Orphan Reach (a contract research organization that focuses on rare disease research) was acquired by CRO Emmes last year. Thomas Ogorka, managing director at Orphan Reach, spoke with Outsourcing-Pharma about the rare disease center’s work, as well as the current state and future of rare disease drug development.
OSP: How has the R&D field’s awareness and understanding of rare diseases evolved over time?
TO: There was always a need to develop new therapies for rare diseases given the fact that there are more than 7,000 diseases for most of which there are no or only insufficient treatments available. With increased regulations and rising costs to develop new products for more common diseases, the once so lucrative blockbuster era came to an end.
For especially smaller but extremely innovative companies it was time to focus on diseases in much smaller patient populations. At the same time diagnoses of rare diseases have vastly improved – although still far away from what we would all like to see – due to progress in genome sequencing.
The incentives offered as part of orphan drug legislation in the USA, Europe, and Japan were attractive enough to kick-start a sharp incline in orphan drug designations followed by clinical trials and consecutive marketing authorizations in this area. Patient groups have successfully supported these initiatives and the “patient´s voice” is louder than ever before.
OSP: Could you share some perspective on how the needs of a rare-disease patient might differ from a patient suffering from a less-uncommon condition?
TO: To be affected by a rare disease very often means that patients think they are alone. If you realize that the average time to receive a correct diagnosis for a rare disease is six years, patients and their families will already have experienced a lot of stress and fear that even the best doctors won´t be able to help.
On the other hand, with only a few patients in the same situation, it is difficult to obtain and exchange information with other patients on how to cope with the situation and where to find the best support.
Clinical trials deliver hope. However, depending on how much a patient´s mobility is affected and how old they are, it may be a big challenge to travel to a study site. This is for example the case if the site is far away from home with only a few specialized doctors knowledgeable in the respective disease. In these cases, families can support, but they also need to be part of the logistical considerations.
Today, we try to reduce the patient´s burden during clinical trials as much as possible by providing travel support as well as home nursing, thereby reducing the need for site visits and the resulting travel.
OSP: COVID-19 had a significant impact on research, in particular the patient-participants. Could you share any ways in which COVID-19 specifically affected rare disease patient-participants?
TO: Travel restrictions during the pandemic have hit all of us, but particularly patients who needed to travel to hospital sites for their scheduled study visits. At the same time in many countries including the UK, governments decided to prioritize the development of COVID treatments which has pushed back site activities in other areas including rare disease trials, and led to delays if not cancellations of clinical trials.
Not being able to receive an investigational drug on time can have a negative impact on the patient´s health and trial data, therefore endangering the outcome of the trial results. In this uncertain time, biopharmaceutical and CRO companies have done their utmost to deal with these challenges introducing measures like remote data monitoring, home nursing, and virtual visits.
OSP: Then, could you please tell us a bit about Orphan Reach—who you are, what you do, locations, key capabilities, and then any achievements you’ve made in recent history you’d like to brag about?
TO: I don´t really like to brag about our achievements so much; however, I am extremely proud of what we have achieved in the past 20 years. Orphan Reach tries to fill the gap between the traditional global CRO, mostly focused on managing larger trials in common diseases, and the needs of companies that develop rare disease treatments for only a few patients.
We have been operating on a global scale in more than 70 countries pretty much from the beginning, and with Emmes onboard we now have a robust presence in North America and can demonstrate over 40 years of experience in Data Management and Biostatistics. We literally build our Sponsor´s studies around each patient in a 360-degree approach and will consider all complexities to make sure the rare disease trial will be conducted in the fastest possible way.
OSP: Last year, you were acquired by Emmes—could you please tell us about how that merger has impacted/benefited your work?
TO: With Emmes we have met an ideal partner to support rare diseases. We have come together because we believe that we can make a huge difference to the outcome of clinical trials in rare diseases, joining forces in a very complimentary way. Emmes´ statistical capabilities alone will bring great advantages to our clients as for example the expertise to design powerful adaptive trials allows rare disease trials to be fit for purpose.
A well-established presence in the US and Canada will allow us to be close to where many of our current clients are located. Data Management teams in the US, Europe, and Asia will now be part of the Orphan Reach full-service delivery. Our rare disease center has been officially launched and we are working hard to make it the go-to destination for any company developing orphan drugs.
OSP: What are some of the reasons a sponsor might opt to partner with a CRO specializing in rare and orphan conditions?
TO: From a CRO perspective, it´s all about the process. Sponsors in the past have selected their CROs predominantly based on their therapeutic know-how. Although this is still the case today, our research indicates that especially for rare diseases, sponsors have learned that therapeutic experience is not the most important criterion when considering a CRO collaboration.
The process to manage a clinical trial in rare disease is complex and involves many additional stakeholders that would not be a part of a more common disease trial. This is all about the patient in the end, and everything we do should have a laser-sharp focus on the patient's needs. Therefore, a specialized niche provider that is global enough to find these rare patients wherever they are in the world, combined with a nimble structure that enables rare disease trials to move fast, is what sponsors really want.
OSP: Do you have any specific plans to commemorate Rare Disease Day on the 28th?
TO: Throughout Rare Disease Awareness Month we have been proactively educating our audiences with key information from across the rare disease community. Whether that be through sharing educational resources and infographics created by Rare Disease UK or NORD, or using our platform to highlight key stories from within the rare disease community to raise awareness in this way.
Internally we have encouraged staff to wear a striped piece of clothing to “show their stripes” in support of the lives of those impacted by a rare disease on the 28th. We have also been speaking with our team to understand their motivations for working within the rare disease space so that we can share insight from the people behind the work we do on Rare Disease Day.
