Latest Drugwonks' Blog
The percent and absolute number of colonoscopies and other procedures performed on colon cancer patients in hospitals has declined. Also, the length of hospital stays and number of in hospital deaths have fallen by 30 percent. Indeed, colon cancer treatment costs in America are growing more slowly than in Europe.
Yet, over the past 30 years, death from colon cancer has declined faster in America than in Europe. Nearly 65 percent of all Americans will live 5 years or longer with colon cancer compared to 59 percent of Europeans.
The difference? Americans get screened and treated faster with newer medicines and diagnostics. The way to saving money and lives is faster innovation, not more regulation.
Yesterday I had the pleasure of participating on the keynote panel at the World Pharma Congress. My fellow panelists were Karim Dabbagh (Executive Director and Head, External R&D and Innovation Research Units, Pfizer Worldwide R&D) and Glen Gaulton (Professor, Pathology and Laboratory Medicine, Executive Vice Dean and Chief Scientific Officer, Perelman School of Medicine, University of Pennsylvania). The panel was expertly chaired by Rick Turner (PGCE Senior Scientific Director, Clinical Communications, Quintiles and Fellow, Society of Behavioral Medicine).
Our topic was, “Advancing Pharma R&D through Communications and Collaboration.” It was an exciting 90 minutes.
The issue of out-sourcing R&D isn’t new – but it’s mighty contentious. And it’s the new reality of drug development. But, if we are to learn any lesson from the CRO experience, it’s that while we say “partnership,” the danger is that it devolves into a vendor-like relationship. It’s the Golden Rule. He who has the gold makes the rules. Will that be acceptable to high-level, big ego Ivy Hall-ers?
And then there’s the issue of academic priorities, specifically tenure. Does industry funding carry the same weight as NIH grants when it comes to advancing a university career? Not at present. That will have to change.
Will university researchers pursue their programs with the same time-driven focus as their pharma counterparts? After all, there aren’t any quarterly analyst calls to worry about. And, as already mentioned, if the funding is viewed as secondary to “tenure-track” funding, how will the work be prioritized?
And then there’s the IP question. According to both Dabbagh and Gaulton, IP negotiations are generally smooth. That’s good news – but there’s a lot of road ahead on the R&D partnership superhighway.
CROs, at least know what needs to be done. Researchers (whether inside a company or a university) don’t. That’s why it’s called research. One issue that arose is the need for total sharing and absolute transparency between industry and academic partners on all levels of the engagement. Is this happening? Sometimes. The good news is that when it does occur – the relationship (and results) are more positive.
But talk with CROs and you’ll hear their frustration over being instructed what to do by their pharma masters. (In most cases their former employers.) Fortunately, that too is changing as the urgency of new and innovative clinical trial protocols and programs becomes self-evident.
Need drives change. Just as CROs are now really partnering with pharma to drive the development of personalized medicine, so too must academic researchers and their industry partners collaborate on the continued evolution of pharmaceutical innovation. It will take discipline and focus. It will be risky. And it will take will. There is a confluence of interest.
To borrow some FDA parlance, will industry/academic partnerships result in expedited pathways in drug development? It must – because if it is only viewed as a cost-saving mechanism it will fail.
“Change is not required. Survival is not mandatory.”
-- W. Edwards Deming
BioCentury reports that the FDA is seeking comments on approaches to make available to non-FDA experts and other interested parties de-identified and masked subject level clinical trial data derived from regulatory applications. According to the notice published in the Federal Register, the agency said the proposal is driven by its "desire to improve the product development process," noting that data may be useful "to address key hurdles in drug development" like validating new trial endpoints or clarifying how products work in different diseases. FDA also noted that pooled data can also be used to identify and analyze safety issues.
FDA is seeking input on strategies to minimize the ability to identify specific products, including making available certain data from a random sample of patients or pooling data for a number of products within a product class. The agency is also seeking input on how to mask data and limitations to making the masked data available, as well as input on de-identifying data. Additionally, FDA is seeking input on situations where disclosing masked data would be the most useful to advance public health. FDA said its proposal does not address making available unmasked safety and efficacy data that can be linked to a specific, identified application, including full study reports. The agency also said it "will not make available business-related confidential commercial information contained in product applications." Comments are due Aug. 3.
By the end of June, EMA is slated to publish a draft policy for public consultation providing more details on its plan to proactively release patient-level clinical data for approved drugs.
The New York Times reports, “Government officials, drug companies and medical experts, faced with outbreaks of antibiotic-resistant “superbugs,” are pushing to speed up the approval of new antibiotics, a move that is raising safety concerns among some critics.”
But the only “critics” the Gray Lady was able to unearth were a former mid-level FDA reviewer and old reliable agency critic Steve Nissen.
Critic #1: “There is really no way of knowing how these drugs are going to perform,” said Dr. John H. Powers, a former F.D.A. antibiotics reviewer who is now an associate professor at George Washington University in Washington.
That is clearly not correct. These “special medical use” products aren’t going direct from bench to bedside – they must still undergo rigorous, FDA-approved development programs -- albeit carefully monitored trunicated ones with robust Phase IV follow-up.
Critic #2: “The big problem is controlling the marketing,” said Dr. Steven E. Nissen, a cardiologist at the Cleveland Clinic and a frequent critic of the F.D.A. “Companies can get a drug on the market for a narrow indication and before you know it, it is being used in everybody.”
This is a recording.
Now on to the real issue – a huge and global public health crisis.
“We are facing a huge crisis worldwide not having an antibiotics pipeline,” said Dr. Janet Woodcock, director of the Center for Drug Evaluation and Research at the Food and Drug Administration. “It is bad now, and the infectious disease docs are frantic. But what is worse is the thought of where we will be five to 10 years from now.”
The compete New York Times article can be found here.
Yesterday, Indiana Governor Mike Pence joined Indiana-based global life sciences and research university executives to unveil the Indiana Biosciences Research Institute, the first industry-led collaborative life sciences research institute in the country.
The Indiana Biosciences Research Institute is a statewide public-private partnership advanced by BioCrossroads and led by Indiana’s life sciences industry, with support from the State of Indiana and partnerships with Indiana’s research universities to discover, develop and deliver biosciences innovations in Indiana. The Institute will serve as the centerpiece project of the BioCrossroads public-private collaboration through its attraction of world-class scientific leaders and life sciences research dollars to Indiana, while focusing on human health solutions for improving the lives of Hoosiers and people around the world.
“Indiana has built a life sciences ecosystem unlike any other state and faces a new season of opportunity as a result,” said Governor Pence. “The Institute will strengthen Indiana’s reputation as a global life sciences hub and produce breakthroughs that will attract new investment to our state and create good-paying jobs for Hoosiers.”
The Indiana Biosciences Research Institute is the result of leadership from industry executives from Eli Lilly and Company, Dow AgroSciences, Roche Diagnostics, Cook Medical, Indiana University Health and Biomet and the Governor of Indiana, with active support in initial development by BioCrossroads. Indiana’s research institutions, including Indiana University, Purdue University and the University of Notre Dame also are participating in the development process.
“This Institute comes at a pivotal time in our state’s evolution as a global life sciences leader. With a bioscience sector that now contributes more than $50 billion a year to the Hoosier economy, Indiana is ranked by BIO and Battelle as one of the top five states in the nation in terms of our total number of life sciences companies and employees. Through the Institute, BioCrossroads believes we have found a bold way to raise our game in Indiana by building the platform that will truly take us to the next level of success,” said David Johnson, president and CEO of BioCrossroads, an organization focused on investment, development and advancement of the state’s signature life sciences strengths.
The Indiana Biosciences Research Institute is developing a novel operating model, with industry providing a major source of funding and defining the Institute’s research focus to optimize commercialization opportunities. The Institute also draws on a life sciences industry cluster that is one of the largest and most diverse in the nation, with global companies that are developing next-generation drugs and pharmaceuticals, diagnostics tests, medical devices, cell-based therapies, agricultural biotechnology and animal health and production solutions. This diversity of industry capabilities creates opportunities for Indiana-based life sciences companies to work in collaboration – not competition – toward common scientific discoveries.
“The research institute will change the bio-landscape of our region,” said Bart Peterson, senior vice president at Eli Lilly and Company. “Indiana’s life sciences companies spend billions of dollars in research and development each year to advance health care innovations for improved human health. The Institute will help us nurture our partnerships across the country and develop more intellectual capital here in Indiana – allowing us to keep more research dollars in the state, attract more federal research funds, and draw top scientific minds to feed our research pipeline and local economies.”
“Having the best talent and the best scientific minds is crucial for growing our life sciences industry cluster in Indiana,” said Steve Ferguson, chairman of Cook Group, which is celebrating its 50th anniversary this year. “World-class scientific talent at the industry and academic level is one of the state’s most powerful economic development tools. Just as companies like Lilly and Cook Medical started as small, entrepreneurial operations, we expect the Institute to draw the best and the brightest to Indiana to further deepen our life sciences industry roots and grow more business opportunities.”
As part of the Institute development process, industry leaders have defined common scientific interests for research and discovery. The Institute will initially focus on the most pressing global and local interrelated human health issues: cardiovascular disease, diabetes, obesity and nutrition. These interrelated metabolic disorders are a major economic burden and a leading cause of death in the United States. Risk factors, such as high blood pressure and insulin resistance, allow for early disease detection, and timely preventive actions, such as through improved nutrition, and early intervention can slow or prevent the onset of disease. This is an important scientific discovery subject for the approximately 35 percent of Americans who suffer from cardiovascular disease and metabolic disorders, and is a significant risk for Hoosiers who suffer disproportionally from these diseases.
“We have an opportunity to not only help millions of people around the world who are battling these metabolic disorders, but we can have significant impact on Hoosiers who suffer more than the average American from diabetes and who rank 8th in the nation in terms of obesity,” said Jack Phillips, president and CEO of Roche Diagnostics. “The Indiana Biosciences Research Institute will provide the platform to deliver significant scientific advancements that could improve Hoosier lives.”
“The ability to better understand metabolic disorders impacts the work of nearly every life sciences company in Indiana,” said Antonio Galindez, president and CEO of Dow AgroSciences. “Together, we can develop a deeper understanding of the pathways leading to metabolic disease and apply those discoveries to not only medical interventions, but also to greatly enhanced nutritional sources developed through advanced crop improvement technologies and other advancements in human health.”
“Patients and clinicians have much to gain by the success of this critically important new venture,” said Daniel F. Evans Jr., president and CEO of Indiana University Health. “The discoveries and inventions generated by the Institute will be used by our physicians to care for patients. To accelerate the pace of innovation from lab bench to patient bedside, we are pleased to make our healthcare system available to researchers to test potential new treatments and therapies.”
The estimated $360-million Indiana Biosciences Research Institute is a non-profit entity that is anticipated to be supported largely by corporate and philanthropic funding with oversight from a largely donor-based board of directors representing the life sciences industry, the state of Indiana, academia and nonprofit donors. The State of Indiana has appropriated $25 million for the biennium for start-up costs. An additional $25 million in start-up funding is being sought from industry and philanthropic sources, which will be used in part to recruit a nationally recognized CEO and research fellows. The remaining capital funding will be sought from corporate and philanthropic sources, and ongoing operating costs will be funded through Institute endowment proceeds, industry-sponsored research and federally funded research.
The Indiana Biosciences Research Institute will attract local and national scientific leaders beginning with the CEO and the recruitment of research “Indiana Fellows.” These research fellows will lead teams of scientists and partner with industry and universities on research projects. These teams will consist of experts across a spectrum of competencies, including bioengineering, bioinformatics, nanotechnology and agriculture. These cross-functional teams will share resources and research laboratories at the Indiana Biosciences Research Institute and will work onsite at industry and research university labs with academic and industry scientists.
“As the scientific discovery process increases in complexity, more companies are looking outside their own walls for multi-disciplined team members to help move innovation forward, at a faster rate, in order to remain competitive in an increasingly global marketplace,” said Jon Serbousek, president, Biomet Biologics. “A cross-industry, statewide partnership focused on commercially translatable innovation on the cutting edge of basic and clinical science will help the state’s life sciences industry move to the next level.”
“The Indiana Biosciences Research Institute provides a new and exciting opportunity for our research universities to work with our statewide life sciences industry,” said Bill Stephan, vice president for engagement at Indiana University. “The Institute will bolster our ongoing efforts to recruit highly regarded local and national researchers and graduate students. We envision the dynamic of researchers from the Indiana Biosciences Research Institute working alongside researchers in industry or university labs, strengthening long-term collaborative research and funding opportunities.”
More information about the Indiana Biosciences Research Institute can be found at www.indianabiosciences.org.
Thinking personalized medicine? Think outcomes.
The Pink Sheet reports on a new study by IMS Health shows payers globally are making more drug reimbursement decisions based on real-world evidence - including 25% of more than 100 identified case studies using biopharma industry-generated data.
IMS Health Inc. has developed a report concluding the data is increasingly influencing how payers are covering pharmaceutical products on a global basis.
The report lists more than 100 non-safety cases from around the world where real-world evidence (RWE) informed payer coverage decisions and notes that while "payers are a powerful stakeholder in setting the RWE agenda, proactive pharma engagement matters: manufacturer-generated RWE influenced over 25% of observed decisions."
The report ranks 10 countries based on their supply of real-world evidence, the demand for it and how RWE is applied. On a scoring scale with a maximum of 20, the leading country - the U.K. - scored only an 11, which reflects that "no country has ideal conditions for RWE use in a scalable manner" and "highlights RWE's infancy," the authors state. "Lower scores indicate that RWE is relatively less available or more costly to generate with less consistent or transparent use in decision making. But even in markets with lower scores, RWE is still relevant."
The U.S. is tied for third in the rankings, sharing a score of 8 with the Netherlands. Among the other countries analyzed, Sweden scored 10; France and Italy both scored 6; Denmark, 5; Canada, 4; Germany, 3 and Spain, 2.
The U.S. is unique from the other countries examined in the study in that owners of real-world data "share it without stipulating how it should be used beyond ensuring individual privacy." Most U.S. insurance companies and providers sell data and only use it to support specific analyses about their own populations," giving pharmaceutical companies broad data access that can be used all the way from clinical trial design to post-market commercial support.
But the U.S. market lacks a clear framework for sharing real-world evidence. "Successful U.S. strategies involve evidence platforms and tools that support multiple internal stakeholders. However, without clear frameworks, the channels for external engagement are more nuanced. Only selected payers engage readily on RWE, and FDA regulations on RWE dissemination are more restrictive than in [other] countries. A differentiated engagement approach is needed, requiring creative thought and investment."
In contrast, the report notes that the U.K., Sweden and the Netherlands "all have strong national [health technology assessment organizations], suggesting an impact from concentrated decision making." In these markets, "pharmaceutical companies should set high ambitions for RWE plans, demonstrating value and engaging stakeholders based on a variety of real-life views (e.g. disease, product, class, cross-care settings, long-term outcomes, payer-relevant quality of care indicators). They must fully exploit RWE beyond traditional evaluations to enable commercial strategy, leverage outcomes-based marketing and use innovative evidence tools with local health systems."
The report notes that countries such as France and Italy have significant demand for real-world data, but limited supply.
"France's bold vision includes using RWE for cost-effectiveness assessments and regular class reviews without detailing how extensive data in the health system can be accessed or levered," the authors observe. "In Italy, there is widespread use of pay-for-outcomes or coverage with evidence development (CED), but how these schemes inform coverage or pricing decisions based on the captured data remains unclear. While manufacturers can react to these limited demands for RWE, the more innovative ones might place a bet that the markets will expand RWE use over time. Pharmaceutical companies must develop some RWE capabilities for payer's current focus areas."
The four countries at the bottom of IMS' list - Denmark, Canada, Germany and Span - "may use RWE more in [the] future but face significant hurdles today," including strong data privacy rules and fragmented health care landscapes, the authors write. "In these markets, pharmaceutical companies benefit from engaging directly with selected stakeholders willing to lead on RWE. Given the limited resources of these stakeholders and the large number of manufacturers, developing a clear value proposition and local RWE capabilities are essential to becoming a preferred partner."
Outcomes talk.
The “Three Cs” of social media are content, context and channel. They are not severable.
The media remains the message.
As discussed previously (“Thug Regulation”), Walgreens is feeling the hot breath of the DEA on its neck. A new letter is designed to explain and assuage physician concerns over the chain's tighter restrictions for controlled substances – particularly opioids. (According to the California Medical Association, the Walgreens’ policy is in response to recent investigations and actions by the DEA.)
According to the Walgreens missive, “We realize that this process may generate questions and comments from both you and the patient and we will do our best to respond in a professional and courteous manner. We recognize that sharing appropriate information with our pharmacists may require additional time from you or your office staff and we want to thank you in advance for partnering with us to provide the best care for patients.”
(The complete Walgreens letter can be found here.)
The issue goes beyond the DEA’s behind the scenes efforts to deputize pharmacists to better control controlled substances (and the important issue of access to pain medications). The bigger issue is re-inventing the role of the 21st century pharmacist.
Some questions to consider:
What will the role of the 21st century pharmacist be in improving drug safety and medication adherence via more proactive (and remunerated) patient education?
How can pharmacists become better integrated (beyond Med Guides) into the FDA’s Safe Use of Medicines initiative?
When will pharmacy synchronization programs really kick into gear, and how will states help to jump-start these important initiatives?
Stay tuned.
When some pharmacists refused to dispense Plan B (aka, “the morning after pill”) because of their personal moral beliefs, reproductive rights advocates and the media swooped down with all guns ablaze. “Denying access!” they cried. “Abusing their state licenses!” shouted others.
And, of course, there was litigation. In Washington, U.S. District Judge Ronald Leighton ruled the Evergreen State cannot force pharmacies to sell Plan B or other emergency contraceptives, saying the state's true goal was to suppress religious objections by druggists — not to promote timely access to the medicines for people who need them.
(Washington State – as do many states -- requires pharmacies to dispense any medication for which there is a community need and to stock a representative assortment of drugs needed by their patients.)
The state argued that the requirements are legal because they apply neutrally to all medicines and pharmacies, and that they promote a government interest — the timely delivery of medicine, including Plan B, which becomes less effective as time passes.
But Leighton ruled that the state allows all sorts of business exemptions to that rule. Pharmacies can decline to stock a drug, such as certain painkillers, if it's likely to increase the risk of theft, or if it requires an inordinate amount of paperwork, or if the drug is temporarily unavailable from suppliers, among other reasons.
Not surprisingly, today the issue of the dispensing of pain medicines is front and center. The California Medical Association has received reports from physicians that Walgreens pharmacists are refusing to fill controlled substances prescriptions without additional information from the prescriber.
Physicians are being asked to provide information on diagnosis, ICD-9 codes, expected length of therapy and previous medications tried and failed. Walgreens has also sent letters to prescribers that provide an overview of its newly revised policy on good faith dispensing of controlled substances and cites a pharmacist’s corresponding responsibility to ensure that every prescription for controlled substances is “issued for a legitimate medical purpose.”
“Overreaching!” claim some. “Second-guessing physicians!” say others.
But, unlike the Plan B debate, this isn’t an issue over morality – it’s being driven by Drug Enforcement Agency thuggery. According to the California Medical Association, the Walgreens’ policy is in response to recent investigations and actions by the DEA.
It seems the DEA, frustrated that the FDA is taking too long to decide if hydrocodone combination products should be further restricted, has decided to take matters into its own hands, strong-arming pharmacists into submission.
And, it seems, they’re taking their cue from Congress. West Virginia Senators Manchin and Rockefeller sent a letter to FDA Commissioner Margaret Hamburg earlier this month complaining that the agency is dragging its feet on “upscheduling” drugs containing hydrocodone from Schedule III to Schedule II. Such a move would bring tighter restrictions on a variety of widely used opioid drugs.
Tighter restrictions, that is, for people that really need the medications, more paperwork for physicians and a heavier workload for pharmacists. Abusers and criminals rarely follow FDA regulations.
When you have a hammer, every problem looks like a nail. The DEA sees opioid abuse and seeks to minimize access to them. That’s a law enforcement solution. They mean well – but are behaving like a bull in a china shop.
The FDA is already providing thoughtful solutions that address the problem of abuse without restricting appropriate access. Recent agency decisions such as its ban on generic forms of the non-abuse-deterrent formulation of Purdue Pharma’s OxyContin show the FDA is actively involved in the fight against addiction.
Here’s a good briefer from FDA News on the recent Journal of General Internal Medicine study on DTC. The “aha” (both missed and misinterpreted by mainstream media reporting) is that this research does not claim DTC advertising causes physicians to inappropriately prescribe medications.
The FDA’s own research, over many years, confirms that same diagnosis. It’s important. The simple fact of the matter (as opposed to the spin) is that statins are not being prescribed to patients who do not have high cholesterol.
DTC Marketing Study Implies Doctors Overprescribe Statins
A new study on direct-to-consumer (DTC) marketing of statin drugs suggests the prevalence of such ads promotes over-diagnosis in patients that may not be at risk for cardiac events. But the study incorrectly implies that physicians are to blame for inappropriate prescriptions, experts say.
A study of 106,000 adults published recently in the Journal of General Internal Medicine showed consumers exposed to advertisements about statins are 16 to 22 percent more likely to be prescribed the drugs, and 16 to 20 percent more likely to be diagnosed with high cholesterol.
These associations were driven almost exclusively by men and women at low risk for future cardiac events, the study states. Use of the popular cholesterol-lowering drugs may lead to an increased risk of Type 2 diabetes, memory loss or confusion, the FDA says.
But don’t blame physicians, Peter Pitts, president and co-founder of the Center for Medicine in the Public Interest and former agency staffer, said Tuesday.
“If you ask your doctor for [AstraZeneca’s] Crestor, they are likely to prescribe Crestor,” he said, adding that he believes the study’s dire conclusions are overblown.
“You either have high cholesterol or you don’t — it’s a simple blood test.”
While reluctant to trumpet brand pharma and its marketing practices, Pitts said anything that drives people to talk to their doctors about symptoms is a good thing and should be applauded.
Drugmakers’ DTC marketing is an ongoing concern at the FDA, with several studies on the issue planned or currently in process. The agency is in the early stages of a survey of healthcare providers about their thoughts on prescription drug advertising and marketing, specifically DTC marketing and how it affects or influences the prescribing of certain medicines.
Earlier this month, the agency launched two studies examining the use of composite score in DTC marketing.
The Direct-To-Consumer Television Advertising Exposure, Diagnosis with High Cholesterol, and Statin Use study, can be found at www.ncbi.nlm.nih.gov/pubmed/23463454.
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.
