Uniting To Accelerate Pediatric Medicine

Drug companies inherently absorb significant risk when developing compounds and biologics for clinical use. A recent article suggests that only one out of every 12 drugs that enters clinical trials succeeds and the cost of a drug successfully reaching the market now exceeds an average of $5 billion when you add up the cost of all those that failed along the way.

For many indications, such as Alzheimer’s and autism, the need for new treatments is enormous, yet investment in these areas is declining due to recent high profile failures. The risks and challenges associated with drug development will continue to be there for the foreseeable future.

Despite these risks, I have noticed a promising trend — the rise of open source drug R&D consortia that include large biotech and pharma organizations that normally compete against each other, along with academic institutions and disease foundations.  These collaborations are coming together, in defined “pre-competitive” spaces, to generate tools and data that can be shared in order to accelerate research at the early stage to collectively reduce costs, minimize failures and shorten the timeline to approval for new drugs — helping bring treatments to the patients who need them the most.

In response to the high cost — 90 percent of R&D costs are spent on phase III trials alone — and inefficient clinical trials process, a community of over ten major pharma companies and federal regulators has come together to create a non-profit company named TransCelerate. The aim is to simplify and accelerate the clinical trials process to benefit the industry as a whole while keeping a level playing field.

In the field of autism, stakeholders such as academic researchers, pharma companies, and patient advocacy groups are coming together to better understand the inner workings of the disease in hopes to increase the development and approval of effective therapeutics. A unified effort in Europe is underway named EU-AIMS — a $40 million, multi-year collaborative program to develop the infrastructure underpinning new treatments for autism. These are just two instances of industry coming together to achieve a mutually beneficial goal and tackle challenges that no one organization can tackle alone.

As an industry, pediatric medicine faces many similar challenges. Addressing chronic and complex diseases in kids can improve health outcomes, quality of life and reduce future medical spending when these children enter their adult lives. Surprisingly, the NIH only allocates five percent of it $30 billion budget to pediatric research and this has been flat for nearly 20 years (See this blog post). .

Other challenges I see include low commercial investment in pediatric medicine and devices, development of effective therapeutics for neurodevelopmental disorders, and clinical interpretation of genomic data into pediatric clinical care.

At the Technology and Innovation Development Office (TIDO) at Boston Children’s, we have the honor and challenge of turning the research discoveries and clinical innovations made by our scientists and clinicians into new products that can benefit our kids and the public.  But we cannot do it alone.

TIDO extends significant effort to set up strategic alliances with pharma and biotech companies to develop better treatments — most recently with Shire around rare diseases. These alliances leverage Boston Children’s research expertise and companies’ development and commercialization capabilities. Similarly, drawing on the expertise of our external advisory board made up of pharma, device, diagnostic and software experts, the Technology Development Fund is an internal funding mechanism set up by the hospital in 2009 that invests in and develops promising innovations. All of these efforts in conjunction with the strength of the research base have led to approximately 16 products on the market with eight more in clinical trials.

But rather than sit back and be proud of our successes, Boston Children’s is interested in breaking down the barriers that remain in pediatric medicine. We can only do that if the innovation community, including researchers, clinicians, foundations and patient groups, companies and the government work together. I know it is a lot to ask — but I can’t think of a better reason to unite than to improve the health of our kids.

This is why Boston Children’s is convening top thought leaders to address the toughest challenges in pediatric health care today.  The National Pediatric Innovation Summit , to be held in Boston on September 26 and 27, will bring together leading clinicians, scientists and leaders in industry, private equity, and health care policy to engage in discussions — collectively taking on problems that no single organization can solve alone. Leaders from companies like IBM, Vertex and Johnson & Johnson will interact with thought leaders from leading children’s hospitals in the US and acclaimed academic institutions like MIT and Harvard.

I am confident that discussions started at the summit will lead to the types of large collaborations and innovative solutions needed to tackle the toughest challenges in pediatric healthcare because the lives and well being of children all over the world is depending on us.

Erik Halvorsen, PhD., is the Executive Director of Technology & Innovation Development Office and Managing Partner, Technology Development Fund at Boston Children’s Hospital. The mission of the Technology and Innovation Development Office (TIDO) is to translate the excellence of the laboratory research and clinical care at Boston Children’s into lifesaving biomedical products, devices and procedures for the public benefit.

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