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Home » Rare Diseases » Why Patient Voices Make All the Difference in Rare Diseases

Raymond Huml

Vice President of Medical & Scientific Strategy, Syneos Health

“When you embed the patient voice into programs, you’re going to not only save money, but you’re also going to make it better for the patient,” says Raymond Huml, VP of Medical Affairs at Syneos Health, a biopharmaceutical solutions organization. “It’s a win-win for both sides of the equation.”

Syneos Health is committed to working with key stakeholders to accelerate clinical research for rare diseases. Their Rare Disease Consortium, a collaborative and independent multifunctional team of experts, shares best practices in all aspects of development for new therapies for rare diseases.

In the past five years, the consortium has conducted 350 projects in rare diseases including real-world evidence projects like registries and non-interventional studies. A quarter of their research experience is in the pediatric population.


Huml, who has over 30 years in clinical and biopharmaceutical industries, knows rare diseases well — his daughter, 27, and his son, 22, both have a form of muscular dystrophy.

He brings both the professional and patient perspective to his work. For example, he understands how important it is to the rare disease community to maximize time. He and his team at Syneos developed “BAM,” a biopharmaceutical accelerator model, a trusted process for speeding up drug development. 

“We realized that in order to bring things to the patient, we have to have a sense of urgency,” says Huml. “I can bring that to the table because I have to live with it every day.”

He continues, “If we can save a patient a burden, if we can help identify an extra rare disease patient for a clinical trial, if we can enroll the trial faster, that means we can get these drugs to kids quicker and to the parents.”

Promising future

While it can be daunting to look at all of the 7,000 rare diseases individually, Huml takes a big-picture approach and looks for commonalities, including potential for gene therapies, similar pros and cons of going into trials and challenges for caregivers. 

He says the future is promising: “We need to focus on the positives and that there is potential for a cure.”

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