#fastercures

Rye provided an overview of FasterCures’ work in this area. The core issue, as Rye described, is that there are several channels for developing promising therapeutics that may not be a strategic fit for an organization or have uncertain commercial value—such as those with unclear reimbursement, weak patent protection, or small patient populations—even if there is promising science. Though these therapies could impact lives, they struggle to garner the needed investments, are cut from industry pipelines, and are fated to sit on a shelf unused.

FasterCures and the Center for Financial Markets, two centers of the Milken Institute, are collaborating to identify new pathways to capitalize the development of these underinvested therapeutics. They are investigating what an “ideal” market for these compounds might look like, how society could unlock fresh capital for innovation, and how new business models can be scaled to the ecosystem.

To open the market for new forms of capital, compounds must be out-licensed between organizations more freely. Traditionally, out-licensing has been a pharma-to-pharma transaction. But other transactions are possible: pharma to nonprofits, to spin-outs, to government agencies, or to virtual drug developers. New players bring new capital sources which could lower the cost of developing new therapeutics.

Dr. Freda Lewis-Hall: Pfizer; Board Member of SpringWorks Therapeutics

Lewis-Hall spoke about her experience with the Pfizer portfolio and the creation of SpringWorks Therapeutics, a drug development company that was spun-out from Pfizer.

Pfizer’s portfolio is large and includes internal R&D, in-licensed compounds, integrations, and collaborations with academic researchers. Such a large portfolio needs to be concentrated on funded, innovative, and strategic assets. Inevitably, many compounds fall outside of Pfizer’s strategic focus and remain unfunded. But, if these unfunded compounds are pharmacologically active, do not present safety concerns, fail their original indication, and have the potential for “off-strategy” indications, then they can be available for strategic partnerships. Since Pfizer has many ongoing partnerships, including collaborations with foundations, NGOs, academia, and venture philanthropists, its executives began to ask if Pfizer could create a fit-for-purpose model to advance deprioritized compounds.

SpringWorks was launched in September of 2017, with a portfolio of compounds in-licensed from Pfizer across several therapeutic areas. It was founded with financial backing from life sciences investors and is led by an experienced team of accomplished drug developers. As Lewis-Hall phrased it, when you put the right assets and the right people in the right place, you create a “sweet spot” for advancing therapeutics. SpringWorks expects to start to pivotal trials with two compounds from its portfolio in the first half of 2019. Both compounds are for rare oncology indications—neither of which currently have an approved therapy. According to Lewis-Hall, SpringWorks is “the beginning of a proof-point that is putting the financial and human resource investments in a novel mechanism, giving an opportunity to speed these therapies to patients with unmet need.”

Dan Liljenquist: Civica Rx

Dan Liljenquist provided an overview of Civica Rx and its efforts to address the shortages and monopolies in the generic drug market. Medication shortages, largely driven by consolidation, has affected many hospitals around the country by disrupting the supply chain for key drugs and created instability. Civica Rx, which Liljenquist describes as a democratized utility company for the manufacturing of generic drugs, will have the important role “to bring competition to the generic drug market focusing on value, price, and quality, and specifically to bring competition to areas… where there is a dominant player that has essentially cornered the market for a particular [generic] compound that we rely on for patient care.” Civica’s three chief objectives are:

Focusing on value and invigorating competition in the market for generic drugs

Ensuring a stable and predictable supply of essential generic drugs to correct shortages

Serving as a check against the aggressive pricing behavior of some generic drug manufacturers

According to Liljenquist, Civica Rx approach will be to establish fair, sustainable, and transparent pricing at all times, with no fees or rebates. The company also intends to become an FDA-licensed manufacturer, offering long-term, guaranteed contracts to hospital systems, with redundant manufacturing and safety stocks to avoid shortages and ensure the continuity of patient care. Finally, Civica Rx will implement a labeling system that, for transparency, indicates where a drug was manufactured.

Since the initiative was announced in January 2018, Civica Rx has received generous amounts of interest, news coverage, and support. In fact, one-quarter of the U.S. hospital market has signed agreements with the company. Liljenquist expects that the company will have its first 14 drugs on the market this year. Furthermore, Liljenquist believes that Civica Rx’s democratized utility model for the manufacturing of generic drugs can be scaled and replicated to other parts of the market.

Q&A

Partnering with patients

When asked how SpringWorks has worked with patient organizations, Lewis-Hall explained that patient input was helpful for the process of choosing compounds and therapeutic areas to advance. There are a variety of considerations in this process aside from just commercial potential, such as the regulatory environment, how to successfully measure outcomes in clinical trials, and how to identify the patient population, especially in the case of rare diseases. Patient organizations have been integral for SpringWorks to prioritize and balance such considerations.

Liljenquist addressed a question about whether the Civica Rx model passes on savings to patients. He said that Civica Rx would publish its market prices with the goal of driving transparency. Patients will be able to see if their health system has marked up the price of a drug.

Partnering with philanthropy

Lewis-Hall and Liljenquist both addressed the role that philanthropy has played for their organizations. In Lewis-Hall’s view, philanthropic organizations and foundations bring value not just for their financial contributions, but for their subject matter expertise, convening power, and relationships with patient groups and other stakeholders. As the system shifts from traditional models of collaboration to a more thoughtful “all hands on deck” model, the deep knowledge and expansive networks of philanthropic organizations will help advance therapies.

Liljenquist said that Civica has incorporated philanthropists onto their board for many reasons, but in particular, to ensure that in the future the capacity being built at Civica Rx will not be monetized by its members.  

The path forward

Responding to a question about what is needed to scale current efforts and create more innovative models, Lewis-Hall noted three things that she would like to see accomplished:

A network or system for facilitating knowledge sharing and connecting different stakeholders so that potential investors would know what assets might be available for investment

A cultural shift in the way we think about creating collaboration in what has traditionally been a go it alone environment. Scientists and industry may be reluctant to release their work to other parties for collaboration and further advancement, but this needs to change.

A recipe or algorithm to discover what types of models fit specific development needs. Variables that might be included, for example, include information on the patient community, unmet therapeutic needs, potential investors, and available assets. The most effective collaboration structure would output a series of opportunities and models that could be a good fit.

Conclusion

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

Background

FasterCures, a center of the Milken Institute, has had a longstanding interest in philanthropy as a source of funding for innovation in medical research. People sometimes wonder why, since philanthropy regularly represents only 2-3 percent of the U.S. investment in medical research every year. However, while philanthropy may be small in size, it can be unique in its role as a source of capital. Like venture capitalists, venture philanthropists are inclined to fund novel, high-risk research. They view philanthropy as a form of capital that can afford to take risk—particularly in the early stages of R&D where costs are relatively low but risks are relatively high.  

For the last dozen years, FasterCures has convened a network of patient-driven foundations that fund medical research and share a desire to be strategic and outcomes-driven with their funding and other resources. Our TRAIN (The Research Acceleration and Innovation Network) program has been a platform for these organizations to share information and best practices and for networking with other stakeholder groups. It also provides insights into the systemic challenges and unmet needs in the biomedical innovation ecosystem that foundations are trying to understand how to address with scarce resources.  

Private Session Discussion

Participants in the conversation at the Future of Health Summit heard an overview of existing philanthropic investment models that are working. . For example, over the last 11 years, the Leukemia and Lymphoma Society’s Therapy Acceleration Program has sought to de-risk the path for young companies by investing $120 million in 60 programs, with two U.S. Food and Drug Administration (FDA) approved products and a third on the way. Funding opportunities go through a very rigorous due diligence process, and terms are flexible depending on the company (e.g., convertible notes, equity, collaborative licensing).

Baiju Shah, CEO of BioMotiv, part of the Harrington Project for Discovery and Development started by the philanthropic Harrington family, provided a different perspective. Harrington features a nonprofit academic granting arm and a for-profit accelerator arm (BioMotiv) and is not disease-specific. It brings not just financial investment, but also development expertise and partnerships. This approach is useful in getting the assets that it invests in across the stages of translational research, where is there a high risk of failure, and into clinical development, which Shah described as “an effective machine for moving really exciting academic discoveries to the point where patients are benefitting from them.”

Participants also discussed several emerging models. Efforts to create hybrid funds, pooling nonprofit and for-profit capital, or staging different types of capital, have been tried in the past but now appear to be gaining traction. For example, Ed Walters and his partner, David Bates, of Tamarisc Ventures have been advising a family office on the creation of a philanthropic entity focused on mental and behavioral health. It is structured to maximize flexibility as a charitable trust with a for-profit LLC and private foundation built in. The foundation can be funded annually, as well as the investment capital for the LLC, and everything eventually flows back into the charitable trust. During the session, Walters explained, “We spent a lot of time asking our network and other experts out there for advice on what the optimal structure was and, to be honest, we're still looking,”, which produced knowing nods from many around the table.

Lisa Ryerson of AARP explained the well-known advocacy organization’s interest in supporting ventures that benefit the aging population, including as a significant investor in the Dementia Discovery Fund, as well as other social impact enterprises. This led to a discussion of the availability of new players to help scale venture philanthropy approaches. Finally, Garen Staglin, founder and chairman of One Mind, gave an overview of a new effort to create a large-scale social impact bond in collaboration with stakeholders such as the World Bank and the Society for Neuroscience. This endeavor will put more resources behind R&D in the high-risk area of neuroscience with the aim of alleviating the enormous economic and social costs of mental health disorders around the globe.  

As individuals in the private session made clear, philanthropy is playing an important role in high-risk therapeutic areas such as neuroscience and rare disease, as well as in particular stages of R&D such as translational science and early-stage clinical research. Later-stage clinical development is also an area in which new hybrid business models are incorporating elements of philanthropy or social responsibility. The group heard from Freda Lewis-Hall, Chief Medical Officer at Pfizer and a member of FasterCures’ advisory board, about a new business model that harnesses socially responsible investing to develop compounds that have been deprioritized by companies but that may still have the potential to be developed into new treatments to address unmet patient needs. “The question was, can you build a fit-for-purpose business model that allowed you to get these late-phase compounds developed as quickly as possible and into the hands of the patients that need them?” One approach described by Lewis-Hall was the creation of SpringWorks Therapeutics, a benefit corporation, which was initially funded by a group of other socially responsible investors, including Pfizer, and which was launched with rights to four compounds licensed from Pfizer. SpringWorks also has partnerships with patient organizations around individual assets.  

Following these discussions, several critical points on the key questions raised at the session’s outset were discussed:

1. Why philanthropy?

It can play a unique role in de-risking promising research for further investment.

Philanthropies can provide value other than financial resources, such as access to key opinion leaders, development expertise, and commercial partnerships.

Philanthropies tend to take a systemic approach and look for levers of change.

By nature, philanthropy can have a lower expectation of financial return than other investor types.

Two interesting questions that surfaced were:

Is there an important distinction between “venture philanthropy” and “impact investing”?

Does investing philanthropic capital potentially dissuade other types of market capital from coming in early or introduce undesirable conditions into the equation?

2. What are the challenges of venture philanthropy? 

Identifying optimal deal structures is hard.

Identifying/sourcing opportunities for investment can be challenging, especially in non-disease-specific contexts.

Philanthropy has a natural interest in openness and sharing, which can come into conflict with intellectual property and other professional/commercial considerations.

Financial and organizational capacity at philanthropies to do this kind of deal-making is usually limited – though success helps build capacity.

As health-care costs continue to rise, many patient-driven organizations are feeling pressure to try to exert influence on pricing and access.

There is little agreement on what reasonable and appropriate return on investment is.

Interest areas of foundations/philanthropists can be narrow.

3. How can these approaches be scaled?

Existing models and best practices should be more widely shared—there is no need to reinvent the wheel.

New players such as AARP could turn their attention and resources to investing in medical research.

A mega-fund of research projects could be securitized for investment.

Setting the stage for data and precision medicine

FasterCures Executive Director Tanisha Carino opened the discussion by explaining why the work of the Accelerator is significant to FasterCures and the health-care field at large.

The role of personal health data is critical to any effort to advance precision medicine. Over the last year, FasterCures has introduced Health Data Basics, a project developed to engage patients with their health information and to help enable the two-way flow of health data between patients and the health-care system. Despite the numerous sources of health information being gathered at regular intervals, there are currently many challenges for patients and caregivers; the majority of individuals still do not have the resources to fully utilize their data in meaningful ways.

Carino introduced webinar speakers Kathy Giusti (Executive Chairman, Board of Directors, Multiple Myeloma Research Foundation [MMRF]; Senior Fellow and Co-Chair, the Kraft Precision Medicine Accelerator at the Harvard Business School) and Gabriel Eichler (Founder and Managing Director, Oak Health Partners; Data & Analytics Workstream Lead, Kraft Precision Medicine Accelerator at the Harvard Business School).

Giusti has been a friend of FasterCures for over a decade. The MMRF, which Giusti founded, is a pioneer in the field of venture philanthropy and has been involved in FasterCures’ network of organizations innovating in that space.  

Eichler has spent the last 15 years working to apply health-care technologies to problems in the pharmaceutical and biotech industry and the delivery of health care. He was formerly the vice president of products at GNS Healthcare. Prior to that, he managed several global client programs at PatientsLikeMe, focusing on patient-centered research and real-world outcomes.

An introduction to the Kraft Precision Medicine Accelerator

The overall goal of the Accelerator is to focus on how new incentives can drive great models in precision medicine. Giusti and her team started their work by looking at the greatest roadblocks facing precision medicine. Their hope is to devise sustainable solutions to these issues and disseminate them through impactful groups such as FasterCures in order to build a strong landscape of organizations innovating in precision medicine.

After many meetings with leaders in precision medicine to decide where they should focus their time, the Accelerator identified four highly integrated work streams: Direct to Patient, Data & Analytics, Innovative Trials, and Investment/Venture. Giusti explained that all four work streams are highly integrated. The Accelerator seeks to bring together many voices to focus on these four areas of need and collectively push precision medicine forward.

Analyzing the Accelerator’s real-world data landscape in oncology  

Eichler’s presentation was broken into two parts: 1) an overview of the Accelerator’s real-world data landscape and 2) a look at data analytics companies doing innovative work in the space.

To set the stage for his work in the Data & Analytics workstream of the Accelerator, Eichler made note that a useful recipe for anyone looking to create data-driven insights is: a well-defined question + a rich data source + an aptly applied, innovative analytical method. What comes out of this model is not answers, but hypotheses. Eichler stated that these hypotheses are useful in and of themselves and that no one walks away from these projects with all the answers, but rather a new place to start.

In 2016, the Accelerator looked at sources of real-world data across the landscape of oncology. Since then, many things have changed in the space—new data assets have been made available, new registries and platform trials have been started, and massive investments have been made in oncology. As such, the Accelerator decided to look at the landscape again in 2018.  

They focused a little differently while conducting the 2018 landscape and were interested in who is integrating molecular, clinical, and longitudinal data assets in the oncology. While talking to Academic medical centers (AMCs) and organizations collecting data, Eichler had an insight. Many people think of AMCs as having neat, accessible, clean, and ready-to go data assets. The reality is that we should think of AMC data more like a field of wild flowers rather than a pre-made bouquet—disbursed, complex, and often inaccessible. Eichler noted that funding is sometimes necessary on the AMC side to reap and harvest this data to be able to answer real research questions.

Overall, the Accelerator team noted several reflections while comparing the 2016 landscape to 2018’s:

Real-world assets are growing in size and sophistication: And patient registries have some of the richest data on specific diseases.

Sharing and linking models are winning: Acknowledging the need to link is a real positive in the field.

Willingness to share and link data are rising: While there may be technical and privacy issues to address, many companies are moving to this methodology.

The frontiers of real-world data are advancing: There are improved data standards and patient re-contact rights and mechanisms that are enabling additional research opportunities.

Leaders among real-world data analytics companies

Data analytics companies have been thrilling to watch in recent years. There is tremendous promise for what AI can do in health care, but Eichler warned that despite the barrage of headlines indicating an exciting future for the space, we should be careful not to get ahead of ourselves. To get an accurate take on what these companies are working on and indicate the emerging leaders in the field, the Accelerator put together a landscape of the top data analytics companies.  

The objective in building the landscape was to reach stakeholders (investors, researchers, non-profits, hospitals/care centers, payers, and patients) with key questions (Where are advanced analytics being most applied? Whom should I invest in? Is it best to build vs. buy vs. partner? When may these technologies be ready for real-world applications?).

They broke down this space into three key areas where AI is being applied (pre-clinical R&D, clinical research, and clinical care) and looked at 46 companies that met their criteria.

After creating this list, they looked at how these organizations were experiencing or demonstrating recent acceleration in their business through financing, job growth, and open positions. They also looked at the establishment of the organizations including publications, partnerships, and overall financing.  

While looking at how established these organizations were and how they were accelerating, Kraft noted the diversity in competencies of companies at different levels of maturity and development. When segmented out on the two-by-two matrix above, it was clear that some companies are industry-standard, highly established organizations, but are not accelerating rapidly. Others are just emerging, having tremendous growth but without much establishment.

Among these patterns, the Accelerator was most interested in companies that reside in the top right-hand quadrant of the above matrix: Hot Companies. These organizations have size and acceleration. Eichler commented that they are the most interesting companies to watch because they are breaking out of the mold and innovating in exciting ways.

Eichler detailed their key findings from this landscape:

Tremendous investments: These companies have raised more than $1.4 billion.

Diverse platform capabilities: Many companies are applying technologies to multiple use-cases.

Paradigm-shifting potential: These companies are young and will take a few years to mature; they have the potential to define the type of stories that will be use-cases for the future of health care.

Where precision medicine is going next

The webinar closed with some time for questions and answers, primarily revolving around the actionable next steps for those funding, conducting, or investing in research and development.

Giusti reiterated that the Accelerator is a huge believer in the data space and where it is going. In order to validate what they want to keep working on, they have to know what other data sets are still out there and whether AI companies have the resources to work with them. Giusti said that the field is in constant need of new data and new AI companies.

Eichler commented that all organizations, especially philanthropies, are trying to figure out their role and how they can bring real-world data into the mix. There is no shortage of challenges in trying to define the right questions to ask. The work that MMRF and others are doing to define, prioritize, and ask the key questions in their disease are going to unlock the most progress in helping patients.

In closing, Carino reminded the audience that precision medicine is where all diseases are headed. The Accelerator and FasterCures will continue their work to catalyze innovation in this space and look forward to collaborating on future endeavors to benefit patients everywhere.

Additional 

 Visit FasterCures’ Health Data Basics project

Learn more about the Kraft Precision Medicine Accelerator

Watch an archived recording of the event and view presentation slides

Take the Pledge

The Pledge to Patients is an easy but significant step that all individuals, no matter their role in the health-care ecosystem, can take to acknowledge their commitment to patient engagement and join an accountable, global community that is driving this work forward. The pledge was created and endorsed by PFMD members. Members of PFMD are institutions or organizations that are eager to see a better future for patients and are working to drive impactful patient engagement worldwide. The Pledge to Patients was founded on the belief that small, individual action can lead to long-lasting, widespread change.

Through this pledge campaign, our goal at FasterCures is to help PFMD get to 1,000 pledges by October 5. PFMD’s goal is to engage the entire biopharma ecosystem worldwide to support the advancement of patient engagement and bring the reality of better solutions and outcomes to patients.

We encourage you to take the pledge and commit to joining the cause of advancing patient engagement and changing the health-care system for the better. Visit the pledge records page to see how others in the field have committed to the movement, and feel free to share with your networks. We are eager to see how this group of like-minded pledge-takers, who understand the power of patients, will shape the future of how medical products are discovered, developed and delivered.

Expanding the Patients Count Resource Library

In addition to sharing the Pledge to Patients, our partnership with PFMD will allow us to expand our Patients Count Resource Library through PFMD’s SYNaPsE platform. This platform is an incredible resource that gathers patient engagement initiatives in a searchable database, allowing individuals and organizations to connect as they seek to push the field forward. In the coming months, FasterCures will transition our resource library into the SYNaPsE platform; PFMD will host the library moving forward.

We are excited about this partnership with PFMD and the opportunity to multiply our patient engagement efforts on a global level. Stay tuned for updates about the resource library transition and the outcome of our Pledge to Patients campaign!  

From our unique vantage point within the ecosystem, we set out to understand the landscape, share best practices, and highlight the essential questions to answer for anyone interested in establishing new collaborative efforts, expanding existing ones, or re-orienting early-stage programs. This resulted in a 2013 Science Translational Medicine paper, Consortium Sandbox: Building and Sharing Resources, and FasterCures’ Consortia-pedia report. In researching, understanding, and writing about these collaborations, we collected information about nearly 500 consortia around the globe and made them public through the Consortia-pedia Catalogue.

Since its debut four years ago, the Consortia-pedia Catalogue has had more than 16,000 unique visitors from 124 countries.

The most popular profiles (and their areas of focus) have been:

1.     Beta Cell Biology Consortium (Type 1 diabetes)

2.     Molecular Diagnosis and Risk Stratification of Sepsis Consortium (Sepsis)

3.     Accelerating Medicines Partnership (Alzheimer’s disease)

4.     Clinical Decision Support Consortium (electronic health records)

5.     TB Drug Accelerator Program (tuberculosis)

The top terms users have searched for have been:

1.     Alzheimer’s

2.     Cancer

3.     Pfizer

4.     Biomarker

5.     Multiple sclerosis

We are thrilled and thankful for the attention and support of all who have used the resource, promoted it, and learned from it. As the field has changed in the last five years, it is also time for FasterCures’ focus to adjust once again to emerging topics. The Consortia-pedia Catalogue will no longer be updated as of August 2018. While the site will remain available through the end of the year, we will not be refreshing or adding any new profiles. If you are interested in the details behind the Catalogue, please reach out to [email protected].