For many, the field of health policy can seem esoteric or abstract. Yet, health policy decisions – made by regulators, federal and state legislators, health plan decision-makers, benefit managers and many other “gatekeepers” – profoundly impact the lives of patients and their access to innovative medicines that can improve their health.
That makes it essential for these decisions to be well-informed. NPC’s work is guided by the understanding that health policy decisions can have a positive impact on patients. Conversely, health policies based on flawed evidence or assumptions can be fraught with unintended consequences – for patients and for the health care system as a whole. It is that understanding that powers NPC’s drive to conduct incisive research that examines our biggest health care problems, challenges assumptions, and digs deep into the data to extract practical insights and durable solutions.
Read NPC's 2020 Annual Report below to learn about our efforts to foster evidence-based health policy conversations and decision-making through high-quality research and analysis on issues related to real-world evidence, value assessment, access to care, innovation and health spending.
Table of Contents
2020 brought challenges and changes for everyone — NPC as well as the rest of the world — and I was honored to lead NPC through this time. I’m proud to be part of an industry that drives innovation and is making a difference in extending and improving the quality of millions of people’s lives.
In 2020, NPC was an influential voice in conversations about access and innovation:
- We championed the importance of appropriately valuing COVID-19 treatments in a widely read Health Affairs article and well-attended webinar.
- With the American Journal of Managed Care, we hosted a webinar that explored how health care innovation affects life expectancy and patient well-being. The webinar featured discussion on independent studies that concluded medicines, along with public health initiatives, are the prime reasons that patient outcomes have improved.
- We addressed access barriers created by benefit design and other challenges faced by patients.
As health care policy evolves under the current Administration, NPC will continue to be a research leader in areas such as:
- Understanding appropriate methods of real-world evidence
- Value assessment and inclusion of the patient perspective
- The impact of benefit design on patient access
- Innovation and health care spending, including identifying and eliminating low-value care
It’s especially gratifying to see a growing roster of member companies support and engage with NPC. I also was proud to lead the search committee that brought new President and CEO John M. O’Brien to the group, and I look forward to helping Mike Ryan of Bristol Myers Squibb onboard as the new chair as he takes the gavel.
I will continue my involvement with NPC, especially on the Board. It’s really important that we support a better health system and a better future for all patients through our work here at NPC.
Blasine Penkowski, MBA
Chair, National Pharmaceutical Council Board of Directors
Chief Strategic Customer Officer, Johnson & Johnson Health Care Systems Inc.
Message From the Interim President and CEO
NPC in 2020: Health Policy Research in the Time of COVID-19 and Beyond
The pandemic that shaped the course of 2020 will leave an indelible mark on each of us. We faced the biggest public health emergency of our time as the novel coronavirus spread rapidly across the globe. It was a year of loss as family members, friends and colleagues were taken from us by COVID-19. It was a year of hardship and endurance in life, in work, and in everyday moments that we used to take for granted — a trip to the store, a meeting with family and friends, a cup of coffee at the local café, a routine visit to the doctor.
Yet, the adversity we faced brought out the best from the biopharmaceutical field. We began the year with few treatment options and no vaccine. The pandemic’s disproportionate toll on underserved communities laid bare the enduring barriers to equity in health care. But innovation in our sector meant that we closed out the year with new treatments for COVID-19, multiple vaccines ready for emergency use authorization, and a deep recognition of the sacrifices and commitment made by the health care workers, researchers and public health officials who worked to create a path back to “normal life.”
At the National Pharmaceutical Council (NPC), the challenges and successes we saw during the pandemic served to reinforce the value of our mission to explore, demonstrate and communicate the role and value of innovative biopharmaceuticals to achieve better patient health. It’s never been clearer that NPC’s member companies create a world where people can thrive. When we fuel biopharmaceutical innovation, while also ensuring patient access to needed therapies, the world is better.
It is with this perspective that the NPC team navigated a particularly challenging year. Like countless other organizations, we closed our offices in mid-March and transitioned to virtual work life. Our team also saw many changes as longtime NPC President and CEO Dan Leonard departed in September and Chief Science Officer and Executive Vice President Robert Dubois stepped in as acting president and CEO. Despite the unusual year, the team has been exceptionally productive. This report highlights our most meaningful accomplishments in 2020 and demonstrates the important role that NPC plays in fostering and shaping the national health care policy dialogue.
NPC’s health care policy research and educational activities remain focused on four strategic areas: real-world evidence, value of medicines, access to treatments, and biopharmaceutical innovation and spending. Each of these areas intersect with the toughest issues facing our health care system and require relevant, credible, and timely research to inform smart, sustainable health policy solutions.
Evidence: Advancing the Use of the High-Quality, Real-World Evidence to Inform Decision-making
Randomized controlled trials are a respected standard for research, but they cannot address all the questions that stakeholders may have about the full impact of a treatment on patients in everyday life. As such, researchers are increasingly examining data from patients’ routine interactions with the health care system, including electronic health records and claims records, to better understand which treatments work best for which patients. Because real-world evidence (RWE) can play an important role in informing health care regulatory, coverage and reimbursement decisions, RWE must be generated using high-quality data and in a transparent way.
NPC’s body of work on high-quality evidence standards, including the GRACE principles and checklist for evaluating the quality and usefulness of observational research studies, is cited and used by key stakeholders, and our leadership in this area has resulted in collaborations on important RWE efforts.
A position paper by the RWE Transparency Initiative describes a plan for improving the transparency of the research process and making registration of real-world evidence study methods easier and more routine.
As a partner on the RWE Transparency Initiative, led by the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), NPC collaborated on a paper describing the need for greater transparency in research and outlining a plan for making registration of RWE study methods easier and more routine. The paper highlights how the lack of transparency in the study methods can make it difficult for decision-makers to assess the quality and credibility of RWE studies. Study registration — particularly for RWE studies evaluating medical treatment effectiveness and safety using existing or secondary data — has been proposed to improve research transparency and build trust in the study findings.
Research & Publications
- Evolution of the AMCP Format for Formulary Submission, Journal of Managed Care & Specialty Pharmacy, June 2020
- Improving Transparency to Build Trust in Real-World Secondary Data Studies for Hypothesis Testing—Why, What, and How Recommendations and a Roadmap from the Real-World Evidence Transparency Initiative, Value in Health, September 2020
Value: Ensuring the Value of Medicines Are Recognized as an Integral Component of Care
As health care stakeholders continue to debate how to measure the value of treatments and interventions, NPC is a leading voice in the discussion. Value assessment can ultimately impact patient access to treatment if health insurers, employers, and other policymakers use these assessments in making coverage decisions. NPC’s research in 2020 took a closer look at how various stakeholders assess the value of medicines — examining the inputs, methods and standards used — to better understand their approaches.
Our efforts provide perspective on the shortcomings of current assessment approaches, the factors that need to be considered for a holistic understanding of value, and the potential pitfalls that could come with relying on any single “formula” to assess value. Our research, analysis and public comment on value assessment — including weighing in on the work by the Institute for Clinical and Economic Review (ICER) — are conducted through the lens of NPC’s Guiding Practices for Patient-Centered Value Assessment.
NPC’s input and recommendations on ICER’s framework has yielded changes to its methodologies. In 2020, those changes included:
- Extending the pilot program for model sharing to all reviews
- Adding a section on controversies and uncertainties to reviews
- Formalizing a process to examine new evidence post-review
- Extending the timeline for class reviews
- Promoting the societal perspective to a co-base case when the impact is substantial
In 2020, NPC produced several research studies that shed light on the challenges with value assessment methodologies and explored alternative ways to assess value by focusing on what matters to patients. One study identified good practices for integrating the patient voice into value assessment through multi-criteria decision analysis (MCDA). Another study assessed patient preferences on breast cancer treatment outcomes to test assumptions commonly used in oncology value frameworks. In recognition of the important context of the pandemic, NPC published an article on the Health Affairs blog about appropriately valuing COVID-19 treatments, highlighting the need to strike a balance between providing widespread access without hindering investment in future innovation.
Research & Publications
- Barriers and Solutions to the Inclusion of Broader Benefits in Biopharmaceutical Value Assessments, Journal of Occupational and Environmental Medicine, February 2020
- Are Value-Based Arrangements the Answer We’ve Been Waiting for?, Value in Health, February 28, 2020
- Improving Patient-Reported Measures in Oncology: A Payer Call to Action, Journal of Managed Care & Specialty Pharmacy, October 2020
- Getting Value Right: The Case for Indirect Benefits, Health Affairs, March 19, 2020
- Underestimating the Value of an Intervention – The Case for Including Productivity in Value Assessments and Formulary Design, Journal of Managed Care & Specialty Pharmacy, May 2020
- It's Time to Talk About COVID-19 Prices, Health Affairs, May 20, 2020
- Do Patient Preferences Align With Value Frameworks? A Discrete-Choice Experiment of Patients With Breast Cancer, MDM Policy and Practice, June 15, 2020
- Multi-Criteria Decision Analysis: Can It Help Make Value Assessment More Patient Centered?, NPC and National Health Council white paper, June 15, 2020
- Patient-Centered Outcomes Research Institute (PCORI): Proposed Principles for the Consideration of the Full Range of Outcomes Data, November 13, 2020
Access: Supporting Meaningful Patient Access to Appropriate Medications
What stands between innovative therapies and the patients whose health could be improved by them? In many cases, health insurance policies intended to promote cost savings serve as barriers to access, unintentionally worsening patient outcomes and increasing overall health system costs via lost productivity, additional doctor visits, preventable hospitalizations and nursing home admissions, and other costs.
In a commentary published in BenefitsPro, NPC Vice President for Health Services Research Kimberly Westrich discussed the need to address the harms of inequitable benefit design and the resulting racial and socioeconomic disparities.
NPC research has long explored the challenges that patients face in accessing care — including high-deductible health plans, restrictive drug formularies and complex coverage approval processes like step therapy. In 2020, NPC launched eight research projects to more closely examine patient financial burdens, copay accumulators and step therapy. Among those projects is an NPC/Pharmacy Quality Alliance (PQA) partnership to fund two research grants to study the real-world challenges that patients face in accessing medications.
NPC continues to make resources available to employers to help foster patient-centered benefit design that promotes value, patient health and successful cost-mitigation approaches.Get the Employer Resource Guide.
The National Alliance of Healthcare Purchaser Coalitions promoted NPC's Employer Resource Guide on its website and in its annual Employers Roundtable on Drug Management Report. In addition, ICER cited NPC resources in its white paper “Cornerstones of ‘Fair’ Drug Coverage: Appropriate Cost-Sharing and Utilization Management Policies for Pharmaceuticals,” and incorporated those concepts into its fair access principles.
Research & Publications
- Racial and Socioeconomic Disparities: Ignoring Inequitable Benefit Design Is Not an Option, BenefitsPro, December 2020
- Evolution of ACO Readiness to Optimize Medication Use: Are We There Yet?, Journal of Managed Care & Specialty Pharmacy, October 2020
- U.S. Department of Health and Human Services: Patient Protection and Affordable Care Act; Notice of Benefit and Payment Parameters for 2021, March 2, 2020
- Centers for Medicare & Medicaid Services (CMS): Advance Notice of Methodological Changes for Calendar Year 2021 for Medicare Advantage Capitation Rates and Part C and Part D Payment Policies – Part II, March 6, 2020
- CMS: Contract Year 2021 and 2022 Policy and Technical Changes to the Medicare Advantage Program, Medicare Prescription Drug Benefit Program, Medicaid Program, Medicare Cost Plan Program, April 6, 2020
- National Quality Forum: Request for Comments on the Patient-Reported Outcomes Draft Recommendations Report, June 29, 2020
- CMS: Proposed Rule on Medicaid Best Price, Value-Based Purchasing, July 20, 2020
- Utilization Review Accreditation Commission: Pharmacy Benefit Manager Accreditation Standards, August 12, 2020
Innovation and Spending: Fostering Biopharmaceutical Innovation That Improves Patients’ Lives
COVID-19 demonstrated to the entire globe the importance of rapid biopharmaceutical innovation, reframing the ongoing debate around health spending and the impact of proposed “solutions” on the pace of innovation. This urgency for innovation has long been felt by individual patients hoping for better ways to navigate their conditions and achieve better health outcomes. That’s why NPC’s work aims to drive a pragmatic discussion that examines the drivers of health spending and the tradeoffs required to balance spending with fostering a health care ecosystem that nurtures innovation.
NPC spurred meaningful conversations on innovation and health spending through our partnerships with MIT's NEW Drug Development ParadIGmS (NEWDIGS) program, Health Affairs, AcademyHealth, the Healthcare Leadership Council (HLC), and the NPC-led Going Below The Surface (GBTS) Forum, a group of experts representing payers, provider groups and patient advocacy organizations.
The NPC/Anthem-sponsored Health Affairs series, Considering Health Spending, generated considerable dialogue with 24 articles and 102 blogs. NPC’s Dr. Dubois continues to serve as a member of the Health Affairs Council on Health Care Spending and Value, which met in 2020 to further the discourse on health spending and develop articles based on their discussions.
As part of the GBTS Forum, the HLC and NPC hosted a virtual town hall meeting for the Austin and Houston, Texas, region to address the essential questions: How much do we want to spend on health care, and how do we want to spend those dollars? The discussion brought to light varying viewpoints but one widely shared perspective prevailed: what’s important is not how much we’re spending, but what benefits we’re getting for the money we’re spending.
In addition, the GBTS Forum developed a roadmap to help navigate the problem of low-value care, which costs the U.S. more than $340 billion each year. The Roadmap for Addressing Low-Value Care is a reference tool to help organizations consider key goals, questions and tactics for prioritizing high-value care, such as life-saving vaccines, and reducing low-value care and wasteful spending, ensuring the health care system is more effective and capable during this unprecedented time.
Research & Publications
- Assessing Consumer and Employer Willingness to Pay for New Medical Technologies, NPC white paper, December 9, 2020
- Roadmap for Addressing Low-Value Care, GBTS Forum, July 2020
NPC Educational and Outreach Activities
Decision-makers need quality research and analyses to inform their work, and NPC’s educational activities are aimed at ensuring stakeholders are well-equipped to navigate complex issues related to evidence, value, access, and innovation and spending.
Our regular columns for Specialty Pharmacy Times, PharmaBoardroom and Chain Drug Review, along with commentaries in Morning Consult and ISPOR's Value & Outcomes Spotlight put timely resources into the hands of stakeholders.
- Value Assessment Frameworks: Is There One “Best” Model?, STAT, January 10, 2020
- Pharmacy Outlook, Chain Drug Review, January 19, 2020
- Evidence: Vital but Seldom Used Consistently by Payers, Specialty Pharmacy Times, March 3, 2020
- Will the pandemic provide the push for positive change?, Chain Drug Review, April 29, 2020
- Accumulating Barriers to Care: Double Jeopardy for U.S. Patients, PharmaBoardroom, July 2, 2020
- Higher Medicaid enrollment can safeguard the vulnerable, Chain Drug Review, July 28, 2020
- Supporting Value-Based Purchasing Through Changes in Medicaid Best Price, PharmaBoardroom, August 9, 2020
- What the Rise of Real-World Evidence Means for the Pharmaceutical Industry: A Closer Look, ISPOR’s Value & Outcomes Spotlight, September/October 2020
- COVID-19 Pandemic Highlights the Need to Maintain Health Care Innovation and Improve Affordability, Morning Consult, December 17, 2020
In 2020, NPC continued its partnership with Duke University's Robert J. Margolis, MD, Center for Health Policy, announcing in July that Salama Freed, PhD, had been named the 2020-22 postdoctoral health policy fellow. The two-year fellowship position, based in Washington, D.C., is designed to bridge a persistent gap between health research and policy analysis. Dr. Freed, the fourth fellow to enter the program since it launched in 2013, is exploring payment reform such as provider rate changes, insurance rate setting and bundled payment initiatives, and how these approaches impact equitable health care access.
Throughout the year, NPC shared research and activities via a broad array of communications channels. We stayed engaged with our followers on social media via Twitter, LinkedIn and YouTube, and we created graphic versions of our research for easy sharing. In addition, NPC continued to offer the CER Daily Newsfeed® — a summary of RWE and health policy research activities and news from around the world — as well as our E.V.I.dently® monthly e-newsletter and engaging content on our blog.
NPC Membership and Board of Directors
NPC is a member-based nonprofit organization supported by leading biopharmaceutical companies committed to facilitating rigorous and timely research that gets to the heart of pressing health policy issues. NPC does not engage in political advocacy. Similar to a think tank, NPC conducts health policy research, which is frequently published in respected peer-reviewed journals, as well as regularly engages in multistakeholder initiatives, conferences and other industry dialogues.
Each member company is represented on NPC’s Board of Directors and helps to shape the research agenda through participation on various Board-level committees, as well as on the Research Work Group and Strategic Communications Work Group.
In addition to our public resources, NPC develops materials exclusively for NPC members, including executive briefs detailing the impact of developments in the health care landscape on the biopharmaceutical industry. We also provide educational resources, practical tools, analytical papers and other information in the members-only section of the NPC website.
2020 BOARD OF DIRECTORS
Blasine Penkowski (Chair)
Chief Strategic Customer Officer
Janssen North America
Michael L. Ryan (Vice Chair)
Head and Senior Vice President, Worldwide Value, Access, Payment & Health Economics and Outcomes Research
Bristol Myers Squibb
Steven J. Romano (Treasurer)
Chief Business Officer
Includes officers listed above and the following members:
Mark J. Nagy (Member at large)
Vice President, Global Patient Outcomes & Real World Evidence
Eli Lilly and Company
Justin McCarthy (Member at large)
Senior Vice President, Patient & Health Impact
OTHER MEMBERS OF THE BOARD
Vice President, U.S. Market Access
Vice President, Global Evidence and Value Development
Senior Vice President, U.S. Commercial Operations
Vice President, Global Health Economics
Vice President, U.S. Value and Access
Senior Vice President, Medical Specialties
Astellas Pharma US, Inc
Executive Director, US Medical Payer Medical
Senior Vice President, US Market Access
Senior Vice President, Head of Market Access
Bayer U.S. LLC
Senior Vice President, Value and Access
Christine G. Marsh
Senior Vice President, Market Access
Boehringer Ingelheim Pharmaceuticals, Inc.
Jan E. Hansen
Vice President, Evidence for Access Medical Unit,
US Medical Affairs
Coy A. Stout, II
Vice President, Market Access & Account Management
Vice President, Corporate Affairs
Vice President, Integrated Account Management
Senior Vice President and Head,
Global Value, Access & Policy
Leigh Anne Leas
Vice President and Head, North America Public Policy
Global Public Affairs
Novartis Services, Inc.
Vice President, Global Head - Global Market Access
Joseph W. La Barge
Chief Business Officer
Spark Therapeutics, Inc.
SVP & Head, U.S. Business Unit Medical Affairs Office
Takeda Pharmaceuticals U.S.A., Inc.
Head, U.S. Corporate Affairs
Vice President, Head of North America Medical Affairs
Tanya Bailey, MS
Membership and Meetings Associate
Michael Ciarametaro, MBA
Vice President, Research
Robert W. Dubois, MD, PhD
Interim President and Chief Executive Officer
Salama Freed, PhD
NPC/Duke-Margolis Center for Health Policy Research
Postdoctoral Health Policy Fellow
Kathryn A. Gleason
Chief Operating Officer and Senior Vice President
Jennifer Graff, PharmD
Vice President, Comparative Effectiveness Research
Amanda Green, MPH
Robert Gutnikoff, MA
Senior Manager, Communications
Andrea Hofelich, MBA
Vice President, Communications
Brian Sils, MPP
Virginia S. Sweeter, CMP
Director of Alliance Development and Education
Kimberly Westrich, MA
Vice President, Health Services Research