CER Daily Newsfeed®
Articles
Payer-Designed Clinical Pathway Program Provides Value for Patients With Breast Cancer (10/19, Zachary Bessette, Journal of Clinical Pathways) reports “...Clinical pathways are designed to be value-driven and support evidence-based therapies that aim at high-quality, affordable care. However, limited real-world data exist to confirm the value of pathway regimens, particularly for patients with breast cancer.” Full
To Innovate on Controlling Health Care Costs: Follow the Money (10/18, Sarah Thomas, Deloitte Insights) comments “...A recent study in the journal Health Affairs found that value-based insurance design — which reduces cost-sharing for high-value services — can improve medication adherence without increasing overall spending. But the value in value-based care has to go beyond cost. A patient recently diagnosed with cancer, for example, might be more concerned with outcomes than with cost. Employers can help their workers navigate care with tools that identify high-quality providers and offer comparable information about prices.” Full
UK: NICE Rejects Lilly's Breast Cancer Drug Verzenios (10/19, Selina McKee, PharmaTimes) reports “...According to the National Institute for Health and Care Excellence, the drug is as effective as both palbociclib and ribociclib, which NICE already recommends as options for treating hormone receptor-positive, human epidermal growth factor receptor 2 (HER2)-negative, locally advanced or metastatic breast cancer as initial hormone therapy. However, ‘once the patient access schemes are taken into account, abemaciclib is not cost effective relative to the others,’ it said.” Full
Press Releases
$3.3 Million Grant to Train Learning Health System Researchers (10/18, Albert Einstein College of Medicine Press Release) “Einstein-Montefiore has received a $3.3 million grant to establish a Learning Health System Center of Excellence, called Excellence in Promoting LHS Operations and Research at Einstein/Montefiore...The center is funded by an award through the Agency for Healthcare Research and Quality and the Patient-Centered Outcomes Research Institute...” Full
McDonough Receives NIH K01 Grant to Study Resistant Hypertension (10/18, UF Health Press Release) “...With support from the 4-year, $502,987 grant, [Caitrin W. McDonough, Ph.D.] will examine electronic health records, and prescription claims data for about 15 million individuals from the OneFlorida Data Trust and REACHnet to identify characteristics in the data that are associated with the disease. Both clinical data research networks are part of the Patient-Centered Outcomes Research network.” Full
Huge Variations Between Countries in Time for Reimbursement Decisions on New Cancer Drugs (10/19, European Society for Medical Oncology Press Release) “Some European countries take more than twice as long as others to reach health technology assessment decisions to reimburse new cancer drugs following their approval by the European Medicines Agency. The average decision time is longer than one year in some countries, according to a study to be reported at ESMO 2018 Congress.” Full
Journals
Patient-Engaged Research: Choosing the "Right" Patients to Avoid Pitfalls Emily A. Largent, Holly Fernandez Lynch, Matthew S. McCoy September/October 2018, The Hastings Center Report Wiley
National Standards for Public Involvement in Research: Missing the Forest for the Trees Matthew S McCoy, et al. October 18, 2018, Journal of Medical Ethics Journal of Medical Ethics
SHERPA: A New Model for Clinical Decision Making in Patients with Multimorbidity Edmund Jack, Neal Maskrey, Richard Byng October 20, 2018, The Lancet The Lancet
Reports
Bringing Real World Evidence into Clinical Research October 2018 UBC
Articles
Video: Real-World Evidence: Powering Health Care (10/17, Deloitte Insights) “Many drug makers are increasingly swimming in new sources of data. Find out how they can channel real-world evidence to influence patient behavior, answer hard questions on what works for whom and at what price, and power the future of health care.” View Video
Real-World Data: Precertification Could Aid Use For Regulatory Decisions (10/17, Sue Sutter, Pink Sheet) reports “...A precertification program for medical data aggregators could help expedite the use of real-world evidence for regulatory purposes. However, such a program could be a cost burden for small data curators, and it's unclear who would do the certifying and what the criteria would be for accreditation, a panel of experts said at a recent Duke-Margolis Center for Health Policy meeting.” Paid Subscription Required
Amgen, Lilly Migraine Drugs Make it onto Express Scripts' Preferred List (10/17, Jacob Bell, BioPharma DIVE) reports “...‘While drug makers have priced CRGP inhibitors in line with independent value-based assessments from organizations such as the Institute for Clinical and Economic Review, they are significantly more costly than previously available preventive migraine therapies and thus require conscientious management to ensure optimal patient outcomes,’ Express Scripts said...” Full
The Trump Administration Hints at New Drug Pricing Regulations on Medicaid, Biologics (10/17, Nicholas Florko, STAT Plus) reports “The White House on Wednesday provided the clearest evidence yet that, eventually, some drug makers might not have to cut Medicaid the best deal on prescription drugs...[T]he scant context clues may signal the administration is moving forward with a plan to exempt certain drug makers from the steep discounts they're required to give Medicaid. It promises changes to the rebate program to ‘support’ value-based payments between states and manufacturers.” Paid Subscription Required
Colorado Moves Toward Outcome-Based Contracts For Medicaid Rx Drugs (10/17, InsideHealthPolicy.com: Inside Drug Pricing) reports “...Cathy Traugott, the pharmacy manager at Colorado Medicaid's department of health care policy and financing, said the state plans to file a State Plan Amendment with CMS soon. ‘The idea of paying based on value is a great concept,’ Traugott told IHP. ‘I want the ability to pursue it if it makes sense.’” Paid Subscription Required
UnitedHealth Group's Value-Based Care Spend Hits $69 Billion (10/17, Bruce Jaspen, Forbes.com) reports “...‘We've been very successful in that migration towards a population orientation,’ [Dan Schumacher, president and chief operating officer of UnitedHealth's UnitedHealthcare business,] told analysts Tuesday. ‘If you look at where that sits today, about half of our value-based spend is in the more progressive relationships that orient around population outcomes. Some of our more progressive relationships are actually with our ACO partners, and in those relationships, we work to share data, share insights, drive better coordination, close gaps and care for people.’” Full
Here's Why You Can Feel Optimistic About The Future Of Healthcare (10/16, Erika Fry, Fortune) reports “...[University Health Network CEO Kevin Smith] said we've got to ‘let the consumer choose based on real data, not drown them in information’ to determine ‘is this the best evidence based care fort the best outcome at the best price?’...‘There is a consumer component,’ he said, noting that some, regardless of evidence of value added, want the latest and greatest medical treatments. It comes down to: ‘Do you want greater benefit for a broader population, or do you want the best service available with the latest technology?’” Full
Industry Groups Sound Off on ACO Program Changes, but Half of Participants Say They Will Stay (10/17, Evan Sweeney, Fierce Healthcare) reports “...A new survey by the National Association of ACOs released this week shows that 60% of ACOs currently in the MSSP wouldn't enter the program under the proposed structure, which includes reducing shared savings from 50% to 25% and shortening the amount of time ACOs can remain in a one-sided risk model from six years to two years. Nearly half of the ACOs surveyed said they are likely to continue even if those changes take effect.” Full
UK: NHS Green Light for Novartis Skin Cancer Combo (10/18, Selina McKee, PharmaTimes) reports “The NHS now has three months to ensure that Novartis' combination therapy dabrafenib/trametinib is routinely available to eligible skin cancer patients, after cost regulators published guidance backing funds for the treatment.” Full
Why the EU Should Support the Standardization of Health Outcomes Measurements (10/18, The Pharma Letter) comments “...EFPIA calls on the EU to drive the evolution towards patient-centered and outcomes-focused healthcare by assessing and benchmarking EU health systems through patient-relevant outcomes data. Few other investments on the part of the EU would have such power to improve the health of patients and reduce health inequalities across Europe!” Paid Subscription Required
Press Releases
Verana Health Debuts New Cloud-Based Software Applications to Accelerate Life Science Innovation and Enhance Patient Care (10/18, Verana Health Press Release) “...The company is also building industry solutions that provide insights derived from structured data elements and clinical notes to enable life science companies to...Understand the patient journey with a research tool that measures comparative effectiveness and disease treatment variations on an aggregated patient population to identify the most efficient and beneficial healthcare interventions.” Full
Journals
Strengthening Health Technology Assessment Systems in the Global South: A Comparative Analysis of the HTA Journeys of China, India and South Africa MacQuilkan K, et al. 2018, Global Health Action PubMed
Comparative Effectiveness of Delayed-Release Dimethyl Fumarate versus Interferon, Glatiramer Acetate, Teriflunomide, or Fingolimod: Results from the German NeuroTransData Registry Braune S, et al. October 16, 2018, Journal of Neurology PubMed
Does Evidence-Based Medicine Training Improve Doctors' Knowledge, Practice and Patient Outcomes? A Systematic Review of the Evidence Simons MR, et al. October 17, 2018, Medical Teacher PubMed
Comparing the Combination Therapy of Ezetimibe and Atorvastatin with Atorvastatin Monotherapy for Regulating Blood Lipids: A Systematic Review and Meta-Analyse Ai C, Zhang S, He Q, Shi J. October 17, 2018, Lipids in Health and Disease PubMed
Cost, Context, and Decisions in Health Economics and Health Technology Assessment Culyer AJ October 17, 2018, International Journal of Technology Assessment in Health Care PubMed
Reports
Incentivizing Pharmaceutical Testing in an Age of Off-Label Promotion October 2018 NYU Law Review
Articles
ICER Is Weighing Early Research Consulting Work With Pharma (10/16, Jessica Merrill, Pink Sheet) reports “...The Institute for Clinical and Economic Review is evaluating a program to provide consulting services to pharmaceutical manufacturers...ICER insisted it would pursue such a program only in a manner that would preserve ICER's independence and integrity and only if it would further its core mission to help patients access high-value care. Additionally, the organization said it would be fully transparent about its funding.” Paid Subscription Required
ICER Weighs Plan to Offer Trial Design Advice to Pharma Companies, for a Price (10/16, Conor Hale, Fierce Biotech) reports “...As to whether this will ultimately affect the drug-pricing watchdog's impartiality, Bernstein analyst Ronny Gal wrote in a note to investors that if ICER does go through with the plan, it would still represent a gain for the pharma industry regardless of its implementation. ‘We do not question ICER staff's integrity, but it is very hard to not grow to like your clients,’ Gal wrote. ‘This funding mechanism is (respectfully) inherently problematic for an organization whose mission is to become the fair arbiter of drug value.’” Full
The Foundation Submits Letter of Support to FDA on Developing Patient-Focused Clinical Outcome Assessments and Endpoints (10/16, Celiac Disease Foundation Blog) comments “On October 15, 2018, the Celiac Disease Foundation submitted a letter of support for the National Health Council's comments to the FDA's Request for Information regarding Standard Core Clinical Outcome Assessment and Endpoint Sets...As a member of the National Health Council, and an advocate for patient-centered outcomes research, the Celiac Disease Foundation was pleased to collaborate on the NHC's comments and to submit a letter in support of this worthy effort.” Full
Brainstorm Health: J&J CEO on Drug Prices, Polio-Like Condition, Pfizer Breast Cancer Drug (10/16, Sy Mukherjee, Fortune) reports “...According to [Johnson & Johnson CEO Alex Gorsky], all of the relevant players, including drug makers, have a part to play in fixing the mess by shifting the way we pay for drugs—for example, by paying for the health value treatments provide rather than how often they're used. ‘We absolutely do need to take the lead,’ said Gorsky. ‘We're going to have to work together in different ways. We need all components of the system working together… How can we get better transparency, and more outcomes-based mechanisms in place?’
Proposed Rule on Prescription Drugs Offers the Wrong Approach to Seniors with Complex Medical Conditions (10/17, Rep. Erik Paulsen, The Hill) comments “...One doctor discussed having to hire an additional nurse at each of his clinics, just to manage the additional paperwork associated with step therapy care. A urologist at North Memorial Health warned of cases where patients are eventually escalated to a different drug, but end up switching insurance plans and having to start the entire step therapy cycle over again...This doesn't just increase costs – it puts patients at risk as they are left waiting for the drugs and treatments they need. And it takes decisions about patients' care out of the hands of doctors and puts it into the hands of insurance companies.” Full
American Patients Face Too Many Hurdles in Regard to Health-Care Access (10/16, Former Sen. Tom Coburn, The Hill) comments “...A recent proposal to allow for Medicare Advantage plans to increase the use of step therapy needs more scrutiny to ensure the doctor-patient relationship is upheld so patients can access the critical medicines they need. Ensuring patients can get the right medicine at the right time is instrumental in containing long-term healthcare costs and policies such as step therapy reform are constructive and commonsense ways to accomplish those two important goals.” Full
Direct-Contracting Won't Hurt Advantage Business, CMMI Director Says (10/16, Susannah Luthi, Modern Healthcare) reports “...[CMMI director Adam Boehler] also said he wants to see more plans use value-based insurance design, or V-BID, which uses low co-pays to nudge patients toward clinical care that's considered better value. He said he would consider promoting the model for prescription drug plans and praised the administration's Medicare Part B step therapy negotiation.” Paid Subscription Required
Novartis Tasks Foundation with Companion Diagnostic Development for Cancer Treatments (10/17, Conor Hale, Fierce Biotech) reports “Novartis has tapped Foundation Medicine to develop companion diagnostic tests for its cancer treatment portfolio, including multiple precision drugs and immunotherapies...‘It is imperative that we collaborate with all of the key stakeholders in oncology to accelerate patient access to personalized medicine,’ said Melanie Nallicheri, Foundation's chief business officer and head of biopharma.” Full
Hybrid Study Designs and the ‘Inextricable Link' Between RWE, Epidemiology (10/17, Melissa Fassbender, Outsourcing Pharma) reports “Hybrid study designs can be advantageous for the generation of real world evidence, providing a potential solution to the challenges presented by observational studies. ‘Real world data and real world evidence generation are inextricably linked to epidemiology,’ said Kerina Bonar, epidemiologist, real world evidence, strategy and analytics, Icon Commercialisation and Outcomes. To further discuss this link, we caught up with Bonar to examine the benefits and challenges of hybrid study designs.” Full
Health Care Chutzpah Vs. Health Plan Literacy (10/17, Peter Pitts, Morning Consult) comments “...Per a study conducted by ConnectiveRx of 503 patients being treated for serious chronic conditions that frequently require specialty medicines, 75 percent of patients who used a copay coupon at least once in the last 12 months said copay accumulator programs would make it harder for them to adhere to their medicines...These patients pay 10 times more out-of-pocket than healthy patients and are forced to try cheaper or more rebate-rich drugs before getting medicines that work best. Faced with higher out-of-pocket costs and barriers to access, people are more likely to stop their treatment, getting sicker and more expensive to treat.” Full
Press Releases
Express Scripts Offers New Value-Based Program for Migraine (10/17, Express Scripts Press Release) “...Express Scripts today announced its new SafeGuardRx SM Migraine Care ValueSM program will begin April 1, 2019. This industry-first program combines a comprehensive clinical care program to help patients achieve better outcomes and experience fewer migraines, while helping plan sponsors more affordably cover new, preventive therapy for their members.” Full
Anthem, HealthCore and Novo Nordisk Launch Large, Real-World Pragmatic Study to Understand the Effectiveness of a New Type 2 Diabetes Drug (10/17, Healthcore Inc. Press Release) “...The randomized Ozempic pragmatic trial, ‘Long-term comparative effectiveness of once weekly SEmaglutide versus standard of care in a real world adult US population with type 2 diabetes - a randomized PRAgmatic clinical trial,’ or SEPRA, will compare the long-term effectiveness of Ozempic to potentially 40 other medications among 2,250 adult consumers with Type 2 diabetes. The trial aims to learn whether Ozempic leads to better outcomes or impacts the need for health services compared to other FDA-approved drugs to treat diabetes.” Full
D2 Consulting Publishes White Paper about Successful Value Based Contracting Strategies (10/17, D2 Consulting Press Release) “...The VBC blueprint offers strategies and guidance for successful negotiations that respond to changes resulting from regulatory and market pressures to reduce cost without compromising safety and quality. D2 is a leading life sciences consulting firm offering both strategic and tactical commercialization services to pharmaceutical, biotechnology and medical device manufacturers.” Full
Cota Healthcare Announces Publication of Two Studies Supporting Need for Real-World Evidence Solutions that Deliver Breakthrough Clinical Oncology Insights (10/16, Cota Healthcare Press Release) “...‘When you think about complex life and death decision-making, we wouldn't expect an airline pilot to fly without navigational tools – but we have expected oncologists to make treatment decisions without a clear view of the real evidence. Treatment protocols have their place – and now we can actually show outcomes and cost among patients with the same disease factors and treatment attributes so that oncologists can be more precise with treatment plans and reduce variation in care, and cost,’ [Andrew Pecora, M.D.] added.” Full
A Research Study to Make Psoriasis Treatments More Patient-Centered (10/16, National Psoriasis Foundation Press Release) “In 2017, the Patient-Centered Outcomes Research Institute awarded an $8.6 million contract to Joel Gelfand, M.D., for a clinical trial called LITE, conducted in partnership with NPF. LITE's purpose is to study the effectiveness and safety of 12 weeks of home-based versus office-based phototherapy for the treatment of psoriasis...The project started this year and is scheduled to end in 2023. LITE could someday provide the data necessary to better inform treatment decisions for hundreds of thousands of phototherapy candidates.” Full
Journals
Addressing High Drug Costs: Integrating Value Assessment Principles Into Clinical Pathways Richard G Stefanacci, DO, MGH, MBA, AGSF, CMD; Matthew Pakizegee, PharmD, MS October 15, 2018, Journal of Clinical Pathways Journal of Clinical Pathways
Sharing and Collecting Hospital-Based Health Technology Assessment Reports Internationally: Is an Extensive Participation of Stakeholders Realistic? Verbeek J, et al. October 16, 2018, International Journal of Technology Assessment in Health Care PubMed
Reports
White Paper: The Need for Value Based Contracting for Pharmaceutical Companies and Challenges in the Current Environment October 17, 2018 D2 Consulting
Federal Opportunities
FDA-Catalyst MyStudies App Alignment with Pragmatic Trials and/or Registries October 16, 2018 Sentinel
Articles
Extending the User Fee Approach to Pharmaceuticals (10/16, David M. Cutler, PhD, JAMA) comments “...To its credit, the pharmaceutical industry supports comparative effectiveness research and even funds some research. However, it is not ideal for an industry to fund research on its own effectiveness. The choice of projects may reflect those areas in which research is likely to benefit the industry, not necessarily areas of most benefit to the public. In addition, researchers who want future funding may tilt their findings to favor the funders. The natural solution is for pharmaceutical firms to contribute to a general fund for comparative effectiveness research. To be fair to pharma, the research could mostly be directed at clinical settings where pharmaceuticals are one potential treatment option." Full
ICER Plots Early Scientific Advice Program for Biopharma (10/15, Zachary Brennan, Regulatory Focus) reports “...‘For some time, ICER has been receiving requests from life sciences companies to help them rethink clinical trial design, so that the trials more adequately measure the types of outcomes that matter most to patients and their families,’ David Whitrap, ICER VP of Communications and Outreach told Focus...‘Some international health technology organizations, such as NICE and CADTH, have offered this ‘early scientific advice' to industry for many years with general success. We are therefore evaluating the options to provide a similar service but have not made any definitive plans,’ Whitrap said.” Full
ICER Plots Early Scientific Advice Program for Biopharma (10/15, Zachary Brennan, Endpoints News) reports “...Whitrap added: ‘We're still evaluating all of our options at this point regarding early scientific advice, including how to support the staffing requirements it would entail … any potential offering in this area would be structured in a way that would safeguard the independence of the drug assessments we conduct at or around FDA approval.’” Full
Michigan Second State To Pursue Outcomes-Based Contracts For Medicaid Rx Pricing (10/15, InsideHealthPolicy.com) reports “...Michigan has asked CMS for permission to enter into outcomes-based contracts with drug manufacturers under its Medicaid program, Michigan Department of Health and Human Services spokesman Bob Wheaton confirmed Monday. If the request is approved, Michigan would be the second state allowed to enter into outcomes-based contracts, behind Oklahoma.” Paid Subscription Required
Parexel Debuts New Patient-Centric Solutions for Drug Development (10/16, Joseph Keenan, Fierce Biotech) reports “...Parexel has been busy recently beefing up its offerings and partnerships. In late September, it announced a partnership with healthcare tech company Datavant to improve the design and operations of clinical studies and generate more real-world evidence. That news came close on the heels of the company opening its Patient Innovation Center focused on boosting efficiency and success in biopharma development areas including clinical trial design, recruitment, execution and market access.” Full
Step Therapy Policy Alarms Cancer Leaders (10/16, Andrew D. Smith, OncLive) reports “...As it stands now, oncology experts do not expect the Medicare Advantage step therapy provision to have a large impact on treatment protocols because anticancer agents can rarely be substituted for one another. However, that's not the case with supportive care drugs, said Stephen S. Grubbs, MD, vice president of clinical affairs for the American Society of Clinical Oncology.” Full
Press Releases
$3.7 Million Grant Aims to Accelerate Move to Patient-Centered Care in Health Systems (10/15, UCLA Press Release) “...A $3.7 million grant to UCLA and three other Southern California health systems aims to help change that. The five-year award from the Agency for Healthcare Research and Quality and the Patient-Centered Outcomes Research Institute funds a Center of Excellence in Learning Health Systems. To be called the SPIRIT K12 program, the efforts will support young scientists who will study how to rapidly evaluate and implement these changes to benefit patients.” Full
Medisafe Launches Outcomes-Based Partnership Model to Pharma (10/16, Medisafe Press Release) “...Medisafe's value-based offering stems from a comprehensive research study the company conducted together with IQVIA, a leading global provider of advanced analytics, technology solutions and clinical development services to the life sciences industry. The study, covering multiple disease states and drug classes, shows Medisafe has a significant positive effect on the frequency of prescription refills and length of time patients remain on therapy.” Full
Journals
Evidence-Based Medicine: Key Definitions and Concepts C. Carrie Liu, Jennifer Shin October 16, 2018, Evidence-Based Management of Head and Neck Vascular Anomalies Springer
Events
2018 PCORI Annual Meeting Breakout Session: A Congressional Perspective on Patient-Centered Outcomes Research November 1, 2018 10:15 - 11:45AM EDT PCORI
Articles
A Nuanced Approach Is Needed to Determine Value of Drugs (10/13, Kara Rosania, Managed Health Care Connect) reports “Recently, CVS Health announced that it will use the value-based drug pricing system of the Institute for Clinical and Economic Review to exclude some high-cost drugs from plans. In a blog post for Health Affairs, Robert W DuBois, MD, PhD, chief science officer of National Pharmaceutical Council, expressed concerns that the approach could restrict patient access to certain medications. We spoke with Dr DuBois about the use of value frameworks to inform formulary decisions and what factors should go into determinations of cost-effectiveness.” Full
Video: Research on Voluntary Patient-Centered Prescription Opioid Tapering (10/15, PCORI) “This PCORI-funded principal investigator's study focuses on patients who wish to reduce their use of prescription opioids. The study aims to help find the best way for patients with chronic pain to self-manage their pain while gently reducing their use of prescription opioids.” View Video
Video: Payer Discussion on Cardiovascular Outcomes Associated with Second-Line Treatments for Type 2 Diabetes Webinar Recording -- October 8, 2018 (10/12, PCORI) “A conversation about cardiovascular outcomes associated with type 2 diabetes. Participant input helped inform on PCORI's efforts to develop materials around a draft observational study protocol.” View Video
On Drug Prices, Pharmacy Benefit Managers Are Not The Problem: A Response To Michael Carrier (10/12, Matthew Eyles, Health Affairs Blog) comments “...[D]rug prices continue to rise because competition is limited, and there often are no alternatives to the life-saving medications patients need. A recent investigation by the Associated Press found that in the first seven months of 2018, there were 96 drug price hikes for every drug price cut. Families find themselves having to choose between putting food on their tables and getting their medications.” Full
Seizing the Opportunity to Create Change in the Rheumatology Field (10/15, Pete Salzmann, M.D., LillyPad) comments “...It is critical for patients to have access to innovative treatments that could help make a difference in their lives. However, it's a complex issue. Take step therapy, for example, which is a practice seen more and more in our health system as financial pressures mount across the board. Despite what the doctor prescribes, when a step therapy program is in place, patients are required to start with a specified medicine which is ‘on formulary.’ Patients must then wait until the formulary product fails before they can begin a non-formulary product, even if there's an indication that it would benefit them more from the start.” Full
Press Releases
National Academy of Medicine Elects 85 New Members (10/15, The National Academies of Sciences, Engineering and Medicine Press Release) “...‘This distinguished and diverse class of new members is a truly remarkable set of scholars and leaders whose impressive work has advanced science, improved health, and made the world a better place for everyone,’ said National Academy of Medicine President Victor J. Dzau. ‘Their expertise in science, medicine, health, and policy in the U.S. and around the globe will help our organization address today's most pressing health challenges and inform the future of health and health care. It is my privilege to welcome these esteemed individuals to the National Academy of Medicine.’” Full
Northwestern University Awarded $3.5 Million to Train the Next Generation of Learning Health System Researchers (10/12, Association of Schools & Programs of Public Health Press Release) “[AHRQ’ and [PCORI] announced $40 million in awards over five years to 11 institutions to support the training of researchers to conduct patient-centered outcomes research within learning health systems (LHS). Northwestern University was among the recipients, with its project entitled, ‘A Chicago Center of Excellence in Learning Health Systems Research Training.’” Full
Journals
Researchers, Patients, and Stakeholders Evaluating Comparative-Effectiveness Research: A Mixed-Methods Study of the PCORI Reviewer Experience Forsythe LP, et al. October 2018, Value in Health PubMed
Unique Review Criteria and Patient and Stakeholder Reviewers: Analysis of PCORI's Approach to Research Funding Forsythe LP, et al. October 2018, Value in Health PubMed
Comparing Use of Health Technology Assessment in Pharmaceutical Policy among Earlier and More Recent Adopters in the European Union Beletsi A, et al. October 10, 2018, Value in Health Regional Issues PubMed
Articles
A Policymaker's Perspective On Comparative Effectiveness Research: History And Prospects (10/11, Tom Allen, Health Affairs Blog) comments “...There is a path to saving PCORI and promoting other aspects of evidence-based medicine, but advocates must play the long game. Policy entrepreneurs—elected, administrative, and expert—will be indispensable to success. Developing good information, proposing potentially effective policy, and building broad coalitions will all continue to be essential and can be productive when the political climate for sensible health policy is more favorable.” Full
Tisagenlecleucel's High Price Aligns With Its Benefit in Pediatric B-ALL, Study Finds (10/11, Laura Joszt, The American Journal of Managed Care) reports “...‘Financing cures in the United States is challenging owing to the high up-front price, rapid uptake, and uncertainty in long-term outcomes; however, innovative payment models are an opportunity to address some of these challenges and to promote patient access to novel and promising therapies,’ the authors wrote.” Full
A New Era for Drug Pricing: The ‘Accountable Choice' (10/11, James C. Robinson, STAT News) comments “...The principles of reference pricing can and, in my opinion, should be applied to price negotiations over specialty drugs, building on methodologically sound comparative analyses...As an alternative, comparative effectiveness and cost analyses could be used to set an upper limit on how much the insurer will pay, rather than to set the drug's price. That gets around the fact that the manufacturer must agree on the price, while the payer has unilateral control over its payment limit.” Full
New Value Assessment Framework Needed for Biosimilars, White Paper Argues (10/11, Kelly Davio, The Center for Biosimilars) reports “...[T]he white paper, authored by Xcenda for the Biosimilars Forum, argues that no current value assessment framework is fully suited to assessing the value of biosimilar medicines; several of the above frameworks focus on oncology drugs alone, ICER's focus on quality-adjusted life years has been questioned for its appropriateness, and each framework is oriented to the perspective of a particular stakeholder.” Full
AcademyHealth and HIMSS Join Forces to Leverage Health and Health Care Data to Support Changes in Policy and Practice (10/11, Lisa Simpson, MB, BCh, M.P.H., FAAP, AcademyHealth Blog) comments “...Through our combined voices, AcademyHealth and HIMSS will work together to expand the programming, audience and reach of the Health Datapalooza, as well as year-round programming that reaches key stakeholders in the health data ecosystem, including policymakers in Washington. We see the important opportunities that emerging technologies and information systems have to broaden our research capabilities, accelerate evidence-development, and bring improved outcomes and value to patient-centered healthcare.” Full
Nordic Procurement Deal Signals New Weapons In Fight For Lower Prices (10/11, Francesca Bruce, Pink Sheet) reports “...A new joint procurement agreement between Norway and Denmark is a signal to drug companies that they will have to increasingly negotiate with more than one country in a new age of collaboration, says Amgros, the Danish medicines procurement agency...While Norway uses a cost per quality adjusted life year approach, Denmark does not use QALYs in its assessments and instead categorizes a product's added therapeutic value. It instead looks more closely at a product's added therapeutic value.” Paid Subscription Required
Press Releases
Biogen Advances Research to Improve Outcomes for Patients With Multiple Sclerosis (10/11, Biogen Press Release) “...Biogen recognizes the importance of real-world evidence to help guide decisions in clinical practice and optimize patient care. The company continues to evaluate its leading MS therapies, TECFIDERA and TYSABRI, to better understand the benefits of using these treatments, including when initiated early within the disease and treatment course.” Full
Institute for Clinical and Economic Review to Publish Upcoming Assessments on Treatments for Secondary Progressive Multiple Sclerosis, Treatment-Resistant Depression (10/10, ICER Press Release) “...The SPMS review is expected to assess the clinical effectiveness and value of siponimod (Novartis), which is currently undergoing FDA review with an approval decision expected in March 2019. The treatment-resistant depression review is expected to assess the clinical effectiveness and value of esketamine (Janssen), which is currently undergoing FDA review with an approval decision expected in May 2019.” Full
PhRMA Foundation and Personalized Medicine Coalition Announce 2018 Value Assessment Challenge Award Recipients (10/10, PhRMA Foundation Press Release) “...A total of $85,000 in funding was provided for the researchers, who answered the question: What are potentially transformative strategies and methods to define and measure value at all levels of decision making that are aligned with personalized/precision medicine?” Full
Pertuzumab in Early BC: No Added Benefit Proven Despite Exemplary Surrogate Validation (10/11, IQWiG Press Release) “...For patients under 65 years of age, the combination therapy of pertuzumab, trastuzumab, a taxane, and, if applicable, an anthracycline is not better than the appropriate comparator therapy. There is even a hint of lesser benefit for patients aged 65 or older. A notable positive aspect was the methodologically solid validation of the surrogate outcome ‘disease-free survival’ and the long follow-up observation period for symptoms and health-related quality of life.” Full
Journals
Engaging Patients in Health Care Epidemiology Research: A Case Example Nicole Brys, MPH, et al. October 10, 2018, American Journal of Infection Control American Journal of Infection Control
Real-World Efficacy: Intravenous Palonosetron Three-Drug Regimen for Chemotherapy-Induced Nausea and Vomiting with Highly Emetogenic Chemotherapy Lee S Schwartzberg, et al. October 11, 2018, Journal of Comparative Effectiveness Research Journal of Comparative Effectiveness Research
Reports
White Paper: Quantifying the Value of Biosimilars October 2018 Biosimilars Forum
Events
Advisory Panel on Patient Engagement Fall 2018 Meeting October 30, 2018 9:30 - 11:30AM EDT PCORI