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In biotech, patient advocates’ voices heard

Foundations help fund drug research

SAN FRANCISCO — The Cystic Fibrosis Foundation, an early investor in Boston-based Vertex Pharmaceuticals Inc.’s drugs to treat the chronic lung disease, caught the attention of other patient advocates in November, when it disclosed that it had made $3.3 billon by selling its royalty rights.

Speaking before a small group of venture capitalists and biosciences entrepreneurs here Sunday night, foundation president Robert J. Beall said his group is plowing the proceeds into new disease-fighting alliances with drug makers ranging from Pfizer Inc. to Shire PLC and expanding a Bedford, Mass., lab where scientists use cell lines from cystic fibrosis patients to test compounds that could become medicines.


“That’s what the monetizing allows us to do,” said Beall, who outlined plans to boost employment at the Bedford lab to 20 employees from 13 in the coming months. “We’re trying to create collaborations. We think the life expectancy of cystic fibrosis patients can be increased by decades.”

The voices of patients and their advocates are growing louder at life sciences industry events like the J.P. Morgan Healthcare Conference, which opened here Monday. While curing and treating patients has always been the focus of the booming business of drug and medical device makers, the patients themselves were conspicuously absent when drug executives in suits and ties gathered in the past.

Now, with the emergence of the “venture philanthropy” model pioneered by Beall’s foundation, and the growing role of patients in lobbying companies, regulators, and lawmakers for more treatment options, patients are becoming a factor to be reckoned with in the biopharmaceutical business.

Like other companies, venture capital firm Polaris Partners of Boston, which hosted a panel of patients, advocates, doctors, and entrepreneurs to talk about patient perspectives as the J.P. Morgan conference got underway, has been seeking out and working with patients who can advise, inspire, and advocate for its portfolio of biotechnology start-ups.


“We hope to be able to convert our knowledge of patient needs into benefits for the companies and the patients,” said Walter M. Capone, president of the Multiple Myeloma Research Foundation. Capone said his group has assembled a tissue bank to do genomic analyses and is using the results to help two companies perform clinical trials with experimental drugs.

Critics have suggested the increasing patient role in funding, developing, and marketing drugs raises some concerns and potential conflicts, particularly when venture philanthropy investors profit from higher drug prices that patients must bear. (The Cystic Fibrosis Foundation has said it was not part of Vertex’s decision to price its Kalydeco drug at $300,000 a year, though its investment clearly benefited.)

But for many patients and businesses, the benefits of greater patient involvement — and the increased visibility of patients telling their stories — are self-evident.

Martha Rhodes, a former New York advertising executive who suffered from treatment-resistant depression, told the Polaris group she has been able to manage the disease and transform her life through transcranial magnetic stimulation, a technique marketed by Polaris-backed Neuronetics Inc. of Malvern, Pa. Rhodes said she has no financial stake in the company.

“This therapy has the potential to save millions of lives of patients who don’t respond to traditional drugs,” she said.

Another patient advocate, Chris Garcia, a global business consultant based in Los Angeles, told the audience that his acne scar treatment from Polaris-based Suneva Medical Inc. in Santa Barbara, Calif., has been a boost to his career.


“Having a clear face with fewer scars played a big role,” Garcia said. “It gives you the confidence that’s important to success in business.”

Polaris venture partner Amy Schulman said the patient perspective is becoming increasingly important in guiding the efforts of investors and entrepreneurs.

“The human dimension underlies all our science and all our business,” she said.

Robert Weisman can be reached at robert.weisman@globe.com. Follow him on Twitter @GlobeRobW.