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An article by STAT journalist Rebecca Robbins on drug pricing could use a lot of re-editing for balance and depth. Or maybe should could find another job.
She contrasts public ‘outrage’ (as represented by some well-dressed protestors) about drug pricing with an orgy of greedy indifference on the part of biotech CEOs.
Public anger at drug companies is “an abomination,” Ron Cohen, chairman of the big industry group BIO (my note: you see, even a trade group of small, money losing companies spending billions on medical research is now BIG as in powerful and dangerous), said at the Biotech Showcase. All the talk about pharma profiteering, Cohen said, is “a perversion of reality.”
Which can also describe Ms. Robbins reporting because of what is NOT included.
She follows Cohen’s comments (which were taken out of context: Cohen actually said that to smear everyone in the biopharma industry as all being “greedy profiteeers” is an abomination and perverse) with context free narrative about drug prices:
“Many drug makers have raised prices in the past year. And a slew of new drugs have (sic) hit the market with eye-popping price tags: cancer drugs at more than $11,000 a month; cholesterol drugs at more than $14,000 a year. Then there’s Martin Shkreli, the pharma executive who bought up a decades-old drug and hiked the price 5,000 percent, turning himself into a target of nationwide protests before he was arrested last month on securities fraud charges.”
Yes, linking Turing turd Shkreli who used the Daraprim price hike to short biotech stocks for the sake of his own portfolio, is now lumped in with companies developing new and important medicines that save lives, reduces health care costs and increase well-being is now part of the narrative.
Robbins is not alone in this perversion of reality. The WSJ’ Peter Loftus runs a me-too story that portrays price hikes as disregarding “mounting criticisms of prescription costs in the U.S”
Loftus claims companies “have raised U.S. prices for dozens of branded drugs since late December, with many of the increases between 9% and 10%, according to equity analysts. The increases are on list prices, before any discounts or rebates that manufacturers sometimes provide insurers and other payers. Some of the increases add thousands of dollars to the cost of already expensive drugs, and come on top of repeated price hikes in recent years.”
Let’s look at the price ‘hike’ in context. I will limit this discussion to the deceptive way in which prices are used. I won’t discuss the fact that neither writer discusses the value of new treatments relative to existing therapies for payers and patients.
Since Ron Cohen , CEO of Acorda Therapeutics,Inc. is one of the main characters of these stories, let’s look at how Loftus reports on the pricing of it’s main product Ampyra, which is used to help multiple-sclerosis patients improve walking. Loftus reports that Acorda raised Ampyra’s price by 11% on Jan. 1, to an annual cost of more than $23,650 a patient.
Ampyra revenues (unaudited) in 2015 were about $ 436 million.
When discounts and rebates to PBMs etc are taken into account, Accorda will gain only about 60% of the price increase.
That does NOT take into account that Acorda provides 2 months of free drug to people with new prescriptions, through our First Step program. At this point, 75% of all new prescriptions are First Step (and a higher percent of all commercial Rxs, as we are not allowed by law to give First Step to Medicare/Medicaid patients). The 10-K notes that 38-43% of patients respond to the treamtment, so First Step ensures the physicians and patients have determined that the patient is a true responder before asking the system to pay for the drug.
It also provides a generous PAP program, giving free drug to a significant portion of the population who are uninsured or underinsured
It also provides co-pay assistance so that no commercially insured patient pays more than $40 for an Rx.
All this an Acorda is not yet not profitable as a company since it is investing 1/3 of net sales n 6 clinical programs for innovative drugs to treat, Parkinson’s, epilepsy, stroke, MS and migraine.
Which means that the money given to PBMs and insurers aren't spent on more R&D. Yet neither Loftus or Robbins acknowledge that PBMs and insurers pocket the rebated portion of these prices. Nor do they note that these organizations then force patients pay to up to 30 percent of the price of the drug which is often marked up by insurers and such pharmacy benefit firms as Express Scripts. Payers know that companies will – after forking over rebates – also pay a big share of the patient’s drug bill. (Which explains why per patient sales are way below list price in many cases.) Indeed, Acorda's copay assistance is provided regardless of where insurers price the drug to patients.
Robbins points to a Senate report claiming Gilead priced drugs so that many people and Medicaid programs could not afford Solvaldi. In fact, payers were pocketing the rebates and deny access to the drugs. The Senate report notes a Gilead memo that states: “While many payers responded to these discounts by opening access broadly, some payers have continued to restrict access despite the discounts. “
Moreover, the Senate report, like Robbins and Loftus, ignored the rebates to the states. A report based on Medicaid data showed that Medicaid rebates for HCV brand medications “typically increased over time and averaged roughly 60% during 2014 across all brand medications.”
Medicaid requires companies to provide rebates (to states) of at least “23.1 % of the Average Manufacturer Price (AMP) per unit” or “the difference between the AMP and the best price per unit” to commercial payers if that rakes in more rebates.
On average, AMP is 59 percent lower than Average Wholesale Price, the so-called ‘retail’ price journalists like Robbins and Loftus use. That means the price used to calculate Sovaldi rebates is $49560 per patient. ($84000 x 59%). Gilead then provided rebates of 33 percent of that price according to the Senate report. That comes out to $33205 per person which is a 60 percent cut from retail price. Which means that drug prices are a vehicle for redistributing income to private and public payers.
Finally, neither Robbins or Loftus put drug price increases (net price or otherwise) into perspective. Loftus states that U.S. prescription-drug spending rose 12.2% in 2014, accelerating from 2.4% growth in 2013. But “price increases for protected brands increased spending by $26.3 billion, contributing 8.2% to total market growth on an invoice price basis; estimated net price growth was substantially lower as rising off-invoice discounts and rebates offset incremental price growth and reduced net price contribution to growth to 3.1%.”
That’s an increase in spending of about $7.1 billion. Total US health care spending increased by $100 billion from 2013-2014. So brand drugs were 7 percent of that amount.
I believe the incremental benefit of this spending is, a Donald Trump would say, huge. But first things first. Reporters should not report on drug prices in a context free zone. Doing so, especially since the factual context is easily available, is a deliberate perversion of reality.
Read More & Comment...
GAO Asked to Assess Viability of FDA’s Complex Generics Pathway
The House Energy & Commerce Committee is calling on the GAO to evaluate whether the FDA’s regulatory pathway for generic versions of complex drugs is sufficient.
Specifically, the committee sent a letter Dec. 10 asking the GAO to assess whether generic versions of nonbiologic complex drugs that are not fully characterized present challenges in meeting generic approval standards.
If the agency concludes that meeting approval standards presents challenges, then the GAO should consult with public and private groups to analyze the following questions:
- What requirements should be established regarding the comparability of the manufacturing process?
- What degree of characterization of the proposed generic version and the reference product should be required to determine safety and efficacy?
- What degree of similarity should be required for the active ingredient of the generic version to be deemed the same as the active ingredient in the reference product?
- What types of evidence should be required to demonstrate bioequivalence?
- How much clinical evidence is needed?
Once those questions are answered, the agency should determine whether current ANDA pathways can address the use of reference products. The study also should make recommendations for the FDA, such as developing policy documents and establishing general principles on evidence needed.
Read the letter here: www.fdanews.com/12-15-15-FDAGenerics.pdf.Read More & Comment...
Compare the approach ASCO takes to the one shaping the Cancer Moonshot 2020 effort.
Read More & Comment...
Today "leaders from large pharma including Celgene and Amgen, biotech including NantWorks, NantKwest, Etubics, Altor Bioscience, and Precision Biologics, major academic cancer centers and community oncologists announced the launch of The National Immunotherapy Coalition (NIC), a historic alliance--in collaboration with Independence Blue Cross, one of the nation’s largest payers and Bank of America, one of the largest self-insured companies in the U.S.--with a singular focus: accelerating the potential of combination immunotherapies as the next generation standard of care in patients with cancer."
This is perhaps the most important scientific initiative undertaken in human history for reasons I will elaborate in another post.
For now, here are the three most important features of this initiative:
1. The QUILT (QUantitative Integrative Lifelong Trial) program is designed to harness and orchestrate all the elements of the immune system (including dendritic cell, T cell and NK cell therapies) by testing novel combinations of vaccines, cell-based immunotherapy, metronomic chemotherapy, low dose radiotherapy and immunomodulators -- including check point inhibitors-- in patients who have undergone next generation whole genome, transcriptome and quantitative proteomic analysis, with the goal of achieving durable, long-lasting remission for patients with cancer.
2. Multiple randomized Phase 2 trials testing genomically and proteomically informed novel combinations of immunotherapy agents, will pave the way to identifying cancer therapy combinations with the lowest toxicity and the highest quality of life.
3. Insurers will be covering whole genome transcriptomic tests in patients receiving immunotherapy. Independence Blue Cross is the first health plan to cover next generation sequencing. The coalition is talking to others, who I think will have to follow.
This is good for patients and innovation. And this approach -- which should be replicated for other illnesses where combinations of treatments - old, new and emerging -- will be gold standard of care.
That mean groups that seek to measure the value of a single cancer drug based on average response -- like ICER, the ASCO Value Framework, and Peter Bach's comical Cancer Drug Abacus etc. will become obsolete. That means Express Scripts effort to use the aforementioned comical Cancer Drug Abacus to establish indication based pricing should be discarded and ignored. Any entity claiming it wants to establish value that does not seek to promote payment and access to precision medicine to match patients to treatments that control and prevent disease most effectively should be considered outdated and obstructionist.
This is a great day for those committed to cures. And that should include everybody.
Read More & Comment...
The death of the transformative musician, artist and filmaker David Bowie to due to cancer underscores once again the value of innovation.
Bowie battled cancer long enough to complete and release his last album on his 69th birthday. You don't have to be a David Bowie to know that using the retail cost of a cancer drug to discourage it's use would sideline many people with a cancer diagnosis.
As Bowie noted: You can neither win nor lose if you don't run the race.
Read More & Comment...
As we move ahead in the US with more biosimilars being submitted for approval – and for interchangeability – attention must be paid to patient outcomes.
Case in point is research presented at the 2013 ACR/ARHP Annual Meeting that hasn’t as yet received the attention it deserves:
Background/Purpose: CT-P13 is an infliximab (INX) biosimilar recently approved by the European Medicine Agency. PLANETAS was a 54-week (wk) randomized double-blind parallel group multicenter Phase I study demonstrating pharmacokinetic equivalence of CT-P13 (5 mg/kg infusion every 8 wks) with INX, in patients (pts) with ankylosing spondylitis (AS) (Park W, ARD 2013;72(S3):516). Here we report results from the extension phase of the Phase I equivalence study, investigating long-term efficacy and safety of extended CT-P13 therapy and switching from INX to CT-P13 in pts with AS.
In this open-label extension study, a total of 174/210 pts who completed PLANETAS entered into the extension phase: 88 were continuously treated with CT-P13 (maintenance group) and 86 were switched from INX to CT-P13 (switch group) for 1 additional year.
Results: At wk 54 ASAS20/ASAS40 and ASAS partial remission rates were similar between groups (CT-P13, 70.5%/58.0% and 20.5%; INX, 75.6%/53.5% and 19.8%, respectively). During the extension, ASAS20/ASAS40 rates were similar in the maintenance group (70.1%/57.5% at wk 78 and 80.7%/63.9% at wk 102) and the switch group (77.1%/51.8% at wk 78 and 76.9%/61.5% at wk 102). ASAS partial remission rates were also similar between groups; 21.8% and 21.7% at wk 78, and 27.7% and 28.2% at wk 102, respectively. An overview of the efficacy data are shown in the Table. Anti-drug antibodies (ADA) were comparable between the two groups and positivity was maintained throughout the study (maintenance group, 22.2%, 24.4% and 25.0%; switch group, 26.2%, 31.3% and 30.7%, at wk 54, 78 and 102, respectively). ADA negative pts achieved higher ASAS40 responses (maintenance group, 62.9%/61.5%/66.1%; switch group, 58.1%/60.0%/71.2% at wks 54, 78 and 102, respectively) compared with ADA-positive pts (maintenance group, 38.9%/36.8%/55.0%; switch group, 41.7%/33.3%/45.8% at wks 54, 78 and 102, respectively) with no differences between the maintenance and switch groups. The proportion of pts with ≥1 treatment-emergent adverse event (TEAE) was lower in the maintenance group (48.9%) compared with switch group (71.4%) mainly due to fewer mild and moderate AEs. Serious TEAEs were identical between groups (4 vs 4 pts). Other tolerability and safety outcomes were similar in both groups (Table). TEAEs due to hypersensitivity and infusion-related reactions were similar in both groups (5 pts in maintenance group vs 2 pts in switch group). There was 1 case of TB in each group and 1 report of prostate cancer in the maintenance group (considered unrelated to treatment).
The efficacy data was good. But the safety data is concerning:
CT-P13 throughout study (N=90)
Switched from INX to CT-P13 in extension phase (N=84)
pts with ≥1 TEAE, n (%)
pts with ≥1 TESAE, n (%)
pts with ≥1 infection, n (%)
ADA positive, n (%)
ADA, anti-drug antibodies; ASAS, Assessment of SpondyloArthritis international Society; ASDAS-CRP, Ankylosing Spondylitis Disease Activity Score C-reactive protein; TEAE, treatment-emergent adverse event; TESAE, treatment-emergent serious adverse event
Biosimilarity and measurement of efficacy is only one dimension. Attention must be paid to effectiveness relative to real-world patient outcomes data. And that means a much closer pharmacovigilance examination of adverse events for both naïve and switched patients.Read More & Comment...
I recently referred to a WSJ article on advances in myeloma drugs by tweeting: Will @joewalkerWSJ @JeanneWhalen @jonathanrockoff ignore PBMs, insurers pocket rebates, make patients pay $1000s http://on.wsj.com/1Oxb69P.. Meaning will these reporters follow up with an article on myeloma treatments hat pins the blame on out of pocket drug costs on drug companies as opposed to or in combination with many other decisions undertaken by PBMs and insurers? Will they look at the impact of such medicines in reducing hospitalizations, infusions, etc that insurers pay for and are more expensive than the drugs patients must partially cover?
Below is a chart that has a link to a specific article on drug costs by each WSJ reporter and whether or not the articles discuss specific issues that would put the cost and value of drugs in context.
Maybe this will clear up the confusion.
Read More & Comment...
The Biotechnology Industry Organization changed its name to the Biotechnology Innovation Organization. The group will continue using the "BIO" acronym.
This is more than just cosmetic. It demonstrates the urgency in both promoting and protecting sustainable innovation in biotechnology. If we are going to argue “value” rather than just “price,” than innovation the most important part of the conversation.
We do what we must, and call it by the best names.
Ralph Waldo EmersonRead More & Comment...
CDC sees the light …
This morning the CDC will ask the National Center for Injury Prevention and Control's Board of Scientific Counselors to appoint a new work group to review the agency's draft guideline for prescribing opioids for chronic pain. It's the next step in CDC's effort to address complaints over the secretive way in which the initial draft was developed. Public comments on the draft guideline are due Jan. 13, and the new group will be tasked with reviewing those, too.Read More & Comment...
Spotlight is a movie about how the Boston Globe's investigative team (called Spotlight) exposed the Boston Diocesan's complicity in protecting child molesters in the priesthood. The most dramatic moment in the film comes when Spotlight's chief, Walter Robinson (played by Michael Keaton) confesses that he buried a story about multiple priests abusing young boys in the in 1970s. At the time, pursuing the story -- compared to easier and less controversial investigations of police corruption, racial discrimination, government incompetency -- went against the ethos of Boston and the narrative that the Church was an integral part of Boston, where most of the reporters were raised.
While nowhere near the level of irresponsibiity, the WSJ coverage about drug costs appears to be shaped by the same set of beliefs and emotions. Every article that the WSJ has written about drug costs in 2015 (with the exception of a blog written by Ed Silverman when Pharmalot was part of the WSJ) ignored the role of pharmacy benefit managers and health insurers in hiking the cost of new drugs used by the most chronically ill patients. The have ignored the fact that PBMs, in particular, pocket 90 billion in rebates, none of which goes to reducing the cost of drugs. They have ignored the fact that the discounted drugs are less than 3 percent of total health plan spending or that as the percent increases, the cost of other services decline. They have ignored the fact that health plans have increasd the number of new targeted medicines -- drugs that work best in specific patients -- by 40 percent since 2014. (See below.)
There are more oversights as well but the list above suggests that the WSJ -- including the most recent WSJ article -- has a blind spot, not a spotlight on the issue of drug costs. The WSJ is burying the real story to sustain a narrative, at the expense of patient well-being.
Our focus in 2016 will be to create greater transparency about how drug prices are set and by who, how those decisions affect patient access to and affordability of such medicines, what affect access has on patient well-being, mortality and health costs and finally, what changes in decisionmaking can reduce spending and increase value (to patients) long term.
Further, we will look at how the reactionary defense of the randomized clinical trial is the greatest rate limiting factor in reducing the time and cost of bringing new medicines, diagnostics and devices to patients. This will NOT be an FDA pinata festival. Rather, the RCT is being protected by leading medical journals, academic research centers, major medical specialty societies and health insurers. Read More & Comment...
Pessimism is necessary in small doses, allowing us to anticipate, adapt to and often avoid failure or danger. It is also an adjuvant for change. (I don’t know what that means but it’s pithy enough to sound smart.) But apocalyptic pessimism, the pessimism that uses every event or fact to confirm certain doom unless we change our ways, is corrosive and unreliable. How do we know?
As Matt Ridley, another professional optimist observes. “For 200 years pessimists have had all the headlines, even though optimists have far more often been right.”
The steam powered train was predicted to bring ruin to England.
The industrial revolution was supposed to bring enduring misery to millions.
The poor will always be poor.
The sky is falling; the ice caps are melting.
We will run out of oil and natural gas.
Drug prices are unsustainable.
If we had followed the pessimists prescriptions (government control or manipulation of wealth, natural resources, population levels and prices) the world would be more like North Korea than North Dakota.
I will focus on the last perennial pessimistic prediction.
We heard and read a lot about drug prices being out of control, skyrocketing, unconscionable, etc.
In every era where new medicines emerge, the same claim is made. Te same bunch of pessimists trot out the same proposals: government price controls and rationing. (Now repackaged as various value frameworks.)
And each time the apocalyptic predictions that new medicines would be unaffordable or would add nothing to our lives or drive up the cost of health care were wrong.
2015 was all about drug pricing because there were so many new and expensive (relative to most consumer purchases) medicines hitting the market. But in fact the high prices – and indeed even the anger about drug prices – are a healthy sign of progress.
It means that diseases that were fatal can be cured for controlled, including Hep C, many cancers and Ebola. It means that more diagnostics are being developed to match people to the right combination of treatments and to monitor health in real time. It means that more investment in new treatments for smaller groups of people will be developed. Nothing sends a signal about the demand for better medicines than high prices.
At the same time, the high prices are forcing drug companies to do something the rest of the health care system will have to do: change business and financial models to make the case for value and increase the pressure to abandon outdated clinical development tools (randomized clinical trials) and find other ways to demonstrate the health benefits of their goods and services.
Prediction is over-rated when it is too precise. But the general trend of medical innovation, the digitization of biology, is part of a broader evolution in how we create and use the things that give us joy, insight and well-being. Our world is better because the footprint for change is getting smaller and faster. In a WSJ op-ed Andy Kessler wrote that “on Dec. 29, 1959, the physicist Richard Feynman delivered a famous speech at the California Institute of Technology titled “There is Plenty of Room at the Bottom.” He predicted almost limitless possibilities if we could “manipulate and control things on a small scale.” He nailed the next five decades of ever-shrinking realms that ultimately produced trillion-dollar markets in microelectronics, nanotechnology and bioengineering through DNA-level manipulation.…The smaller technology shrinks, the bigger the world can grow. Smaller transistors, faster processors, cheaper sensors will all allow innovators to tackle problems with tremendous precision. It’s as if, because there is plenty of room at the bottom, now there is plenty of room at the top.”
We are increasingly able to navigate and understand what Lee Hood calls “the dark matter of human biology.” New medicines are the result of our ability to explore health, wellness and disease at a molecular level.
Here’s a non-pessimistic prediction: the next trillion-dollar market will be new treatments and algorithms that control disease and keep people healthy. You have to try really hard to despair about that! Read More & Comment...
“The price can’t be considered a stagnant proposition. It needs to be a vibrant, living thing,” said FDA consultant Peter Pitts.
A key unknown, however, is whether drug makers will become more transparent about their pricing strategies when negotiating with these payers. This is a “very important” part of determining value for patients, noted Peter Pitts, a former Food and Drug Administration official who now heads the Center for Medicine in the Public Interest.Happy New Year Read More & Comment...
I bet you didn’t because not one media outlet covering the release of CMS drug spending data mentioned it: Instead, most of the articles started off like… oh, wait why don’t I just share with you the highest form of journalistic misdirection by the WSJ's Peter Loftus.
“Hefty price increases for a number of prescription drugs contributed to higher spending by the U.S. Medicare program in 2014, according to new government data released Monday. The Centers for Medicare and Medicaid Services identified at least five drugs that were covered under Medicare’s Part D drug benefit and had increases of 100% or more in cost per unit from 2013 to 2014. At the top of the list was Vimovo, a pain reliever whose cost-per-unit rose more than 500% after Horizon Pharma PLC purchased rights to the drug from AstraZeneca.”
Still waiting for a detailed breakdown of who did what? Don’t look under the Christmas tree because Santa isn’t delivering it this year. Neither is the WSJ or Bloomberg (who produced what can only be called a deceitful piece of reporting on the relative price of drugs in Europe and the US).
Instead after providing a description of the new CMS dashboard, Loftus concludes: “The new data emerge amid a rising debate over drug pricing. Cancer doctors have complained that the high costs of new drugs don’t seem to be tied to the benefits provided by the drugs. Presidential candidates Bernie Sanders, Hillary Clinton and Marco Rubio have criticized drug prices on the campaign trail. A U.S. Senate committee recently held a hearing about companies that have dramatically raised prices after acquiring older drugs.”
Just enough ‘information’ to reinforce the narrative that drug prices are wildly out of control because they don’t reflect value if they are new or R&D costs if they are old. Either way, the real goal of the piece is to imply that drug profits are too high, a view stoked by previous articles by Loftus and others. Any word about how PBMs and other third parties pocket $90 billion in drug rebates never shared by patients? Come on.
The late Christopher Hitchens observed that he had “become too accustomed to the pseudo-Left new style, whereby if your opponent thought he had identified your lowest possible motive, he was quite certain that he had isolated the only real one. This vulgar method, which is now the norm and the standard in much non-Left journalism as well, is designed to have the effect of making any noisy moron into a master analyst.”
Noisy morons as master analysts. Now THAT’s accurate reporting.
Read More & Comment...
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."
Read More & Comment...
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 Barrie Read More & Comment...
President 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.Read More & Comment...
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.Read More & Comment...
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:
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.Read More & Comment...
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.
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