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
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).
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.
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
Build a think tank. A guide for policy entrepreneurs
Using this guide 8
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
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
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.
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.
Pharmaceutical innovation sourcing
Figure below shows that 23% of new medicines came from public bodies and private-private collaboration and they didn't apply for any marketing authorisation.
A decade ago I posted this: Gestionar el declivi. John Kay said in 2011:
"When an industry model is broken, the best business strategy may be to manage its decline"
Now a new report confirms that the process continues after a decade.
From IQVIA report:
The composite success rate across all development phases and therapy areas declined to 5.0% in 2021, which can be attributed to an appetite for increased scientific risk in clinical development programs as the bar for efficacy and safety rises, as well as increased pauses in product development due to the pandemic.
Across disease areas, probability of success varies considerably, and 2021’s composite success rate fell below the 10-year trend in all areas except for vaccines and cardiovascular.
That's all folks.
Today my suggestion is to read the post by Eric Topol with the same title.
It begins with this statement:
We’re now well over 20 years since the first human genome was sequenced, but with few exceptions the massive amount of data that has been generated has not been transformed to routine patient care.
So, why?
