Phasing Out Phase 3What if drugs were released to the public earlier, then graded on their performance in the real world?

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December 12–13, 2017 at MIT

Biomedical science is outpacing the system. Barriers prevent patients’ access to innovations.Are you ready to innovation how we innovate?

The Next Wave in Adaptive Biomedical Innovation:Advancing Platform Trials into End to End Rapid Learning Systems

Featuring keynote speakers Janet Woodcock, MD Robert Califf, MD Jonathan Gruber, PhD Sandy Pentland, PhD …and senior thought leaders from other innovation centers. 

Details and registration at

BLOOMBERG: Novartis’s $475,000 Cancer Breakthrough Heralds New Era of Cures

There are more than 600 gene and cell therapies in clinical trials today, according to Mark Trusheim, a strategic director at the Massachusetts Institute of Technology’s Center for Biomedical Innovation. Those that make it to approval are likely to come with similar costs, in part because of the complex manufacturing process required to make these treatments.

“Gene therapies are different because you’re treated once and then you’re done, as compared to regular drugs where you take them every month or every day,” Trusheim said. “There’s no mechanism for the developer to get paid every month, so the price points start to look pretty high.” He called Novartis’s...

Industry leaders, patient advocates, regulators, policymakers collaborate to identify strategies for better data use in healthcare innovation

CAMBRIDGE, MASSACHUSETTS—The MIT NEW Drug Development ParadIGmS (NEWDIGS) initiative, an international biomedical innovation “think and do tank” involving a range of stakeholders in the healthcare system, hosted a collaborative Design Lab to explore the challenges and opportunities presented by Efficacy to Effectiveness (E2E) strategies for the development and delivery of new medicines. 42 people, representing pharmaceutical companies, patient advocacy groups, regulators, payers, healthcare providers, and policymakers, participated.

The Design Lab, held July 19 and 20 at the Massachusetts Institute of Technology, was part of an ongoing NEWDIGS project called WISDOM. The project is...

MIT NEWDIGS Director and advisor speak at LIGHT Forum on AI in Healthcare

CAMBRIDGE, MASSACHUSETTS—MIT NEW Drug Development ParadIGmS (NEWDIGS) director Dr Gigi Hirsch, and program advisor R. John Glasspool spoke this week at the inaugural Leaders in Global Health & Technology (LIGHT) Forum hosted by Stanford Medical School in California. NEWDIGS is an international “think and do tank” dedicated to re-engineering pharmaceutical innovation to deliver more value faster to patients, in ways that work for all stakeholders.

Hirsch and Glasspool were members of a panel entitled, “Real World Evidence: Hype or Reality?” They were joined by Hans-Georg Eichler, MD, Senior Medical Officer, European Medicines Agency; Lloyd B. Minor, MD, Dean of the Stanford...

Industry leaders, patient advocates, policymakers collaborate to lower barriers to potentially curative treatments

CAMBRIDGE, MASSACHUSETTS—The MIT NEW Drug Development ParadIGmS (NEWDIGS) initiative, an international biomedical innovation “think and do tank” involving a range of stakeholders in the healthcare system, hosted a collaborative Design Lab to explore ways to make potentially curative—but expensive—therapies available to patients as they emerge from development. 69 people, representing pharmaceutical companies, patient advocacy groups, payers, healthcare providers, and policymakers, participated.

The Design Lab, held April 11–13 at the Massachusetts Institute of Technology, was part of an ongoing NEWDIGS project called Financing and Reimbursement of Cures in the US (FoCUS). The...

MIT NEWDIGS director Dr Gigi Hirsch and her colleague R. John Glasspool will address the LIGHT Forum on the impact of artificial intelligence and Big Data on healthcare. They will explore the evolution from an emphasis on efficacy to a focus on value, and how all healthcare stakeholders can collaborate to improve outcomes for all parties. Learn more about the Forum at

Regulatory and business leader will help accelerate system-wide biomedical innovation

CAMBRIDGE, MASSACHUSETTS, 1 MARCH 2017—The MIT New Drug Development Paradigms Initiative (NEWDIGS) announced today that Hilary Malone, Chief Regulatory Officer for pharmaceutical company Sanofi, has joined the NEWDIGS Executive Committee. Sanofi is a strategic partner of NEWDIGS, which is an international, multi-stakeholder consortium to design modern systems that connect scientific discovery to patient care. In her role on the Executive Committee, Dr. Malone will help lead the initiative in its drive to advance the evolution of more adaptive approaches to biomedical innovation.

“NEWDIGS welcomes Dr. Malone to the Executive Committee,” said Dr. Gigi Hirsch, Executive Director of...

Biomedical Innovation Initiative Supporting New NIH-Funded Trial Innovation Center

CAMBRIDGE, MASSACHUSETTS—The MIT NEW Drug Development ParadIGmS (NEWDIGS) initiative, an international biomedical innovation “think and do tank”, co-hosted an “Efficacy to Effectiveness Clinical Trial” Design Lab in partnership with the Tufts Clinical Translational Science Institute (CTSI) and the new Johns Hopkins University (JHU) – Tufts Medical Center Trial Innovation Center (TIC), part of the CTSA Trial Innovation Network.

The JHU-Tufts TIC, recently funded through a $25 million NIH grant, provides high-quality design and operational support to investigators conducting multi-center clinical trials. The strategic goal is to create new frameworks and practices for clinical...

Industry veteran will lead development of new decision support technologies

CAMBRIDGE, MASSACHUSETTS—The MIT New Drug Development Paradigms Initiative (NEWDIGS) announced today the hire of Dr. Kay M. Larholt as their Director of Integrated Knowledge Solutions. Dr. Larholt will lead a portfolio of activities focused on “Knowledge Generation and Use 2.0,” a critical component of Adaptive Biomedical Innovation.

Dr. Larholt is a seasoned statistical professional with over 25 years of experience in the Pharmaceutical, Biotechnology, and Medical Device industry. She has global experience with all phases of clinical trials and regulatory submissions. An experienced statistical collaborator providing strategic input to clinical program design and analysis, she...

How the US Can Beat Hepatitis C: Don’t buy the pills, buy the company
Pharma veteran and physician advance provocative solution to overcome treatment costs

CAMBRIDGE, MASSACHUSETTS—In an article published today in Forbes, Visiting Scientist at the MIT Sloan School of Management Mark Trusheim and co-author Peter B. Bach, a physician at Memorial Sloan Kettering Cancer Center in New York, propose a startling response to the prevalence of Hepatitis C and the high cost of the leading therapy: The US government should not just pay for the pills—they should buy their maker.

The pharmaceutical...

Expert views on strategies for getting new science to patients faster, safely, and affordably

CAMBRIDGE, Mass., Nov. 9, 2016—Clinical Pharmacology & Therapeutics (CPT), a cross-disciplinary journal in experimental and clinical medicine, in collaboration with the MIT Center for Biomedical Innovation (CBI) and its New Drug Development Paradigms (NEWDIGS) initiative, published a special issue today focusing on the emerging practice of “Adaptive Biomedical Innovation (ABI).”

Featuring articles by diverse healthcare system stakeholders, this special issue embodies a key principle of ABI: working across traditionally fragmented and siloed approaches to innovation to optimize the benefit and access of new medicines for patients, while ensuring a more...

CAMBRIDGE, Mass., Oct. 27, 2016 — Patient advocates, healthcare payers, industry, and academic stakeholders gathered today at the Massachusetts Institute of Technology (MIT) to map out a new strategy for designing and piloting innovative financing and reimbursement models for promising, and possibly curative, new medicines. The project is called Financing and Reimbursement of Cures in the US (FoCUS).

FoCUS will initially target three urgent medical areas—Curative Gene Therapies, Durable Oncology Treatments, and Antibiotics for Multi-Drug Resistant Infections. Working groups will use components of a dynamic design “toolkit” to tailor innovative finance and reimbursement models...

International collaboration aims to improve healthcare for people living with a rare disease

BOSTON, PARIS, 1 JULY 2016—MIT’s NEW Drug Development ParadIGmS (NEWDIGS) program announced today that EURORDIS-Rare Diseases Europe, the European Organisation for Rare Diseases, has joined its international effort to accelerate delivery of new, better, affordable therapeutics to patients faster. NEWDIGS brings together pharmaceutical and biotech companies, regulators, insurers, patient advocacy groups, and other healthcare stakeholders to design modern systems that connect scientific discovery to patient care.

“We are excited to welcome EURORDIS to the NEWDIGS community,” said Dr Gigi Hirsch, Executive Director of NEWDIGS and the MIT Center for Biomedical Innovation. “NEWDIGS is...

Listen to a conversation between Dr. Alberto Grignolo, Deputy Editor of DIA Global Forum, and NEWDIGS Executive Director and DIA 2016 Conference Co-Chair Dr. Gigi Hirsch discuss the challenges of healthcare system redesign in an era of groundbreaking science and new medical therapies.

Dr Hirsch is also participating in the DIA 2016 panel discussion “Europe and the US: Making Outcomes-Based Health Care Possible.

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Mark Trusheim, Director of NEWDIGS Strategic Initiatives, is quoted in the Nature News article “Promising gene therapies pose million-dollar conundrum“. He spoke at BIO on June 8th presenting on “Curative Therapies: Aligning Policy with Science to Ensure Patient Access.”

NEWDIGS will soon be launching a project on financing and reimbursement of cures in the US to focus on these issues.

The inaugural meeting of the NEWDIGS Data Program was held in January 2015, and brought together international experts from academia, government, information technology, and the healthcare sector. A series of panel discussions focused on identifying the gaps and opportunities that might be addressed through better international cooperation in data system design and use. Through discussion, participants joined in formulating a research agenda with associated pilot programs to develop the tools, methods and policies needed to leverage real world evidence for better decision-making.

The workshop referenced three important paradigm shifts currently unfolding in biomedical and healthcare innovation, which are converging in a way that is forcing advancements in the world of health data that many believe are long overdue. These change drivers include:

Increasing pressures to accelerate access to new and better treatments for patients in need. The advancement of translational and “precision” medicine,” associated with erosion of the traditional blockbuster model of innovation toward one that targets small subpopulations and individually tailored treatments. Growing demands from reimbursement/coverage decision-makers for...
Scientists argue that scientific and political changes will make adaptive pathways the preferred approach to make new treatments available

“Adaptive pathways should be the preferred approach in the near future to bring new medicines to patients.” A number of scientists, including members of the European Medicines Agency (EMA) and its scientific Committees take this position in a co-authored article published in Clinical Pharmacology and Therapeutics.

The concept of adaptive pathways foresees an early approval of a medicine for a restricted patient population based on small initial clinical studies. The first approval is followed by progressive adaptations of the marketing authorisation to expand access to the medicine to broader patient populations based on data gathered from its use and additional studies.


NICE (the UK National Institute for Health and Care Excellence) has joined a global group of organisations whose goal is to speed up the development and delivery of new, effective and affordable drugs to patients.

The New Drug Development Paradigms (NEWDIGS) program, led by the Center for Biomedical Innovation (CBI) at the Massachusetts Institute of Technology (MIT) in Cambridge, Massachusetts brings together pharmaceutical companies, academic researchers, patient advocacy groups, government agencies involved in healthcare and other experts to collaborate on ways to reliably and sustainably deliver new, better, affordable therapeutics to the right patients faster.

“Through NEWDIGS we’ll work with others to explore how our guidance development processes can contribute to faster and better drug development and delivery,” said Professor Sarah Garner, NICE’s associate director for R&D. “The...