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At an ever-quickening pace, biopharmaceutical companies and patient advocacy groups are banding together to hasten development of new drugs for rare diseases.


Patient-Finding Approaches for Rare Disease | Blog

Mark Baglin, Vice President of Global Marketing at Alnylam Pharmaceuticals, works in ultra ultra rare diseases, like Hereditary ATTR Amyloidosis.


Opening a New Era in Rare Disease Medicines | In The Media

Jeanine O'Kane and Marie Emms, senior executives at Syneos Health, address rare disease pricing and the ties between manufacturers, payers and patient advocacy groups.


Harnessing Social Media to Reach Today’s Social Media-savvy Rare Disease Patient and Caregiver | In The Media

Marie Emms, Rowena Kam, and Michael Pearlman of Syneos Health discuss how social media can be used by patients with rare diseases and by those who recruit for rare disease clinical trials.


How payers and manufacturers can find common ground in rare diseases | Perspectives

Health insurers have raised challenging questions about how drugs for rare diseases are defined, regulated, and priced in the U.S. market.


Real World Value: Advancing Payer Understanding of RWE in Rare Disease | Perspectives

Drug developers and regulators are expanding the use of real world evidence to design, test and review rare disease treatments.


November 2019: Real World Value: Advancing Payer Understanding of RWE in Rare Disease | Newsletters

Drug developers and regulators are expanding the use of real world evidence to design, test and review rare disease treatments.


Why Payers Lag Drug Developers and Regulators in Embracing Real-World Evidence | Perspectives

Catch Meg Alexander, Head, Reputation & Risk Management Practice at Syneos Health, on RARECast - a podcast focused on the intersection of rare disease with business, science, and policy.


What Do You Get When You Combine Scientists and Patients? | Blog

This summer, students in California Institute for Regenerative Medicine’s Bridges to Stem Cell Research and Therapy program at California State University (CSU) San Marcos had new lab partners: families impacted by rare diseases.


Jazz Pharmaceuticals Creates New Education Center for Rare Cancer Patients | Blog

A new unbranded disease campaign Find the Right Fit launched by Jazz Pharmaceuticals seeks to support rare disease patients with myelodysplastic syndrome (MDS) and secondary acute myeloid leukemia (sAML) as they move throughout their diagnosis and treatment journey.


Wearable Devices Enter Clinical Trials | Blog

Huntington’s disease is a rare, fatal neurodegenerative disease that is desperate for treatment options and doctors are doing their best to help improve the quality of life for the more than 30,000 people living with the disease and over 200,000 at-risk of inheriting it.



Efforts to bring treatments to market for the approximately 7,000 rare diseases affecting more than 350 million people globally have been stepped up in recent years.


Orphan Drugs More Promising than Ever | Blog

The growing appeal of orphan drugs to manufacturers isn’t a groundbreaking story, but a new report identifies some head-spinning facts behind the focal shift from blockbuster to rare-disease drugs.


June 2018: Vision 2020 - The Orphan Drug Forecast | Newsletters

For some 30 million Americans living with one of 7,000 known rare diseases, there has never been a more hopeful era.


Patient Advocacy at the Center of Patient-Centered Design | Blog

Representatives from small and big pharma, and those fighting both rare diseases and more prevalent ones, came together in San Diego recently for WIB-Southern California: The Growing Importance of Patient Advocacy in BioPharma.



There’s no doubt the “pricing crisis,” has taken a heavy toll on the reputation of the biopharmaceutical industry.


Executive Summary - Vision 2020: The Orphan Drug Forecast | Perspectives

Market Landscape for Rare Diseases


Medicine Meets Minecraft | Blog

Pfizer brings both fun and learning to the hemophilia community through a Minecraft-based experience


Lost in Translation: Why Your Teams Don’t Execute Your Strategy | Blog

Live from eyeforpharma Philadelphia 2019— A dozen fresh soundbites from a panel of pharma executives exploring the gap between strategy and execution.


Orphan Medicines: Averting Price Debacles And Winning Payer Support | In The Media

In this collaborative piece, Meg Alexander, Managing Director of our Reputation & Risk Management practice and Susan Suponcic, Managing Director of our Pricing and Market Access practice, highlight misalignments in perception that derail access to orphan drugs.


Tap into HCPs on TikTok for Your Next Awareness Campaign | Blog

As a social media millennial at Syneos Health™, connecting the world of TikTok—one of the hottest social media channels today—and healthcare was a challenge I couldn’t pass up.



Since the fall of 2015, and against a backdrop of harsh media attention to pharmaceutical pricing, patient advocacy groups that form partnerships with pharma companies have received negative coverage in The New York Times, USA Today and other publications.


Pfizer, Novartis Align Against Liver Disease | Blog

Pfizer and Novartis have been independently developing therapies for nonalcoholic steatohepatitis (NASH), and they’re now set to join forces to yield a combination therapy for the fatty liver disease linked closely with metabolic disorders.


BIO International 2018 | Perspectives

We’ve pulled together for you all our best thinking from our Reputation & Risk Management team - comprised of healthcare communicators, policy-shapers and crisis response specialists.


New Outcomes-Based Payment Models Pave Way for Expensive Therapies | Blog

That figure represents the total cost to cover the 2,200 (approx.) patients with a rare form of blindness with Spark Therapeutics new one-time gene therapy, Luxturna.


The Week That Was: Could sunny days sweep the opioids away? | Blog

Missed the most recent analysis conducted by ICER?


LifeCapsule Advances Regenerative Medicine | Blog

Regenerative medicine is growing fast, and was a hot topic at this year’s SXSW event in Austin, TX.


What Biogen’s Alzheimer’s Drug Approval Means for Life Sciences Companies | Blog

In a highly anticipated decision, the Food and Drug Administration (FDA) last week cleared Alzheimer’s disease drug aducanumab on an accelerated, conditional approval program.


What Patient Advocacy Groups Want | Blog

New York, NY.


Trend Tracking: Dr Batman and AI Robin | Blog

One of our top 2017 Healthcare Trends, “Dr Batman and AI Robin” outlined the ways in which artificial intelligence is really starting to change the way diseases are assessed, diagnosed, and treated.