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(ED NOTE: Ayesha is doing a Hangout tonight, October 2, 2014, at 9 PM, EST at www.BostonMedical.TV.)


June 18, 2014

Ayesha Khalid, MD, MBA, is the co-organizer of Hacking Medicine.  She writes about how we can improve rare disease research by looking to the private sector and streamlining adaptive techniques.

A secret is getting out, orphan disease breakthrough pathways have accelerated the race for cures in a wide-array of diseases. This is a disruptive opportunity in a tough space. Researchers in rare disease constantly struggle for funds and the opportunity to create life saving drugs.

The rare disease community questions how “rare” they truly are since 10% of all chronic disease in the US is through one of 7,000 rare diseases. As a group, about 95% of these diseases lack a cure or even treatment.

NIH research dollars are competitive, now more than ever. The community is calling for funding of early stage ideas and innovation in clinical research. Later stage research is happilyfunded by big pharma given there is a much greater chance of success. But the early stage groundbreaking research stalls while searching for funding. It is a classic “chicken and egg dilemma” – in order to get large-scale National Institutes of Health Funding, you need to have effectively run pilot data, and in order to get pilot data you need funding – herein lies the dilemma.

The Private Sector Steps In

Enter a group of financial engineers, investors, venture capitalists and the private equity world. Frustrated by the scientific foundations and the NIH for early stage funding, there is now a growing clamor for direct funding of innovative technologies, and drug development. Frankly, even cures for individual patient diseases that have come forward.

I recently researched several of these companies and with the advent of social media and crowdfunding; it is increasingly apparent that adaptive clinical trial design and more importantly adaptive financing mechanisms should be explored as avenues of providing early stage funding.

Applying Business Principals to Research

Through recent research, I learned the pace of such innovative funding mechanisms remains slow for a number of reasons. One of the key findings is the engine of clinical research lacks the infrastructure to be adaptive – that is to adopt the same mindset as an entrepreneur or an options broker.

The FDA has a rigorous approach to research; the regulatory hurdles faced by investigators lead to a very strict path with a well-defined trajectory designed to dot all the i’s and cross the t’s. Allowing researchers to adaptresearch pathways as data accumulates is difficult. How do we adhere to FDA requirements and interject variability when needed?

We need to find a way.

This piece is a call to action. Pharma statistics shows it can take 2 billion dollars (yes billion with a “B”) to develop one drug. It is imperative that we do something differently in order to move forward.

We need more collaborative models where investigators and institutions are financially incentivized to work together. Otherwise, the widespread adoption of adaptive designs for clinical trials will not occur. And as such, cures for the 95% of rare diseases will remain elusive.


As a physician, what do you think about funding for rare disease research? Have you seen promising early stage funding thwarted by competition for research funding?

Ayesha Khalid, MD MBA


Ayesha Khalid is a sinus surgeon with an MBA from the MIT Sloan School of Management. Ayesha is interested in the re-design of the health care system with a patient-centered focus, specifically in the area of clinical trials and rare diseases. At present, she is working on a project to evaluate adaptive clinical trial design. She is a co-organizer at Hacking Medicine, an interdisciplinary group at MIT that seeks to spark change in healthcare. She also serves as the Young Physician Chair for all ear, nose and throat physicians in the United States.


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