#PatientsEU2019

Addressing the urgent need for PLS for clinical trials and

medical publications Dawn Lobban, Envision Pharma Group

Nicholas Brooke, Patient Focused Medicines Development

Lay summaries of clinical trials: Regulatory requirement and key considerations

• The EU Clinical Trials Regulation No. 536/2014 requires trial sponsors to provide PLS of clinical trial results• These will be housed in the EU clinical trial portal and database, under development by the EMA

Follow specific EU/FDA/ academic/industry guidelines

(EUAnnex V of Regulation 536/2014; MRCT toolkit)

Use neutral, objective language; must not be perceived as promotional

Include visuals/tables to make the data easier to understand

Consider structure; Criteria must be met, but can combine categories,

change order of headers, add subheaders

Clinical trial identifiers (include trial title, protocol number, EU trial number and any other identifiers)

Type and frequency of adverse events Name and contact details of the sponsor

Overall results of the clinical trialGeneral information about the clinical trial (include where and when the trial was conducted, the main objectives of the trial, and the reasons for conducting it) Comment on the outcomes of the clinical trial

Population of participants (include the number of participants in the EU Member State concerned, EU nations and nations with third-country relationships with the EU; age group and gender breakdown; and inclusion and exclusion criteria)

Indicate if follow-up clinical trials are planned

Investigational medicinal products used

Indicate where to find additional information

1

62

73

8

4 9

5

10

https://ec.europa.eu/health/sites/health/files/files/eudralex/vol-10/2017_01_26_summaries_of_ct_results_for_laypersons.pdf

Do we need anything else?

W H AT D ATA W O U L D W E M I S S ? W H AT C O N T E X T W O U L D W E M I S S ?

Laysummary PLS

Real-wor ld evidence

Systematic reviews

Case studies

Global studies without an EU site

Reviews on disease burden

(Some) patients want to understand published research:

but it isn’t that easy

*Amelot V et al. Pharm Med. 2017;21:329-37 (study included 28 patients and 35 parents of patients).

I N A S U R V E Y O F P E O P L E W I T H F R I E D R I C H ’ S A T A X I A A N D T H E I R C A R E R S : *

Most patients and carers were interested in scientific publications related to their condition

Few could understand scientific publications

Few considered the Internet (Facebook, discussion forums, etc) to be a useful source for better understanding

Patients

67.9%

Patients

12%

Patients

32.1%

Carers

78.8%

Carers

6.3%

Carers

5.7%

Publication: to make content available to the public

(Some) patients aren’t waiting for the published data: they are attending conferences and writing their own summaries

One of my biggest takeaways from the APSS 2018 SLEEP meeting last month was the great amount of drug development underway for narcolepsy! Below is a list of some of the emerging treatments “abuzz” at #SLEEP2018.

Please remember, I failed high school biology (sorry mom!). However, I hope this post helps provide access to information.

When navigating a serious condition like narcolepsy, information is power.

http://julieflygare.com/2018-exciting-update-advancing-novel-treatment-options-for-narcolepsy/

Addressing the need for PLS

Information that is easy to access and understand can prevent misinformation

People have a right to information that is about them: transparency builds trust

PLS can increase the reach of data to different audiences and are often shared on SoMe

By empowering patients, PLS can facilitate shared decision making

W H Y ? H O W ?

Overcoming compliance concerns Discuss the Code (early) - communication is not promotion

FDA: “It has long been FDA policy not to consider a firm’s presentation of truthful and non-misleading scientific information about unapproved uses at medical or scientific conferences to be evidence of intended use when the

presentation is made in non-promotional settings and not accompanied by promotional materials…”FDA Memorandum – Public Health Interests and First Amendment Considerations…January 2017 p 21

Our approach to PLS is evidence based and constantly evolving

MRCT Center Return of Aggregate Results to Participants Guidance Document Version 3.1 22 November 2017

Annex V of Clinical Trial Regulation (EU No 536/2014)

Expertise is required Team approach is needed to co-create PLS of publications

P L A I NL A N G U A G E

W R I T E R

S C I E N T I F I CC O N T E N TE X P E R T S

V I S U A LC O M M U N I C A T I O N

E X P E R T

P A T I E N TP A R T N E R S H I P

E X P E R T

Co-creation workshop: London, Nov 2018 B R O U G H T K E Y S T A K E H O L D E R S T O G E T H E R T O D I S C U S S

Patients

Publishers

Industry

Publicationprofessionals

Evidence relevantto PLS

Real-worldcase studies

Co-creation of practical tools for PLS

How to ensure access, quality and integrity of PLS

The Workshop was a ‘tools fest’ not a ‘talk fest’

Agreement and recognition of the need to prioritise patient preferences and take an evidence-based approach

A collaborative event where the voice of the patient was

powerful throughout the day

Key learnings: Ensuring ethical and effective preparation

Industry and publishing codes reinforce that communication does not equal promotion

Avoid ‘cherry picking’ which publications have PLS. Transparency builds trust

Identify the ‘right’ patient reviewer – ‘lived’ experience, lay perspective, or both?

Drive awareness – make PLS available and accessible to all who need them

Use an experienced and specialist PLS team to deliver at scale, on time, and to industry standards

Output: The future of PLS

EV I D E N C E B I B L I O G R A P H Y

G LO S S A RY

1 P G D ATAV I S I O N U P D AT E

1 P G P L S V S L AY S U M M A RY

Q C C H E C K L I S T

P L S T E M P L AT E

S P O N S O R R EV I E W E R G U I D E

PAT I E N T R EV I E W E R G U I D E

A W O R L D - F I R S T C O - C R E AT E D P L S O F P U B L I C AT I O N S T O O L K I T W I L L B E R E L E A S E D I N E A R L Y 2 0 1 9

Leverage publication software for the optimal management of PLS

Addressing the need for PLS

Information that is easy to access and understand can prevent misinformation

People have a right to information that is about them: transparency builds trust

PLS can increase the reach of data to different audiences and are often shared on SoMe

By empowering patients, PLS can facilitate shared decision making

W H Y ?

Work with legal teams from the outset to ensure a compliant plan that avoids ‘cherry picking’ of data for PLS

Consider the optimal format and communication channels for PLS including open access journals and congresses

Combine scientific understanding, plain language expertise, graphical communications skills and patient partner insights

H O W ?

#PatientsEU2019

For Further information please contact:

Dawn Lobban Scientific Divisional Lead Envision Pharma Group

+44 (0) 7557238550 Dawn.Lobban@envisionpharmagroup.com