As part of our continued efforts to showcase the people behind the work we do within the rare disease space, we are delighted to share with you the thoughts of four integral members of our newly formed Rare Disease Center team.
With February being official Rare Disease Awareness Month, we asked the team a selection of question about the work they do to serve rare disease communities as part of Orphan Reach. Click below to read what each of them had to say!
Thomas Ogorka
Managing Director – Orphan Reach: Emmes’ Rare Disease Center
Q&A with Thomas Ogorka
"As long as our work contributes to improving the lives of patients and their families who have to live with a rare disease, I could not be more motivated to give everything we have. I have worked with an incredibly passionate team in the past 20 years mastering all sorts of challenges in the field of rare diseases, with the purpose always focused on working towards a world where all rare diseases have a treatment or cure."
Read here
1. What role do you play within Orphan Reach – Emmes’ rare disease Center?
How does your role contribute to the center and goals of OR/Emmes
As the Head of the Orphan Reach rare disease center I am responsible for harnessing the expertise of Emmes’ global workforce in critical areas so that rare disease clinical trials can be supported in the most effective way.
2. How does your work within Orphan Reach contribute to serving the rare disease community? Insight into the how the center enables developments within rare
Rare diseases are complex and require further planning and logistical considerations in addition to the requirements for trials in more common diseases. Identifying these additional needs and implementing them will help to improve the speed of trials and the probability of orphan drug marketing approvals. This in turn then benefits patients, with access to treatments hopefully becoming available more quickly.
3. Can you tell us how you came to work within rare diseases? Brief back story about your path into the field
As the founder of Orphan Reach Ltd. (acquired by Emmes in May 2021) I have seen too many orphan drug trials fail as often not enough attention is paid to small patient populations. This inspired me to create a CRO that focuses on these trials and eventually helps affected patients and their families to see urgently needed orphan products registered in the market.
4. What makes working within rare diseases different from other research areas in your opinion? Highlight key differentiating factors that set rare disease research apart from other areas.
As only a small number of patients are affected by rare diseases, patients and their individual needs become much more “visible”. On the one side we do need data to confirm safety and efficacy of a drug, but trials in rare diseases are so much more than just collecting data. Fully understanding the disease in the first place and designing a tailor-made trial almost around each patient is a good starting point to conduct a successful clinical study in rare.
5. What experience do you have in rare diseases? Give some examples of the work you have done within rare diseases – key highlights and achievements
I have worked with an incredibly passionate team in the past 20 years mastering all sorts of challenges in the field of rare diseases. Our expertise in more than 60 rare disease trials includes neurodegenerative diseases like DMD and Rett Syndrome as well as mitochondrial and rare endocrinological and haematological disorders.
6. What motivates you when working within this space?
Being part of an organization that can make a true difference to biopharmaceutical companies and – even more importantly – to patients´ lives in an area of vast unmet medical need.
As the Head of the Orphan Reach rare disease center I am responsible for harnessing the expertise of Emmes’ global workforce in critical areas so that rare disease clinical trials can be supported in the most effective way.
2. How does your work within Orphan Reach contribute to serving the rare disease community? Insight into the how the center enables developments within rare
Rare diseases are complex and require further planning and logistical considerations in addition to the requirements for trials in more common diseases. Identifying these additional needs and implementing them will help to improve the speed of trials and the probability of orphan drug marketing approvals. This in turn then benefits patients, with access to treatments hopefully becoming available more quickly.
3. Can you tell us how you came to work within rare diseases? Brief back story about your path into the field
As the founder of Orphan Reach Ltd. (acquired by Emmes in May 2021) I have seen too many orphan drug trials fail as often not enough attention is paid to small patient populations. This inspired me to create a CRO that focuses on these trials and eventually helps affected patients and their families to see urgently needed orphan products registered in the market.
4. What makes working within rare diseases different from other research areas in your opinion? Highlight key differentiating factors that set rare disease research apart from other areas.
As only a small number of patients are affected by rare diseases, patients and their individual needs become much more “visible”. On the one side we do need data to confirm safety and efficacy of a drug, but trials in rare diseases are so much more than just collecting data. Fully understanding the disease in the first place and designing a tailor-made trial almost around each patient is a good starting point to conduct a successful clinical study in rare.
5. What experience do you have in rare diseases? Give some examples of the work you have done within rare diseases – key highlights and achievements
I have worked with an incredibly passionate team in the past 20 years mastering all sorts of challenges in the field of rare diseases. Our expertise in more than 60 rare disease trials includes neurodegenerative diseases like DMD and Rett Syndrome as well as mitochondrial and rare endocrinological and haematological disorders.
6. What motivates you when working within this space?
Being part of an organization that can make a true difference to biopharmaceutical companies and – even more importantly – to patients´ lives in an area of vast unmet medical need.
Traci Clemons
Chief Research Officer
Q&A with Traci Clemons
"Although the diseases are rare the total number of people suffering collectively from rare diseases represents a significant number of the global population. I want to be a part of making potential treatments options available to people with rare diseases."
Read here
1. What role do you play within Orphan Reach – Emmes’ rare disease Center?
How does your role contribute to the center and goals of OR/Emmes
I provide scientific leadership to Orphan Reach. I review study information and provide feedback primarily on the study design and statistical analyses. I also support the strategic direction of the Center when needed, whilst serving on the Steering Committee to ensure we are doing everything we can to move rare disease research forward.
2. How does your work within Orphan Reach contribute to serving the rare disease community? Insight into the how the center enables developments within rare
I hope that I am supporting the business process and ultimately supporting the growth of the Center through the work we do and the patients we positively impact. In addition, the work we are doing with MedGenome to develop specific rare disease cohorts will provide additional opportunities to expand our rare disease portfolio and the patient communities we ultimately serve through our projects.
3. Can you tell us how you came to work within rare diseases? Brief back story about your path into the field
Rare disease research is one of the reasons I went into public health. I have Sickle Cell Trait which is not a disease in itself, but means that I inherited the sickle cell gene from one of my parents. I do not have any symptoms of SCD but did have to worry about passing SCD to my children. I have several family members who have suffered with debilitating complications of SCD. This background shaped my passion for rare disease research.
4. What makes working within rare diseases different from other research areas in your opinion? Highlight key differentiating factors that set rare disease research apart from other areas.
As a statistician the biggest difference is designing trials to deal with small sample sizes and heterogeneity of a specific population under study.
5. What experience do you have in rare diseases? Give some examples of the work you have done within rare diseases – key highlights and achievements
I am currently working on a Phase 1 gene therapy trial in Sickle Cell Disease. I also worked on a program for a potential treatment for Macular Telangiectasia (MacTel) type 2, which is an eye disorder affecting the macula that causes loss of central vision. We are currently on the home stretch and expect the Phase 3 study to end by Q3 of 2022.
6. What motivates you when working within this space?
Although the diseases are rare, the total number of people suffering collectively from rare diseases represents a significant number of the global population. I want to be a part of making potential treatment options available to people with rare diseases.
I provide scientific leadership to Orphan Reach. I review study information and provide feedback primarily on the study design and statistical analyses. I also support the strategic direction of the Center when needed, whilst serving on the Steering Committee to ensure we are doing everything we can to move rare disease research forward.
2. How does your work within Orphan Reach contribute to serving the rare disease community? Insight into the how the center enables developments within rare
I hope that I am supporting the business process and ultimately supporting the growth of the Center through the work we do and the patients we positively impact. In addition, the work we are doing with MedGenome to develop specific rare disease cohorts will provide additional opportunities to expand our rare disease portfolio and the patient communities we ultimately serve through our projects.
3. Can you tell us how you came to work within rare diseases? Brief back story about your path into the field
Rare disease research is one of the reasons I went into public health. I have Sickle Cell Trait which is not a disease in itself, but means that I inherited the sickle cell gene from one of my parents. I do not have any symptoms of SCD but did have to worry about passing SCD to my children. I have several family members who have suffered with debilitating complications of SCD. This background shaped my passion for rare disease research.
4. What makes working within rare diseases different from other research areas in your opinion? Highlight key differentiating factors that set rare disease research apart from other areas.
As a statistician the biggest difference is designing trials to deal with small sample sizes and heterogeneity of a specific population under study.
5. What experience do you have in rare diseases? Give some examples of the work you have done within rare diseases – key highlights and achievements
I am currently working on a Phase 1 gene therapy trial in Sickle Cell Disease. I also worked on a program for a potential treatment for Macular Telangiectasia (MacTel) type 2, which is an eye disorder affecting the macula that causes loss of central vision. We are currently on the home stretch and expect the Phase 3 study to end by Q3 of 2022.
6. What motivates you when working within this space?
Although the diseases are rare, the total number of people suffering collectively from rare diseases represents a significant number of the global population. I want to be a part of making potential treatment options available to people with rare diseases.
Eve Wallace
Projects Director
Q&A with Eve Wallace
"I have been working on rare disease clinical trials for the past 6 years. It is such a privilege to be part of a community and space that advances scientific and medical knowledge whilst placing the patient at the heart of every step of the process. What makes rare disease research unique is how truly patient centric it is, which I find makes it such a rewarding area to work in."
Read here
1. What role do you play within Orphan Reach – Emmes’ rare disease Center?
How does your role contribute to the center and goals of OR/Emmes
My role within Orphan Reach is as a hands-on Project Director. I have oversight across all the rare disease projects we are currently working on, whilst also taking an active role in a Project Management capacity in certain trials. I am extremely committed to delivering projects for our biotech clients and of course our patient groups.
2. How does your work within Orphan Reach contribute to serving the rare disease community? Insight into the how the center enables developments within rare
By harnessing our expertise in a way that positively impacts the clinical trial process, I hope we in turn positively impact the patients within the rare community as the trial outcomes will ideally result in treatment options for them in future that are currently not available.
3. Can you tell us how you came to work within rare diseases? Brief back story about your path into the field
I joined Orphan Reach as a Study Monitor, looking for a new challenge. Rare disease patients are truly at the heart of every project here which is why am delighted to have worked within this field for the past 6 years.
4. What makes working within rare diseases different from other research areas in your opinion? Highlight key differentiating factors that set rare disease research apart from other areas.
Rare disease studies are truly global, finding those patients affected by the disease that are eligible for your study can be challenging.
Working with multiple external stakeholders such as travel/ concierge vendors, site staff, training experts or central review services (e.g. ECG, Labs) to deliver projects. These factors are just examples of a few of the key stand out differences that come with working within this space.
5. What experience do you have in rare diseases? Give some examples of the work you have done within rare diseases – key highlights and achievements
I have spent the last 6 years working within rare disease and therapeutic areas with an unmet medical need, as a clinical trial monitor, project manager and now project director. I have delivered multiple global studies with different clients.
6. What motivates you when working within this space?
Providing patients with the opportunity to participate in clinical trials and advancing scientific knowledge.
My role within Orphan Reach is as a hands-on Project Director. I have oversight across all the rare disease projects we are currently working on, whilst also taking an active role in a Project Management capacity in certain trials. I am extremely committed to delivering projects for our biotech clients and of course our patient groups.
2. How does your work within Orphan Reach contribute to serving the rare disease community? Insight into the how the center enables developments within rare
By harnessing our expertise in a way that positively impacts the clinical trial process, I hope we in turn positively impact the patients within the rare community as the trial outcomes will ideally result in treatment options for them in future that are currently not available.
3. Can you tell us how you came to work within rare diseases? Brief back story about your path into the field
I joined Orphan Reach as a Study Monitor, looking for a new challenge. Rare disease patients are truly at the heart of every project here which is why am delighted to have worked within this field for the past 6 years.
4. What makes working within rare diseases different from other research areas in your opinion? Highlight key differentiating factors that set rare disease research apart from other areas.
5. What experience do you have in rare diseases? Give some examples of the work you have done within rare diseases – key highlights and achievements
I have spent the last 6 years working within rare disease and therapeutic areas with an unmet medical need, as a clinical trial monitor, project manager and now project director. I have delivered multiple global studies with different clients.
6. What motivates you when working within this space?
Providing patients with the opportunity to participate in clinical trials and advancing scientific knowledge.
Guisela Torres
Associate VP, Program Management Office
Q&A with Guisela Torres
"Knowing that the work you do could change someone’s life is truly a privilege. 50% of Rare Diseases affect children which in turn affects a family. That ‘truly privileged’ becomes a passion that makes me thankful each day that we are working towards a treatment or hopefully a cure that will impact these families’ lives."
Read here
1. What role do you play within Orphan Reach – Emmes’ rare disease Center?
How does your role contribute to the center and goals of OR/Emmes
My role allows me to contribute to the operational excellence of Orphan Reach, ensuring our clients receive the best service possible. I also work closely with our steering committee on our rare disease strategy, which enables us to chart a clear path to helping more patients through our work.
2. How does your work within Orphan Reach contribute to serving the rare disease community? Insight into the how the center enables developments within rare
Everything we do at Orphan Reach is aimed at ensuring our clients have the most efficient, effective route to progressing the development of their orphan product. This is turn we believe provides hope to countless families in the rare disease space as we actively try to advance research and available treatment options.
3. Can you tell us how you came to work within rare diseases? Brief back story about your path into the field
6 years ago I was asked to be part of a team that was establishing a Rare Disease and Paediatric Center of Excellence. It opened my eyes to another side of research where teams share a great passion for the work they do. I am now lucky enough to be part of the of Orphan Reach Emme’s Rare Disease Center and working with a great team who share the same desire and are committed to the work they do.
4. What makes working within rare diseases different from other research areas in your opinion? Highlight key differentiating factors that set rare disease research apart from other areas.
There are over 7000 rare diseases with a very small percentage of then having treatment much less cures. Since they affect small groups of patients they can be hard to find and therefore difficult to enrol. Identifying patient pathways is key and ensuring that trials are patient centric is crucial for retention.
5. What experience do you have in rare diseases? My experience as indicated has been working within organizations with therapeutic area leads and SMEs with expertise in various rare disease indications. Establishing Centres where we as a team are a resource for our internal teams as well as sponsors.
I have spent the last 6 years working within rare disease and therapeutic areas with an unmet medical need, as a clinical trial monitor, project manager and now project director. I have delivered multiple global studies with different clients.
6. What motivates you when working within this space?
It’s the children… most rare diseases affect children and that is something that tugs my heartstrings. Knowing that I may contribute to making a difference is everything.
My role allows me to contribute to the operational excellence of Orphan Reach, ensuring our clients receive the best service possible. I also work closely with our steering committee on our rare disease strategy, which enables us to chart a clear path to helping more patients through our work.
2. How does your work within Orphan Reach contribute to serving the rare disease community? Insight into the how the center enables developments within rare
Everything we do at Orphan Reach is aimed at ensuring our clients have the most efficient, effective route to progressing the development of their orphan product. This is turn we believe provides hope to countless families in the rare disease space as we actively try to advance research and available treatment options.
3. Can you tell us how you came to work within rare diseases? Brief back story about your path into the field
6 years ago I was asked to be part of a team that was establishing a Rare Disease and Paediatric Center of Excellence. It opened my eyes to another side of research where teams share a great passion for the work they do. I am now lucky enough to be part of the of Orphan Reach Emme’s Rare Disease Center and working with a great team who share the same desire and are committed to the work they do.
4. What makes working within rare diseases different from other research areas in your opinion? Highlight key differentiating factors that set rare disease research apart from other areas.
There are over 7000 rare diseases with a very small percentage of then having treatment much less cures. Since they affect small groups of patients they can be hard to find and therefore difficult to enrol. Identifying patient pathways is key and ensuring that trials are patient centric is crucial for retention.
5. What experience do you have in rare diseases? My experience as indicated has been working within organizations with therapeutic area leads and SMEs with expertise in various rare disease indications. Establishing Centres where we as a team are a resource for our internal teams as well as sponsors.
I have spent the last 6 years working within rare disease and therapeutic areas with an unmet medical need, as a clinical trial monitor, project manager and now project director. I have delivered multiple global studies with different clients.
6. What motivates you when working within this space?
It’s the children… most rare diseases affect children and that is something that tugs my heartstrings. Knowing that I may contribute to making a difference is everything.