The RPM Report :: Elsevier Business Intelligence

Mylan’s Autor-de-Force Move into Quality “Differentiation” and Sterile Injectables

cole.werble@previsionpolicy.com — Thu, 16 May 2013 13:40:01 -0400

Mylan is investing heavily to create a competitive advantage based on manufacturing quality – and public policy. The firm’s recruitment of FDA’s Deborah Autor highlights a strategy to spend more on quality while aiming at higher margins, especially in sterile injectables where the company sees an opening after widespread quality breakdowns across the industry.

Medicare’s Independent Payment Advisory Board: First, Do No Harm

Tue, 14 May 2013 13:40:00 -0400

The Affordable Care Act promises some important improvements in the US healthcare system. However, argues a biotech CEO, cutting spending by targeting the lifeblood of biopharma R&D is not one of them.

Which Came First? Alogliptin Avoids Possible Exclusivity Dispute

michael.mccaughan@previsionpolicy.com — Fri, 10 May 2013 11:00:00 -0400

An unusual footnote in FDA’s approval letter for Takeda’s long-delayed diabetes medicine means that the company won’t have to worry about losing five-year exclusivity on the molecule.

Digesting Caronia

michael.mccaughan@previsionpolicy.com — Thu, 09 May 2013 16:55:00 -0400

A December appellate court ruling in an off-label promotion case prompted headlines about sweeping implications for FDA’s regulatory authority. How does the ruling look four months later?

Medicare Rx and the Sequester Impact: Making Headlines, But is There a Commercial Impact?

Wed, 08 May 2013 11:39:00 -0400

Two large pharmaceutical manufacturers weigh in on whether the sequester is impacting their injectables business. The answer so far: not really.

FDAAA Impact Analysis (Year 5): The REMS Paradox

michael.mccaughan@previsionpolicy.com — Wed, 08 May 2013 11:39:00 -0400

FDA is approving new molecules at the fastest rate of the REMS era, while actually imposing REMS obligations less frequently than ever. That is good news for sponsors—but it doesn’t make a lot of sense if the REMS are working as intended.

Declaring Victory: How FDA Releases REMS

michael.mccaughan@previsionpolicy.com — Tue, 07 May 2013 14:25:00 -0400

There is a new category of FDA action letter: a REMS release. Dozens of sponsors of “MedGuide-only” REMS received them after an FDA policy change.

Commissioner Hamburg as Congressional Witness: Mixed Success With GOP Thus Far

Laura.Helbling@PrevisionPolicy.com — Fri, 03 May 2013 10:54:00 -0400

FDA Commissioner Margaret Hamburg has had some trouble at Congressional hearings, but she has made considerable headaway in her interactions with one key legislator, House Appropriations Committee Chair Rogers.

A New Direction for Alzheimer’s R&D: FDA Outlines Early Disease Pathway

krawson3@yahoo.com — Mon, 29 Apr 2013 11:26:00 -0400

Alzheimer’s R&D has suffered multiple failures over the last decade, with no drug found to slow or halt the underlying progression of the disease. Industry’s focus is now on patients at the very earliest stages of Alzheimer’s in the hopes of finding an effective drug before too much damage is done.

Defense-to-Offense: PhRMA Readies to Protect Government Markets

ramsey.baghdadi@previsionpolicy.com — Tue, 23 Apr 2013 13:46:00 -0400

White House budget targeting biopharma with new cuts hits just as the Pharmaceutical Research & Manufacturers of America’s annual meeting gets under way.

Landmark Trial for Personalized Medicine Coming Soon: NGS Approval May Be One Offshoot of Lung Cancer “Master Protocol”

michael.mccaughan@previsionpolicy.com — Wed, 17 Apr 2013 13:50:00 -0400

A Friends of Cancer Research/Brookings proposal for a multi-drug, multi-sponsor registrational study of targeted cancer drugs is moving forward. Squamous cell lung cancer will be the initial indication.

Making Pain Relief Affordable – Or Abuse Cheaper?

Laura.Helbling@PrevisionPolicy.com — Fri, 12 Apr 2013 14:02:00 -0400

There are many arguments against generics of opioids, especially if you’re Purdue Pharma. Here’s a new one: they’re lower the cost for abusers.

Former FDA ODE II Director Meyer Building New Regulatory Sciences Center at UVA

Jacqueline.Fitton@PrevisionPolicy.com — Thu, 11 Apr 2013 15:00:42 -0400

Bob Meyer, a veteran of both sides of the FDA drug regulatory process from top-level positions in FDA’s drug review office and the Merck regulatory strategy and policy group, is trying to create a new regulatory science center at the University of Virginia that could add to FDA’s close-by academic support resources.

Testing Part B Payment Cuts: Sequester Means Providers Get ASP+4%

Thu, 11 Apr 2013 12:00:00 -0400

Cutting the payment level for Medicare Part B drugs is a perennial budget proposal, but never gets very far in Congress. Now, thanks to the sequester, Part B payments have been cut from ASP+6% to ASP+4.3%. Will it stick?

CMS’ Tavenner Makes Her Case For Confirmation

Tue, 09 Apr 2013 15:10:00 -0400

Marilyn Tavenner has been running the Centers for Medicare & Medicaid Services since Don Berwick stepped down in December 2011. She finally got the chance to begin the formal process of applying for the job, with a Senate Finance Committee confirmation hearing.

DEA Delays in Scheduling Are Offsetting FDA’s Improved Decision Making

Laura.Helbling@PrevisionPolicy.com — Fri, 05 Apr 2013 17:40:00 -0400

FDA is showing a renewed commitment to meeting review deadlines for new molecular entities, but that is not translating into earlier commercial availability for all NMEs. Some products also need a controlled substance scheduling decision from the Drug Enforcement Administration; those actions have become a new regulatory purgatory that can be painful and frustrating.

Compromise On “Pay For Delay”? Supreme Court Hunts for Middle Ground

krawson3@yahoo.com — Thu, 04 Apr 2013 11:58:00 -0400

During Supreme Court oral arguments in the AndroGel pay-for-delay case, the government and drug manufacturers each pushed for a one-size-fits-all antitrust interpretation of brand-generic reverse payment settlements. But the most likely outcome will probably fall somewhere in the middle: allowing the deals to continue, but with additional scrutiny to ensure they are not anticompetitive.

The Biology Police: The Plavix Investigation and Pharmacogenomics

michael.mccaughan@previsionpolicy.com — Thu, 28 Mar 2013 13:10:00 -0400

The discovery of a genetic marker for Plavix resistance is one of the milestones in the regulatory application of pharmacogenomics. Is it also a crime drama?

Patient Feedback Pioneers: Chronic Fatigue Advocates Prepare for First PDUFA V Meeting

Jacqueline.Fitton@PrevisionPolicy.com — Thu, 28 Mar 2013 10:55:00 -0400

FDA has selected CFS/ME as the first topic for the PDUFA V patient-centered drug development initiative. The lessons learned from this session will inform future work on patient engagement in drug development both for FDA—and for patient advocates.

FDA Plants OB-GYNs on Implant Addiction Therapy Committee; Titan Harvests Yes Vote

Laura.Helbling@PrevisionPolicy.com — Wed, 27 Mar 2013 14:30:00 -0400

An advisory committee reviewing Titan’s Probuphine for abuse dependence leaned heavily on reassuring safety perspectives of two guest members invited for their particular expertise. No, they weren’t addiction experts.

Explaining FDA’s Caution on New Surrogates to New Patient Constituencies

Tue, 26 Mar 2013 16:31:00 -0400

When is a surrogate marker good enough for approval, accelerated or otherwise? That is not an easy concept to explain, but FDA is doing its best to educate patient advocates on the nuances of the regulatory process.

Growing Pains for Generic Drugs: OGD Gains Respect, But Loses Geba

krawson3@yahoo.com — Thu, 21 Mar 2013 16:52:00 -0400

FDA’s Office of Generic Drugs was at the start of a turnaround: $299 million in new user fees to clear a big ANDA backlog and fund overseas inspections, an elevated status within the Center for Drugs and a brand-new director to steer the ship. But there have been some bumps in GDUFA implementation, and OGD’s new director, Gregory Geba, has abruptly left the agency.

PDUFA V and Investor Communications

michael.mccaughan@previsionpolicy.com — Thu, 21 Mar 2013 15:50:00 -0400

The Prescription Drug User Fee Act reauthorization made a number of important changes to the new drug review process and programs for stakeholder engagement with FDA. It also created a number of new investor communication challenges for sponsors.

The Subjectivity Of Severity: Would Re-Labeling Opioids Make A Difference?

Laura.Helbling@PrevisionPolicy.com — Tue, 19 Mar 2013 00:00:00 -0400

Proposals to address the opioid abuse problem include restricting non-cancer use to “severe” pain, but one major source for opioid prescription dispensing (the chain drug stores) argues “severe” isn’t an appropriate way to separate patients in a subjective category like pain treatment.

Salmon Calcitonin and the Challenge of Life-Cycle Regulation

michael.mccaughan@previsionpolicy.com — Mon, 18 Mar 2013 16:45:00 -0400

An advisory committee is recommending pulling the osteoporosis indication for salmon calcitonin. The product is, to put it bluntly, outdated: approved on standards FDA no longer accepts and now commericially irrelevant.

Much “Ado” About Biosimilars

michael.mccaughan@previsionpolicy.com — Mon, 18 Mar 2013 16:25:00 -0400

The approval of Kadcyla gives Roche/Genentech yet another new breakthrough cancer therapy in the US. It also offers some clues about the future of biosimilars at FDA.

Regulating Formularies: OIG Wants Closer Scrutiny of Process; CMS Says Focus on Results

Mon, 18 Mar 2013 14:35:00 -0400

The HHS Inspector General is concerned about lax oversight of conflict-of-interest standards on formulary committees for Medicare Part D plans. CMS is defending its approach, which emphasizes review of the formularies themselves, rather than the process to construct them.

The Companion Diagnostics Dilemma

michael.mccaughan@previsionpolicy.com — Wed, 13 Mar 2013 14:40:00 -0400

An Institute of Medicine workshop on companion diagnostics suggests that regulatory standards are evolving at FDA to encourage companion diagnostic development. But almost everything else is still a challenge.

DoJ and GMPs: A Prosecutor’s Take on Enforcing Product Quality

Tue, 12 Mar 2013 12:33:17 -0400

The wave of marketing promotions undertaken by the Department of Justice over the past decade may be abating, but prosecutors are looking at manufacturing violations as fertile new territory for cases. One prosecutor shares her thoughts on how companies can protect themselves.

Genentech’s Cancer Product Line Expands, As Do Its Post-Market Manufacturing Obligations

michael.mccaughan@previsionpolicy.com — Fri, 01 Mar 2013 15:45:00 -0500

Genentech appears to be having significant manufacturing problems for some of its new cancer therapies. FDA hasn’t taken enforcement action yet, but the agency has delayed two approvals and tied them to post-market requirements to address QC issues.

Pain Drug Development Becomes Personalized

m.wortman@elsevier.com — Mon, 25 Feb 2013 00:00:00 -0500

Advances in pain neurobiology point to the promise of more specific drugs with fewer side effects, yet the field continues to show few successes. Nevertheless, fast-advancing knowledge, a huge market, and better clinical study de-risking strategies should eventually overcome investor shyness about backing emerging personalized pain drug developers.

CER “Substantial Impact” Expected in Three-to-Five Years: NPC’s Annual Comparative Effectiveness Survey

Thu, 21 Feb 2013 00:00:00 -0500

CER is slowly beginning to enter the public lexicon. Respondents to the National Pharmaceutical Council annual survey on comparative effectiveness report being more familiar with CER now than two years ago; but they still see the major impact several years away – not right on the horizon.

GAIN Begins (Part 2): Infectious Disease Society and Start-Up Companies Push for More

Wed, 20 Feb 2013 17:28:00 -0500

The ink is barely dry on new antibiotic incentives in the GAIN Act, but the anti-infective development community is pushing for more. New ideas include “pull” incentives modeled on biodefense programs.

Part D Rebates: Talking Points Continue, But Deficit Deal Elusive

michael.mccaughan@previsionpolicy.com — Wed, 20 Feb 2013 13:35:00 -0500

Once again the President is calling for rebates on prescription drugs sold under Medicare Part D. But without a big deficit deal involving entitlement reform, is there any way to enact them?

GAIN Begins (Part 1): FDA Completes Turnaround On Antibiotic R&D

krawson3@yahoo.com — Wed, 20 Feb 2013 10:05:00 -0500

In less than two years, FDA has moved from requiring unrealistic approval standards for antibiotics to exploring innovative ways to shorten the pathway for the most-needed drugs. And, surprise: Congress is on board too.

Senator Manchin Goes to White Oak

Laura.Helbling@PrevisionPolicy.com — Wed, 20 Feb 2013 00:00:00 -0500

The open public hearing portion of an advisory committee is often emotional and sometimes persuasive. It is not usually a forum for a give-and-take with a sitting US Senator.

Why NMEs and Therapeutic Biological Products Fail in the First FDA Review Cycle

Fri, 08 Feb 2013 12:25:00 -0500

PAREXEL Consulting looks at the underlying causes of multi-cycle reviews.

How Not To Develop A Drug

michael.mccaughan@previsionpolicy.com — Wed, 06 Feb 2013 16:20:00 -0500

What is the best way to encourage drug development in an unmet medical need? Chronic Fatigue Syndrome advocates argue that FDA should approve a drug—specifically Ampligen—even if it falls far short of reaching typical standards of evidence.

The Consequences of Caronia: Impact On FDA’s Off-Label Regulation

krawson3@yahoo.com — Tue, 29 Jan 2013 09:14:00 -0500

The government has decided not to appeal the Second Circuit’s Caronia off-label promotion ruling – no doubt in part because of concerns about the broad reading of the First Amendment currently in favor at the Supreme Court. Officially, FDA has adopted a low-key response.

A Regulator’s Perspective For 2013: FDA’s Temple Discusses FDASIA, Caronia, and Breakthrough Development

ramsey.baghdadi@previsionpolicy.com — Tue, 22 Jan 2013 14:48:33 -0500

FDA’s dean of drug development Robert Temple offered his perspective on a number of different issues facing the agency and the drug industry at The RPM Report’s FDA/CMS Summit, including the impact of FDASIA, the Caronia off-label promotion ruling, and new approval pathways. Below is an edited transcript of his comments.

FDA’s Very Big Year: Drug Approvals Approach Record Levels

michael.mccaughan@previsionpolicy.com — Wed, 16 Jan 2013 13:55:00 -0500

In 2012, FDA new drug approvals reached levels not seen since the peak years of the 1990s. FDA counts 39 NMEs; The RPM Report tracks a different metric—ICTs—and FDA topped 30 for the first time since 1999.

Part D: “Very Successful” Does Not Mean “Perfect”

Wed, 16 Jan 2013 13:35:00 -0500

Medicare’s Jonathan Blum says Part D has been a success but says there are areas where improvements could be made. CMS will continue to emphasize access, but is paying more attention to over-utilization in some categories.

Turpentine, Arsenic and Xyrem: The Caronia Dissent

Tue, 15 Jan 2013 10:50:00 -0500

In December, the federal appellate court in Manhattan overturned the conviction of a former Orphan Medical sales rep, issuing a broad ruling declaring that truthful, off-label communication is protected by the First Amendment. There was one dissent on the three-judge panel—and FDA officials like Bob Temple hope that the dissent gets more attention than the majority ruling.

FDA’s Drug Review Challenge For 2013: Managing Sponsor Expectations

krawson3@yahoo.com — Tue, 15 Jan 2013 08:27:00 -0500

The first late-cycle meetings under FDA’s new drug review process are scheduled to start in February, and the agency is scrambling to manage expectations. What agency officials don’t want: a contentious fight over approvability, followed by a flood of new submissions and appeals.

Rx Spending Continues To Lag Overall Health Growth; New Drugs Have Positive Impact in 2011 Data

Laura.Helbling@PrevisionPolicy.com — Mon, 14 Jan 2013 00:30:00 -0500

Prescription drug spending modestly increased in 2011 but continues to lag historically slow rates of overall health spending growth. But it could have been worse: government actuaries cite spending on new brands as driving what growth there is.

What To Do About Caronia

krawson3@yahoo.com — Thu, 10 Jan 2013 10:14:00 -0500

While pharmaceutical marketers wait to hear how the government will respond to the Second Circuit’s off-label communication Caronia court ruling, PhRMA is considering ways to take the initiative. On the table: working proactively with FDA to re-frame the rules around off-label promotion.

Breaking Down the First “Breakthrough”

michael.mccaughan@previsionpolicy.com — Wed, 09 Jan 2013 14:05:00 -0500

Vertex’ cystic fibrosis research is the recipient of the first two “Breakthrough” designations issued by FDA. That underscores the likelihood that the primary value of the new pathway will be as a badge for investors.

Invaluable Guidance: FDA’s Dean of Drug Reviews on “Enrichment Strategies”

Fri, 04 Jan 2013 15:17:00 -0500

FDA’s draft guidance on “Enrichment Strategies for Clinical Trials” fulfills an overdue goal from PDUFA IV. It also captures decades of wisdom from the agency’s senior drug review manager.

CMS Proposed National Coverage Decision Topic List Exempts Biopharma

michael.mccaughan@previsionpolicy.com — Mon, 17 Dec 2012 08:38:00 -0500

The Centers for Medicare & Medicaid Services’ new list of “potential” topics for National Coverage Decisions includes 33 separate ideas, most proposed by the public. Not one is directly related to a biopharma topic.

FDASIA Implementation Progress Report

m.laffler@elsevier.com — Mon, 17 Dec 2012 00:00:00 -0500

Center for Drug Evaluation and Research senior officials discuss early metrics related to “breakthrough therapy” designations, contacts with dedicated communications liaison staff under PDUFA V, biosimilar development meetings and generic drug user fee collections.