BOSTON – In the mid-20th century, a cancer patient could die without ever hearing his or her diagnosis. Many doctors, in fact, considered that the ethical thing to do. Since patients had no voice in their treatments, why saddle them with the knowledge of their diseases – which, at the time, could carry considerable social stigma?

Today, patients are making treatment decisions with their doctors. And increasingly – especially in rare diseases, but also in some common ones where good options are in short supply – they are becoming involved in creating the next generation of treatments for what ails them.

The recent approval of Kalydeco (ivacaftor) marks the first drug that was developed in close collaboration between a company and a disease foundation – Vertex Pharmaceuticals Inc. and the Cystic Fibrosis Foundation. (See BioWorld Today, Feb. 1, 2012.)

At last week's BIO International Convention, several panels looked at how companies can benefit from such patient involvement, tapping not just research funds, but access to clinicians and patients themselves.

At a panel on "Disease Foundation-Funded Drug Development Partnerships: Structuring for Success," Skip Irving, a board member of the Michael J. Fox Foundation (MJFF), said the approach a foundation takes to funding research will depend on the disease.

Its colalboration with Vertex worked for the Cystic Fibrosis Foundation, Irving said, because the biology of cystic fibrosis was both clear and fairly simple. "They could put a lot of dollars in a single bucket," he said, because they knew which gene to target.

Parkinson's disease, which is the focus of MJFF, is still much more of a medical mystery. "There aren't good targets, biomarkers or pathways," Irving said.

As a result, MJFF has spread its bets much more than the Cystic Fibrosis Foundation. It has funded 87 different companies, in addition to academic researchers, in the past decade – many of them for tools and preclinical model development as well as therapeutics development.

But whether a given foundation makes one big bet or many small ones, a common pattern is that "foundations are becoming a lot more sophisticated" about funding research, Irving said. While they used to limit themselves to straight research grants, these days funding mechanisms can include sponsored research grants, but also milestone-based investments, venture investments and participation in research consortia.

Whether a partnership between a disease foundation and a company or academic lab works out depends on a number of factors. "We have our priorities, much like a company would," Irving said.

If the company fits with those priorities, MJFF will do due diligence on both the quality of the underlying science and the company. And while disease foundations are an example of venture philanthropy, they are definitely looking for a return on their investments.

Irving said that surprises some people, whose attitude is "'You're a nonprofit, what do you care about returns?' Well, we're not going to solve Parkinson's disease with the first drug that comes out. We're going to be in this business for a long time. And we're looking for that money to give us a path forward."

Access to patients can be another advantage of working with disease foundations. That access can lead to such straightforward things as a patient pool for clinical trials. But especially in rare diseases, patients can develop expertise on their conditions that rivals or surpasses that of their doctors. When such patients organize, often in online support groups, they can become a valuable source of information about which aspects of a disease are the most distressing to patients and so, the most urgent to treat.

"The people who have the biggest stake here are the patients," Daniel Sands, who is both a primary care physician and a senior medical informatics director at Cisco Systems, told the audience at a panel on "New Rules of Patient Engagement: The role of patient influence in driving novel partnerships and business models."

And "as you go to such online communities, you harness the wisdom of the crowds," he added.