signal to noise ratio issues are profound, but we can’t ignore it because within all that noise there are gems that we must capture. The challenge is how to put robust modern pharmacovigilance or pharmacoepidemiology programs in place for established drugs and decide who leads the effort. We need to do a better job of capturing and sharing data in real time. This requires better staffing and an openness to finding new things.
A couple of years ago I was talking to a pharmaceutical company in New Jersey, and someone said to me, “We don’t use social media because we don’t want to find adverse events.” We must embrace negative information, because it’s happening in the real world.
Don Therasse: Everybody talks about how we can do these things and manage the entire post-approval safety environment more effectively and efficiently. The fear is not that we will find new information; it’s that we would overwhelm our current systems and capacity with poor quality information – and just generate more noise as opposed to real signals. That’s the primary concern about the social media space.
The goal of the pharmacovigilance enterprise is not to simply generate more signals; it’s to generate better signals and to apply our finite resources to identifying safety issues and implementing programs to help mitigate them. In terms of regulatory convergence, or divergence in this area, we need work not only in this and other areas that define sources of information for signal detection, but also for how we manage the data, how it’s reported, how you’re inspected and how you implement your risk mitigation programs. Everybody has the same goals, but everyone seems to want to put their own spin on it which causes redundant effort and diverts way too many resources from the important work at hand creating a very inefficient system.
There are many good ways to advance the science. At the same time we have to be disciplined and willing to give up older, less effective activities. There are finite resources on both industry and regulatory sides, and we’re diverting too much of our attention to nonproductive activities at the expense of fully implementing powerful new tools.
Pitts: We have a tool at our disposal that is poorly used, which is physicians. How many hours do medical students spend learning about drug safety management and adverse event reporting? None. How much time do physicians spend in their daily practices thinking about adverse event reporting? How friendly is the MedWatch system? Obviously, the first mission of doctors is to address their patients’ immediate needs. Once that’s done there is an obligation to share information that can advance the science and ultimately help to prevent similar issues for future patients. We need greater awareness and more effective, user-friendly processes to encourage and enable them to do so.
To paraphrase Homer Hickam (of "October Sky" fame), “A docket won't fly unless somebody lights the fuse.”
Here are my comments to the CDC’s docket Proposed 2016 Guideline for Prescribing Opioids for Chronic Pain:
Comments
In determining appropriate guidelines, CDC should engage as part of an intramural HHS team. FDA has issued labeling changes for this category along with thoughtful and strategic roadmaps for how and why they can be successfully implemented. The FDA’s statements and actions on the approval, labeling, and future development of abuse-deterrent products and technologies are designed to both protect and advance the public health relative to the balance of benefit/risk and access. Further, FDA has identified prescriber, dispenser, and user education as a major focus for pursuing a safe-use agenda for opioids. CDC must broaden its vision when it comes to the development of opioid guidelines to include the thoughtful, dynamic, patient-focused programs already instituted by the U.S. Food & Drug Administration.
From the fine folks at RAPS:
CDER’s Woodcock Outlines Priorities for 2016
With a banner year for new drug approvals under its belt in 2015 (42 approvals and counting), the US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) is now looking at how it can not only negotiate three new user fee agreements but fill an enormous number of staff vacancies.
CDER Director Janet Woodcock, speaking Monday at the FDA/CMS Summit in Washington, DC, outlined 2015’s achievements and a number of the agency’s top priorities for 2016, particularly as CDER, with its $1.2 billion budget, continues to work with Congress on legislation like the House-passed 21st Century Cures Act.
2015 Recap
As far as meeting CDER’s priorities for 2015, Woodcock said the agency did “pretty well,” noting that there were some serious concerns in the year prior on addressing the backlog of abbreviated new drug applications (ANDAs), though she said there’s been “a huge turnaround.”
FDA has been particularly interested in reducing the number of multiple cycle reviews for generic drugs, she said, noting that in the not-too-distant past the average was four review cycles, though the reduction in such cases has occurred over the past year as more companies understand what it takes to submit a complete ANDA.
John Jenkins, Director of the Office of New Drugs at CDER, also outlined a number of the drug approval statistics, particularly as CDER has seen a steep rise in orphan drug approvals, though he noted that such approvals aren’t always to the benefit of public health (some of his slides can be viewed here).
On the international front, Woodcock highlighted FDA’s work with the International Conference on Harmonisation (ICH), noting that it “was headed off a cliff,” though after “strenuous efforts,” FDA was able to help sign new agreements and make the group more inclusive, which should be a boon for negotiating more global standards for drugs.
And while many in Congress and industry are still waiting on new biosimilar draft guidance documents on labeling and interchangeability, among others, Woodcock said that CDER has been working extremely hard behind the scenes to ensure the US market for biosimilars can be robust in the near future as education efforts continue.
She also highlighted the success of the new “drug trials snapshots” program under CDER, which offers a look at the demographics of clinical trial data for newly approved drugs, which she said has been “very popular” for consumers.
A Look Ahead
As far as what to expect for 2016, Woodock offered a wide-ranging laundry list of topics that the agency will look to address, though the 680 vacancies at CDER seem to be one of the focal points.
The re-negotiations of the prescription drug, generic drug and biosimilar user fee programs will also be at the forefront of CDER’s work next year as these agreements with industry need to be ironed out before the programs expire in 2017.
In addition, CDER is looking to:
- Reevaluate the regulations on drug advertising and promotion in light of current jurisprudence around the First Amendment
- Issue draft guidance on generic versions of abuse-deterrent opioid formulations
- Integrate the Sentinel Network into routine drug safety activities
- Further implement statutory provisions on track and trace legislation
- Continue drug label improvement initiative as “a lot of labels are out of date,” Woodcock said
- Develop a more robust process and policy documents on how to evaluate a biomarker as a surrogate endpoint for accelerated approvals
- Streamline clinical trial monitoring and data cleaning practices
- Conduct more outcomes assessments of breakthrough therapies, modernize clinical evidence assessments and further utilize electronic health data (which Woodcock said is a priority for FDA commissioner nominee Robert Califf)
- Make significant progress on FDA’s mutual reliance initiative with the European Medicines Agency
- Improve combination product inter-center review process
- Develop an implementation plan and training for a new pregnancy/lactation labeling rule
- Continue to push standards development and standardized electronic submissions to CDER
From the pages of Politico:
CDC opens opioid prescription guidelines to public comment
The CDC plans to publicly post draft guidelines on opioid prescribing after being criticized by pain advocates, professional associations and some within HHS for developing them behind closed doors and with limited public input.
A notice posted this morning provides 30 days for public comment, which means that the guidelines - a key part of the Obama administration's response to the opioid abuse and overdose epidemic - won't be released in January as the agency had planned.
"Opening a docket will tack months on to the process [and also] increases the likelihood that the guideline may never be released," said Andrew Kolodny, a founder of Physicians for Responsible Opioid Prescribing and the chief medical officer at Phoenix House. "This is an enormous win for the opioid lobby."
CDC hadn't planned to publish the document ahead of its release in January but came under fire earlier this month from members of the administration's Interagency Pain Research Coordinating Committee, which announced that it would file a formal objection to CDC's prescribing guideline after agency officials briefed them at a meeting.
The level of evidence cited to support the guidelines, which are non-binding on physicians and apply to chronic pain patients outside of the cancer or palliative care treatment, "is low to very low and that's a problem," Sharon Hertz, FDA's director of the Division of Anesthesia, Analgesia and Addiction Products, said at the meeting.
AHRQ health scientist Richard Ricciardi, another member of the interagency committee, called the recommendations "ridiculous" and "an embarrassment to the government."
The document had already been circulated among HHS agencies for final review ahead of its planned release in January, but it had drawn criticism from the American Cancer Society Cancer Action Network and the American Medical Association after it was previewed for some health care groups at a webinar in September.
CDC didn't immediately respond to a request for comment.
Dear Santa:
I’ve been a good policy wonk this year, so here’s my FDA wish list for 2016 …
· Thoughtful guidance on differential nomenclature for biosimilars that recognizes that “similar” doesn’t mean “identical” and prioritizes the need for savvy pharmacovigilance
· Further agency thinking on biosimilar label extrapolation
· Physician notification x 50 states, Puerto Rico, and the District of Columbia (not under FDA authority -- but since we're on the subject)
· 21st century bioequivalence written guidance for generic drugs (including tighter ranges for narrow therapeutic index medicines and critical dose drugs)
· A super effort from the FDA’s new Super Office of Pharmaceutical Quality
· Aggressive intramural efforts on biomarker development and validation
· A first quarter meeting on off-label communications
· Smart efforts on abuse-deterrent opioid PROs
· Next-step traction for Patient-Centered Drug Development
· More attention to combination products
· And IVDs
· Continued sophisticated thinking (from management) – and action (on the Division level) on adaptive clinical trials
· Completion of the Generic Labeling Rule
· Better oversight of compounding pharmacies
· And drug shortages
· For-real updating of the PMA and 510k processes
· Continued sanity on drug importation
· And medical mobile apps and social media
· Sharper teeth on dietary supplements
· 21st Century Cures legislation with both mandates and budget
· A standardized benefit/risk analysis grid
· Wider lanes on expedited pathways
· A potent PDUFA that recognizes that FDA resides at the center of the precision medicine ecosystem.
· A potent PDUFA that doesn’t use “fast and clean” as it’s sole metric for success.
· A swift, positive vote for Rob Califf
And Santa, this is only a partial list.
Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.
