Patient advocate engagement in early stage development (part 1)

By: Jessica Cerullo Merrill, MPP

Twice a year, MK&A associates attend an offsite meeting to plan, improve our skills and stay up-to-date on the latest in patient, professional and consumer trends and research.

At our March meeting we invited a panel of advocates to discuss why and how their organizations are investing time and resources in the development process of therapeutic options.

Over the next couple of blog entries, we will take a look at different ways patient advocates are influencing early stage development with the goals of:

  • Identifying promising molecules and advancing them from promising translational research to early clinical trials,
  • Speeding up drug development and
  • Increasing the treatment options for patients


Changing Role of Advocates
Historically, patient advocates did not invest donor dollars in the early drug development process. Companies often did not engage patients or their advocates until they were recruiting patients into a registration trial or the product was being prepared for submission to regulatory authorities or sometimes even closer to launch.

However, both pharmaceutical companies and patient advocates realized that this needed to change; as a result advocates are increasing their activity in early stage development. This only makes sense as no group has a greater interest in accelerating the speed of research and ensuring that all appropriate avenues are explored in moving promising translational research into clinical development.

Advocates Taking Action
Some organizations such as the Pancreatic Cancer Action Network are investing in molecular profiling of tumors to identify promising targets for drug development. The Know Your Tumor project is a personalized medicine service that will provide pancreatic cancer patients and their oncologists with information about the biology of their specific tumor. This information can be used to identify treatments that may be valuable to an individual patient, including clinical trials.

An interesting consortium of patient groups has formed in the neurodegenerative disease community to leverage the individual strengths of their organizations while pooling financial resources to provide seed funding to identify and support early translational research. This approach is intended to compliment, not to compete, with the work being done by pharmaceutical companies. This collaborative approach allows researchers to continue to thoroughly examine promising compounds that may not have been moved forward by academics or small biotech companies due to lack of resources. Patient organizations can seed this early clinical development and if promising, they will present this work to larger pharmaceutical companies to continue the work and move promising compounds on to phase II or III trials; often prohibitively expensive investments for patient groups, academics or small biotech companies.

Real-life Example
One of the guest speakers at our Associates’ meeting was Mike Johnson, director, corporate partnerships, MRC Technology (MRCT). Established in 2000, MRCT handled the technology transfer needs of the United Kingdom’s Medical Research Council. The MRCT has transformed into an independent charity that works to bridge the gap between basic research and commercial application by connecting universities, medical research charities, and pharmaceutical and biotechnology organizations around the world.

MRCT is now leading the Neurodegeneration Medicines Acceleration Program (Neurodegeneration-MAP) a consortium of nine patient advocacy organizations*, launched in 2014 (see launch video), who have “joined forces to turn existing science into patient treatments” for the estimated 50 million people worldwide who suffer from a neurological condition.

Mike Johnson shared that MRCT “approaches pharmaceutical companies with the knowledge that certain projects have been shelved, we offer to fund these projects and move them to a pre-agreed upon place where the company can pick the project back-up and bring it to the patient. As a charity we believe every patient deserves the best solution science can offer.”

Neurodegeneration-MAP charities plan to collectively raise funds to bring promising compound(s) though early stage clinical research and if of value, approach pharmaceutical companies to further invest and move these projects forward. The consortium has just begun their work with the hope to have at least one promising compound launched within the next three years.

At MK&A, we can assist in connecting patient advocates with appropriate knowledge and background who may wish to explore and potentially engage in a similar type of collaboration in their therapeutic area.

* Alzheimer’s Association; Alzheimer’s Research UK; Alzheimer’s Society; ALS Association; Michael J Fox Foundation; Motor Neurone Disease Association; Northern Health Science Alliance; MRC Technology and Parkinson’s UK

About the Author:

Jessica Cerullo Merrill, MPP

Jessica Cerullo Merrill, MPP is an associate at MK&A.  She can be reached at +1 212 620 2768 or

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