MAGI Moments: Panel Perspectives on Rare Disease Research

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MAGI Moments: Panel Perspectives on Rare Disease Research

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MAGI Panel Discussion – Rare Disease Clinical Research 

There are inherent challenges to the conduct of clinical trials and these are magnified in the rare disease space. At the recent MAGI-East (Model Agreements and Guidelines International) Conference held in Boston, Massachusetts, Joan Chambers, Vice President of Marketing and Strategy, ClinX, moderated a compelling panel that discussed technology’s rapidly evolving place in rare disease research and other relevant topics.

From left to right, Harsha Rajasimha, Joan Chambers, Scott Schliebner and Derek Ansel.

Panelists included:

  • Scott Schliebner, Senior Vice President, Center for Rare Diseases, PRA Health Sciences
  • Harsha Rajasimha, Founder and Board Director, Organization for Rare Diseases India, USA
  • Derek Ansel, Clinical Strategy Lead, PRA Health Sciences

Joan opened the session with some thought-provoking statistics:

“An estimated 350 million individuals suffer with a rare disease, worldwide, which includes about 10% of the population of the United States.”

Technology is developing at an incredible rate and it’s changing the face of clinical conduct across all areas. It’s assisting researchers to connect with rare disease patients more easily, but there are still significant challenges with enrollment, patient travel and participation.

“Ironically, I don’t think technology is the sole answer,” said Scott. “Think from the patient’s point of view.” A patient-centric focus looks at moving from a traditional clinical study model of site visits to a virtual approach in order to alleviate the burden of participation for patients. Technology now enables clinical researchers and patients to do many tasks, remotely. The rise of E-consent and telemedicine means that some visits can be eliminated and replaced with mobile based, wearable technologies that collect data points in the billions, in real time. Auto-reminders can prompt patients about scheduling and reporting.


“The intention is not to eliminate sites entirely – we’re just trying to make patient participation easier by removing barriers. After all, patients are the primary stakeholders and without them, we cannot have clinical trials,” observed Scott.

Another emerging technology that is impacting the conduct of rare disease clinical trials is blockchain, which facilitates the use of patient data wallets for healthcare management.

“We’re looking at a trend of empowered patients who have their health information at their fingertips,” said Harsha. Currently, patient data wallets are not seamless and can be challenging to navigate. But they can be a single source of access to all health data from disparate sources.

Blockchain is an immutable system of data ledgers that ensures trust, traceability and transparency among participating stakeholders including regulators, pharmaceutical companies and CROs. Patient data, shipments of medication and dosing records are all kept secure.

Derek conducted a Q & A session during his portion of the panel. One question from the audience that provoked discussion was “How much is too much when it comes to asking patients who participate in virtual trials to monitor wearables and other devices?”

“We can’t ask patients to input data everyday for the next 15 years,” said Derek. “You have to have a patient-centric focus.”

Sites need to understand that there can be pitfalls with vendor technology. Additionally, it’s beneficial for sponsors and sites to have an educational discussion about what technology works for which demographic. As society adapts, some technologies, such as tablets, are becoming universally used across generations.

Next, the conversation turned to patient preferences. “We may need to be flexible in a given study and meet people where they are,” commented Derek. For example, some patients may not want a nurse visiting them in their home and may wish to preserve a separation of their home life and their life as a participant in a clinical trial study. It’s vital to compare site and patient satisfaction to design studies that work for everyone involved. 

As the session’s time came to a close, other conversations touched upon the challenges of reaching rural populations and the overall lack of diversity in clinical studies. “There needs to be a democratization of clinical trials,” said Derek.

Clinical trials provide the opportunity for participants to receive life-changing treatments and access to cutting edge medical technology. Individuals diagnosed with more prevalent indications are presented with a far greater number of studies that may well serve their healthcare objectives than patients in the rare disease space. Education and strategic coordination of resources is key.

“In many instances, patients with rare diseases are not aware that there are avenues to treatment and it can be challenging for sponsors, CROs and sites to get the word out that they are conducting a clinical trial. Given the inherent tendency for rare disease enrollment to be of international proportions, there are many moving parts to navigate. The right combination of digital and traditional marketing and advertising, educational materials and community outreach drives enrollment and retention, making study completion possible,” explained Joan Chambers.


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Authored by Melissa Daley, Marketing Content Specialist, ClinEdge

Melissa Daley

Post by Melissa Daley -

Melissa develops social media and marketing content strategies and produces a variety of collateral with creative, effective messaging. Melissa has served as an educator in higher education for close to two decades.

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