Patient Advocacy Strategies

Attention Massachusetts cholesterol patients currently on PCSK9 inhibitor therapy. We're working with the patient community to submit letters to protect appropriate, doctor-prescribed patient access to PCSK9 therapies and to address access barriers to patients in MA. Please reach out if you are interested to [email protected] for more information.

It's easy for drug development companies to say they're committed to supporting the patient engagement movement – but research shows otherwise.

A recent survey from Tufts Universityrsity shares that of several hundred companies polled, only 27% are routinely soliciting patient input into protocol design decisions.

Aside from that, a recent C.I.S.C.R.P study found:

◾ Only 35% of clinical trial volunteers report receiving a formal verbal or written “Thank You."
◾ Less than 30% of clinical trial volunteers report receiving any information about the results of their clinical trial.

If you want to become more patient-centric but don't have the bandwidth, resources, or know-how, we'd love to help. You can connect with us here: https://patientadvocacystrategies.com/contact/



https://www.appliedclinicaltrialsonline.com/view/calling-out-check-the-box-patient-engagement

Calling Out ‘Check-the-Box’ Patient Engagement Spotlight on poor compliance, access, and usefulness of trial results summaries.

What if you could pick up your prescription and register for a clinical trial at the same time?

Though that may sound unrealistic now, retailers like Kroger, Walmart, and Walgreens have their sights set on expanding into the clinical trial space.

"The reality is, we're trying to build the right highway into our communities to bring trials closer to where they are," shares Ramita Tandon, Walgreens' chief clinical trials officer.

With current recruitment challenges, including diversifying participation to underrepresented groups, having the ability to register for a clinical trial while visiting one of the nation’s largest retailers could have significant positive impacts.

Would you want to see major retailers participate in clinical trial recruitment?

Retailers seek to shake up clinical trials business Big retailers see a growth opportunity tapping their huge customer bases to recruit participants into clinical trials.

What clinical trial trends are on the rise in 2024?

While science journalist Ayesha Rashid shares several, we had to point out a trend we are especially hopeful for.

◼️ Patient-centric approaches are on the rise - A survey from C.I.S.C.R.P found that 41 percent of respondents found it very important to know that patients and caregivers with their condition had shared insight into the study design before enrolling any study volunteers.

What clinical trial trends are you most looking forward to in the coming year? Let us know in the comments.

engagement

Top 10 Clinical Trial Trends for 2024 The top trends in clinical trials in 2024 will include growing use of AI tools and real-world evidence (RWE) in regulatory decision-making.

There’s no denying the power and passion of patient advocacy groups, but John Hu, CEO and co-founder of Pepper Bio, shares a thought-provoking perspective.

“Through letters to the FDA, targeted advertisements, appearances at advisory committee meetings, email campaigns, engagement with celebrities, meetings with Congress, and more, patient advocacy groups spend significant time and money to influence the FDA. Whether these drugs are approved due to the efforts of patient advocacy groups, lobbyists, politicians, or other outside factors, the problem remains the same. When we make decisions that lack the necessary scientific rigor to establish robust conclusions, nobody wins.”

Put plainly, Hu argues that patient advocacy groups, while well-intentioned, could more greatly benefit from a more thorough, yet slower drug approval process.

The obvious counter to his arguement is time. It can take several years to approve a rare disease drug, however, not all rare-disease patients have years to wait.

We’d love to hear your perspective. Let us know your thoughts in the comments.

Alzheimer’s drug approvals show we need a reevaluation of patient advocacy The current state of patient advocacy needs reform so that the best, safest drugs come to market, writes Jon Hu of Pepper Bio.

Patient attrition can have far-reaching and negative impacts on patients, sponsors, and investigators.

So, what’s the solution for keeping patients in a clinical study?

It’s as easy as listening to their concerns and helping them find a realistic solution.

For example, patients often drop out of clinical trials because of travel and lodging barriers. Solutions to these issues could include providing travel to the trial sites, offering stipends, and reimbursing expenses.

While these solutions may be costly, it’s likely more affordable than replacing a patient, which costs 2.5x as much as recruiting a patient.

Check out the infographic below for more insights from Scout Clinical.

Thank you to Halloran Consulting Group for inviting PAS's Josh Argall to speak at this year's CORE East, an event for leaders in life sciences to talk through the most pressing industry issues.

Josh will be taking the stage twice: Once to discuss his experience as a caregiver to his son Devin, who lives with Duchenne Muscular Dystrophy, and the second time to examine the use of social media in clinical trials.

There’s no denying the power of social media, or the impact it's had on clinical trial patient recruitment.

Not sure if social media is the right platform to create awareness about your clinical trial? Consider these factors:

1. Researchers can reach prospective participants who may not have known about a clinical trial through social media ads and sponsored posts.

2. Prospective participants can ask questions and get answers in real-time through social media comments and messages, fostering a greater sense of trust.

3. By joining relevant patient or support groups on social media, partaking in conversations, and sharing information about a study, researchers can connect with engaged individuals who are looking for opportunities to contribute to medical research.

Learn more:

How Social Media Influences Clinical Trial Patient Recruitment Social media platforms have revolutionized the way we connect and interact with each other. Beyond personal connections, social media has also proven to be a powerful tool for businesses and organizations.

From clinical trial recruitment to drug creation, it seems like AI is the future of health sciences and medicine.

The first AI generated drug, created by InSilico Medicine to treat idiopathic pulmonary fibrosis, recently entered clinical trials with human participants in a groundbreaking Phase II trial. Insilico Medicine’s founder Alex Zhavoronkov shares:

“It is the first fully generative AI drug to reach human clinical trials, and specifically Phase II trials with patients. While there are other AI-designed drugs in trials, ours is the first drug with both a novel AI-discovered target and a novel AI-generated design.”

What are your thoughts on the role AI is playing in medical advancements?

Let us know in the comments.

The first fully A.I.-generated drug enters clinical trials in human patients This week, generative AI hit a new milestone: The first fully AI-generated drug reached clinical trials in human patients.

“I believe that incorporating patients into every step of the process is integral to overcoming obstacles related to drug development, clinical trials, and manufacturing.”

As Kinnari Patel, president and COO at Rocket Pharmaceuticals Inc shares, patient-centricity is the key to success, and we could not agree more.

So, what does this look like in practice?

Patel states that companies should:

1. Meet with patients and their caregivers before designing a human study to hear about the challenges they face
2. Engage patient advocacy groups and physician communities when designing clinical protocols
3. Invite caregivers, physicians, and disease experts to attend meetings with health authorities, including the FDA
4. Prioritize patients in drug manufacturing
5. Collaborate with industry peers to tackle the toughest challenges together

As a patient, caregiver, or physician, how else would you like to see patient-centricity included in clinical trials?

Patient-Centric Clinical Development For Rare Disease Treatments Incorporating patients into every step of the process is integral to overcoming obstacles related to clinical development of rare disease treatments. Obtaining early patient input, designing meaningful clinical trials, and considering manufacturing strategy early is crucial.

There are dozens of factors impacting the creation and reliability of rare disease patient registries – and while challenging, experts like rare disease dad and former The National Institutes of Health date scientist Harsha K Rajasimha, are dedicated to tackling the issue.

So, where does one begin?

Rajasimha shares:

“I believe the ideal scenario is for nonprofit patient groups to receive the funding to lead the creation of a global patient registry for a given rare disease(s), supervised by academic principal investigators (e.g., medical geneticist research doctor). Following proper good clinical practice (GCP) guidelines with institutional review board (IRB) approved protocols in the U.S., or ethics committee approved protocols in the rest of the world, would make the registry most useful in the long term for clinical research, guiding public health policy development, and controlling data of patients enrolling in clinical trials.

What’s more, the patients enrolling in patient registry studies have to be properly informed and consented on what data is collected and how that data will be used for research and development in the future. Defining those intended uses of the data is a critical hallmark of usable and useful patient registries.”

Learn more about the complexities of rare disease registries and Rajasimha's work:

Advancing Rare Disease Research With Patient Registries In this Q&A, “Rare dad” Harsha Rajasimha, Ph.D. of IndoUSrare discusses the current state of rare disease research and explains how patient registries can help propel drug development for rare disease therapies. 

From clinical trial recruitment to drug creation, it seems like AI is the future of health sciences and medicine.

The first AI generated drug, created by InSilico Medicine to treat idiopathic pulmonary fibrosis, recently entered clinical trials with human participants in a groundbreaking Phase II trial. InSilico Medicine's founder Alex Zhavoronkov shares:

“It is the first fully generative AI drug to reach human clinical trials, and specifically Phase II trials with patients. While there are other AI-designed drugs in trials, ours is the first drug with both a novel AI-discovered target and a novel AI-generated design.”

What are your thoughts on the role AI is playing in medical advancements?

Let us know in the comments.

The first fully A.I.-generated drug enters clinical trials in human patients This week, generative AI hit a new milestone: The first fully AI-generated drug reached clinical trials in human patients.

Patient enrollment is one of the biggest challenges in launching clinical trials – and it’s one many tech and medical professionals, including UPMC’s Chief Healthcare Data and Analytics Officer Oscar Marroquin, are confident AI can help tackle.

Marroquin shares that UPMC’s Center for Connected Medicine (CCM) has “already seen the benefits of using natural language processing to harness and analyze the vast quantities of unstructured data in healthcare to better understand the conditions of our patients. Applying these techniques for clinical trial matching would be an advantage for health systems, industry, and patients."

With more than 80% of trials missing initial enrollment targets of timelines, the impact of AI could be significant. Learn more about what AI could mean for the future of healthcare:

AI and EHR Could Expedite Matching Patients with Clinical Trials Combined technology reduces time spent searching for appropriate patients for trials

What hinders life sciences companies from prioritizing patient-centricity?

ZS's associate partner and patient-centricity lead, Sharon (Mandler) Suchotliff, MPH shares:

“The reality is that unless we can make a connection between the investments we make in patient centricity and some sort of impact, that investment's going to go away.”

In short, if there’s no obvious ROI in patient-centricity efforts, companies aren’t going to prioritize it.

So, how do companies, especially those that are early in their patient-centricity journey, show its immense value?

1. Show your leadership the opportunity cost of not engaging with patients and the potential benefits of patient engagement.

2. Get your leadership on board, because for change to happen, it has to come from the top.

3. Make it easy to engage with a group of diverse and representative individuals, get feedback on your program, get input into your protocol, talk about your messaging, and bring people in.

Find more patient-centric insights from Suchotliff here:

Discussing the rise of patient-centricity in life sciences If anybody knows about the importance of a patient-centric approach to life sciences, it is Sharon Suchotliff. | What is driving the shift towards patient centricity in life sciences, and how can the link between investing in patient-centric strategies and business impact be proven? ZS’ Sharon Suc...

How do life science companies ensure their clinical trial participants represent the patients their drugs are designed to help?

“They can start by listening to conversations at scale during the recruitment process,” shares Authenticx founder and CEO Amy Brown.

The critical advantages of doing so include:

1. Patient recruitment strategists can glean insight from conversational data to guide protocol reviews and adjust endpoints (if possible) as the clinical trial progresses to ensure the study is answering participant questions, offering reassurance, and clearly articulating the benefits each participant receives.

2. Organizations can gain a more granular view of specific emotions conveyed by tone and words.

Ready to start improving your clinical trial recruitment? Find more reasons for utilizing patient conversations and advice on doing so here:

Listening at Scale and Leveraging Conversational Data to Improve Clinical Recruitment How can managers ensure their clinical trial participants represent future patients?

As an organization dedicated to addressing and improving unmet patient needs, especially within the rare disease community, we are thrilled by the FDA's recent accelerated approval of Sarepta Therapeutics' ELEVIDYS to treat Duchenne muscular dystrophy (DMD) in patients aged 4-5.

This news is especially meaningful to PAS, as our senior director of engagement, Josh Argall's son, Devin Argall, lives with DMD.

Of the news, Devin shares, "The FDA's approval of ELEVIDYS provides hope that the FDA will expand approval to include others like me."

Learn more about this groundbreaking approval here:

Sarepta Therapeutics Announces FDA Approval of ELEVIDYS, the First Gene Therapy to Treat Duchenne Muscular Dystrophy | Sarepta Therapeutics, Inc. ELEVIDYS (delandistrogene moxeparvovec-rokl) is approved for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne based on expression of ELEVIDYS micro-dystrophin observed in patients treated with ELEVIDYS ELEVIDYS is a one-time treatment designed to treat the

Lack of participant diversity in clinical trials is a prevalent issue, and it’s one that Alekhya Pochiraju and TransCelerate BioPharma Inc. are working to combat.

Transcelerate has identified four potential considerations that sponsors may consider for improved diversity within clinical trials.

1. Increase a sponsor’s capacity to build effective partnerships with community-based sites: Community-based sites may have less clinical trial experience than larger, established sites. Sponsors should work with community-based site staff to determine if they need to allocate more resources to the site.

2. Develop community engagement and sustainable partnerships to build trust: Sponsors and sites should identify key community stakeholders, minority-serving healthcare providers, and other trusted voices who can support the understanding of healthcare needs and interests of racially and ethnically diverse communities in the site catchment area.

3. Identify the site’s capacity-building needs focused on site staffing: Sponsors should evaluate existing site networks to understand staff capacity to recruit and retain diverse candidates or partner with minority-serving professional societies and organizations that can support the identification of diverse candidates.

4. Identify site-level capacity-building needs focused on support services: Sponsors should proactively solicit input from sites and the diverse patients they serve to better understand the need for supplemental services like childcare and eldercare that they may not be currently providing.

Learn more about increasing diversity within clinical trials:

Start Early, Collaborate Often: Key Steps To More Diverse Clinical Trials Overcoming the diversity gap in clinical trial research requires multilevel approaches and collaborative partnerships. Genentech's Alekhya Pochiraju reveals four easy ways sponsors can together with their site sites foster more patient diversity and shows us the importance of prioritizing div...

How do you improve clinical trial recruitment?

Patient engagement.

In a review of studies on improving clinical trial success, only three studies with high-certainty evidence were identified, with the following strategies found to be effective:

◾️ Open trials rather than blinded, placebo trials had an absolute improvement of 10% in recruitment

◾️ Telephone reminders to people not responding to postal invitations had an improvement of 6% (for trials with low underlying recruitment).

Want to learn more? Find more key considerations here:

Patient Recruitment in 2023: What Works and What Doesn’t in Clinical Trial Enrollment ⋆ Vial Patient recruitment and enrollment for clinical trials is challenging. We guide you through best practices and explain why enlisting a CRO is the way to go.

As AI continues to increase in popularity across nearly every industry, IQVIA VP Global Head, Integrated Health Practice, Real World Technology Solutions, Marie E Lamont, shares the headway it’s helping to make in oncology clinical trials through:

◾️ Decreasing staffing turnover rates through mitigated feelings of burnout
◾️ Enabling site workers to have more availability for training and development and engaging in higher-quality workloads
◾️ Making the most of tight budgets

Has your organization started utilizing AI in its clinical trials? If so, let us know how in the comments!

Accelerating Clinical Trials Through AI-Enabled Precision Oncology Through precision oncology solutions, such as AI-enabled software, physicians and staff can automate traditionally manual processes...

Pharmaceutical companies lean into four pillars when working in the rare-conditions space:

1. Demonstrating commitment to the rare-condition community
2. Effectively finding rare-condition patients
3. Fostering treatment accessibility
4. Providing support to patients and caregivers alike

Roche's rare-conditions team has created an Infinity model—a patient-centric, decentralized, and networked way of organizing go-to-market teams—to understand how its deployment in real-life scenarios resonates with these different pillars.

When asked how the infinity model demonstrates a commitment to the patient community, Hsien Ping Hu, rare-conditions partner for Taiwan, shared an impactful example:

“We had the opportunity to work with one of three major patient advocacy groups that had the particularity of being led not by a practicing doctor but by a patient with spinal muscular atrophy (SMA).

Bringing patients’ voices into the debate proved powerful. Among other things, it led to the production of a documentary about people living with SMA in Taiwan. The personal lens and insight this provided helped raise awareness for the need to drive access—not only to the treatment but also to the day-to-day care these patients require.

"We believe it is critical to listen to patients’ needs, making their voice heard so that we can co-create solutions that matter and ultimately drive value for them, and for society in general.”

Find more insights from McKinsey & Company on how Roche is putting patients at the center of its rare disease efforts.

Developing a customer-centric approach to rare conditions Roche Pharmaceuticals’ approach to rare conditions highlights industry efforts to make a difference for patients, their caregivers, and healthcare professionals.

Knowledge is power, especially when it comes to rare diseases.

That’s why University of Minnesota College of Pharmacy assistant professor, Reena Kartha, created a course on the subject.

"On average, 17% of undergrads in the pre-med track get into medical school. We are missing the remaining 83% of students who are going into other professions. The biology of a disease is just one aspect of it, there is more to it.”

It takes more than just scientists and doctors to move the needle on rare disease. It’s a massive undertaking that also requires dedication from policymakers, educators, economists, and more.

Kartha is hopeful that if undergraduate students learn about rare diseases, they could be inspired to make rare disease the focus of their career – no matter what that career may be.

Learn more:

Turning Undergrads Into Rare Disease Allies The University of Minnesota offers a “Grand Challenge” course to build awareness about rare diseases as a significant global issue.

The relationship between drug developers and the patient populations they serve is critical – especially when it comes to the rare patient population.

“Working with advocacy groups enables sponsors to access an enthusiastic and appropriate candidate pool while increasing patient access and participation. Additionally, patients and patient advocates offer a unique and credible perspective for clinically meaningful input disease burden and trial design. This is especially valuable for the sparse populations targeted by rare disease trials, which 39% of our survey respondents noted as a priority therapeutic area,” shares Thermo Fisher Scientific Scientific’s Rodrigo Garcia, M.D., M.S.H.S.

Input from patients and advocates during clinical trial design also helps create more patient-centric studies, making study participation easier for patients and helping to yield truly meaningful study results.

Keep reading to learn how to improve clinical trials by prioritizing patient needs.

Lessons Learned in 2022 and the Impact on Drug Development in the Year Ahead DCTs and RWD among topics for industry to focus on moving into 2023.

Though Autoimmune Disease Awareness Month is coming to a close, raising awareness for the more than 100 known autoimmune diseases that impact approximately 50 million Americans should remain a priority throughout the year.

Autoimmune Association president and CEO Molly Murray, CAE, shares why:

1. When more people are talking about autoimmune disease, it generates more attention from researchers who are looking for new ways to address the issue. This increased interest can result in more funding for research, as well as more opportunities for researchers to collaborate and share their findings.

2. Raising awareness can help doctors stay up to date with the latest research and treatment options for autoimmune diseases. This can be particularly important because autoimmune diseases are often complex and multifaceted, and new treatments are continually being developed.

3. Many people with autoimmune diseases report feeling isolated and misunderstood. By raising awareness and increasing understanding of these diseases, we can help create a more supportive and inclusive environment for those affected.

Thank you to all those, including our friend Lilly Stairs, Autoimmune Association board chair, who use their voice to bring awareness to autoimmune disease all year long.

Raising Awareness During March Autoimmune Awareness Month - National Health Council Raising Awareness During March Autoimmune Awareness Month By: Molly Murray, President and CEO, Autoimmune Association Autoimmune Awareness Month is observed every March with the aim of promoting awareness and understanding […]

Thank you to Karen A. Jones and the wAIHA Warriors for inviting the PAS team to its inaugural annual meeting this past weekend in New Orleans.

We were able to learn from dozens of patients, caregivers, physicians, and researchers to help better understand the wants and needs of people living with warm Autoimmune Hemolytic Anemia (wAIHA).

Having played an instrumental role in wAIHA Warriors creation - PAS CEO Mike Walsh was honored to receive the Founders Award. Mike shares:

"When PAS set out to galvanize the fragmented wAIHA patient community, we knew there was a clear need for this patient population to become connected to one another, and we knew launching the wAIHA Warriors at the American Society of Hematology conference in 2020 was a significant step forward in that pursuit. To witness the passionate engagement and support for one-another was powerful, and the great work Karen Jones and the team are undertaking will no-doubt improve the lives of patients and families living with AIHA across the world."

We know patient-centricity is a non-negotiable in rare disease research – but how often do we remind ourselves why?

Richard Scheyer, Medpace VP, Medical Affairs, Neurology, highlights critical insights that only the rare disease patient community can offer, including:

◾️ They can share what matters most to them — Is it the symptoms that clinicians think are important, or are there other symptoms that are actually more important?

◾️ They can help identify what to measure and when to measure it in clinical studies.

◾️ They can help develop patient-reported outcomes if necessary when clinicians cannot utilize the established scales.

What are the most meaningful insights you’ve learned directly from rare disease patients in rare disease research? Let us know in the comments.

How important is patient-centricity in rare disease studies? In this post, Dr. Richard Scheyer discusses the importance of patient-centricity in rare disease studies. Read Now.

Medable, a software platform for decentralized clinical trials, created its Patient Caregiver Network (PCN) to help facilitate equitable, inclusive, and diverse clinical trials.

Within a 12-month span, the PCN attended more than 25 industry events and collected the following key patient insights:

◾️ Decentralized and hybrid clinical trials rose 28% from 2021 to 2022. This growth is promising for patients and caregivers as it helps reduce time away from work, family, and school.

◾ Patients and caregivers want as much information as possible from the get-go, including, at minimum, the purpose of the trial, potential risk factors, and info about the study drug itself. Additionally, they want to learn about the benefits of the drug, the overall progress of research, and have the flexibility to schedule study visits.

◾ Simplicity is key. Don’t use medical jargon, make the informed consent form as easy to understand as possible, and write at 6th grade reading level.

Learn more here:

Patient perspectives: a year of pharma webinars showcase what’s working and what needs work | Medable See patients and caregivers' thoughts on what pharma has gotten right with digital technology, and what needs more work in this blog.

The PAS team was honored to run in recognition of 👟

In recognition of , we want to spotlight PAS's senior director of engagement and operations, Josh Argall, and his son, Devin Strong, who lives with Duchenne Muscular Dystrophy (DMD), a rare neuromuscular disease.

Since Devin's diagnosis at 3-years-old, the Argalls have become outspoken advocates, participating in a clinical trial for the first FDA-approved medication to treat DMD, and working on behalf of the Muscular Dystrophy Association, the Jett Foundation, Parent Project Muscular Dystrophy and EveryLife Foundation for Rare Diseases, fighting for treatments and cures for families impacted by rare disease and DMD.

"I'm dedicated to serving and protecting the DMD community, and the advocacy work I’m engaged in helps me do that. I want to make sure people living with DMD can receive adequate treatments and care and can access all the things that anyone else can," shares Josh.

How are you recognizing Rare Disease Day this year. Let us know in the comments.