Rewardable pharmaceutical innovation

A Review of Current Approaches to Defining and Valuing Innovation in Health Technology Assessment

Key messages, 

Although, formerly, denoting a new drug as innovative was based on the drug having received patent protection or being a new molecular entity, this criterion is no longer sufficient from the perspective of many stakeholders. Rather, it became widely accepted that the central criterion to identify a drug as truly innovative should be the drug’s benefit or usefulness. With usefulness being a relative quality though, innovation is often defined by the particular view of what is deemed important or valuable. 

On the one hand, there is the notion that the value of innovation derives exclusively from the effect that the innovative drug has on the therapeutic benefit. What matters most for patients is whether a drug is the best choice to achieve treatment goals. It is this basic principle that guides most of the work on how to define rewardable innovation in the context of drug value assessments. The main argument is that, assuming a limited budget for healthcare expenditures, valuing anything beyond this therapeutic benefit in pricing and reimbursement decisions would reduce population health by displacing more cost-effective therapies. Furthermore, as a consequence, pharmaceutical companies might be incentivized to increasingly invest in R&D of slightly modified but basically similar products as opposed to drugs with a potentially larger positive therapeutic impact on patients.

On the other hand, it is argued that there might also be other, less obvious benefits from innovation in a wider sense. First, there may be benefits related to health or well-being that are not captured in measures of health outcomes typically used in clinical trials. A novel drug with less side effects or one that allows a more convenient treatment, for example, oral versus intravenous administration, may have the same effect on the therapeutic outcome but still improve the subjective well-being of the patients.

 

Tuscany, man made landscape

Health expenditure before COVID

 Spending on health in Europe: entering a new era

This report analyses health spending in 53 countries in the WHO European Region from 2000 to 2018 (the latest year for which internationally comparable data are available). It reviews key patterns and trends in health spending over time and across countries

 

Pharmaceutical utilization research

Methods to analyse medicine utilization and expenditure to support pharmaceutical policy implementation 

Reproductive genomics paradigm

The End of Genetics. Designing Humanity's DNA

Although human genetics has already advanced far enough to provide parents with information on which they may choose to act, key gaps in our knowledge make it very difficult to anticipate the consequences of the actions likely to become possible. These gaps mean that any reproductive engineering that is performed beyond the most straightforward elimination of strongly acting disease-causing mutations will be performed without a complete understanding of the likely consequences of those changes. This is a prospect that I find deeply troubling, and this book above all represents my best effort to empower non-specialists to develop their own opinions about this most central question for the future of humanity.

In response to this deep uncertainty, I have developed a thought experiment in reproductive genomics to help illustrate the kind of genome engineering that could be entertained in the not too distant future. Throughout the book I will refer back to this thought experiment to help make clear that we will have the technological ability of making some kinds of adjustments to the genomes of children, without having a matching ability to accurately predict the consequences of those adjustments. You will be in a position to understand this thought experiment more fully later in the book, but as a motivation in the reading that follows, consider the following possibility.

A very controversial book, glups!

This is the outline, 

Introduction

Chapter 1. The Future of Reproduction

Chapter 2. Learning to Read the Human Genome

Chapter 3. The Nature of Human Genetic Variation

Chapter 4. DNA and Human Disease

Chapter 5. Writing the Genomes of Our Children

The financial crash of 2008 never really ended

 The Lords of Easy Money. How the Federal Reserve Broke the American Economy

Ten years on, the gap between the rich and poor has grown dramatically, stock prices are trading far above what’s justified by actual corporate profits, corporate debt in America is at an all-time high, and this debt is being traded by big banks on Wall Street, leaving them vulnerable—just as they were during the mortgage boom. Middle-class wages have barely budged in a decade, and consumers are buried under credit card debt, car loan debt, and student debt.

Finding network effects desperately

 The Cold Start Problem. How to start and scale network effects.

Precision medicine (2)

Discovering Precision Health: Predict, Prevent, and Cure to Advance Health and Well-Being

Introduction The Power of Precision Health 1

Chapter 1 The State of U.S. Health and Health Care Delivery 15

Chapter 2 There’s More to “Health” Than Health Care 33

Chapter 3 The Innovation and Disruption Powering Progress in Health 43

Chapter 4 Fundamental, Discovery‐Focused Research: The Foundation of Biomedical Breakthroughs 111

Chapter 5 Peering into the Future: Leveraging The Powers of Prediction to Help Prevent Illness 147

Chapter 6 Prevention as a Pathway to Health and Wellness 177

Chapter 7 Curing Disease with More Precise Medical Therapies 207

Conclusion Achieving Precision Health: The Opportunities—and Challenges—Ahead 237

Paying for healthcare

Better Ways to Pay for Health Care

Public health budget evolution

 Last September I posted the data on per capita health expenditure. In 2020 we spent 1786€ per capita. The total budget for 2021 was 13.162m€, 1700€ per capita. Now the budget for 2022 says that we are going to spend 1.446€. This is an absolute nonsense. The budget approval has no relationship with former incurred expenditures...

Somebody should explain it clearly to the population and take measures. Forget this figure, it has no relationship with reality.

Bundled payments update (2)

 Year 1 of the Bundled Payments for Care Improvement–Advanced Model

A NEJM article shows a negligible effect of bundled payments. Unfortunately, I haven't seen any comment about the flaws in the design. A design mistake for not taking into account a holistic view.

If you reduce 78$ out of 27,315 $ per episode, this is an absolute FAILURE! (it is not a small reduction!!!)

However, the conclusion is:

In this study, we found that the BPCI-A program was associated with small reductions in Medicare payments among participating hospitals. Longer-term evaluation is needed to determine the full effect of the program.

Jean Pierre Capron

 

 

Integrated care and population health (2)

 Handbook Integrated Care

Opioid epidemic as a crime (3)

 Unsettled. How the Purdue Pharma Bankruptcy Failed the Victims of the American Overdose Crisis

Medtech regulatory affairs

 Medical Regulatory Affairs. An International Handbook for Medical Devices and Healthcare Products

Platforms, a business model (7)

 Platform Business Models. Frameworks, Concepts and Design