Worldwide, hundreds of patient organizations are holding awareness-raising activities based on the theme of research, because “with research, possibilities are limitless.” Rare disease research is crucial to providing patients with the answers and solutions they need, whether it’s a treatment, cure or improved care. Patient involvement in research has resulted in more research, which is […]
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February 28, 2017
Rare Disease Day 2017 Focuses on Research and Patient Involvement
Worldwide, hundreds of patient organizations are holding awareness-raising activities based on the theme of research, because “with research, possibilities are limitless.”
Rare disease research is crucial to providing patients with the answers and solutions they need, whether it’s a treatment, cure or improved care. Patient involvement in research has resulted in more research, which is better targeted to the needs of patients. Patients no longer solely reap the benefits of research; they are empowered and valued partners from the beginning to the end of the research process.
The idea for our Patients as Partners conference arose seven years ago, when our Disrupting Clinical Trials (DPharm) audience was asked, “When was the last time you thanked your patients for being in a clinical trial?” and no one raised their hand. Immediately we saw the need for a conference on service for patients in clinical trials. However, more importantly, we saw the urgent need for the patient perspective in driving a conference on collaborating in clinical research.
Patients and patient research taught us empathy not only with their disease, but with their lifestyle and struggles in finding and experiencing a clinical trial. In particular, we learned the need to:
- Understand and incorporate the patient’s voice in designing clinical trials and developing a clinical endpoint.
- Vastly improve the patient’s entire experience in a clinical trial.
The Conference Forum is proud to participate in 2017’s Rare Disease Day as we continue to advocate for patient-led research. If it were not for patient research, we would not have a conference 100% designed for the benefit of the patient. Join us this week to learn about new research into creating patient centricity as the ecosystem within pharma at the 4th Annual Patients as Partners US, March 2-3. Learn more about #Patients2017 and register here: http://bit.ly/12nEslG
February 15, 2017
Takeaways from Inaugural Patients as Partners Europe Meeting
The Conference Forum held the inaugural Patients as Partners Europe (Patients EU) conference in London on February 6-7, 2017, in response to requests from European attendees at the previous Patients as Partners US events to bring the conference abroad. Patients as Partners is Patients Included-accredited and the research is driven equally by patients and advocacy alongside […]
The Conference Forum held the inaugural Patients as Partners Europe (Patients EU) conference in London on February 6-7, 2017, in response to requests from European attendees at the previous Patients as Partners US events to bring the conference abroad.
Patients as Partners is Patients Included-accredited and the research is driven equally by patients and advocacy alongside industry, government and academia. The Patients as Partners conference, both in the EU and the US, is devoted to understanding and incorporating the patient’s voice in designing clinical trials and developing a clinical endpoint, as well as vastly improving the patient’s entire experience in a clinical trial.
Through the on-site activities, including the Patient Engagement Toolbox, each attendee walked away with a “toolbox” full of strategies to continue to support their patient involvement initiatives. Listed below are many takeaways discussed during Patients EU. We look forward to seeing everyone again for the 2nd annual event in 2018!
Day One takeaways:
- The interests of patients are best served when industry and patient groups work in partnership
- Key to the success of such partnerships are trust and transparency
- Several examples exist that exemplify best practice that should give confidence to those about to embark on such a partnership
- Engaging with Patients;
- Helps bridge the gap between clinical trial data and real world data which ultimately leads to more meaningful outcomes for all concerned
- Increases transparency and trust in the system
- Engagement should be mutually beneficial; use a flexible step-by-step approach, to learn together what format works best
- Provide support – define roles – manage expectations – give feedback
- Everyone benefits from knowledge sharing
- Opportunities to engage patients exist across all aspects of drug development and the patient journey
- To improve drug and device development the patient voice should be routinely solicited
- Patient involvement in medicines development and assessment is essential to ensure that the added value of a new medicine to patients is clearly understood
- Patients have unique experiential knowledge about living with the condition that can inform clinical trial design to ensure the outcomes that matter most to patients are studied
- Patient organizations can invest in research and contribute to comparative effectiveness programs to fill evidence gaps or uncertainties in value assessments.
- It is critical to assess outcomes defined by patients living with the disease and its treatment
- Considering “Patient-Centered Outcomes” requires us to have a comprehensive understanding of patients’ needs and priorities. This information can be coupled with the needs of others to develop an outcomes measurement plan that evaluates outcomes that matter
- Outcomes may include concepts that are best measured using patient-reported outcome measures (PROMs), but not all patient-centered outcomes are best-measured in this way.
- Engaging in and implementing a novel patient-centric service requires commitment, dedicated time, cooperation, collaboration and investment by sponsor and partner vendors
- Results can be fruitful-ambulant care at-home visits can reduce the burden on study participants by making it more convenient and comfortable for them and their families- ultimately resulting in faster enrollment, better compliance and higher retention and higher satisfaction for all parties
- Feedback to patients is essential to encouraging data sharing
- Transparency about how data are shared and giving patients control will reveal new possibilities
Day Two takeaways:
- Patient organizations can help you find patients to work with and offer guidance and support to develop meaningful involvement.
- Patient engagement should be as early as possible, preferably before a clinical trial protocol has been finalized.
- Patient groups tend to have the best databases of patient details, which is essential for rare disease trials.
- Build trust & manage expectations (a new drug with maximum efficacy and no side effects is rarely a realistic goal)
- Set aside enough funds for patient travel and subsistence and remember some of us need careers too. Allow time to explain things. Slowly and clearly. And probably twice.
- Keep patients engaged. Tell us what’s happening with the study (and why), tell us the outcomes, and tell us what difference we made (if any).
- Identify the joint goals (=mutual values) through open & transparent discussion. This will create a win/win situation.
- Need to listen to and communicate with the patient(s)
- Culture change – we need to continue to evolve and work beyond constraints of “the process”
- Strive to “remove barriers” for effective patient engagement
- Patients get ruled out by one of many hurdles to cross, not lack of interest
- Empower the patient
- The culture of the treating institution is central to how likely patients are to volunteer
- Many patients would welcome general information at diagnosis about clinical trials so that treatment planning includes consideration of developing options
- The most crucial element of the Informed Consent Process is interaction between investigator and patient
- The investigators need to be made aware of the relevance of an optimized IC process
- A complete overhaul of the IC preparation process is needed
- The proposed “10 IC Steps to Success” Plan should be implemented as new standard in clinical trials
- Informed Consent is a window of opportunity for researchers
- Write with target audience in mind
- Involve patient organizations from start
- Informed Consent process needs input from multiple stakeholders
- Sponsors have a role to play and should utilize the available guidance, best practice and shared knowledge to support the process that ultimately benefit the patient
- EFPIA support reinforcement of the elements for “10 IC Steps to Success”, in particular:
- Putting efforts in simplification
- Utilizing modern technology
- Taking patients on board
February 1, 2017
Tackling rare disease with the voice of patients
Proponents of big data methodologies are excited about their potential applications in rare disease research, including in: drug discovery; the discovery of disease-related genes, genetic mutations and biomarkers; matchmaking of rare disease cases to help diagnose patients; and drug repurposing. But while great insights can be gleaned from huge datasets, equally valuable and complementary intelligence […]
Proponents of big data methodologies are excited about their potential applications in rare disease research, including in: drug discovery; the discovery of disease-related genes, genetic mutations and biomarkers; matchmaking of rare disease cases to help diagnose patients; and drug repurposing.
But while great insights can be gleaned from huge datasets, equally valuable and complementary intelligence can be derived from rigorous interrogation of datasets that are relatively small.
Interestingly, a small data movement has emerged in the past few years; its loudest cheerleader being Martin Lindstrom, the Danish author of “Small Data: The Tiny Clues That Uncover Huge Trends”. Mr Lindstrom’s world is that of marketing and branding, but it doesn’t take a huge leap to apply his principles of keen observation of small samples to people living with rare disease.
And recent work in the field of patient-reported outcomes (PROs) has provided evidence that patient-generated medical data can be of comparable quality to data gathered from traditional sources. A group of US researchers conducted a proof-of-concept study using the chronic lymphocytic leukemia (CLL) community of PatientsLikeMe, a patient-powered research network. There are several PRO instruments specific to CLL, meaning the supporting literature contain data the researchers could use as comparators. Using a combination of online surveys and telephone interviews, they found good alignment between the symptoms that members of PatientsLikeMe’s CLL community said were important to them, and those identified through traditional interviews and patient focus groups.
Raremark has also been exploring how to involve patients in the area of data sharing and donation, the reasons for doing so, and the implications for the patient community. In line with the small data model, we posed a series of well-defined questions to small groups of patients, using a combination of telephone interviews and online surveys. The study sample comprised Raremark users with an interest in three rare diseases: adrenoleukodystrophy, myasthenia gravis and Sanfilippo syndrome.
Work conducted from November 2016 to January 2017 revealed an understanding of the importance of data sharing for the benefit of others, and a willingness to do so: 94% of participants said they would feel comfortable sharing selected health-related information about themselves with the community and the pharmaceutical industry.
Raremark’s findings reflect the results of a larger study by RD-Connect, an EU-funded research initiative in rare disease, that included similar themes. As long as the right governance systems are in place, RD-Connect discovered, the rare disease patient community generally has a positive view on the sharing of data to support medical research. “All the participants understood the incentive for [rare disease] in sharing data and samples; in fact, there were several pleas for research systems to be standardised across the EU in order to make data sharing easier,” the authors wrote in the European Journal of Human Genetics.
I’ll be discussing the Raremark project in the context of these industry trends at Patient as Partners Europe, which takes place in London on February 6-7, 2017.
Pete Chan is Head of Research & Analysis at Raremark, a patient-powered research network in rare disease. This blog is an edited extract from Raremark’s white paper: Tackling rare disease with big and small data (http://pages.raremark.com/whitepaper-big-small-data).
December 7, 2016
Patients as Partners Opens in Europe this February in London
At the request of our European attendees at the Patients as Partners US event to bring the conference to Europe, we are delighted to announce we are doing just that on February 6-7, 2017 in London. Patients as Partners EU is co-produced with patients, industry, academia, government and nonprofit organizations to establish a well-rounded program […]
At the request of our European attendees at the Patients as Partners US event to bring the conference to Europe, we are delighted to announce we are doing just that on February 6-7, 2017 in London. Patients as Partners EU is co-produced with patients, industry, academia, government and nonprofit organizations to establish a well-rounded program that addresses the needs of all stakeholders seeking to implement and advance patient involvement across the entire clinical development continuum. The US conference will take place March 2-3, 2017 in Philadelphia.
Each session at the 2017 conference seeks to put ideas into action and must demonstrate the what, where, when and how behind the topic. For example, when addressing the topic of, “How to Incorporate Patient Insights in Clinical Research,” the speaker representing AstraZeneca will show the planning, process and tools used to obtain the patient insights. They will also demonstrate how these insights were used to co-create solutions with patients and measure the impact of those initiatives.
Most of the sessions have at least one confirmed patient speaker to ensure the patient perspective is represented for the topic at hand.
Introducing “Ask the Patient”
We will introduce for the first time in Europe, the “Ask the Patient” program. This is an opportunity for attendees to meet face-to-face with patients who experienced at least one clinical trial. These patients, our Medical Heroes, will be attending the conference, providing a unique opportunity to learn about the realities of a clinical trial directly from the patient’s point of view. Opportunities to meet with patients will be both assigned and unassigned throughout the program.
“Patients Included” Accredited
Patients as Partners Europe is accredited by “Patients Included” (patientsincluded.org) to help address what matters most to patients to enable them to support clinical research. “Patients Included” provides a blueprint for:
– Understanding and incorporating the patient’s voice in designing clinical trials and developing a clinical endpoint
– Vastly improving the patient’s entire experience in a clinical trial
– Empowering the patient in the new medicines development process
Patients as Partners Europe is co-chaired by:
Clare Grace, PhD
VP, Site and Patient Access, INC Research
Anton Hoos, MD, PhD, MBA
Head of Medical, Amgen Europe
The goal of creating patient-centricity as the ecosystem within pharma cannot be achieved without patient involvement.
A special thanks to our patient, industry and academic advisors for their unwavering guidance and expertise in helping us develop an agenda that puts the “discussion” around patient involvement into action across the entire clinical development continuum. For more information, visit here or go to www.theconferenceforum.org. Take note there is a 15% launch discount off the first early bird with code PEUL15.
November 16, 2016
Patients as Partners Speakers Collaborate on Inaugural Patient Engagement White Paper
Patients as Partners EU and US speakers including Marc Boutin, National Health Council, Tony Hoos, Amgen EU, Andrew Garvey, GSK, Jan Geissler, EUPATI, Veronica Todaro, Parkinson’s Disease Foundation, Roslyn Schneider, Pfizer and Sarah Krüg, Cancer 101, collaborated on an inaugural white paper focusing on culture and process change as a priority for patient engagement. Patient Focused Medicine’s […]
Patients as Partners EU and US speakers including Marc Boutin, National Health Council, Tony Hoos, Amgen EU, Andrew Garvey, GSK, Jan Geissler, EUPATI, Veronica Todaro, Parkinson’s Disease Foundation, Roslyn Schneider, Pfizer and Sarah Krüg, Cancer 101, collaborated on an inaugural white paper focusing on culture and process change as a priority for patient engagement.
Patient Focused Medicine’s Development (PFMD) published the white paper this week in the peer reviewed journal Therapeutic Innovation & Regulatory Science (TIRS).
The authors of the white paper are lead advisors to both Patients as Partners programs and will be participating in case studies and panel sessions at Patients as Partners Europe taking place February 6-7, 2017 in London and Patients as Partners US taking place March 2-3, 2017 in Philadelphia.
Read the white paper here.
For more information on Patients as Partners Europe, click here.
For more information on Patients as Partners US, click here.