Insights on the research and publication “Patient involvement in the development, regulation and safe use of medicines”. What are the recommendations?
Dr Lembit Rägo
Secretary-General
CIOMS
ragol@cioms.ch
Council for International Organizations of Medical Sciences�
Founded in 1949 by WHO and UNESCO�In official relations with WHO | UNESCO associated partner | ICH Observer since 2016
CIOMS mission
is to advance public health through guidance on health research including ethics, medical product development and safety
https://cioms.ch
CIOMS Working Group XI:
Patient involvement in the development, regulation and safe use of medicines
Started in April 2018
Objective | To discuss benefits of patient involvement during the whole life-cycle of a medicine, from early development through regulatory process and post-marketing use until (potential) retirement: life-cycle approach |
Composition | International, with involvement of patient representatives, regulators (including US FDA and EMA), industry, academia and international/national organizations including WHO |
Transparent process | All full WG meeting minutes made public, public meetings organized for input (Geneva, Kampala), public consultation of the final text with structured analysis and use of comments |
Report content | 11 chapters with annexes: numerous case studies, a very comprehensive glossary (>90 terms and definitions) |
Accessibility | The publication is a ‘public good’: �open-access online publication �limited hard copies at postal costs |
Ethical considerations for patient involvement |
Executive summary |
Chapter 1: Introduction |
Chapter 2: Landscape |
Chapter 3: Guiding principles |
Chapter 4: Advancing treatments |
Chapter 5: Use of real-world data and evidence |
Chapter 6: Product labeling |
Chapter 7: Rapid safety communication |
Chapter 8: Additional risk minimization |
Chapter 9: Clinical practice guideline |
Chapter 10: Low- and middle-income countries |
Chapter 11: Pandemic considerations |
Appendices:
|
Comprehensive report
236 pages
Appendices
1 | Glossary |
2 | Case studies |
A | Medication formulation created to meet patients’ and doctors’ needs (AdrenalNET) |
B | A regulatory agency involving patients; public hearing on valproate (EMA) |
C | Pilot collaboration between Lareb and a patient organisation in communicating a signal (Lareb) |
D | Creating partnerships between industry and patient groups for therapy development (Roche) |
E | Example of a pharmaceutical company working with patients to develop an additional risk minimisation measure |
F | Engaging patients in early development plans for a novel treatment (Takeda) |
G | Patient activism to counter AIDS denialism and improve access to HIV medicines in South Africa |
3 | (…) |
Each case study describes: |
Purpose and objective of the case study |
Pharmacology |
Indication/disease treated |
Stage of the drug development lifecycle |
Why were patients involved? |
How was contact established with the patients? |
What did the patients do? |
Was the process adjusted to the patients’ needs? |
If patients were asked to help disseminate information, how was it done? |
Did the patients receive payment or compensation? |
Were any patient requests or recommendations discarded and why? |
Conclusion |
Contact details |
3. Patient participation is needed in planning, testing, reviewing, approving, and monitoring treatments throughout the lifecycle of medicines.
Life cycle approach
Chapter 4. Figure 4. Patient involvement in the medicines lifecycle at European Medicines Agency
Conclusion
1
2
3
4
5
The CIOMS Working Group report is a unique resource:
Recommendations
Readers should select those best practices that fit with their current organisational needs |
Patient engagement should be non-tokenistic (not just for appearance’s sake) |
No “one-size-fits-all” approach: The report recommendations require nuanced interpretation according to circumstances |
Sharing the lessons learned will advance patient involvement further |
… |
Thank you!
Questions?