Drug pricing overhaul

 Pricing for Medicine Innovation: A Regulatory Approach to Support Drug Development and Patient Access

The message:

We introduce a regulatory approach we term “Pricing for Medicine Innovation” (PMI), which departs dramatically from the market-equilibrium assumptions of conventional (neoclassical) economics. The PMI approach recognizes the centrality of collective investments by government agencies and business firms in the productive capabilities that underpin the drug development process. PMI specifies the conditions under which, at the firm level, drug pricing can support both sustained investment in these capabilities and improved patient access. PMI can advance both of these objectives simultaneously by regulating not just the level of corporate profit but also its allocation to reinvestment in the drug development process. PMI suggests that although price caps are likely to improve drug affordability, there remain two potential issues with this pricing approach. Firstly, in an innovation system where a company’s sales revenue is the source of its finance for further drug development, price caps may deprive a firm of the means to invest in innovation. Secondly, even with adequate profits available for investment in innovation, a firm that is run to maximize shareholder value will tend to use those profits to fund distributions to shareholders rather than for investment in drug innovation. We argue that, if implemented properly, PMI could both improve the affordability of medicines and enhance the innovative performance of pharmaceutical companies.

 David Hockney

Professionalism, current challenges (2)

 Medical Professionalism In An  Organizational Age: Challenges And Opportunities

This article aims to start a dialogue on how these changes may affect the key responsibilities of medical professionalism: putting patient interests first, maintaining and enhancing physicians’ medical  competence, and sustaining trust in the doctor-patient relationship. We identify several potentially effective strategies. They include policies to promote an institutional culture committed to professionalism and to enlarge physicians’ role in institutional leadership. We also address how the principles of professionalism might guide physician compensation formulas, policies governing transparency, and best practices for strengthening the relationships between physicians and newly empowered patients.

 

The long and bumpy road to CRISPR (5)

 CRISPR: Genome Editing and Engineering And Related Issues

Pandemethics (3)

 The Ethics of Pandemics. An Introduction

Table of Contents

1. Introduction: Why Ethics of Pandemics?

2. The General Principle of Pandemic Response

3. Rationing of Scarce Health Care Resources

4. Pandemics in an Unequal World

5. Restricting Freedom

6. Inducing Voluntary Behavioral Change

7. Moral Mathematics under Uncertainty.

The architects of choice

Nudging

How “nudges” by government can empower citizens without manipulating their preferences or exploiting their biases.

We're all familiar with the idea of “nudging”—using behavioral mechanisms to encourage people to make certain choices—popularized by Richard Thaler and Cass Sunstein in their bestselling 2008 book Nudge. This approach, also known as “libertarian paternalism,” goes beyond typical programs that simply provide information and incentives; nudges can range from automatic enrollment in a pension plan to flu-shot scheduling. In Nudging, Riccardo Viale explores the evolution of nudging and proposes new approaches that would empower citizens without manipulating them paternalistically. He shows that we can use the tools of the behavioral sciences without abandoning the principle of conscious decision-making.

A controversial approach. 

Pharma, big pharma (16)

 The Truth About the Drug Companies. HOW THEY DECEIVE US AND WHAT TO DO ABOUT IT

From the former editor-in-chief of the New England Journal of Medicine and now a member of Harvard Medical School’s Department of Global Health and Social Medicine, Marcia Angell

The decline of pharma R&D productivity (2)

Science needs to move beyond luck if it is to design better drugs for the brain

The Economist:

Between 2011 and 2020 the likelihood of a drug in psychiatry being approved by the Food and Drug Administration was 7.3%. In neurology it was 5.9%. (The industry average is 7.9%.) As well as being less likely to succeed in trials (see chart), neurology drugs also take much longer, on average, to develop, further decreasing their appeal

According to the Global Burden of Disease project 12 mental-health disorders affect about 970m people. Their prevalence has increased by 48% since 1990 as the population has grown. With more than one in ten people on the planet affected, it is a global problem, although what data are available suggest it is more marked in Western countries (see map).

Paying for rare diseases drugs

The next generation of rare disease drug policy: ensuring both innovation and affordability

The study finds that while 5% of drugs with an orphan indication cost more than $500,000 per year in US, these drugs make up only 0.08% of all patients treated by drugs with an orphan indication. The majority (52%) of patients who receive treatment an orphan drug get the treatment for <$50,000.

Pharmaceutical contracts and prices

  Introduction to Market Access for Pharmaceuticals

Contents:

Chapter 1: Health as a Good

Chapter 2: Decision-Making in Public Health

Chapter 3: Definition and Concepts

Chapter 4: HTA Decision Analysis framework

Chapter 5: Early HTA Advice

Chapter 6: Overview of Market Access Agreements

Chapter 7: External Reference Pricing

Chapter 8: Gap between Payers and Regulators

Chapter 9: Early Access Programs

Chapter 10: Market Access of Orphan Drugs

Building a think tank

 Build a think tank. A guide for policy entrepreneurs

Using this guide 8
The reasons for creating this companion guide 8
Using this companion guide 8
Introduction 11
The importance of supporting new think tank development 11
Unravelling the definition of think tanks 13
What’s in the name? 13
Defining think tanks 14
Think tank functions 15
Summing up 16
The Why? questions 18
Why do you want to set up a think tank? 18
Why do think tanks aim to influence policy? 23
Are you sure you want to establish a think tank? 25
The What? questions 26
What will your think tank do? 26
What is the context? 27
What do you want to achieve by setting up a think tank? 33
What issues will the think tank focus on? 37
What will the think tank want to influence? 38
What will its business model be? 40
The Who? questions 46
Who will govern it? (And how?) 46
Who will lead the think tank? 51
Who will engage with it? 56
Who will work for it? 60
Who will fund it? 65
Who will support it? 71
The How? questions 72
How will it carry out research? 72
How will it be managed? 79
How will it communicate? 82
How will it monitor its progress? 88
How will you ensure its credibility? 92
How will it adapt to change? 95
How will it engage with evolving technology? 99
The ‘When’? questions 101
When to start? 101
When to let go? 106
Checklist: Establishing a think tank 108
References and resources 110
On the definition and functions of think tanks 110
Boards and governance 111
Creating and managing think tanks 111
Policy impact 111
Communications 112
Funding and financial management 112
Context 113
Evidence-informed policy 113
List of Boxes, Figures and Tables 114

https://buildathinktank.org/

Market access for expensive therapies (or how to overcome prices) (2)

 Gene and Cell Therapies-Market Access and Funding

Contents:

Chapter 1        Introduction to cell and gene therapies concepts and definitions in US and EU

Chapter 2        cell and gene therapies: genuine products and potential for dramatic value

Chapter 3        cell and gene therapies: Regulatory aspects in US and EU

Chapter 4        the need for new HTA reference case for cell and gene therapies

Chapter 5        How to mitigate cell and gene therapies uncertainties and HTA risk adverse attitude?

Chapter 6        Cell and gene therapies funding: challenges and solutions for patients’ access

Chapter 7        Conclusion

Market access for expensive therapies (or how to overcome prices)

 Managed Entry Agreements and Funding for Expensive Therapies

Key reference to understand what's going on in this topic. Recommended.

Contents:

1. Introduction to Managed Entry Agreements

2. Definition and Classification of MEAs

3. From Coverage with Evidence Development to Individual Performance-Based Agreements in Italy

4. Coverage with Evidence Development for Multiple Sclerosis Drugs in the UK: A "Costly Failure"?

5. Country Comparison of the Implementation of Managed Entry Agreements

6. Novel Funding Models for Expensive Therapies

7. Managed Entry Agreement for Cell and Gene Therapies

The decline of pharma R&D productivity (how much are we willing to pay for failure (95%) or success (5%)?)

Global Trends in R&D . Overview through 2021 

The productivity of the clinical development process can be considered as a measure of trial outputs (drugs, innovation, trial success, etc.), compared to a measure of trial inputs or resources dedicated to obtaining those outputs (e.g., aspects of trial complexity, duration, monetary investments, etc.). Such measures of success, complexity and trial duration were selected for inclusion in the productivity index as described above. Increases in success will increase productivity overall as will decreases in complexity or duration. Conversely, decreases in success will drive down the productivity index, as do increases in complexity and duration.

Clinical development productivity — a composite metric of success rates, clinical trial complexity and trial duration — declined in 2021, continuing an overarching 10-year trend. Despite a second year of decreasing trial complexity, the ongoing decline in success rates has resulted in reduced average trial productivity. Trial success rates fell to their lowest in more than 10 years to an average 5% likelihood of progressing successfully through all phases.

Health systems design

 Building a High- Value Health System

Countries and institutions worldwide face the challenge of planning and paying for health care that effectively meets the needs of citizens and employees. While there are many criticisms of existing healthcare models, current literature offers little guidance for individuals who want to carry out the work of redesigning and improving their health system.