“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:

1. 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
2. FDA Patient Representative Program [6]: Offers patients and caregivers the opportunity to provide critical advice to the agency as it regulates medical products
3. 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

Link: https://www.ema.europa.eu/en/committees/working-parties-other-groups/chmp/patients-consumers-working-party 

6. FDA Patient Representative Program

Link: https://www.fda.gov/patients/learn-about-fda-patient-engagement/about-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