Three challenges precision medicine faces before it can scale up

Different aspects of precision medicine therapyDoctors, scientists, consumers, entrepreneurs and others came together recently for the Precision Medicine 2017 symposium at Harvard Medical School, now in its third year. This year’s theme was “breakaway business models.” What are challenges in developing targeted treatments based on clinical and genetic data, and how do we overcome them?

Here were some of the speakers’ main concerns and takeaways:

No1Regulatory requirements need to accommodate precision medicine initiatives

FDA and other governmental regulations around drug approval and trial design were brought up frequently.

From the left: Pamela Gavin, Jamie Heywood, Noga Leviner, Matthew Might
From the left: Pamela Gavin, Jamie Heywood, Noga Leviner, Matthew Might (Photos courtesy Isaac Kohane, MD, PhD)

Matthew Might, co-founder of Pairnomix (a genetic research company that helps patients with rare disease find treatment options), said the FDA’s agenda should include “improving regulations that favor more small patient trials.” As the father of a child who was the first ever to be diagnosed with NGLY1 deficiency, his story is extreme but not unusual in the rare disease world. While the number of drug approvals given by the FDA has gone up, it is still not enough.

At the moment, a further challenge is that around 35 percent of positions at the FDA are unoccupied.

“You could work on new pathways, you can look at legislation, but if you don’t have the resources to implement it, it’s really hard,” said Pamela Gavin, COO, National Organization for Rare Disorders.

No2Academia and industry need to go hand in hand


Ethan Perlstein, CEO Perlara
Ethan Perlstein, CEO, Perlara

Ethan Perlstein, CEO of Perlara (a drug discovery platform for rare diseases), compared the relationship between industry and academia to “allopatric speciation,” in which members of the same species get so isolated from each other that reproduction between the two is no longer possible. “That means you’ve got scientists in the industry, scientists in academia and they’re no longer mixing,” he added. He went on to propose one way to bridge the gap: adding an industry internship to a graduate science student’s rotations.

Joe Pickrell, co-founder of Gencove (a genome testing service and information provider to researchers) and a core member of the New York Genome Center, suggested that scientists should pick a research problem before deciding if they want to go the industry or academic route; not the other way around. The focus should be on finding the solution, not necessarily the approach taken.


Increasing patient involvement is a necessity


Patient engagement, panelists agreed, is a central aspect to developing any precision medicine effort.

Kathy Giusti, co-chair, HBS Kraft Precision Medicine Accelerator wishes for “instant knowledge among the patient community that their data is on the preliminary path to finding cures.”

Kathy Giusti, co-chair of HBS Kraft Precision Medicine Accelerator
Kathy Giusti, co-chair, HBS Kraft Precision Medicine Accelerator

Additionally, Gavin believes patient empowerment — knowing what options are out there — will bring with it patient involvement. Only with the latter can precision medicine grow: patient contribution and scientific research need to feed into each other.

“We have to continue to work towards transparency and allowing all stakeholders that should have a voice or take a seat at the table to be a part of that discussion,” said Gavin.

Beyond these fundamental issues, challenges lie ahead: building infrastructure for precision medicine services and scaling them up in clinical practice. The researchers, entrepreneuers and families involved in this arena are personally invested in successful outcomes, and it is their enthusiasm that will move the needle.

More coverage of the Precision Medicine Symposium from Harvard Medical School.