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These tools are described below. Before continuing, we recommend that you read our background blog on PFDD meetings and FDA resources and visit our PFDD Resources webpage if you haven’t already.

The PFDD Readiness Assessment

How do you know when your organization and your community are ready for a PFDD meeting? This self-guided PFDD Readiness Assessment is designed to help you answer that question by asking you to reflect on the knowledge and capacity of your organization and the context of therapy development for your community. To use the Assessment, answer each question and tally your points as you go. At the end, evaluate your status using the scoring key. We’ll continue to update the Assessment with links to new resources.

PFDD Meetings: Smart Practices from Patient Community Leaders

This extensive guide details common practices, lessons learned, and considerations for planning PFDD meetings. The guide is based on interviews conducted with community leaders that have planned and hosted externally led PFDD meetings, and it has been checked for accuracy by FDA. It is organized chronologically into three planning sections: Section 1: Steps to Take Before Submitting a Letter of Intent, Section 2: Planning Your PFDD Meeting, and Section 3: Preparing for Post-Meeting Outcomes. Each section includes topics relevant to that planning period depending on where you are in your planning.

The PFDD Community Toolbox

Many organizations developed tools and resources for PFDD meetings during their planning process. The PFDD Community Toolbox is a collection of materials that were donated by organizations that have hosted meetings. You’re encouraged to reference or adapt these tools for your PFDD planning purposes.

A Note on These Tools

FasterCures developed these three tools based on internal expertise and interviews with patient advocacy groups that have hosted PFDD meetings. While FDA provided input on the accuracy of the content in the Smart Practices guide, all of these tools represent the perspective of FasterCures alone and do not necessarily reflect the perspectives of the organizations involved or the FDA. These tools do not set standards and will continue to evolve along with patient-focused drug development.

Regulating software products

FDA regulates thousands of medical devices—everything from popsicle-stick tongue depressors to electrical brain implants. The swell of digital health is creating waves of new and complex software technologies, such as mobile medical applications. However, the FDA’s current regulatory process is not built to accommodate the fast-paced, iterative development that software products go through. Imagine if the weekly updates for the apps on your phone had to each go through the FDA approval process—app developers would be discouraged, and the FDA overwhelmed.

To avoid delays in getting new software products to patients while ensuring the application of the same standards of safety and effectiveness to all devices, FDA is developing a new regulatory approach that will first be tested on one type of software called software as a medical device, or SaMD. SaMD is software used for a medical purpose—to treat, diagnose, drive, or inform clinical management that operates independently of hardware. For example, the software internal to an infusion pump that controls the motors and the dispensing of medication is not SaMD because it is a part of and necessary for the pump to work. However, the software of a mobile app on a smartphone that analyzes images from an MRI to diagnose a patient is SaMD since that same software could also operate on your laptop.

General wellness apps are not SaMD since they are used to maintain or encourage a healthy lifestyle and not used to treat, diagnose, drive, or inform clinical management.

FDA launched the Digital Health Software Precertification (Pre-Cert) Pilot Program in mid-2017 and revised the model based on public feedback, with a second version, v0.2, released in June 2018. To ensure that patient perspectives inform the development of the program, on Nov. 27, 2018, FasterCures convened a small group of patient representatives to provide their perspectives on this pilot program to FDA staff. On January 7, 2019, FDA released a new working model 1.0, a test plan describing how FDA intends to iterate the model, and regulatory framework for conducting the pilot program.

About the Pre-Cert Program

The Pre-Cert Program evaluates software developers on whether they meet certain standards of quality and organizational excellence. Software developers are pre-certified if they demonstrate excellence in five principles: (1) product quality, (2) patient safety, (3) clinical responsibility, (4) cybersecurity responsibility, and (5) proactive culture. Pre-certified developers must also have a robust method for collecting and analyzing real-world data, and they must commit to monitoring these data to assure product safety and effectiveness.

Pre-certified developers can bring their SaMD products to market either without pre-market review or through a streamlined pre-market review. Which pathway a product takes depends on (1) the risk category of the product, (2) the level of pre-certification of the developer, and (3) whether the product is a new device or an iteration of an existing device. After entering the market, developers must follow the product’s real-world performance by tracking (and sharing with FDA): (1) real-world health analytics (e.g., clinical safety and health benefits), (2) user experience analytics (e.g., user satisfaction), and (3) product performance analytics (e.g., cybersecurity).

Comments from Patient Representatives

During FasterCures’ convening, patient representatives described their individual views on the pre-cert program; the meeting was not meant to elicit a consensus of views. Below is a selection of comments we heard during the discussion about elements of the Pre-Cert Program:

Excellence Certification Assessment:

Companies should be evaluated on their patient engagement efforts and should be encouraged to engage patients earlier in the development pathway for medical devices. FDA should include guidance for companies and patients on what specific actions meet the excellence standard for patient engagement.

The proposed organizational elements listed in Appendix B (of working model v0.2) to demonstrate excellence resonate but may be difficult for a lay audience to understand.

FDA should consider the disease state when creating measures to establish excellence. For example, for patients facing a decline in function, holding steady or even a small decline in function could be a benefit, so the measure should not require improvement.

Healthcare provider understanding of the product is important, especially for some diseases where the healthcare provider may be a user as well.

FDA should make it clear to patients and other users if only a part of a company is pre-certified or different parts of the company have different pre-certification ratings. The pre-certification could be related to each product to try to make this clear.

FDA should give guidance to developers on what the company can and can’t say about its pre-certification status (which might differ among products), to avoid the public assuming all products from a pre-certified company are pre-certified.

Excellence appraisal should include whether the company proactively engages with users to identify issues and how it reports (including to users), abets, and resolves product issues to them.

Real-World Data Monitoring:

User experience is important in assessing safety. System downtime and customer satisfaction are not the same things; clinical safety and accuracy are not the same things.

Patients want companies to reach out proactively to users regarding known issues.

Many device errors currently go unreported because of the burden of reporting on users, so companies should try to reduce the burden on patients to report errors. Companies should tailor error reporting for different patient populations (e.g., reflect any physical limitations of that population).

Patients expressed that cybersecurity is a top concern given that the correct performance of these devices and the protection of user privacy is very important.

Product performance is the most important of the three listed real-world data domains (the other two are user experience and real-world health).

FDA should develop monitoring requirements that are flexible enough to allow a product on the market (and stay on the market) even if it is only helpful to a subset of the patient population (i.e., it doesn’t meet the needs of all users).

 Transparency:

FDA should define transparency and encourage companies to share data with patients in a format they can use. FDA should also consider data access granted to healthcare providers and the complex webs of data flow between SaMD products and electronic health records.

Algorithmic transparency is important; patients should be able to know the inputs of an algorithm (in a way that does not compromise intellectual property protection).

Companies should tell users when they recognize inaccuracies or flaws in their products and that they are working to fix them. It is also important for companies to relay to users why they make changes to the product.

FDA should make sure that the description of its pre-certification approach and review process (on its website and other materials) should be easily located and described in lay terms for the public. The description should balance simplicity and depth: simple explanations and labels should build awareness for all consumers, while comprehensive information should be easily obtainable for consumers who want it.

How to Voice Your Opinion to FDA

o Patients and caregivers hope that using patient-centered PROs will advance medical products that present better treatment options and/or can be used to inform care decision-making.

o Drug and device companies use PROs to demonstrate impacts of medical products that matter to patients and/or to differentiate their products from others in the same market.

o Clinicians frequently use PROs to assess how well individual patients feel or function as a basis for shared decision-making about care.

o Hospitals and clinics use PROs and other tools to understand clinical care outcomes or the way particular care approaches affect patient health and health-care utilization at the population level.

o The Food and Drug Administration (FDA) can use PROs, as long as they meet certain data requirements, as part of its benefit-risk determination and as evidence of unmet medical need.

o Payers use comparative and cost-effectiveness data, which can include PROs, to make reimbursement decisions.

At the end of 2017, FasterCures surveyed organizations that are using patient-centered PROs in their patient registries. The goal was to better understand the role patient organizations are playing in advancing the use of PROs.

Methods

We conducted the survey through the Patients Count Network, a digital directory of patient organizations maintained by FasterCures. Patients Count Network involves nearly 150 patient organizations of all sizes and areas of focus. The network serves as a one-stop shop to find patient organizations and as a mechanism for these organizations to showcase their programmatic and research resources.

We sent a survey on patient-centered measurement to 142 current members in late 2017. The goal was to better understand the types of work that they are doing related to PROs, who they are partnering with, and the purpose of their efforts. Thirty percent of organizations (42) responded. Most of the organizations are small, with 10 or fewer staff.

 Conclusions

Three-fourths of patient organizations surveyed are working on PROs.

Patient organizations are using their resources in multiple ways to advance the development and use of PROs. They indicated many uses for PROs and patient-perspective data and are partnering with different organizations and stakeholders to accomplish their work. Patient organizations are positioned to work within and possibly across diseases and conditions and may be an underappreciated partner in the development and use of patient-centered measurement.

Based on our findings and our prior work in this area, we recommend three key actions to drive patient-centered PROs forward:

1. Collaborate: Find the patient organizations that represent your disease area(s) of interest. They may already be doing great work, but if not, work with them early and often to make their needs and perspectives a starting point.

2. Find success and build on it: Where have PROs been successfully aligned with patients and effective for decision-making in R&D or care delivery? Don’t recreate the wheel, but do make sure existing PROs are aligned with what patters to patients. 

3. Consider technology: Electronic delivery of PROs and technology to manage and make use of the data are going to be critical aspects of the future viability and success of PROs.

For more of FasterCures’ work on patient-reported outcomes, check out the following resources:

· Webinar: Patient-Centered Measurement: How Close Are We?

· White paper: Patient-Reported Outcomes: Design with the End in Mind

· Blog post: Patient-Reported Outcomes and the Elephant in the Conference Room

Kyle Brown of PatientCrossroads noted that the conversation about registries has changed quickly in the last several years. He used to have to start presentations by explaining what a patient registry is.

Brown's passion is "knocking down silos to make the data more useful for everybody," he said. His company partners with many groups, from academics to "two moms sitting around the kitchen table." One of those moms, Megan O'Boyle of the Phelan-McDermid Syndrome International Registry, has been working with Brown for six years. O'Boyle was persuaded by another parent to go to a meeting about patient registries at the National Institutes of Health because she happened to live closest. That one meeting turned into a six-year passion. Of the 1,300 diagnosed cases of Phelan-McDermid Syndrome in the world, 844 are in the registry, and one-third have uploaded records. The registry was recently awarded a Patient-Powered Research Network grant from the Patient-Centered Outcomes Research Institute (PCORI), "which has catapulted us into the world of big data," said O'Boyle.

Another registry that has received PCORI funding is Citizen Pscientist, which is run by the National Psoriasis Foundation. Leah McCormick Howard explained that users can contribute data, run queries, and analyze and discuss findings.

Jaye Bea Smalley of PCORI, whose organization was established through the Affordable Care Act, aims to produce high-integrity information and answer questions that are important to patients and caregivers. PCORnet, the National Patient-Centered Clinical Research Network, works to increase the speed at which information is produced. "Engagement is key," said Smalley. "Collaboration is a major part of this network."

These collaborations are opening doors to new ways of collecting and using data.

O'Boyle pointed out that 97 percent of the families in her registry are interested in participating in clinical trials. Through the registry, her foundation can directly contact those families who have identified particular symptoms, such as seizures. And the families feel that getting their aggregated data back is a great reward.

Howard agreed that people are more willing than ever to share data. She has noticed that, in their online platforms, her disease community encourages people to use the registry. "Our registry lets our community play a role in research that matters to them," she said. In addition to connecting users, Smalley said, registries can add value by distributing data in an understandable language. 'It's about creating value for people."

According to O'Boyle, six years ago clinicians thought patients weren't qualified to answer their own questions. Now they recognize that patient-reported data are legitimate. "There is so much missing from an electronic health record," echoed Brown. "You have to have patient input."

"Having complete data is key," agreed Smalley. If you are creating data that will be used by researchers and clinicians, for example, those groups need to be involved in the registry.

Brown added that more data are not better, especially if it's fragmented. "More data in one place that people can access is better," he said. For example, there are 27 pharma companies doing research for muscular dystrophy, and it doesn't make sense for all of them to have their own registries.

"The biomedical research paradigm has changed," said Margaret Anderson, executive director of FasterCures. “The innovation connectivity sweeping the world is showing us a path forward. That and just plain bold, disruptive, and tenacious efforts by everyone in the system."

Francis Collins, M.D., director of the National Institutes of Health, noted, "We are at a remarkable moment in biomedical research, where we're figuring out the fundamentals -- how life works and how disease occurs. Now we need to turn this opportunity into medical advances -- health promotion, disease prevention, and treatment of illness when it strikes."

Recognition of the growing demand for patient input echoed across the meeting. The healthcare establishment, which has been slow to accept that personal data have value for research, is beginning to embrace elements of the consumer revolution, and find effective ways to more meaningfully integrate patient perspectives into drug development and regulatory decision-making.

Dana Ball, Executive Director and Co-Founder of T1D Exchange and CEO of Unitio, Inc., remarked, "There is now substantial evidence about what patients need, where they see value, and what they're willing to pay for."

Punctuating the value of patient engagement and cross-sector collaboration, FasterCures today released the following new resources:

Consortia-pedia - FasterCures analyzed more than 400 medical research consortia around the globe, and found that they address 165 diseases and engage 3,000 unique sponsors and partners. Nearly half of those consortia develop tools, 94 of them conduct biomarker research, and 135 engage in data sharing. These consortia include collaborations such as the more than $5 billion Innovative Medicines Initiative, the National Institutes of Health-driven Accelerating Medicines Partnership, and the pre-competitive Biomarkers Consortium. Find this first-of-its-kind free, searchable, online resource at www.consortiapedia.fastercures.org.

The Power of Ideas - Fifteen compelling essays that include David Panzirer's candid take on why he's determined to find a cure for Type 1 diabetes, Anne Wojcicki's vision of what it means to have a consumer-driven healthcare revolution, and Tom Insel and Kafui Dzirasa's insights on redefining mental illness. This collection of big ideas will deliver a good dose of optimism grounded on innovation, resiliency and purpose.

Patients Count: the Science of Patient Input - Learning from health economics, marketing and engineering, a new science of patient input has emerged, one that embraces data as a means for measuring patient-centered outcomes and quantifying patient preferences. Today, FasterCures released From Anecdotal to Actionable: The Case for Patient Perspective Data, which presents a model for advancing the collection and application of Patient Perspective Data, along with initial ideas for how such data might inform drug development and regulatory processes.

Held at the Grand Hyatt New York, Partnering for Cures is open to the public and to members of the media (registration is required). For general registration information, go to www.partneringforcures.org/registration. Members of the press may apply here to receive complimentary registration.

The Partnering for Cures meeting consists of panels on hot-button issues, presentations about compelling personal experiences, facilitated working sessions, town hall-style sessions, informal conversations among creative innovators, and more. Watch videos of today's sessions and check out tomorrow's program at www.partneringforcures.org.

About FasterCures

FasterCures, a center of the Milken Institute, is an action tank, determined to remove barriers to medical progress. We have only one goal: to save lives by speeding up and improving the medical research system.

www.fastercures.org; @FasterCures

About the Milken Institute

The Milken Institute is a nonprofit, nonpartisan think tank determined to increase global prosperity by advancing collaborative solutions that widen access to capital, create jobs and improve health. It conducts data-driven research, convenes action-oriented meetings and promotes meaningful policy initiatives.

www.milkeninstitute.org; @milkeninstitute

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