6th Annual Patients as Partners US Conference – Collaboration in Action

by Joe Ternullo

Patients as Partners conferences are the only conferences in the US and Europe dedicated to demonstrating how to systematically involve patients throughout the entire drug development life cycle. They aim to improve efficiency in clinical research and speed lifesaving, quality-of-life-improving therapies to those in need. Dr. Femida Gwadry-Sridhar and I recently returned from 6th annual Patients as Partners US conference in Philadelphia in March.

Collaboration and Efficiency Lead to Faster Treatments

Collaboration and efficiency are at the center of Pulse’s mission. That’s why it was so important for Pulse to participate in both conferences – our colleague, Dr. Tamara Howard presented at Patients as Partners EU. Networking is critical in the medical field to stay on top of the latest research, talk to a wide range of people, and exchange new ideas. At Patients as Partners,  we connected with patients, advocates, industry representatives, academic researchers, government officials, and non-profit organizations.

Patients as Partners US enjoyed a capacity, standing-room-only crowd. The collegial and engaging esprit de corps among all participants was apparent. Listening to one solid speaker after another, whose stories stayed with me, helped clarify that something was different—there was a bigger picture that everyone was aligned and working  towards—real progress was afoot.

A Few Highlights from Patients as Partners US

Judy Perkin’s Immunotherapy Trial: A Cancer Veteran’s Remarkable Story

Who

  • Judy Perkins, Metastatic, Breast Cancer Veteran and Patient Advocate

What

Judy shared her remarkable 16-year journey battling breast cancer and the success that resulted upon enrolling in an immunotherapy clinical trial at the National Cancer Institute. Within six months of beginning immunotherapy, Judy had been cleared of all cancer. In fact, she’s the first person to be declared free of all metastatic cancer after a course of immunotherapy. Judy’s story was gripping, relevant and an inspirational patient engagement message. She is the first of many, many more to come.  The future is now.

Appraising Initiatives to Raise Public and Patient Awareness and Education

Who

  • Ken Getz, MBA, Director and Associate Professor of Tufts Center for the Study of Drug Development and Founder and Board Chair of the Center for Information and Study on Clinical Research Participation

What

Ken reviewed global initiatives to educate the public and patients and to engage them as partners in the clinical research process. Ken highlighted  15 years of progress and educational excellence in this informative and comprehensive keynote address that ran the gamut of a broad cross section of educational programs and services, media outreach, awareness campaigns, and plain language clinical trial result summaries. This remarkable resource—at once pointing the way forwarding, making things happen, and catalouging results—is so essential to the advancement of the patients-as-partners movement.

FDA Patient Engagement Synergistic Efforts

Who

  • Anindita Saha, Director of Clinical Expertise and Partnerships, FDA Center for Devices and Radiological Health
  • Michelle Tarver, MD, PhD, Director of Patient Science & Engagement, FDA Center for Devices and Radiological Health
  • Megan Moncur MS, Senior Advisor for the Science of Patient Input at the FDA Center for Biologics Evaluation and Research
  • Pujita Vaidya, Senior Advisor for Patient-Focused Drug Development Program and the FDA Office for the Center Director

What

This panel discussion focused on the FDA’s efforts to fully integrate the patient voice in all aspects of drug development. In summary, the FDA is systematically working to help ensure that patients’ experiences, perspectives, needs and priorities are captured and meaningfully incorporated into the development and evaluation of medical products throughout the medical product life cycle. These are committed and engaged people who are going to make it happen!

Do You Advocate for Patients? Work with Patients?

With its welcoming and committed community of interest and chock-full of high-value content, Patients as Partners is a must-attend event. Pulse Infoframe will return to Patients as Partners in 2020. Let’s not wait until then to talk. I will be at Clinical Research as a Care Option in Raleigh Durham in April. Femida and I will be at the Great Plains Rare Disease Conference in May. If you attend any of these events, look for us (or, better yet, email us). Let’s see how we can help you.

Joe Ternullo is the head of business development for Pulse Infoframe, a member of NQRN Advisory Committee and Co-Chair of the Society for Participatory Medicine

About Pulse Infoframe Inc.
Focusing on rare disease, cancer and patient reported outcomes, Pulse Infoframe, a new style health informatics company, delivers real-time analytics and visualization to improve outcomes, generate real-world evidence, and provide data insights and precision knowledge. Pulse aggregates data from multiple sources, making it available to relevant stakeholders and offering numerous benefits including dramatically improved efficiency, productivity, and collaboration. Pulse Infoframe is headquartered in Canada, with offices in the United States, and India.

More information is available at www.pulseinfoframe.com

Real-World Evidence and Personalized Medicine

For Julian Summers, it was a long time coming.  For his brother John it was too late: he had passed away a year ago, succumbing to Alstrom Syndrome. What could have been done to save him?

In December 2018 the FDA published a Framework for its Real-World Evidence (“RWE”) Program.  Among other matters, the Framework provides guidance to assist researchers interested in using real-world data (“RWD”) to develop RWE to support FDA regulatory decisions.  The Framework applies to both drugs and biologics products.  It defines RWD as: “data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources”; RWE is defined as “the clinical evidence about the usage and potential benefits or risks of a medical product derived from analysis of RWD”.  With personalized medicine, both John and Julian could have been diagnosed earlier. While there is currently no treatment for Alstrom Syndrome, many of its potentially fatal symptoms could have been managed better, if people knew the cause.

Alstrom Syndrome is typical of many rare diseases in that it often takes years for a patient to get a diagnosis and this delay allows the symptoms to proceed unchecked.  Sadly, like most rare diseases, there is not yet a treatment or cure. RWE provides not only a route to aid diagnosis, but provides information about the course of the disease that is useful for researchers and pharmaceutical companies. Properly collected and curated, RWE offers more information and insights around causes and treatments, even a cure, for rare medical conditions.  The FDA has taken a significant step with its announcement of a framework for RWE, enabling both clinical researchers and commercial entities to focus on the collection of this vital information. 

A key component of the Framework is the use by the FDA of RWE/RWD to support regulatory decisions about drug effectiveness, as opposed to its current use of RWD primarily toevaluate drug product safety.  In other words, the FDA is trying to shift the emphasis from “how safe is this drug?” to “how well does this drug work?”  To do so, the FDA also recognizes the necessity of developing common data standards to maximize the utility of RWD; merely because information is stored in a database does not mean that the data is meaningful. Sources of data such as patient registries need to be curated to ensure that the data is valid so that what is captured is a good source of RWD that can then be used to generate RWE.  As noted in the Introduction, the FDA has, in part, created the Framework to implement its mandate under the 21st Century Cures Act which was “designed to accelerate medical product development and bring new innovations and advances faster and more efficiently to the patients who need them.”  Pioneering efforts using RWE/RWD have already demonstrated the effectiveness of this approach.

The Framework’s themes of “real”, “reliable” and “relevant” can be achieved.  There are already numerous examples of well-curated RWD, providing RWE in several current and successful collaborative rare-disease research efforts.  These groups work together to accelerate medical product development and have brought new innovations and advances faster and more efficiently to patients who need them most: those who suffer from rare diseases, such as Castleman’s, LMGD2 and CDKL5 and melanoma, lung and breast cancers.

Traditionally, advances in the clinical study of disease etiology and treatment in these areas have been hampered by lack of collaboration, incentive misalignment and the selection of research studies based on an individual’s area of expertise and/or interest. These approaches did not tend to prioritize the research questions that need to be asked and answered to advance knowledge of a given disease and its treatment and/or cure.  Patient numbers are small so that global reach is required.  Key to addressing these challenges is the development of a patient registry that can manage data from diverse inputs, including patient-reported outcomes (“PROs”). These patient-reported data can support clinical trial recruitment, accelerate treatment outcomes for patients, provide information to payers to support their decision-making processes, inform clinicians who need to decide of a particular treatment will be effective for their patient and to pharmaceutical companies to identify the most promising areas of drug development.

It is apparent that a number of the rare disease and cancer research challenges are similar to those that must be overcome before we as a society can transition from a disease/population-based model of healthcare to a patient/person-based model (i.e. “personalized medicine”).  Advances in technology and genetics enable a personalized approach; the recognition that medical treatments designed for the “average patient” can be successful for some patients but not for others is a driver for change.  Moreover, not only may some treatments be ineffective, they may be harmful to certain groups of individuals.  RWE can proactively identify the characteristics of those patients who will benefit most from treatments, while experiencing the least adverse effects.

This blog series will identify and discuss common challenges in rare disease and cancer research, personalized medicine and the FDA’s RWE Framework to demonstrate how successful solutions implemented to address the former may be applied to achieve the present and future vision of “the right medicine to the right person, at the right does and at the right time”.[1] 

We begin with an in-depth discussion of the data.


[1] Canadian Institutes of Health Research, and Institute Affairs. “Personalized Medicine”. Canadian Institutes of Health Research, Government of Canada, March 24, 2017. http://www.cihr-irsc.gc.ca/e/43627.html

Rare Disease Week on Capitol Hill

by Joe Ternullo

I just returned from Rare Disease Week on Capitol Hill, a spectacular and unique event that Pulse Infoframe was privileged to support. Over 500 patient advocates trekked to Washington, D.C., last week for this extraordinary event that has grown steadily since the inaugural annual event in 2010. Their mission—to amplify the voice of those living with rare disease—a mission Pulse Infoframe endorses and supports fully.

Commencing Sunday, Feb. 24 and continuing through Thursday, Feb. 28, this was an organized, potent combination of a collection of great thinkers and the best that society offers – collegiality, active listening, accessible resources, and a shared strategy for moving forward.

Rare Diseases May Be Rare, But They Affect Millions

There are over 7,000 rare diseases. More than 30 million Americans live with a rare disease. Ninety-five percent of rare diseases do not have an FDA approved therapy. Much more work needs to be done to improve those numbers. Therefore, throughout the week, we all united around these three pillars:

  1. Support robust funding increases for NIH and FDA
  2. Reauthorize the Newborn Screening Saves Lives Act
  3. Join the bipartisan rare disease congressional caucus

Hats off to the Everylife Foundation, the NIH and to the cadre of US Senators, US Representatives and congressional staffers who gave of their time so freely to give voice to living with, caring for or otherwise supporting the rare disease community.

Rare Disease Research Needs to Speed Up

It was refreshing and inspiring to witness Washington, D.C., united in its resolve to accelerate clinical research and improve the lives of those living with rare disease. Christopher Austin, M.D. Director, National Center for Advancing Translational Science made the point that at the present rate of research, it would take over 2,000 years to develop appropriate treatments for all rare disease–a disturbing fact to all of us.

Sharing Stories and Knowledge

The week started with a wonderful Sunday welcoming reception at the Ronald Regan Building and International Trade Center. A documentary, My Turn, which tells the story of Scott Matzka, a former professional hockey player and his firsthand perspective on how his life changed upon being diagnosed with ALS, followed.

Thereafter came the following events and activities:

  • an informative conference, planning and exhibits, of which Pulse Infoframe was pleased to be a part
  • a Lobbying Day breakfast, followed by scheduled meetings with members of the House and Senate
  • a young adult meet-up Tuesday evening
  • a Rare Disease Congressional Caucus Briefing in the Russell Senate Office Building
  • a group photo on the Capital steps
  • a rare artist reception

Thursday, Feb. 28, International Rare Disease Day, was a daylong meeting at the National Institutes of Health in Bethesda.

Help End Rare Diseases

Far from a “one-and-done” annual event, there are ample opportunities to bring this community of interest together and to increase its ranks. To help educate the next generation of rare disease advocates, the Everylife Foundation is supporting Rare on the Road in Boston, Birmingham, Denver and Sioux Falls.

Surely, Rare Disease Week reinforces one’s faith in the goodness of human nature. If you are not involved, get involved. Don’t wait until next year. Pulse Infoframe will continue to be involved and we look forward to seeing you at future events.

Joe Ternullo is the head of business development for Pulse Infoframe, a member of NQRN Advisory Committee and Co-Chair of the Society for Participatory Medicine

Group photo at Capitol HillGroup photo on steps of Capitol Hill

About Pulse Infoframe Inc.
Focusing on rare disease, cancer and patient reported outcomes, Pulse Infoframe, a new style health informatics company, delivers real-time analytics and visualization to improve outcomes, generate real-world evidence, and provide data insights and precision knowledge. Pulse aggregates data from multiple sources, making it available to relevant stakeholders and offering numerous benefits including dramatically improved efficiency, productivity, and collaboration. Pulse Infoframe is headquartered in Canada, with offices in the United States, and India.

More information is available at www.pulseinfoframe.com

Newest Pulse Infoframe Collaborators

This summer, Pulse welcomed: Friedreichs Ataxia Research Alliance (FARA), a non-profit organization dedicated to curing Friedreich’s Ataxia and the related ataxias. The FARA registry will facilitate dialogue between the public and scientific communities globally and promote information exchange that drives medical advances.

Myonexus Therapeutics Logo

Myonexus Therapeutics, a clinical stage gene therapy company focused on treating distinct forms of Limb-Girdle Muscular Dystrophy, and Pulse Infoframe signed a multi-year agreement encompassing a multitude of Pulse products and services, including a global registry focused on identifying new treatment options for this devastating illness. These new initiatives demonstrate Pulse’s commitment to the rare disease and cancer communities, contributing what we can toward cures and quality of life improvement.

Myonexus Therapeutics Logo

This story first appeared in our February 2019 newsletter. Get Pulse Infoframe news delivered to your inbox. Email us to sign up today.

New Pulse Office, Added Staff, Conferences

Supporting this growth, Pulse Infoframe has added more specialist staff and moved to new, larger offices to better support its clients. In 2018, Pulse Infoframe attended, presented or exhibited at Patients as Partners, BIO2018, CDKL5 Forum, ESMO 2018 Congress, World Orphan Drug Conference, HIMSS2018, Society for Participatory Medicine Conference, DPharm and more. Look for us this year at the 15th Annual Columbia Business School Healthcare Conference, Patients as Partners EU, Patients as Partners US, Clinical Research as a Care Option, Patient Experience Symposium, BIO2019, and more.

This story first appeared in our February 2019 newsletter. Get Pulse Infoframe news delivered to your inbox. Email us to sign up today.

Updates on 2 Pulse Infoframe Registries

We have some exciting updates on two Pulse Infoframe registries.

Pan-Canada Cancer Registry

The success of the Canadian Melanoma Research Network, powered by Pulse and currently encompassing 10 major Canadian cancer centres of excellence, has resulted in a series of discussions to expand the registry to include breast, lung, prostate, and other cancers, transforming it to a powerful pan Canada cancer collaborative research network. At 2:50 to 4:10, see Dr. Scott Ernst, London Regional Cancer Program, present at the Global Melanoma Research Network and Real-World Evidence.

Preventing Sudden Cardiac Arrest in Children

Simon's Heart Logo

Taking the lives of thousands of children every year, Sudden Cardiac Arrest is the #1 killer of student athletes. Research shows that heart screenings, coupled with a good medical history and physical exam, are effective at detecting heart conditions and preventing sudden cardiac arrest. Simon’s Heart has screened over 18,000 children for undiagnosed cardiac disorders and changed over 120 lives. The data and images collected in the Simon’s Heart national youth cardiac registry are made available to qualified researchers free of charge.

This story first appeared in our February 2019 newsletter. Get Pulse Infoframe news delivered to your inbox. Email us to sign up today.

Two Pulse Infoframe Rare Disease Registries Expand Their Global Reach

Castleman Disease Collaborative Network Logo Orphan Disease Center Logo

Proving the power of global collaboration, the Castleman Disease Collaborative Network, an initiative dedicated to accelerating research and treatment for Castleman disease, convened 42 experts from 10 different countries to establish the first-ever treatment guidelines for idiopathic Multicentric Castleman Disease, based on data from over 300 iMCD patients. This landmark study was pre-published in Blood, the top hematology journal in the world.

The Orphan Disease Center in the Perelman School of Medicine at the University of Pennsylvania has launched a global registry for CDKL5 Deficiency, a severe epilepsy disorder with infantile onset. It involves cyclin-dependent kinase-like 5 (CDKL5), a protein whose gene is located on the X chromosome. The purpose of the registry, which has united hundreds of patients since its launch earlier this year, is to enable international collaboration to better understand CDKL5 Deficiency and to accelerate and improve clinical studies focused on developing new therapies to combat CDKL5.

This story first appeared in our February 2019 newsletter. Get Pulse Infoframe news delivered to your inbox. Email us to sign up today.

What’s up Down Under: Updates on Healthcare Innovation in Australia

Empowering 76,000 Clinicians in Australia With New Patient Insights

NSW eHealth Logo

The voice of NSW’s 7 million citizens is rising thanks to NSW’s forward-thinking leaders committed to improving patient care across Australia’s largest health system.

There is a lot of health care innovation in Australia where there has been a strong focus on listening to patients. Patients take centre stage in New South Wales (NSW), where evidence-based, clinically validated patient reported outcome measures (PROMs) and patient reported experience measures (PREMs) are being deployed across the entire health system. In a first-of-its-kind initiative globally, working with NSW Agency for Clinical Innovation and eHealth NSW, Pulse Infoframe is deploying 15 different PROMs and PREMs, empowering 76,000 NSW clinicians with new patient insights.

Empowering Patients With Colorectal Cancer

Bowel Cancer Australia is the leading community-funded NGO and patient advocacy organisation dedicated to prevention, early diagnosis, research, quality treatment and the best care for everyone affected by bowel (colorectal) cancer. Through the deployment of the International Consortium of Health Outcomes Measurement (ICHOM) Colorectal Cancer Standard Set, Bowel Cancer Australia is empowering patients with tools and knowledge, to be advocates for their own health and to express what matters most to them about their treatment and care. Colorectal cancer is the third most common cancer globally, affecting men and women, young and old. Pulse’s technology platform, with an enhanced e-healthie payment gateway for clinicians, facilitates the collection of valuable information direct from patients using standardised questionnaires to help clinicians better understand patient perceptions about the impact colorectal cancer has on their lives.

These two new Pulse clients join Ramsay Health Care hospitals in deploying the Pulse Infoframe technology platform to capture the patient voice in Australia through PROMs and PREMs. They’re two more examples of healthcare innovation in Australia.

This story first appeared in our February 2019 newsletter. Get Pulse Infoframe news delivered to your inbox. Email us to sign up today.

Executive Assistant to the CEO

Position Title: Executive Assistant to the CEO.

Region: London, Ontario, Canada.

Compensation: Full Time Position with salary, commensurate with experience. Benefits package included.

Job Description:
The Executive Assistant is an essential person within the organization who will coordinate logistics and provide essential communication for the CEO. A successful candidate must be detail-oriented, process-oriented, and must have the ability to juggle multiple responsibilities at one time. Candidate will be responsible for coordinating logistics for the CEO, communicating with team members, and communicating on behalf of the CEO to prospects, strategic partners, and clients. You must have the ability to create process and systems, then make sure that those systems are followed by members of the organization. You must be detail oriented and take good notes. You must have strong written and verbal communication skills and be comfortable communicating over the phone and through emails. You need to be comfortable with deadlines and handling pressure, and you must have the ability to juggle multiple tasks at one time. You must also have the ability to handle many different types of personalities and have thick skin. You must have the ability to handle adversity, think on your feet, be flexible, and manage multiple relationships at once. A successful candidate will be a self-starter, willing to learn on the job, and can take on different tasks without being micro-managed. You will occasionally be asked to do things outside of traditional work hours.

Company Description:
Pulse Infoframe is a healthcare IT company that has built a collaborative ecosystem for the healthcare world that enables researchers, doctors, pharmaceutical companies, and patients to be heard and pertinent information to be shared as appropriate. Our goal is to help cure disease and improve healthcare outcomes through collaboration. And it really works! Already our global disease ecosystems have helped advance treatments and connect patient communities around the world. Effective, global collaborative data exchanges are managed with our Healthie™ solution. Our focus is in cancer and rare disease.

Qualities You Need:

  • College degree
  • Ability to work remotely
  • Proficiency in all Microsoft systems
  • Specialized computer skills including the ability to use various applications such as database, spreadsheets, report writing, project management, graphics presentation creating/editing, and scheduling software
  • Ability to follow instructions
  • Self-starter
  • Experience multi-tasking
  • Strong written and verbal communication
  • Detail oriented
  • Team player

Local Candidates only.
No recruiters or agencies.

Candidates must submit:
– Resume
– Cover letter

We are an equal opportunity employer.

Society for Participatory Medicine Adds Pulse Infoframe’s Digital Tool-Set and Supercharges Corporate Member Benefit Offering

Patient Portals, Evidence-Based Patient Reported Outcome Measures, Health Registries, Real Time Analytics, & Data Visualization Capabilities Offering Augments Member Benefit

(Boston, Mass. and London, Ont., Oct 25, 2017) Senior executives from the Society for Participatory Medicine, the preeminent organization in promoting true partnerships between patients and healthcare professionals, and Pulse Infoframe Inc., a leading cloud-based healthcare data insights company, announced a partnership to promote the use of patient portals, patient reported outcomes, and registries to enable the open exchange of information between patients and caregivers.

“A growing body of research is showing that engaged and informed patients have better outcomes and reduced costs,” said Dr. Danny Sands, a co-founder and board chair of the Society for Participatory Medicine. The Society is a catalyst for positive change in healthcare and is thrilled to offer this new member benefit providing specialized pricing and domain expertise on an essential digital toolset that facilitates dialogue, enables needed ecosystems, and improves human health. I am delighted with our new relationship with Pulse Infoframe.”

“The Society for Participatory Medicine’s activities are routed in community, conversation, advocacy, research, and education. The Society’s mission and methods are fully aligned with Pulse Infoframe’s objectives. We are committed to serving the Society’s growing membership. Together, we can empower the voice of the patient and bring about a stronger, collaborative and learning healthcare system,” said Dr. Femida Gwadry-Sridhar, Founder and CEO, Pulse

Infoframe. “Through technology-enabled ecosystems, research, analysis, reporting and collaboration globally become easier, efficient and much less labor intensive.”

About the Society for Participatory Medicine
Created by a passionate group of professionals and patients with a vision to start a movement to transform the culture of healthcare, The Society for Participatory Medicine is a 501(c)(3) not-for-profit membership organization devoted to promoting the concept of participatory medicine, a movement in which activated empowered patients engage as drivers of their health, and in which providers encourage and collaborate with them as full partners in their care.  SPM does this by stimulating dialogue, influencing policy, advocating research, and educating patients, health care professionals, and others. SPM members include stakeholders from across the healthcare continuum.

More information is at participatorymedicine.org

About Pulse Infoframe Inc.
Focusing on rare disease, cancer and patient reported outcomes, Pulse Infoframe, a new style health informatics company, delivers real-time analytics and visualization to improve outcomes, generate real-world evidence, and provide data insights and precision knowledge. Pulse aggregates data from multiple sources, making it available to relevant stakeholders and offering numerous benefits including dramatically improved efficiency, productivity, and collaboration. Pulse Infoframe is headquartered in Canada, with offices in the United States, and India.

More information is available at www.pulseinfoframe.com