What Rare Disease Day Means to Trinity Partners

Rare Disease Day falls on Wednesday, February 28th. At Trinity, we work closely with many life sciences companies developing treatments for rare disease patients. Research is crucial to the rare community, and we are committed to help raise awareness around the importance of medical representation for individuals with rare diseases and their families. Keep an eye on our social media channels this week for some rare disease-focused updates and read on to learn what Rare Disease Day means to Trinity employees:

“I hope that, one day, rare diseases have as many treatment options and cures as any other disease. Rare diseases deserve to receive attention and be understood like any other condition.” Rachel Halperin, Senior Associate Consultant

“From helping companies develop business cases for rare inherited disorders, to my personal life where I function as a caretaker for a loved one with a rare autonomic/hypermobility disorder, I deal with the challenges presented by rare diseases on a daily basis. I hope that the pharmaceutical ecosystem continues to evolve so that the business cases for these agents become more attractive, spurring innovation in underserved patient populations.”  Consultant

“Rare Disease Day is a reminder of the millions of patients and families affected by rare diseases. It’s also a reminder of how the treatment of rare disease is catalyzing innovations that will revolutionize treatment of rare and non-rare diseases.” Herman Sanchez, Partner

“[Rare Disease Day means] that no one gets left behind. Just because it is a rare disease doesn’t mean we don’t care. I hope more companies will invest in rare diseases and find help for patients who have been overlooked all this time.” Nandini Hadker, Principal

“Rare Disease Day means raising worldwide awareness of rare conditions to help improve the ability to identify and diagnose patients and increase research and development to find effective treatments.  I’ve personally watched my brother struggle for years to find a cause for his symptoms and, now diagnosed with a rare disease, he continues to suffer due to lack of treatments. I hope for him and all others with rare diseases that research continues and the great progress over recent years continues.” Maria Gumina, PhD, Engagement Manager

“Rare Disease Day means an opportunity to recognize the tireless work and strength of communities across the world who fight for recognition and awareness of their conditions that consume their lives. A good friend of mine has Phenylketonuria (PKU), and I’ve seen her overcome obstacles in her everyday life that most people never face. In researching rare diseases as a focus of my work, I’ve learned to be so much more grateful and feel so much more blessed to have two happy, healthy children.” Krista Perry, Principal

Experts from Trinity Partners and Janssen Publish Article in Dermatology and Therapy

A team of experts from Trinity Partners and Janssen have published an article in Dermatology and Therapy. The paper, titled, “Dermatologist and Patient Preferences in Choosing Treatments for Moderate to Severe Psoriasis,” describes the results of a study to determine dermatologist and patient preferences in choosing treatments for moderate to severe psoriasis. The study concluded that comprehensive education regarding treatment attributes may help minimize the influence of subjective biases and facilitate appropriate and patient-centered treatment selection in psoriasis.

The paper was authored by Gordon Lau, Gretchen R. Chiu, Nandini Hadker, Aparna Deshpande, Stephen Fleming, and Nicola Vance, all of Trinity Partners, along with Seina Lee and Steve Fakharzadeh of Janssen Scientific Affairs. At the time of the study, Matthew Alcusky was a postdoctoral fellow with Thomas Jefferson University and Janssen Scientific Affairs.

To view the full text of the article, please visit http://bit.ly/2hJqe9k

Trinity Partners Launches New Whitepaper to Assess Value in Ultra-Orphan Markets

Trinity Partners, a leading global life sciences consulting firm, today announced, at the NORD Rare Disease and Orphan Products Breakthrough Summit in Washington, D.C., a new whitepaper titled ‘Assessing Value in Ultra-Orphan Markets’.

In the last five to 10 years, the concentration on orphan development has exploded. From acquisitions to creation of separate rare disease units, pharma companies of all sizes have an increased focus on the orphan market. Yet, while rare diseases have the attention of the industry and the incentives continue to be strong, finding success in the orphan market is not easy. This new whitepaper outlines the crucial clinical and commercial attributes that drive success for ultra-orphan, non-oncology products.

Key insights include:

    • Potential products (to acquire or for portfolio planning) must first be assessed on several different commercial and clinical criteria specific to ultra-orphan markets.
    • Success in the ultra-orphan market does not follow one formula. Soliris (Alexion) and KUVAN (BioMarin) are two assets that score high with regard to clinical and commercial opportunity, yet followed very different paths to success.
    • The complexities surrounding success in the ultra-orphan market will continue to grow as competition rises alongside increased payer and pricing sensitivity.

“There are more than 7,000 rare diseases with only 500 approved treatments, which is a clear indicator of a large unmet need and opportunity for success in the orphan market space,” said Herman Sanchez, Partner, Trinity Partners, and lead author of the report. “However, being aware that there is a large market opportunity and truly realizing that opportunity are very different things. Our whitepaper outlines key insights and findings based on our deep commercialization experience in the market, providing companies with a proven roadmap to recognize the critical metrics that determine success and the efforts needed to achieve them.”

Trinity Partners, headquartered in Waltham, MA, with additional offices in San Francisco, New York City and Princeton, New Jersey, has extensive experience in orphan and ultra-orphan markets globally including NA, EUMEA, LATAM and APAC. The firm has more than 150 employees and has worked with six of the top 12 global pharmaceutical companies and 12 of the top 20 specialty and biopharmaceutical companies.

“The industry is driving to more targeted therapies, including new and exciting gene therapies, and many of them could become success stories with the right considerations. We’ve worked with many companies in the U.S. and around the globe on their commercialization strategies and are excited to share key learnings in our new whitepaper,” added Neal Dunn, Partner, Trinity Partners and co-author of the whitepaper.

NORD’s Rare Diseases and Orphan Products Breakthrough Summit is the largest and most meaningful multi-stakeholder event of its kind – featuring more than 20 speakers from the FDA, participation from over 80 patient organizations and the Pharma/Biotech industry’s foremost experts in orphan product innovation, investment and commercialization.

To read the full whitepaper and access all key insights, click here.

Trinity Client Akcea Therapeutics Publishes Interim Results from Pivotal IN-FOCUS Research Study

Trinity Partners, a leading global life sciences consulting firm, announced that their client, Akcea Therapeutics, a wholly owned subsidiary of Ionis Pharmaceuticals, Inc., has published results from one of the first-ever research studies measuring the considerable burden of patients with familial chylomicronemia syndrome (FCS) and the impact the disease has on their activities of daily living and employment. The results, from an interim analysis of the largest survey in patients with FCS, were published in Expert Review of Cardiovascular Therapy. 

FCS, also known as lipoprotein lipase deficiency (LPLD) or type I hyperlipoproteinemia, is a rare genetic disease characterized by extremely high levels of triglycerides and increased risk of other health complications, including pancreatitis. IN-FOCUS, a comprehensive web-based research survey of patients with FCS, was designed to capture the associated burden of illness and impact on quality of life. Sixty patients from the United States diagnosed with FCS participated; patients described multiple symptoms spanning across physical, emotional and cognitive domains.

An analysis of the survey data found that patients with FCS saw five physicians on average with FCS-related symptoms before receiving a proper diagnosis. Nearly all respondents reported frequent, debilitating symptoms leading to reduced quality of life. Respondents also indicated that FCS significantly influenced their career choice and employment status, and resulted in substantial work loss.

Learn more about the study results: http://bit.ly/2oNlzbE

Read Endocrine Today’s article on the findings: http://bit.ly/2oaDxDE

Commentary from Trinity’s Ryan Million and Jillian Godfrey Scaife Featured in November/December PharmaVOICE Year in Preview

Trinity’s Ryan Million and Jillian Godfrey Scaife discuss the future of precision medicine and gene editing, respectively, in the November/December PharmaVOICE Year in Preview issue. View Ryan’s insight on precision medicine: http://bit.ly/2fZOamA. Read Jillian’s comments on gene therapy: http://bit.ly/2gaV0ZA.

Trinity Partners Co-Founder and President John Corcoran Featured on Bloomberg Radio

Trinity Co-Founder and President John Corcoran discussed Trinity’s 2016 Drug Index and 20th anniversary on the Bloomberg Baystate Business Hour with Anne Mostue and Tom Moroney. Listen to the full program here: http://bloom.bg/2gzr59P. John’s segment begins at 7 minutes, 20 seconds.