Insights | Patient Centricity and the Patient Voice
“Our industry has long spoken of “patient centricity” as a key to progress. But the concept has fallen short – largely due to well-meaning conversations about patients rather than with patients. It is a relatively recent phenomenon wherein true “patient voice” is recognised as an essential asset in the development of medicines that matter to patients.”
[Maryna Kolochavina – Rare and Orphan Disease Expert and Patient Advocate]
‘Patient Voice’ and ‘Listening to the Patient Voice’ are terms we hear quite often, but what does this mean? What is the ‘patient voice’?
Put simply, patient voice refers to the opinions and experiences of your patients and how these views inform the medical treatment they receive [1].
Why is the Patient Voice so Important to Non-Interventional Studies?
Patient voice makes up at least 50% of patient engagement. If patients don’t feel their voice is being heard they won’t feel engaged and satisfied with their healthcare. Poor engagement levels lead to worsened public health through [2]:
- Non-adherence to recommended care
- A greater likelihood of bad lifestyle decisions
- A lack of knowledge regarding both treatment options and preventative measures
The solution, in the context of the design and conduct of NIS, is to listen to the viewpoint of patients and caregivers’ by [3]:
- Using patient advocacy groups
- Using patient/caregiver surveys
- Insertion of patients/caregivers into the protocol steering committees
- Attendance at regulatory advisory committee
Engaging Patients in Medicines Regulation
The European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) both consider that the voices of patients in medicines regulation are essential, as they bring the unique perspective of someone living with a disease, as a patient or carer [4].
Regulator and Regulatory Initiatives Include:
- EMA Patient’s and Consumer’s Working Party [5]: Provides a platform for exchange of information and discussion of issues of common interest between EMA and patients and consumers
- FDA Patient Representative Program [6]: Offers patients and caregivers the opportunity to provide critical advice to the agency as it regulates medical products
- Section 204 of the proposed 21st Century Cures 2.0 Act – Patient Experience Data [7]: Requires drug manufacturers/sponsors to collect and report on patient experience data as part of the clinical trial. Requires FDA to fully consider all patient experience data collected during the clinical trial
Why is the Patient Voice so Important to Innovation, Drug Development and Real World Research?
Patients and caregivers have a unique perspective of the disease and the needs of that patient group that are vital to drug development [3].
The future of clinical research relies on our ability to listen and respond [to patients] – this is the sweet spot where innovation happens. It’s also a crucial part of creating remarkable patient experiences [1].
References
1. MD Group –Why We Need to Listen to the Patient Voice
Link: https://mdgroup.com/blog/why-we-need-to-listen-to-the-patient-voice/
2. Jayex – What Is Patient Voice and Why Does it Matter?
Link: https://www.jayex.com/en-au/blog/why-patient-voice-is-key-to-improving-patient-engagement
3. Lee, D. 11 – Listening to the patients’ voice, Editor(s): Philippe Auby, In Woodhead Publishing Series in Biomedicine, Clinical Research in Paediatric Psychopharmacology, Woodhead Publishing, 2020, Pages 173-182, ISBN 9780081006160,
Link: https://www.sciencedirect.com/science/article/pii/B9780081006160000113
4. Mavris M, Furia Helms A, Bere N. Engaging patients in medicines regulation: a tale of two agencies. Nat Rev Drug Discov. 2019 Nov;18(12):885-886. doi: 10.1038/d41573-019-00164-y. PMID: 31780842.
Link: https://media.nature.com/original/magazine-assets/d41573-019-00164-y/d41573-019-00164-y.pdf
5. EMA Patient’s and Consumer’s Working Party
6. FDA Patient Representative Program
7. Section 204 of the 21st Century Cures 2.0 Act – Discussion Draft
Link: https://degette.house.gov/sites/degette.house.gov/files/Cures%202.0_DISCUSSION%20DRAFT.pdf
Insights – Document Management and Archiving Requirements for Observational Studies
5 NOVEMBER 2021 – Did you know that there is a DIA TMF Reference model for observational studies?
- [Lean Version] Real-World Studies Document Index Provides a proposed Document Index for use on real-world studies, based on the TMF Reference Model for clinical trials (v1.0 Approved 29-July-2020) [1]
- [Detailed Version – with guidance notes] Study Master File [2]
For years, those of us who have been running observational studies (also known as non-interventional studies) have been the ‘poor relation’ with regards to document management tools and offerings. We’ve had to ‘bootstrap’ existing clinical trial tools to fit the significantly different document requirements for observational studies. This is why, in 2018 a group of frustrated experts got together with the blessing of the TMF Reference Model Steering Committee…and the rest, as they say…is history…
Why is a Stand Alone Study Master File Needed?
Those of you who are involved in Real World Studies (RWS) know that trying to file your documents using the existing TMF Reference Model (TMF RM) based on ICH E6 can be challenging. Your study isn’t a clinical trial and as a result, at least 40% of the artifacts in the index are irrelevant. The result? You start from the beginning every time you set up a new study and ‘switch off’ the tabs that aren’t applicable. Alternatively, you may be required to use the existing TMF RM in accordance with your organisation’s policy and disregard tabs that aren’t relevant. Additionally, you hope that an auditor of your ‘real world study TMF’ understands your study and doesn’t penalize you for missing documents (or documents that were never generated, because your study is not a clinical trial).
Real World Studies are increasingly being used to support drug development, product licensing, label claims and reimbursement. Consequently the need for an audit-ready framework has become a critical requirement to effectively collect and manage the documents that evidence integrity in relation to study conduct and study data.
Designing a Solution
To address this, a working group of RWS experts* convened in March 2018 to develop a framework for filing essential documents for non-interventional and observational studies. In designing the Real World Study-Document Index (RWS-DI), the group took into account the ethical standards, regulatory requirements, guidelines and industry best practices applicable to non-interventional studies (NIS). The group developed the RWS-DI based on a prospective study design to provide maximum coverage of the potential documents (or artifacts) across the range of real world study designs, from non-interventional studies, retrospective chart reviews, to prospective product registry studies.
The result is a listing of core and recommended artifacts relevant to Real World Studies (which are not clinical trials) while remaining as consistent as possible with the TMF-RM format and structure. The RWS-DI adapted the TMF RM removing artifacts specific to clinical trials such as Investigational Product Dossier (IMPD) or Investigator’s Brochure (IB) and replaced terminology such as ‘trial’ with ‘study’ and ‘subject’ with ‘patient’.
The RWS-DI was sanctioned under the auspices of the DIA TMF Reference Model Steering Committee throughout its development. It was endorsed by the DIA TMF Reference Model Steering Committee as a ‘stand-alone’ deliverable for launch to the wider research community. It is anticipated that, as the RWS-DI becomes widely adopted, it will evolve to reflect user community requirements. Feedback, enquiries, and suggestions for enhancements for incorporation in future versions can be made using the e-mail address stuart.mccully@phoenix-rwr.co.uk.
The Real World Studies Working Group comprised of the following members, all of whom work within the Real-World Study environment and contributed their expertise throughout the duration of the project.
Shelley Brigstock Study Project Manager, Novo Nordisk
Kath Firth Head of Quality Operations, GSK
Tara Isherwood Senior Director, Regulatory Advice and Delivery, Syneos Health
Russell Joyce Director and Principal Consultant, Heath Barrowcliff Consulting
Jeff Kirsch* Senior Director, Quality & Risk Management and Governance, GSK
Stuart McCully Founder, Phoenix RWR
Linda Rudolph Principal Consultant, Quality Werx, LLC
____________________________________________________
*Currently-Director and Founder, Jeff Kirsch Consulting, Ltd
Want to Learn More?
Speak to the Master Architect – Russell Joyce. Russell is one of the founders of the TMF Reference Model and the chair of the Study Master File working group (Real World Study Document Index)
Email: russell@heathbarrowcliffconsulting.co.uk
Web: https://heath-barrowcliff-consulting.co.uk/
LinkedIn: https://www.linkedin.com/in/russell-joyce-18524918/
References
1. Real-World Studies Document Index Provides a proposed Document Index for use on real-world studies, based on the TMF Reference Model for clinical trials (v1.0 Approved 29-July-2020)
2. Study Master File
