An Exploration of the Impact of Emicizumab on the Lives of People With Haemophilia and Inhibitors and Their Families
Conditions: Hemophilia A With Inhibitor
Sponsor: Haemnet

 
Emi and Me Study Protocol V4 
 
 
1
 
 
An Exploration of the Impact of Emicizumab on the 
Lives of People with Haemophilia and Inhibitors 
and their Families 
 
 
Principal investigators: Simon Fletcher 
Co-Investigators: 
Dr Kate Khair, Luke Pembroke  
Study Sponsor:  
Haemnet (Registered Charity No 1152241) 
Study Coordinator: 
Mike Holland, Haemnet 
Version date: 
 
4 November 2019 
Version number: 
Version 4 
 
 
 
 
 
 
CONFIDENTIAL 
Information and data included in this document contain privileged or confidential 
information, which is the property of the sponsor. No person is authorised to make it 
public without written permission of the sponsor. These restrictions on disclosure will 
apply equally to all future information supplied to you, which is indicated as privileged or 
confidential. This material may be disclosed to and used by your staff and associates as 
may be necessary to conduct the clinical study. 
 
 
 

 
Emi and Me Study Protocol V4 
 
 
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List of Abbreviations 
 
Abbreviation 
Definition 
CCC 
Comprehensive Care Centre 
EAMS 
Early Access to Medicines Scheme 
Emi 
Emicizumab 
Haemo-QOL 
Haemophilia Quality of Life scale 
Hep-Test-Q 
Haemophilia and Exercise Project Test Questionnaire 
HJHS 
Haemophilia Joint Health Score 
HRQoL 
Health-Related Quality of Life 
HTC 
Haemophilia Treatment Centre 
PWH 
People with Haemophilia 
QoL 
Quality of Life 
SO-FIT 
Study Of physical Function In adolescenTs with haemophilia 
 
 
 
 
 
 

 
Emi and Me Study Protocol V4 
 
 
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Study Synopsis 
Title 
Emi and Me: an exploration of the impact of emicizumab on the lives 
of people with haemophilia and inhibitors and their families 
Short Title 
Emi and Me 
Study aim 
This study aims to examine the real-life experience and impact of 
using emicizumab in a cohort of patients with haemophilia and 
inhibitors, who are being prescribed emicizumab in routine clinical 
practice as well as those who have been in the clinical trials. We also 
intend to capture the impact of emicizumab use on the lives of close 
family members (parents/carers/children/siblings). 
Primary 
Objective 
To capture the real-life experience of using emicizumab for 
haemophilia inhibitor therapy for the patient with haemophilia and 
his close family members 
Secondary 
Objectives 
To describe difference patient satisfaction with injections (numbers of 
injections, how to remember treatment dates, and so on). 
To understand user’s expectations of emicizumab and how they see 
future haemophilia care (less frequent injections, impact on home 
storage, number of bleeds, reduced hospitalisations and so on). 
To describe the as yet ‘unseen’ impact on the extended family – and 
to seek their views of innovative therapy for haemophilia. 
Design 
Non-interventional qualitative study 
Number of 
Centres 
N/A (recruitment via social media) 
Duration 
6 months data collection 12 months total study duration 
Number of 
Subjects 
25 ‘dyad’ pairs 
Inclusion Criteria 
Study participants will be people with inhibitors using emicizumab 
(prescribed by treating clinicians) in usual clinical care. Family 
members of participants will also be included. 
Exclusion 
Criteria 
Participants will be excluded from the study if they do not have an 
inhibitor, are not being treated with emicizumab, do not speak English 
(for the interviews) or do not consent. 
Statistical 
Analysis 
This is a qualitative study using established qualitative research 
methodologies (grounded theory). Statistical evaluation is not 
appropriate 
Operational procedures 
Ethical approval 
NHS ethical approval will be sought from HRA  
Data analysis 
To be conducted by Haemnet 
Write up and 
publication 
Lead researcher and research team  
 
 

 
Emi and Me Study Protocol V4 
 
 
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Emi and Me 
An Exploration of the Impact of Emicizumab on the Lives of People 
with Haemophilia and Inhibitors and their Families 
Index 
1 
Background ............................................................................................................. 5 
1.1 
Disease Introduction .................................................................................................... 5 
1.2 
Rationale for the study ................................................................................................. 6 
2 
Aim and Objectives .................................................................................................. 6 
2.1 
Aim ............................................................................................................................... 6 
2.2 
Primary Objective ......................................................................................................... 6 
2.3 
Secondary Objectives ................................................................................................... 7 
3 
Study design ............................................................................................................ 7 
3.1 
General Design ............................................................................................................. 7 
3.2 
Primary Endpoints ........................................................................................................ 7 
3.3 
Secondary endpoints .................................................................................................... 7 
3.4 
Statistical analysis and sample size calculation ............................................................ 8 
4 
Study Group ............................................................................................................ 8 
4.1 
Inclusion Criteria ........................................................................................................... 8 
4.2 
Exclusion Criteria .......................................................................................................... 9 
5 
Recruitment, Screening and Study Procedures .......................................................... 9 
5.1 
Study visits 1 (inclusion) ............................................................................................... 9 
5.2 
Study duration ............................................................................................................ 10 
5.3  
Safety reporting .......................................................................................................... 10 
6 
Data analysis plan .................................................................................................. 10 
6.1 
Data analysis ............................................................................................................... 10 
7 
Ethics ..................................................................................................................... 11 
7.1 
Informed consent ....................................................................................................... 11 
7.2 
Anonymity/confidentiality ......................................................................................... 11 
7.3 
Ethical approval .......................................................................................................... 12 
7.4 
Reward for participants .............................................................................................. 12 
7.5 
Stakeholder consultation ........................................................................................... 12 
8 
Data Protection ..................................................................................................... 12 
9 
Finance and Funding .............................................................................................. 12 
9.1 
Study sponsor ............................................................................................................. 12 
10 
Dissemination ........................................................................................................ 13 
11 
Study Personnel ..................................................................................................... 13 
11.1 
Steering Committee Members ................................................................................... 13 
11.2 
Study Lead Investigators ............................................................................................ 14 
12 
References ............................................................................................................. 15 
Appendix 1: Interview Guide .................................................. Error! Bookmark not defined. 
Appendix 2: Haemnet Lone Worker Policy ...................................................................... 17 
Appendix 3: Safety Reporting Definitions ........................................................................ 18 
Appendix 4:  Roche Adverse Event and Special Situation Reporting Form ........................ 19 
 
 
 

 
Emi and Me Study Protocol V4 
 
 
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1 
Background 
1.1 
Disease Introduction 
Haemophilia A is a rare congenital disorder caused by an inherited genetic defect of the X 
chromosome that results in a deficiency in factor VIII production. Factor VIII plays a pivotal 
role in the coagulation cascade, bringing together the clotting factors IXa and X, a critical 
step in the formation of a blood clot to help stop bleeding. Haemophilia A results in impaired 
clot formation and can lead to uncontrolled and often spontaneous bleeding. It affects 
approximately one in every 5,000 males [Srivastava et al, 2013]. Different types of severity 
are recognised:  
• 
“severe” (factor activity is less than 1%) 
• 
“moderate” (factor activity is 1-5%  
• 
“mild” (factor activity is 6-25%). 
In its severe form, haemophilia results in recurrent joint and muscle bleeds that predispose 
to arthropathy, muscle contracture and disability. Treatment of affected individuals in the 
UK is with FVIII prophylaxis, which decreases spontaneous bleeding events and resultant 
joint damage [Richards et al, 2010]. Prophylaxis requires frequent intravenous infusions, 
which can be as often as daily but are usually 2-3 times per week. 
A serious complication of treatment is the development of inhibitors to infused factor VIII 
replacement therapies. Inhibitors are antibodies developed by the body’s immune system 
that bind to and block the efficacy of replacement factor VIII, making it difficult, if not 
impossible to obtain a level of factor VIII sufficient to control bleeding. Up to 40% of patients 
receiving replacement factor develop inhibitors [Iorio et al, 2017]. 
Of these patients, who are predominantly children, between 50 and 86% can be tolerised 
through extended periods of very high treatment [Holstein et al, 2016]. Unfortunately, for a 
small number of these children, this treatment fails leaving a cohort of young 
children/adolescents who join a group of adults who never had access to this treatment 
[Khair et al, 2018]. For this ever-growing cohort of individuals treatment is with bypassing 
agent therapy either when bleed occur (on-demand or episodic therapy) or as prophylaxis 
[Dekoven et al, 2013]. 
Treatment of inhibitors is burdensome for the patient and his family [Lindvall et al, 2014; 
DeKoven et al, 2014] as it requires repeated intravenous infusions. Furthermore, treatment 
does not always alleviate bleeding, which can result in haemophiliac arthropathy, reduced 
mobility and pain and frequent episodes of hospital delivered care [Berntorp et al, 2011]. 
Emicizumab is a bispecific factor IXa- and factor X-directed humanised monoclonal antibody 
that has been co-developed by Chugai, Roche and Genentech. It has been designed to mimic 
the activity of factor VIII bring together factor IXa and factor X, proteins that are required to 
activate the natural coagulation cascade and restore the blood clotting process for 
haemophilia A patients. Emicizumab is a prophylactic (preventative) treatment that can be 
administered by an injection of a ready-to-use solution under the skin (subcutaneously) once 
weekly or potentially once monthly following initial weekly ‘loading’ doses [Shima et al, 
2016]. In a pivotal phase III study, which compared emicizumab with on-demand (no 
prophylaxis) and prophylaxis use of bypassing agents (BPAs) in adults and adolescents with 
haemophilia A with inhibitors, the primary endpoint showed a clinically meaningful and 
statistically significant reduction in treated bleeds of 87% (risk rate [RR]=0.13, p<0.0001) 
with emicizumab prophylaxis [Oldenburg et al, 2017]. Furthermore, over a median 
observation time of 31 weeks, substantially more patients experienced zero bleeds with 
emicizumab prophylaxis. 

 
Emi and Me Study Protocol V4 
 
 
6
Consequently, patient expectation of emicizumab is significant. On haemophilia-specific 
social media websites, many patients have expressed optimism for a treatment likely to 
result in reduced treatment burden and fewer bleeds, with a consequent impact on mobility 
and pain. Treatment burden also includes the time commitment required for treatment, 
which is reported to impact on treatment adherence [Thornburg, 2018]. The frequency of 
infusion of current therapy is often cited as influencing adherence to treatment; decreased 
frequency of infusions and sub-cutaneous administration may encourage adherence and 
better treatment outcomes [Hacker et al, 2001]. 
 
1.2 
Rationale for the study 
Emicizumab offers patients protection from bleeding with fewer injections, this should 
reduce treatment burden. While patient expectation is high (as evidenced by the 
conversations on UK haemophilia social media sites) there are risks that patients may forget 
to treat and thus experience bleeds. During clinical trials participants have been monitored 
closely and, through questionnaire completion, have described improvement in quality of 
life [Mancuso et al, 2018, Oldenburg et al 2019]. 
What is also apparent to clinical teams is the dramatic change to life experience of not just 
the patient but his family – the ability to travel, to plan attending events knowing that 
bleeds will not occur, even potentially to have another child because of the reduction in 
treatment burden, particularly the time needed for treatment and rehabilitation. 
This therapy represents a substantial shift in the entire life experience of living with and 
managing haemophilia with inhibitors. As such, there is a need to look beyond the 
quantitative data collected in clinical trials and to assess the real impact of therapy on the 
everyday lives of patients and their families, gathered using qualitative research techniques. 
Emicizumab has been available in the UK in clinical trials and as part of an Early Access to 
Medicines Scheme (EAMS) for non-trial eligible patients since spring 2018. It was recently 
licensed in the UK and is now available for routine clinical care for any person with 
haemophilia A and an inhibitor.  
We hypothesise that patients and their carers/families will be excited about this new 
treatment option and that for most, if not all, the promise of improved care and quality of 
life will be a reality. 
2 
Aim and Objectives  
2.1 
Aim  
This study aims to examine the real-life experience and impact of using emicizumab in a 
cohort of patients with haemophilia and inhibitors, who are being prescribed emicizumab in 
routine clinical care or as part of the EAMS process as well as those who have been in the 
clinical trials. We also intend to capture the impact of emicizumab use on the lives of close 
family members (parents/carers/children/siblings). Each participant and his family members 
will be deemed a study ‘dyad’. 
2.2 
Primary Objective 
To capture the real-life experience of using emicizumab for haemophilia inhibitor therapy 
for the patient with haemophilia and his close family members.  

 
Emi and Me Study Protocol V4 
 
 
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2.3 
Secondary Objectives 
• 
To describe difference patient satisfaction with injections (numbers of injections, 
how to remember treatment dates etc). 
• 
To understand user’s expectations of emicizumab and how they see future 
haemophilia care (less frequent injections, impact on home storage, number of 
bleeds, reduced hospitalisations etc). 
• 
To describe the as yet ‘unseen’ impact on the extended family – and to seek their 
views of innovative therapy for haemophilia.  
3 
Study design  
3.1 
General Design  
This is a prospective, observational cohort qualitative research study to be conducted 
among patients using emicizumab in routine clinical practice.  
The study is designed to allow English-speaking patients and their families to tell their own 
life stories through narrative accounts. The narratives represent a true sharing of 
experiences valued by the tellers, listeners and gatherers [Hardy et al, 2009], and therefore 
offer insight into how these patients and families cope with haemophilia. 
Most experienced practitioners familiar with haemophilia have preconceived ideas and 
theories that may potentially bias data capture. Grounded theory is a research methodology 
that allows identification and description of the broad spectrum of life with haemophilia 
from the perspectives of the patient and their families. This methodology enables rich data 
capture across a large age range and allows for deep consideration of data, allowing 
research questions to be re-shaped as evolving themes and new concepts emerge. 
The study will form part of a PhD by published works undertaken by the PI and supervised by 
Dr Kate Khair.  
3.2 
Primary Endpoints   
To gather a deep and thorough understanding about how switching from factor based 
products to emicizumab for routine treatment in “real-world” settings meets the needs and 
changes the perspectives of the patient with haemophilia and his immediate family 
members. 
3.3 
Secondary endpoints  
• 
To gather a deeper understanding about how emicizumab is used in real life, how 
patients remember to treat, and what impact this has had on their ability to engage 
fully in daily activities. 
• 
To describe patients’ views about treatment patterns; given days vs. tailored 
individualised treatment (once weekly to once monthly injections) 
• 
To understand differences and experiences between participant sub-groups 
(children/siblings, carers, adult and child patients). 
• 
To develop a greater understanding of where emicizumab fits in the future 
treatment landscape, which might include use in patients with haemophilia without 
inhibitors. 

 
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3.4 
Statistical analysis and sample size calculation  
This is a qualitative study using established qualitative research methodologies. Statistical 
evaluation is not appropriate. 
We will start with an interview guide developed by the lead researcher and a patient co-
researcher, and tested on three pilot participants (one child, one teenager, one adult).  
All interviews will be recorded digitally so that the researcher can pay full attention to the 
subject, without the need to write down verbatim comments [Balen et al, 2000]. Following 
each interview, the researcher will also make notes of any thoughts, reflections or 
observations that arose during the interview. 
After each of the interviews, the sound files will be transcribed verbatim; the lead 
investigators will then analyse the transcripts using a grounded theory approach. Direct 
quotes are analysed into topics, and topics are coded into themes to identify recurring 
themes seen as important aspects of life with haemophilia. Each interview is coded as it 
occurs, following analysis the interview guide is then amended so that emerging themes 
may be further investigated in later interviews [Charmaz 2006]. 
This continues until no new data are described (data saturation). This is recognised as being 
a very effective way to gain deep insightful and rich data from study participants [Bryant and 
Charmaz, 2010; Charmaz, 2013; Charmaz, 1990, Sim et al, 2018]. The study team have used 
this methodology in previous haemophilia research studies [Khair et al, 2013; Khair et al 
2019 (HOPE study, in press)]. 
Inferential testing will be used to describe how outcomes differ between groups (e.g. 
patients vs. parents). Correlation between groups may be achievable with explanatory 
factors (e.g. age, treatment regimen, bleeds, joint health etc.) 
4 
Study Group   
The study population will comprise people with haemophilia A, of any age or severity, in the 
UK who have an inhibitor, and their family members. According to the UKHCDO annual 
report for April 2016 to March 2017, there are 217 people with an inhibitor listed on the 
National Haemophilia Database (UKHCDO, 2017). 
We estimate that in the UK, around 20 patients have been treated with emicizumab in 
clinical trials (principally in the major London centres). Around 40 people received therapy 
with emicizumab as part of the Early Access to Medicines Scheme (EAMS) for non-clinical 
trial eligible patients. Another small cohort of people have received emicizumab since it was 
licensed in the UK in May 2018.  
From this sub-population of <100 people, we would hope to recruit around 25 to the study, 
together with at least one family member. 
Prescribed doses and frequency of treatment will be decided and prescribed by the patient’s 
clinical team. No patient will switch to emicizumab to gain entry to this study.  
4.1 
Inclusion Criteria  
• 
People with haemophilia A and an inhibitor aged >8 yrs who are prescribed 
emicizumab therapy in routine clinical practice. 
• 
Parents and siblings of children aged <16 years with haemophilia A who are 
prescribed emicizumab. 

 
Emi and Me Study Protocol V4 
 
 
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• 
Partners/carers/siblings/children of adults with haemophilia A who are prescribed 
emicizumab will become patient ‘dyads’ 
• 
Those who have given written consent to be in the study. 
4.2 
Exclusion Criteria 
• 
Patients prescribed treatment other than emicizumab for their haemophilia 
• 
Non-English speakers 
• 
Those who do not consent to be in the study. 
5 
Recruitment, Screening and Study Procedures  
The study will be advertised via Haemnet to nurses in haemophilia centres where patients 
who are being prescribed emicizumab will be seen for direct clinical care. Once identified 
potential participants will be given information about the study by their clinical team and 
will be invited to participate. If they agree to participate they will be contacted by the 
research team to organize a mutually convenient time for their interview which could be 
undertaken at hospital, home or other mutually convenient site. The Haemnet lone worker 
policy (appendix 2) will be followed to ensure interviewer safety. 
5.1 
Study visits 1 (inclusion) 
There will be a single study visit, at which all study data will be collected. This is summarised 
in the panel. 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
Patient 
Parent/carer 
Document informed 
consent 
•Demographics: age 
•Haemophilia history: age at diagnosis, 
age when prophylaxis started, joint 
procedures including replacements, 
fusion and synovectomies  
•Review other medical conditions, 
including HIV, hepatitis C etc.  
•Current treatment characteristics  
•Recent treatment history:  product / 
dose / frequency of infusion; doses 
missed and impact on bleeds 
•Review emicizumab history: when 
started, dose, number of injections per 
treatment 
•Discuss impact on bleeds, how it feels 
to administer drugs using subcutaneous 
route 
•Review patient’s reported joint health 
(recent bleeds, target joints, surgical 
procedures) 
•Demographics:  relationship to patient 
treated with emicizumab; age; gender; do 
they have an inhibitor? Do they carry the 
haemophilia gene 
 
• Other long-term conditions? 
 
Proceed to 
interview 

 
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5.2 
Study duration  
The study will last for 6 months, during which time each dyad will be interviewed once only. 
 
5.3  
Safety reporting 
 
Emicizumab is a newly licensed therapeutic option for people with haemophilia as such safety 
data is still being collected.  
Although adverse event information is not being actively solicited via this protocol, 
physician/consumers are reminded to report any adverse reactions (for which they suspect a 
causal role of a medicinal product) (see Appendix 3) that come to their attention either to the 
marketing authorization holder of the suspected medicinal product, via the Roche Adverse 
Event and Special Situation Reporting Form to be supplied to the physician (for Roche 
medicinal products) (see Appendix 4), or to the concerned competent authorities via the 
national spontaneous reporting system. 
In addition, the following should also be reported if occurring during exposure to a marketed 
medicinal product, even in the absence of adverse events: 
• 
Pregnancy 
• 
Breastfeeding 
• 
Abnormal laboratory findings 
• 
Overdose, abuse, misuse, off-label use, medication error or occupational exposure 
• 
Reports of lack of efficacy 
• 
Product quality defects and falsified medicinal products 
• 
Data related to a suspected transmission of an infectious agent via a medicinal 
product 
• 
Drug interactions (including drug/drug, drug/food, drug/device and drug/alcohol) 
When a subject is not exposed to a marketed medicinal product, but the physician/consumer 
becomes aware of the potential for a medication error, or an intercepted medication error, 
this should also be reported. 
If during one of the interviews a participant mentions an adverse event, or any of the special 
situations described above, the interview will continue without interruption. Once the 
interview is complete, an appropriate adverse event report will be prepared and submitted. 
 
6 
Data analysis plan  
6.1 
Data analysis 
This study will be analysed using grounded theory. Grounded theory involves gathering rich 
data, using a variety of methods, including interviews, ethnography and textual analysis 
identifying themes, coding and analysing these finally developing theories about the data 
that has emerged [Charmaz, 2006]. Grounded theory has been used extensively in research 
with children and families [Neill, 2007]. 
Taped Interviews: 

 
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• 
The interviews will be transcribed and anonymised during the transcription process. 
• 
Once the interviews are transcribed, the text will be read and re-read whilst 
listening to the recordings to ensure correct transcription. Following this review 
process and analysis the tapes will be destroyed and the transcripts will become the 
source data 
• 
The interviews will be coded into themes by the whole research team, the themes 
will then be analysed further using a transformational framework, identifying over-
arching themes or concepts, summarising and synthesising the data and using 
descriptive analysis to represent the findings/dyads views [Spencer et al, 2003]. 
• 
A table of the themes will be produced 
• 
These themes will characterise recurring ideas and thoughts from the participants 
and will form the basis for further analysis and publications. 
• 
Individual quotes may be used – these will be anonymised, this will be in the 
information sheet(s) and consent. 
Field notes: 
• 
Immediately after the interview, the researcher will record any thoughts, reflections 
or observations made during the interview. In particular any thoughts/ideas about 
emerging themes will be recorded.  
• 
These notes will be analysed as part of the framework analysis [Ritchie et al, 2003]. 
7 
Ethics  
There is minimal risk to participants or researchers from this study. Participants will be 
invited to one interview to discuss their hopes, fears, expectations and the realities of the 
new treatment.  
In the event that any patient or family member becomes distressed by this they will be 
referred (with consent) to the psychology services affiliated with the haemophilia centres 
from which they have been recruited.  
National guidance on interviewing children [MRC, 2004; RCPCH, 2000] will be followed. 
7.1 
Informed consent 
Study participants will be required to consent to be in the study; their consent can be 
withdrawn at any stage and will not have any impact upon their haemophilia care; as such 
consent will be reaffirmed before the interviews take place. Informed consent will be 
required from the adult participants and parents of all child participants.  
Age appropriate information will be used to gain assent from child participants, this will 
either be in a written format, for those old enough to comprehend the written assent form, 
or verbal from those who are pre-literate. [Gibson and Twycross, 2007]. If a child says ‘no’ 
this will be taken as non-assent and they will not be requested to participate again. 
7.2 
Anonymity/confidentiality 
The use of interviews raises issues of confidentiality especially when direct quotes and/or 
the circumstances of quotes may be used in reports and publications. It is therefore 
imperative that individuals are anonymised. This will be achieved by the individual reports 
and quotes using study numbers which are known only to the researcher. 

 
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7.3 
Ethical approval 
The study will be registered with the research and development office at Oxford University 
Hospitals NHS Foundation Trust. 
Ethical approval will be sought from the Health Research Authority using the standard IRAS 
application forms.  
This study will be registered with the research and development department at GOS.  
7.4 
Reward for participants 
At the end of each interview, participants will be given a £50 voucher as a ‘thank you’ 
present for participation (maximum £100 per family).  This will be included in the participant 
information sheets.  
7.5 
Stakeholder consultation  
This Steering Committee developing this project will include patient representation: our 
patient representative will also assist in recruiting study participants and conducting the 
patient interviews. All appropriate Disclosure and Barring Service (DBS) checks will be 
undertaken. The primary patient interview schedule will be derived from three current users 
of emicizumab (one child, one teenager, one adult) and their family members 
8 
Data Protection  
Participants in the Emi and Me study will be anonymised and will be known by study number 
only and managed in line with the EU General Data Protection Regulation (GDPR) (successor 
to the UK Data Protection Act 1998). 
• 
All audio-recordings will be transcribed verbatim by a professional transcriptionist 
unknown to the study participants. The transcriptionist will have signed a 
confidentiality agreement. 
• 
All data (paper records and audio recordings) will be kept in locked cupboards by 
Haemnet for the duration of the study.  
• 
Recordings will be deleted by Haemnet once the study has been analysed. 
• 
All personal identifiable data will be kept for 12 months after the study. After this 
period they will be shredded.  
• 
All anonymised data, including transcripts of interviews will be kept for 15 years 
after the study. After this period they will be shredded. 
• 
Any data on computers will be password protected in line with NHS data protection 
procedures.  
Haemnet will act as sponsor to the study and will monitor data quality and undertake site 
audits as necessary.  
9 
Finance and Funding 
Funding for the study is being sought under the Roche/Chugai investigator initiated research 
programme. All study funds will be managed by Haemnet. 
9.1 
Study sponsor 
The study will be sponsored by and indemnified by Haemnet.  

 
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10 
Dissemination 
We will submit abstracts to the 2020/2021 national and international haemophilia 
conferences, such as EAHAD, WFH, and ISTH. 
In addition, we will submit the study results for publication in peer-reviewed journals serving 
medical/nursing/allied health professionals who work with people with haemophilia. 
We will also disseminate our results directly to study participants through a final report. We 
will also publicise the results via the UK Haemophilia Society member newsletters. 
All investigators will contribute to study publications and will be named as co-authors. 
Authorship will be confirmed in line with journal publication guidance such as ICMJE.   
11 
Study Personnel 
11.1 Steering Committee Members 
The following people have contributed to the preparation of the study design and protocol: 
Dr Kate Khair is Clinical Academic Careers Fellow, The Centre for Outcomes and Experience 
Research in Childhood Health, Illness and Disability, Great Ormond Street Hospital for 
Children, London, and Visiting Professor of Health and Social Care London South Bank 
University. Formerly Nurse Consultant in Haemophilia at GOSH, she has extensive 
knowledge in qualitative research with children and young people with haemophilia. She will 
oversee the study including ethical approval, study staff training, participant enrolment, 
study completion including evaluation of all outcome measures used and ensure timely 
results presentation and publication.  
Simon Fletcher: is a clinical trials/research nurse in the haemophilia centre at The Churchill 
Hospital in Oxford. He is interested in the impact of new therapies on patients with bleeding 
disorders and is undertaking a PhD by published works; this study will form part of this body 
of work.  He has research expertise including assent/consent, data analysis and 
presentation/publication skills. He also has clinical expertise in the care of adults with 
haemophilia and inhibitors.  
Luke Pembroke is Communications Officer and “patient researcher” with Haemnet. Luke has 
experience working within the medical education sector of the healthcare communications 
industry. Living with haemophilia himself, Luke is an active patient advocate, and has 
worked extensively with a network of patient groups within the UK, Europe and globally. 
Luke writes educational materials for HCPs and patients, as well as creative blogs, social 
media posts, and clinical papers. 
Haemnet is a registered charity (No 1152241) that supports health and social care 
professionals to ensure that excellent care becomes an everyday experience for people with 
bleeding disorders. The charity provides education, undertakes research and drives service 
innovation. Haemnet has previously managed and successfully delivered the Pfizer 
EUROASPIRE-funded SO-FIT study, as well studies and projects funded by Novo Nordisk (the 
SO-HEROIC study), the Roald Dahl Marvellous Children’s Charity (the SO-LIFE study), and the 
Burdett Trust for Nursing (the Transforming Transition programme).  
Haemnet is currently engaged in a multi-methods study assessing the prevalence and impact 
of chronic pain in PWH. Previous research studies and publications have also included: 

 
Emi and Me Study Protocol V4 
 
 
14
• 
Khair K, Pollard D, Harrison C, Hook S, O’Driscoll M, Holland M. HOw Patients view 
Extended half-life products: impressions from real world experience (The HOPE 
study). In press. 
• 
Khair K, Holland M. The Kids' immune thrombocytopenia Tool is not suitable for 
assessing quality of life in children with platelet function disorders. Haemophilia 
2018;24(4):e259-e261. doi: 10.1111/hae.  
• 
Khair K, Klukowska A, Myrin Westesson L, Kavakli K, Escuriola C, Uitslager N, Santoro 
C, Holland M, von Mackensen S. The burden of bleeds and other clinical 
determinants on caregivers of children with haemophilia (the BBC Study). 
Haemophilia 2019 March 29. doi: 10.1111/hae.13736.  
• 
von Mackensen S, Myrin Westesson L, Kavakli K, Klukowska A, Escuriola C, Uitslager 
N, Santoro C, Holland M, Khair K. The impact of psychosocial determinants on 
caregivers' burden of children with haemophilia (results of the BBC study). 
Haemophilia 2019 Apr 11. doi: 10.1111/hae.13684. 
• 
Khair K, Holland M, Bladen M, Griffioen A, McLaughlin P, von Mackensen S; SO-FIT 
Study Group. Study of physical function in adolescents with haemophilia: The SO-FIT 
study. Haemophilia 2017;23(6):918-925. doi: 10.1111/hae.13323. 
• 
Khair K, Holland M, Pollard D. The experience of girls and young women with 
inherited bleeding disorders. Haemophilia 2013;19(5):e276-81. doi: 
10.1111/hae.12155. 
• 
Forrester C, Bielby H, Johns S, Efford J, Holland M, Khair K; United Kingdom 
Haemophilia Nurse's Association. Potential for development of haemophilia link 
nurse role within UK hospitals. Haemophilia 2013;19(4):578-82. doi: 
10.1111/hae.12144. 
• 
Khair K, Holland M, Vidler V, Loran C, Harrington C. Why don't haemophilia nurses 
do research? Haemophilia 2012;18(4):540-3. doi: 10.1111/j.1365-
2516.2012.02749.x. 
• 
Khair K, Holland M, Carrington S. Social networking for adolescents with severe 
haemophilia. Haemophilia 2012;18(3):e290-6. doi: 10.1111/j.1365-
2516.2011.02689.x. 
Haemnet also runs a core education course (Contemporary Care of People with Bleeding 
Disorders), a nurse leadership programme (ASPIRE), and hosts a European nurses network 
facilitating development of haemophilia practice across Europe. 
 
11.2 Study Lead Investigators 
Simon Fletcher  
Research nurse in the haemophilia centre at The Churchill Hospital in Oxford. 
Simon.fletcher@ouh.nhs.uk 
 
Dr Kate Khair 
Clinical Academic Careers Fellow, The Centre for Outcomes and Experience Research in 
Childhood Health, Illness and Disability, Great Ormond Street Hospital for Children, London 
kate@haemnet.com 
 
 

 
Emi and Me Study Protocol V4 
 
 
15
12 
References 
Balen R, Holdroyd C, Mountain G, et al. Giving children a voice: methodological and practical 
implications of research involving children. Paediatric Nursing 2000; 12 (10) 24-8. 
Berntorp E. Importance of rapid bleeding control in haemophilia complicated by inhibitors. 
Haemophilia 2011 Jan;17(1):11-6.  
Bryant B, Charmaz K. (Eds) The Sage Handbook of Grounded Theory: Paperback Edition 
(Sage Handbooks) Feb 2010. 
Charmaz K. Constructing Grounded Theory (Introducing Qualitative Methods series) Nov 
2013. 
Charmaz K. 'Discovering' chronic illness: using grounded theory. Soc Sci Med. 
1990;30(11):1161-72. 
Charmaz K. Constructing grounded theory: a practical guide through qualitative analysis. 
Sage Publishing 2006. 
Dekoven M, Wisniewski T, Petrilla A, Holot N, Lee WC, Cooper DL, von Mackensen S. Health-
related quality of life in haemophilia patients with inhibitors and their caregivers. 
Haemophilia. 2013 Mar;19(2):287-93. 
DeKoven M, Karkare S, Lee WC, Kelley LA, Cooper DL, Pham H, et al. Impact of haemophilia 
with inhibitors on caregiver burden in the United States. Haemophilia . 2014 Nov;20(6):822-
30. 
Gibson F, Twycross A. Children’s participation in research. Paediatric nursing 2007; 19: 14-17 
Hacker MR, Geraghty S, Manco-Johnson M. Barriers to compliance with prophylaxis therapy 
in haemophilia. Haemophilia. 2001;7(4):392-6. 
Hardy S, Gregory S, Ramjeet J. An exploration of intent for narrative methods of inquiry. 
Nurse Researcher 2009; 16 (4) 7-19. 
Iorio A, Fischer K, Makris M. Large scale studies assessing anti-factor VIII antibody 
development in previously untreated haemophilia A: what has been learned, what to believe 
and how to learn more. Br J Haematol. 2017 Jul;178(1):20-31. 
Khair K, Chaplin S, Holland M. Living with an inhibitor: Results from the Study of Haemophilia 
Experiences, Results and Opportunities in Children and young adults with long-standing 
inhibitors (the SO-HEROIC study). J Haem Pract 2018; 5:24-24. DOI: 
https://doi.org/10.17225/jhp00109. 
Khair K, Collier C, Meerabeau L, Gibson F. Multi-methodology research with boys with severe 
haemophilia. Nurse Res. 2013 Jul;20(6):40-4. 
Khair K, Pollard D, Harrison C, Hook S, O’Driscoll M, Holland M. HOw Patients view 
Extended half-life products: impressions from real world experience (The HOPE 
study) in press (Haemophilia 2019) 
Lindvall K, von Mackensen S, Elmståhl S, Khair K, Stain AM, Ljung R, Berntorp E. Increased 
burden on caregivers of having a child with haemophilia complicated by inhibitors. Pediatr 
Blood Cancer. 2014 Apr;61(4):706-11. 
Mancuso ME, Mahlangu J, Sidoni RF, Trask PC, von Mackensen S, Shima M, Uguen M, Chang 
T, Young G, Oldenburg J. Emicizumab prophylaxis in paediatric persons with Haemophilia A 
(PWHA) with inhibitors: impact on health related outcomes and caregiver burden in the 
HAVEN 2 study J Haemophilia 2018 (S1) p28 DOI 10.111/hae.13389. 

 
Emi and Me Study Protocol V4 
 
 
16
Medical Research Council (2004) MRC Ethics Guide. Medical research involving children. 
London: Medical Research Council. Available at: 
www.mrc.ac.uk/Utilities/Documentrecord/index.htm?d=MRC002430. 
Neill S. Grounded theory sampling: ‘whole’ family research’. Journal of Research in Nursing 
2007; 12; 435-443. 
Oldenburg J, Mahlangu JN, Kim B, et al. Emicizumab prophylaxis in hemophilia A with 
inhibitors. N Engl J Med 2017;377(9):809-818. 
Oldenburg J, Mahlangu JN, Bujan W et al. The effect of emicizumab prophylaxis on health-
related outcomes in persons with haemophilia A with inhibitors: HAVEN 1 Study. 
Haemophilia 2019;25(1):33-44 doi:10.1111.hae13618 epub 14 Nov 2018 
Royal College of Paediatrics and Child Health Guidelines for the ethical conduct of medical 
research involving children. Archives of disease in childhood; 2000 (2); 177-182. 
Richards M, Williams M, Chalmers E, Liesner R, Collins P, Vidler V, et al. A United Kingdom 
Haemophilia Centre Doctors’ Organization guideline approved by the British Committee for 
Standards in Haematology: guideline on the use of prophylactic factor VIII concentrate in 
children and adults with severe haemophilia A. British Journal of Haematology. 
2010;149(4):498-507. 
Ritchie J, Spencer l, O’Connor W. Carrying out qualitative analysis in Qualitative Research 
Practice (eds) 2003 Ritchie J, Lewis J. Sage publishing.  
Shima M, Hanabusa H, Taki M, Matsushita T, Sato T, Fukutake K, et al. Factor VIII-Mimetic 
Function of Humanized Bispecific Antibody in Hemophilia A. N Engl J Med. 2016 May 
26;374(21):2044-53. doi: 10.1056/NEJMoa1511769.  
Sim J, Saunders B, Waterfield J, Kingstone T. Can sample size in qualitative research be 
determined a priori? Int J Soc Res Methodology 2018 DOI: 
https://doi.org/10.1080/13645579.2018.1454643. 
Spencer L, Ritchie J, O’Connor W. Analysis: practices, principles and processes. Qualitative 
Research Practice (eds) 2003 Ritchie J, Lewis J. Sage publishing.  
Srivastava A, Brewer AK, Mauser-Bunschoten EP, Key NS, Kitchen S, Llinas A, Ludlam CA, 
Mahlangu JN, Mulder K, Poon MC, Street A; Treatment Guidelines Working Group on Behalf 
of The World Federation Of Hemophilia. Guidelines for the management of hemophilia. 
Haemophilia 2013; 19(1): e1-47. 
Thornburg CD. Physicians' perceptions of adherence to prophylactic clotting factor infusions. 
Haemophilia. 2008;14(1):25-9. 
United Kingdom Haemophilia Centre Doctors’ Organisation. UKHCDO Annual Report 2017 
includingBleeding Disorder Statistics for 2016/2017. UKHCDO, 2017. 
 
 
 

 
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Appendix 1: Haemnet Lone Worker Policy  
 
Haemnet staff will frequently work by themselves without close or direct supervision. 
Such work is usually home-office based. However, there may be occasions when 
Haemnet staff will need to work alone, visiting and interviewing service users, or 
running meetings with previously unknown colleagues, whether in or out of usual 
office hours. 
The Haemnet Lone Worker policy aims to ensure the health, safety and welfare of all 
staff who are required to work in such circumstances. The health and safety of lone 
workers depends on good risk assessment practices, effective communication, and 
shared responsibility. 
When working as a lone worker on Haemnet-related work, lone workers must: 
• 
ensure that someone else knows the whereabouts of the lone workers and 
what they are doing; 
• 
ensure they do not take unnecessary risks; 
• 
care for their own health and safety and that of others who may be affected by 
their acts or omissions; 
• 
seek and follow advice from their manager; 
• 
follow all health and safety policies; 
• 
comply with requests for information on whereabouts from managers; 
• 
report any incidents including threats and potentially dangerous situations; 
• 
ensure that the Team Administrator is aware of any changes to their contact details.  
 
 
 

 
Emi and Me Study Protocol V4 
 
 
18
Appendix 2: Safety Reporting Definitions 
 
Adverse Events (AE) 
According to the International Conference of Harmonisation (ICH), an AE is any untoward 
medical occurrence in a patient or clinical investigation patient administered a pharmaceutical 
product, regardless of causal attribution. An AE can therefore be any of the following: 
• 
Any unfavorable and unintended sign, symptom, or disease temporally associated with 
the use of a medicinal product, whether or not considered related to the medicinal 
product 
• 
Any reports of new disease or exacerbation of an existing disease (a worsening in the 
character, frequency, or severity of a known condition).  
Recurrence of an intermittent medical condition (e.g., headache) not present at baseline. 
 
Assessment of Causality of Adverse Events 
For patients receiving combination therapy, causality will be assessed individually for each of 
the medicinal products. 
The PI will use her knowledge of the patient, the circumstances surrounding the event, and 
an evaluation of any potential alternative causes to determine whether or not an AE is 
considered to be related to a medicinal product. The following guidance should be taken 
into consideration: 
• 
Temporal relationship of event onset to the initiation of study medicine 
• 
Course of the event, considering especially the effects of dose reduction, 
discontinuation of study medicine, or reintroduction of study medicine (when 
applicable) 
• 
Known association of the event with the study medicine or with similar treatments 
• 
Known association of the event with the disease under study 
• 
Presence of risk factors in the patient or use of concomitant medications known to 
increase the occurrence of the event 
• 
Presence of non-treatment-related factors that are known to be associated with the 
occurrence of the event.  
 
 

 
Emi and Me Study Protocol V4 
 
 
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Appendix 3:  Roche Adverse Event and Special Situation 
Reporting Form 
 
Instructions: 
This form is to be used for reporting Adverse Events (AEs) and Special Situations originating from a spontaneous source, a Non-
Interventional Study (NIS), a Market Research and Patient Support Program (MAP), a Pre-Approval Access (PAA) / Compassionate Use 
(CU) Program and a Post-Trial Access Program (PTAP). 
 
AEs and Special Situations will be collectively referred to as "adverse events" hereafter. 
For AEs and Special Situations originating from:  
• Spontaneous sources: Omit section A, B,C and D and start to complete the form with Section 1 – Reporter Details. 
• NIS: Complete section A first in addition to the other sections. 
• MAP: Complete section B first in addition to the other sections. 
• PAA/CU Program: Complete section C first in addition to the other sections. 
• PTAP: Complete section D first in addition to the other sections. 
The four essential elements for AE/Special Situations reporting are marked with * . 
Once completed, forward the form to your Roche Local Safety Unit (LSU) or to Roche Safety Operations as applicable. 
For dates, spell out the first three letters of the month, DD/MMM/YYYY, e.g., 07/APR/2015. 
Note: The format/wording of this form may be modified by the Roche LSU as needed as long as the requirements on essential data 
that needs to be collected is not omitted. 
A. Non-Interventional Study (NIS) REPORTS ONLY 
NIS Protocol Number: 
      
Site Number: 
      
Patient Number: 
      
Did the patient receive a studied medicinal product as per the NIS protocol?  
 Yes      
 No 
Are any of the adverse events reported on this form exempted from collection as per the NIS protocol?  
 Yes      
 No 
B. Market Research and Patient Support Program (MAP) REPORTS ONLY 
Note: Even if there is no identifiable patient, complete as many details as possible. 
MAP Identifier: 
      
Project 
Title: 
      
MAP Service Provider: 
      
Respondent ID:        
Address: 
      
E-mail Address: 
      
Telephone 
Number: 
      
Country: 
      
Fax Number: 
      
C. Pre-Approval Access (PAA)/Compassionate Use (CU) Program REPORTS ONLY 
Program Number: 
      
Patient Identifier: 
      
D. Post-Trial Access Program (PTAP) REPORTS ONLY 
Program Number: 
      
Patient Identifier: 
      
1. REPORTER DETAILS *Note: If available please provide reporter’s occupation. If data privacy allows please provide name, address and/or phone 
number. 
Reporter First Name: 
      
Occupation: 
Reporter Surname: 
      
 Physician (specify speciality):  
Address: 
      
      
 Pharmacist 

 
Emi and Me Study Protocol V4 
 
 
20
Postal/Zip Code: 
      
 Nurse 
Country: 
      
 Consumer/Patient 
E-mail Address: 
      
 Legal 
Telephone Number: 
      
 Company Representative 
Fax Number: 
      
 Other (specify):       
Has the Regulatory Authority been notified of this report? 
 Yes 
 No 
 Unknown 
2. PERMISSION TO CONTACT HEALTHCARE PROFESSIONAL (HCP) 
If the reporter is a consumer/patient, permission to contact HCP 
regarding adverse event(s)?   
 Yes   
 No 
HCP Contact Details:       
 
 
3. PATIENT DETAILS *  
Note: If data privacy allows please provide at least one descriptor. Please ensure the patient’s age or age group is captured wherever possible.  
Name/Initials: 
      
Weight: 
       
kg   
lb 
Gender: 
 Male  
 Female 
 Unknown 
Height: 
       
cm  
inch 
Date of Birth: 
or age at time of 
event: 
      
       
 Year(s)  
 Month(s)  
 
Day(s)  
Ethnic Origin: 
 Asian      
 Black      
 Caucasian     
 
Hispanic      
 Other (specify):       
4. SUSPECT PRODUCT * - If more than 4, continue in Additional Relevant Information, Section 8. 
Product Name 
(report brand name if 
available) 
Indication/ 
condition  
for which the 
product has been 
prescribed 
Dose and 
Unit 
Route 
Frequency 
Start Date 
 
Stop Date 
(or ongoing) 
Batch/ 
Lot Number 
A 
      
      
      
      
      
      
      
      
Was the suspect product discontinued due to the adverse event?  
 Yes 
 
No     
If so, did the patient’s condition resolve/improve? 
 Yes 
 No 
 Unknown 
Was the suspect product reintroduced?  
 Yes 
 No    
If so, did the event recur? 
 Yes 
 No 
 Unknown 
B 
      
      
      
      
      
      
      
      
Was the suspect product discontinued due to the adverse event?  
 Yes 
 
No     
If so, did the patient’s condition resolve/improve? 
 Yes 
 No 
 Unknown 
Was the suspect product reintroduced?  
 Yes 
 No    
If so, did the event recur? 
 Yes 
 No 
 Unknown 
C 
      
      
      
      
      
      
      
      
Was the suspect product discontinued due to the adverse event?  
 Yes 
 
No     
If so, did the patient’s condition resolve/improve? 
 Yes 
 No 
 Unknown 
Was the suspect product reintroduced?  
 Yes 
 No    
If so, did the event recur? 
 Yes 
 No 
 Unknown 
D 
      
      
      
      
      
      
      
      
Was the suspect product discontinued due to the adverse event?  
 Yes 
 No     
If so, did the patient’s condition resolve/improve? 
 Yes 
 No 
 
Unknown 
Was the suspect product reintroduced?  
 Yes 
 No     
If so, did the event recur? 
 Yes 
 No 
 Unknown 
5. ADVERSE EVENT(S)/SPECIAL SITUATION(S) * - If required, continue in Additional Relevant Information, Section 8. 
Adverse Event (AE) 
(list primary first) 
Onset 
Date 
Outcome 
(enter number as per 
the key below) 
Resolved/ 
Improved 
Date 
Seriousne
ss 
Causality 
Y=Yes, N=No, U=Unknown, NP=Not 
Provided (specify all suspect products that may 
have caused the adverse event). If causality is 

 
Emi and Me Study Protocol V4 
 
 
21
If outcome is 
unknown, use key 
6 and add further 
details in Section 
8 
(enter one or 
more numbers 
as per the key 
below) 
unknown, specify “unknown” and add further 
details in Section 8 
Suspect Product 
A 
B 
C 
D 
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
Key for outcomes: 
1. Fatal,     2. Not Recovered/Not Resolved,     3. Recovered/Resolved,     4. Recovered/Resolved with sequelae,     5. 
Recovering/Resolving,          6. Unknown 
Key for seriousness:  
1. Death (if yes, provide date):               2. Life-Threatening (use only if patient was at immediate risk of death due to adverse event)        3. 
Initial/Prolonged Hospital Admission        4.Congenital Anomaly/Birth Defect        5. Persistent or Significant Disability        6. Medically 
Significant (important medical event that may jeopardize the patient and may require medical/surgical intervention to prevent the other outcomes)        
7. Non-serious Adverse Events of Special Interest (AESI) as per NIS protocol         8. Non-serious 
6. CONCOMITANT MEDICATIONS 
Also include herbal, homeopathic medications and supplements as well as OTC products. If more than 6, continue in Additional Relevant Information, 
Section 8. 
Product Name 
(report brand name if 
available) 
Indication 
Dose and Unit 
Route 
Frequency 
Start Date 
 
Stop Date 
(or ongoing) 
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
      
7. TEST(S) PERFORMED TO EVALUATE ADVERSE EVENT(S)  
E.g., baseline results prior to product. If required, continue in Additional Relevant Information, Section 8. 
Test 
Date of Test 
 
Test Result  
(include units if applicable) 
Reference 
Range 
Result Pending? 
      
      
      
      
 
      
      
      
      
 
      
      
      
      
 
      
      
      
      
 
      
      
      
      
 
      
      
      
      
 
8. ADDITIONAL RELEVANT INFORMATION  
 
Provide a description of the adverse event(s), severity, concurrent conditions and relevant medical history (including start and end date if applicable), 
clinical course, causality (if unknown), treatment for adverse event(s) and outcome.  
      
 
 

 
Emi and Me Study Protocol V4 
 
 
22
 
 
 
 
 
 
 
 
 
 
9. MEDICATION ERROR INFORMATION 
Please complete for all reports of intercepted or confirmed medication error. If any other information is considered of relevance, please provide it in 
section 8. 
 
A. 
Brief description of the medication error: 
      
B. 
Stage where the error first occurred: 
 
 Storage 
 Prescribing 
 Dispensing 
 Preparation for administration 
 Administration 
 Drug monitoring 
 
C. 
Was the patient exposed to the error? 
 Yes 
 No 
If the answer is Yes, continue to section C1. If the answer is No, continue to sections C2 and C3. 
C1. Did the error have any clinical consequences? 
 Yes 
 No 
 
If the answer is yes, please provide the numbers of the associated adverse events (as per section 5) below: 
 
C2. Stage where the error was intercepted: 
 
 Storage 
 Prescribing 
 Dispensing 
 Preparation for administration 
 Administration 
 Drug monitoring 
 
C3. Please describe the potential for harm that might have occurred if the error had reached the patient: 
      
D. 
Setting(s) where the error occurred (list more than one if applicable, e.g. pharmacy, hospital, private home…): 
      
E. 
Contributing factors and root causes: 
      
F. 
Mitigating factors that prevented or moderated the progression of the error towards harming the patient: 
      
G. 
Corrective and/or preventative actions taken in response to the error: 
      
ROCHE USE ONLY OR VENDORS ACTING ON BEHALF OF ROCHE 
LRN:                                    AER:                               Local Received Date:                                Company Received Date:  
       
Report Type 
 Initial     
  Follow-Up  
 Spontaneous 
 Literature     
 Other (specify):       
NIS Protocol Number:               MAP ID:               PAA/CU Program 
Number:       
PTAP Program Number:                
Ancillary Documentation?  
  Yes    
  No 
Supplementary Form attached?  
  Yes    
  
No 

 
Emi and Me Study Protocol V4 
 
 
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