June 02, 2018
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Collaborative investment may expedite drug development for pediatric cancer

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Andrew Lo

CHICAGO — Investment from the private sector, the government and philanthropic organizations holds the most promise for new drug discovery for children with cancer, according to data presented at the ASCO Annual Meeting.

“As a new era of drug development dawns, scientists are finding cures and treatments for different types of cancers every day. But, for a variety of reasons, childhood cancers are being left behind," Andrew Lo, PhD, Charles E. and Susan T. Harris professor and director of the Laboratory for Financial Engineering at MIT Sloan School of Management, said in a press release. “Spurring innovation in this field requires us to be more creative in our approach to financing. And if there is one patient population that really needs our support and advocacy, it’s our children.”

Lo and colleagues conducted simulations — simultaneously published in JAMA Oncology — to evaluate economic viability of different approaches to therapeutic development for pediatric cancer. The simulations included factors such as philanthropic support, government guarantees and portfolios of assets in different clinical phases

The researchers found that a private sector-only portfolio of pediatric oncology assets exhibited low to moderate returns, ranging from –24.2% to 10.2% depending on the price charged for approved therapies. To achieve an average return of 10.2%, pricing for one course of treatment would have to be at least $1 million per patient for each approved drug.

“When lives are at stake, such lofty prices may seem like price gouging, even though such prices may be justified from a cost-effectiveness perspective,” Lo said in the release. “The political ramifications could be immense, so the issue is treated like a third rail — no one wants to touch it.”

However, when researchers included philanthropic and government support in their simulations, and both early- and late-stage assets were included in the portfolio, the data showed much more attractive returns, even at lower price points (–5.6% to 22.5%).

“Our findings show that a public/private partnership is especially effective for developing pediatric oncology therapies, and the key to making this partnership work is that oncologists need to continue coming up with new ideas and strategies for treating cancer, particularly those that aren't already being pursued,” Lo told HemOnc Today. “Having ‘multiple shots on goal’ isn't enough — we need the shots to be as statistically independent as possible.

“Everyone agrees that we’re at an inflection point in treating cancer; now is not the time to reduce funding at the early stages of drug discovery but, rather, to double-down and invest even more resources to get us over the finish line,” Lo added. – by Cassie Homer

Reference:

Das S, et al. Abstract 10528. Presented at ASCO Annual Meeting; June 1-5, 2018; Chicago.

Disclosures: Lo reports leadership with BridgeBio Pharma and Roivant; stock/other ownership interests with BridgeBio Pharma, Ignite Biosciences, ImmuneXcite, KEW, LS Polaris Innovation Fund, MPM Capital, Roivant and Royalty Pharma; consulting/advisory roles with BridgeBio Pharma and Roivant; and other financial relationships with Beth Israel Deaconess Medical Center, Milken Institute and MIT Whitehead Institute. Please see the abstract for all other authors’ relevant financial disclosures.