Not Just Patients

Join us for this special Rare Disease Day episode of Not Just Patients, where we speak with Jo Balfour, Managing Director and Co-founder of Cambridge Rare Disease Network (CamRARE), about research in rare disease.  

Jo is the driving force behind the CamRare's innovative events program, designed to raise awareness about rare diseases and encourage cross-sector collaboration. She manages the organisation's Companies Forum, a networking and knowledge-sharing platform for pharma, biotech, and life science companies, and oversees all other aspects of the charity's research and project work. Additionally, Jo founded the charity's regional rare disease community, Unique Feet, bringing families together in an inclusive and supportive environment. 

Listen to this insightful conversation where we discuss the unique challenges faced by people living with rare disease, overcoming barriers to rare disease research, and the value of collaboration between researchers and rare disease patient organisations.

Chapters

0:00 - Introductions
1:30 - Jo's journey into rare disease advocacy
7:07 - Challenges of living with a rare disease
11:34 - Challenges to rare disease research
15:58 - The numbers game and need for data
21:29 - Collaborating with patient organisations
28:10 - Navigating the rare disease advocacy landscape
40:12 - Initiatives supporting collaborative research
47:47 - Jo's dream for rare disease research
50:53 - Advice for researchers
51:14 - Advice for patient communities
53:50 - Reflections with Caitlin and Clarinda

Resources and abbreviations mentioned:

• UK Rare Diseases Framework
• CoRDS - Coordination of rare diseases at Stanford
• Duchenne UK
• PROMs - patient-reported outcome measures 
• Rare Diseases International
• Global Genes
• EURORDIS
• Genetic Alliance UK 
• Cystic Fibrosis Trust
• Cambridge Rare Disease Network (CamRARE)
• Department for Health and Social Care Disease Research Landscape Report
• RING 20
• Rare Disease Research Network
• Cambridge Rare Summit
• MRC - Medical Research Council, UK
• NIHR - National Institute for Health and Care Research, UK
• LifeArc
• Additional suggested: The Power of Being Counted

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Join us for an insightful conversation with Maya Zlatanova, CEO and Co-founder of FindMeCure and TrialHub, as we discuss patient-aligned clinical trials that consider both scientific rigour and patient needs.

Maya is a health-tech entrepreneur and pharma industry speaker with over 15 years of experience in feasibility, operations, and patient recruitment and engagement. Guided by her strong sense of empathy and love of storytelling, Maya champions patient-aligned trial design and is passionate about improving the way clinical research is conducted.

This episode highlights the barriers patients face in accessing trials, the importance of awareness and education, and the benefits of improving clinical trial design to enhance patient experiences.

Resources and abbreviations mentioned: 

• UK Clinical Trials Gateway
• FindMeCure
• TrialHub 
• EUPATI - European Patients Academy on Therapeutic Innovation
• Maya's publication on a statistical model for quantifying patient experiences
• Clarinda's publication on patient journeys

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Join us for this special (unedited) recording of Not Just Patients, which was streamed LIVE on LinkedIn!

To celebrate our 10th episode, we were joined by two incredibly inspiring advocates, Danielle Drachmann and Sumaira Ahmed. Listen to this uplifting, raw, and real conversation as these two amazing women share how they turned their adversity into advocacy, and the unspoken realities of their deeply personal work.

About our guests: 

Danielle Drachmann is a recipient of the 2022 Young Patient Advocate Award at the Black Pearl Awards, and Executive Director at Ketotic Hypoglycemia International. As a rare disease patient and mother of three children with a rare disease, she is deeply passionate about patient engagement, partnering, citizen science, and patient-driven research. At Evidera, part of Thermo Fisher Scientific, Danielle specialises in fostering multi-stakeholder relationships and advancing patient-centred research. Additionally, she contributes to various health committees and has helped shape Europe's rare disease policy through her work with EURORDIS.

Sumaira Ahmed was diagnosed with seronegative neuromyelitis optica spectrum disorder (NMOSD) in the summer of 2014, after experiencing sudden and severe vision loss and weakness/numbness. Less than two months after her diagnosis, she founded The Sumaira Foundation (TSF) dedicated to generating global awareness of NMOSD, MOGAD and other rare neuroimmune conditions, building communities of support for patients and their loved ones, supporting research and advocating on behalf of patients. Sumaira currently serves as the Executive Director of The Sumaira Foundation.

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Join us for a compelling conversation with Professor Lara Bloom, President and CEO of the Ehlers-Danlos Society. A strong patient advocate, Lara has a passion for pushing boundaries and fighting for progression DE&I in healthcare and LGBTQIA+ rights.

Throughout our discussion, we dissect the complex relationship between health equity and inclusion on a global scale. The episode also explores the transformative power of patient engagement in achieving health equity, emphasising the importance of true representation and inclusion.

Chapters
00:00:00 - Introductions
00:01:20 - Lara's journey into advocacy
00:04:16 - State of DEI in healthcare
00:10:45 - Challenges viewed through a global lens
00:15:00 - Interplay between health equity and diversity & inclusion
00:17:40 -  Understanding diverse communities and minorities
00:24:05 - Need for better representation in healthcare
00:29:09 - Evolution of DEI in healthcare
00:35:13 - Initiatives working to further DEI in healthcare
00:37:18 - Understanding privilege in the context of healthcare
00:40:42 - Mindset for overcoming challenges
00:44:03 - Future and advice for healthcare stakeholders
00:47:00 - Advice for patients and patient advocates
00:48:56 - Power of patient organisations
00:54:53 - Reflections with Caitlin and Clarinda

Resources and abbreviations

• Ehlers-Danlos Support Group (EDS UK)
• EDS - Ehlers-Danlos Society
• EDNF - Ehlers-Danlos National Foundation, US
• HSD – hypermobility spectrum disorders
• DEI - diversity, equity, and inclusion
• NHS - National Health Service, UK
• Global Genes conference
• CF – cystic fibrosis
• ESG – environmental, social, and governance
• World Health Assembly
• Global Patient Movement Catalyst
• EDS Road to 2026
• World Health Summit

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On this episode, Dr. Anke-Peggy Holtorf joins us to help demystify the complex processes of regulatory affairs and health technology assessment (HTA) and explain the crucial role of patients in these processes.

Anke acts as Secretary of the Board at Health Technology Assessment international  and is a Steering Committee member and project coordinator for the Patient and Citizen Involvement in HTA Interest Group. She is co-editor of the 2nd edition of the book on Patient Involvement in HTA, which will be published in 2025. Anke has also taught graduate courses in International Health Policy, HTA, and the role of patient involvement.

Anke's global experience and depth of knowledge spanning decades shine through as she simplifies some highly technical and complex concepts for our listeners. 

Chapters:
0:00 - Introductions
1:34 - Overview of regulatory affairs and HTA
6:48 - The regulatory approval process
12:09 - Patient involvement in regulatory affairs
22:19 - Overcoming barriers to patient involvement
27:32 - Regulatory success rates
30:30 - The HTA process
32:32 - Differences in healthcare models
36:46 - Factors evaluated in HTA
43:45 - Patient involvement in HTA
54:32 - Advice for patients and stakeholders
59:08 - Reflections with Caitlin and Clarinda

Resources and definitions:

• HTA - Health Technology Assessment 
• Reimbursement: the process by which healthcare providers are compensated/paid back for the medical services they provide, either by the government or through insurance
• Out-of-pocket costs: when patients pay for their own medical costs
• FDA - US Food and Drug Administration
• EMA - European Medicines Agency
• Chinese FDA 
• QALY - Quality adjusted life years
• WHO - World Health Organization
• NICE - National Institute for Health and Care Excellence, UK
• CF Trust - Cystic Fibrosis Trust 
• EUPATI - European Patients' Academy on Therapeutic Innovation 
• EUCAPA - European Capacity Building for Patients 
• Anke's report on Patient Involvement in HTA in Europe - full report and short version

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On this episode, we speak to Mark Duman, Chief Patient Officer at MD Healthcare, about digital health and health data, how digital therapeutics get developed and approved, and how challenges around access and equity can be navigated.

Mark is a clinician, management consultant, and patient advocate who works with several healthcare organisations to harness digital health, improve market access, and embed patient engagement in healthcare products and services.

Join us for this insightful conversation on a hot and rapidly evolving topic.

Chapters
0:00 - Introductions
1:47 - Mark's story
3:46 - Scope of digital health and health data
6:11 - Enhancing patient care
12:15 - Patient engagement
14:44 - Regulations and reimbursement
21:59 - Challenges around access and equity
28:55 - Development process and patient inclusion
37:35 - Data privacy and ethics
47:18 - Future of digital health
52:57 - Advice for patients/stakeholders
56:39 - Reflections with Caitlin and Clarinda

Resources/abbreviations

• Cerner system for electronic health records
• QALY – quality-adjusted life years
• WALY – wellbeing-adjusted life years
• Salford Lung Study
• PREMS – patient-reported experience measures
• PROMS – patient-reported outcomes measures 
• ABPI - Association of the British Pharmaceutical Industry
• ABHI - Association of British HealthTech Industries
• EUPATI Patient Engagement Roadmap
• DTA - Digital Therapeutics Alliance
• DIME – Digital Medicine Society
• NICE – National Institute for Health and Care Excellence, UK
• Sleepio
• Nesta report
• CE Mark
• DTAC – Digital Technology Assessment Criteria
• DTS (data security and protection) toolkit
• Scotland’s Patient Experience Programme
• Flatiron Health Patient Voices Panel
• GDPR - General Data Protection Regulation
• Information Governance Toolkits
• HMO - health maintenance organization
• ZOE Study
• Lloyd’s Bank Consumer Digital Index
• Patient Innovation

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On this episode, we speak to Avishek Pal about how patients can better access and contribute to medical publications.

In parallel to his day job as Global Medical Director of Cell & Gene Therapy at Novartis, Avishek is affiliated with the Institute for Biomedical Ethics at University of Basel, investigating how medical research information in plain language may improve health literacy and autonomy in patients with chronic diseases.

Join us as we discuss how patients seek medical information, the role of generative AI, and initiatives fostering better patient access and involvement in medical literature.

Chapters:

• 0:00 - Introductions
• 1:48 - Patients' information-seeking patterns
• 4:38 - Role of health literacy
• 6:21 - Importance of accessing medical literature
• 8:44 - Information overload & reliability
• 15:24 - Plain-language summaries
• 26:43 - Publication bias in medical research
• 30:54 - AI for medical information
• 39:25 - Patients as authors
• 49:53 - Future of medical publications
• 53:15 - Advice for different stakeholders
• 56:44 - Reflections with Caitlin & Clarinda
  
  

Resources and abbreviations:

• Cochrane Library
• Pubmed
• Google Scholar
• CF - cystic fibrosis 
• Avishek’s landscape analysis paper on plain-language resources
• Future Science Group, now a part of Taylor & Francis
• Becaris Publishing
• Envision Group
• A&E - accident and emergency
• CF Trust - Cystic Fibrosis Trust 
• NHS - National Health Service, UK
• CDC - US Centres for Disease Control and Prevention
• NIH - National Institutes of Health, US
• FDA - US Food & Drug Administration
• EMA - European Medicines Agency 
• Chat-GPT
• Avishek's paper on ethical considerations for plain-language materials
• EUPATI - European Patients’ Academy on Therapeutic Innovation
• PIF TICK
• Discern
• ICMJE (International Committee of Medical Journal Editors) authorship criteria
• Clarinda’s Patient Perspective paper
• Coalition S

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In this episode, we speak to Dr. Victor Montori about the importance of shared decision-making in healthcare.

Dr. Montori is the Robert H. and Susan M. Rewoldt Professor of Medicine at Mayo Clinic. An endocrinologist, health services researcher, and care activist, he is the author of more than 750 peer-reviewed publications and is among the most cited researchers in clinical medicine and in social science. He is a recognized expert in evidence-based medicine, shared decision making, and minimally disruptive medicine. He is also the author of the book Why We Revolt, and is leading a movement, a Patient Revolution, for Careful and Kind Care for all.

In our conversation, we discuss the need for patient-centred care that involves and respects individuals as holistic beings, the challenges that inhibit shared decision-making in practice, and the broader societal need to foster self-care and community support.

Chapters
0:00 - Introductions
1:40 - What is shared decision-making?
5:30 - Careful and kind care 
9:24 - Forms of shared decision-making
14:58 - Victor's motivation
18:50 - How much time should doctors spend?
23:20 - Is shared decision-making for all patients?
27:15 - Who is responsible for shared decision-making?
31:28 - Where can clinicians learn shared decision-making?
35:46 - A Patient Revolution
44:25 - Barriers to overcome
50:08 - Tackling an insurmountable challenge
55:43 - Reflections with Caitlin & Clarinda

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On this episode, we talk to Robert Joyce about the critical role of patient involvement in clinical research and the various ways in which patients can bring rich perspectives to the research and development process.

Robert has lived with chronic illness all his life, but following a traffic accident in 2014, he was left with debilitating symptoms. No longer able to work, Robert started his blog, a 30 minute life, to share his experiences, and also has a podcast of the same name.

In 2019, Robert also became involved in clinical research, eventually earning the title of Embedded Patient Researcher and becoming a co-applicant on the trial of a therapy for Multiple Sclerosis. Unfortunately he was not permitted to continue in this role, but is now an advocate for more patients to be involved in research.

Chapters:

0:00 - Introductions

1:25 - Role of an embedded patient researcher

8:10 - Benefits of patient involvement in research

15:56 - Different ways patients can be involved in research

23:08 - Role of country/disease-specific patient organisations

25:08 - Challenges and initiatives for rare diseases

27:37 - Initiatives to bring patients closer to research

29:10 - Robert's story and personal challenges

33:55 - Challenges to patient involvement

35:44 - What needs to be done to overcome barriers

44:22 - Engaging funders in patient involvement

51:30 - Advice for patients looking to get involved

53:01 - Advice for stakeholders looking to involve patients

55:38 - Reflections with Caitlin and Clarinda

Resources and abbreviations mentioned:

• MS - multiple sclerosis
• SWOT - strengths, weaknesses, opportunities, and threats (analysis)
• PPI - patient and public involvement
• PPI Ignite Network

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Email: [email protected]

On this episode, we speak with Bronwyn Lewis, Global Head of Patient Engagement at Boehringer Ingelheim.

Drawing on her 20+ years of experience across diverse roles in the pharmaceutical industry, Bronwyn offers deep insights into how the industry is perceiving and  responding to the call for deeper patient engagement, the challenges and opportunities for better multi-stakeholder collaboration, and her vision for the future.

Note: In the episode, Bronwyn mentions  Boehringer Ingelheim’s Research & Development (R&D) investment data for the year 2022. In 2023, Boehringer Ingelheim’s R&D investments increased by 14.2% to EUR 5.8 billion. R&D investments were substantial, at 22.5% of net sales (source: Boehringer Ingelheim strong growth pipeline acceleration 2023 | Boehringer Ingelheim (boehringer-ingelheim.com)).

Chapters

• 00:00 - Introductions
• 01:03 - Defining patient engagement from the industry perspective
• 04:06 - Bronwyn's journey into patient engagement
• 06:51 - The Patient Engagement function at Boehringer Ingelheim
• 10:39 - Composition of Patient Engagement teams in pharma    
• 13:22 - Aligning the motivations of pharma and patient groups
• 16:42 - Barriers to pharma–patient collaboration
• 20:23 - Justifying return on investment (ROI) 
• 26:17 - Patient engagement touchpoints and activities
• 29:16 - Matching best-fit patients to targeted activities 
• 32:21 - Systemic integration of patient insights into all pharma operations 
• 36:25 - Examples of patient insights influencing decisions
• 38:28 - Boehringer Ingelheim's Global Patient Partnership Summit
• 44:26 - Collaboration among industry stakeholders
• 47:56 - Visualizing the future
• 56:14 - Advice for patients and industry stakeholders 
• 58:13 - Reflections with Caitlin and Clarinda

Resources and abbreviations mentioned

• Boehringer Ingelheim
• Canadian Pulmonary Fibrosis Foundation
• IPF - Idiopathic pulmonary fibrosis
• PFMD - Patient-Focused Medicines Development
• EUPATI Connect 
• ROI - Return on investment
• HTA - Health Technology Assessment 
• FDA - U.S. Food and Drug Administration 
• EMA - European Medicines Agency
• PFMD - Pharmaceuticals and Medical Devices Agency
• GPPS - Global Patient Partnership Summit
• Patient Information Forum
• The PIF TICK
• Atomic Habits by James Clear
• EUPATI Fundamentals training

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Email: [email protected]