Recruiting Rare and Oncological Patients? Top Pharma Turns to Inspire

Recruiting Rare and Oncological Patients? Top Pharma Turns to Inspire By Jeff Terkowitz Did you know that 80 percent of Inspire’s members agreed to receive information from Inspire about clinical trials? That’s about 1.6 million members with conditions in oncology, rare diseases, autoimmune disorders and chronic conditions who want to know if and when trials affecting their condition might be recruiting. Pfizer needed to find rare patients with genetic resilience to lung cancer. On Sept 23, the Breakthrough Staff at Pfizer wrote, Normally, discovering a small cohort or one or two families who meet such criteria might [...]

Launching a New Drug During a Pandemic? Successful Launches Included a Rapid Pivot to Social Media

Launching a New Drug During a Pandemic? Successful Launches Included a Rapid Pivot to Social Media By Paul Nash How has the pandemic affected pharmaceutical product launches? Experts say that social media and research have been the key factors for this year’s successful launches. In a webcast on the PharmaVoice Webcast Network, “The State of Product Launch During and Post COVID-19,” speakers Maya Desai, Director at Guidehouse; Lead Launch COE, Eduardo Schur, Partner and life sciences global practice lead, Guidehouse; and Justin Zamirowski, Chief Commercial Officer, Better Therapeutics, described key results from their studies into recent pharmaceutical [...]

How Has COVID-19 Impacted Patients with Rare Conditions?

How Has COVID-19 Impacted Patients with Rare Conditions? By Judy Chandler Over 600,000 Inspire members are affected by rare conditions. As a vital partner to 40 rare disease non-profit advocacy organizations, Inspire’s rare disease patient and caregiver members represent over 3,000 rare conditions. Serving as a common point across these diverse conditions, Inspire was uniquely positioned to learn how rare disease communities were being affected by COVID-19.   First, we noticed a surge of COVID-19 posts across all of Inspire through the month of March. Inspire’s research team began by reviewing some of these member communications to identify [...]

Patients Are The Best Resource For Defining Study Endpoints

Patients Are The Best Resource For Defining Study Endpoints By Robert Gardner In September, the World Orphan Drug Congress USA Fall Webinar series opened with a program called: “Understanding the Novel Challenges in Rare Disease Development: Perspectives From Experts.” They took an audience poll, asking attendees to identify their “biggest challenge in rare disease studies” from five choices, including competition for patients; lack of understanding of disease progression; development of appropriate endpoints; lack of data; and global regulatory pathways. Almost half the respondents -- 46% -- chose “development of appropriate endpoints” as the biggest challenge in rare [...]

Online Health Communities Change Patients’ Journeys: A Live Virtual Roundtable

Online Health Communities Change Patients’ Journeys: A Live Virtual Roundtable By Dana Deighton In the fable of the Ugly Duckling, the duckling feels isolated by his differences until he discovers that he is a swan too. Patients with undiagnosed conditions also feel isolated, with some even describing not being believed by their doctors or even their friends or families. One member with scleroderma shared her story: I spent 27 years seeking help --- every type of specialist, tests, hospitalizations, medications of every type, anything to put a name to my misery, not being treated like an attention-seeking patient. [...]

Next Generation Online Health Communities

Next Generation Online Health Communities: Data empowering patients and caregivers improves care and accelerates research By Richard Tsai The World Orphan Drug Congress USA 2020 (WODC 2020) provided four days of powerful panels and keynotes about collaborations, successes and failures in creating what rare disease patients and families need and desire most – effective treatments for their conditions. In one failure turned to success, Kim Stephens shared her story involving inappropriate clinical trial endpoints. Stephen’s son has Hunter Syndrome, an extremely rare inherited condition where children lack an essential enzyme in their cells that eliminates waste . [...]

Accelerating Rare Disease Research

Accelerating Rare Disease Research By Richard Tsai COVID-19 has challenged the non-profit arena’s fundraising arm, which is largely based on face-to-face events like galas, runs and walk-a-thons. This limit on the number and amount of donations has occurred just as rare disease patient organizations have reached a tipping point in their influence on government and industry. According to Malcolm Gladwell, the “tipping point” is “the moment of critical mass, the threshold, the boiling point,” where “ideas...spread like viruses do.” 1In this case, the tipping point refers to the efforts made by rare disease patient organizations to provide [...]

COVID-19 and a Future with Fundamental Design Shifts: Incorporating Virtual into Clinical Trials

COVID-19 and a Future with Fundamental Design Shifts: Incorporating Virtual into Clinical Trials By Kathleen Hoffman, PhD, MSPH On a recent Pharma Talk Radio podcast called, The Impact of COVID-19 on Clinical Trials: Where are We? And Where are We Going?, three subject matter experts discussed how they see COVID-19 impacting the future of clinical trial research. What needs to change?1 The experts were Ray Dorsey, MD, the David M. Levy Professor of Neurology and Director of the Center for Health & Technology at the University of Rochester; Matt Kibby, President and Principal of BBK Worldwide; and Craig [...]

Customer Loyalty and Service Insights for the Pharmaceutical Industry

Customer Loyalty and Service Insights for the Pharmaceutical Industry By Dana Deighton “Only nine percent of U.S. consumers believe pharmaceutical and biotechnology companies put patients over profits.” This sentiment, found in the 2016 Harris Poll of Reputation Equity and Risk Across the Health Care Sector, has stayed fairly consistent in Harris polls until recently.1 In this year’s April and May series of surveys around the COVID-19 pandemic, Harris asked the question, “How has your view of [the Pharmaceutical Industry] changed since the start of the coronavirus pandemic?” In both the polls, 40% of respondents had a more [...]

Patients Discuss and Debate the Medical Outcomes from Generic Formulations

Patients Discuss and Debate the Medical Outcomes from Generic Formulations By Jeff Terkowitz When mature products face the introduction of generics, patients are often considering, or asked to consider, switching their medications. At that transition, there is a high need for Real World Data documenting patient experiences. What happens to medical outcomes when patients switch medications? Where does a business get the Real World Evidence (RWE) about patient experience that is so needed during any transition from an exclusive formulation to the availability of competitive generics? A logical place to start is with the patient consumers. After [...]