Andra Stratton has never met a pharmaceutical company that wasn’t patient-centric, or at least one that didn’t declare itself to be so.

The reality for Stratton, president and co-founder of Lipodystrophy United, is that all of them are quick to declare that they are patient centric while few conduct themselves as such.

Lipodystrophy is a genetic metabolic condition that prevents the body from properly storing fat as under the skin. There are several forms of the disease, but the absence of fat tissues results in the body’s failure to produce adequate amounts of the hormone leptin and brings complications of heart and liver disease from the accumulation of fat in organs, as well as diabetes.

After Stratton began to deal with her own diagnosis of partial lipodystrophy and helped co-founded Lipodystrophy United, she found herself in more and more contact with pharmaceutical companies. There is one drug that won approval as a treatment for lipodystrophy— metreleptin (a synthetic analogue of the hormone leptin that regulates fat storage)—and the strange tale of that drug’s life has provided Stratton with up close and personal education on the inner working of drug companies and what it means to be patients centric.

Amgen licensed the drug in from Rockefeller University in the hopes of developing it as a treatment for obesity. But it wasn’t effective because the problem with obesity is that people have grown resistant to the effects of leptin and do not suffer from the absence of it. The drug, though, had potential as a treatment for diabetes and Amylin Pharmaceuticals licensed it from Amgen in 2006.

Bristol Myers Squibb and Astra Zeneca acquired Amylin in 2012 in a diabetes alliance and metreleptin along with it. BMS later divested itself of the alliance and claims to metreleptin along with it. AstraZeneca won approval for the drug in February 2014, but sold it nine months later in November 2014 to Aegerion. Aegerion, suffering financial problems, merged with QLT in 2016 and the combined company Novelion Therapeutics now markets the drug under its brand name Myaetpt.

In all, six different companies owned the drug as Myalept. Some of those companies gained control of Myalept as an ancillary asset and weren’t interested in marketing a rare disease therapy. Along the way, Stratton has had close contact with several other drug companies developing other therapies for lipodystrophy.

Stratton said six days after she became a patient advocate she was drafted on to a patient advisory board for Amylin Pharmaceuticals. That was her first exposure and for her that set the bar. “It was very clear that the patient ad board was all about understanding the burden of the disease,” she said. “My interactions with them were infrequent, but always very patient-focused.”

As she had exposure to a number of different pharmaceuticals and saw the different role they saw for the patient, she developed strong opinions for both how drug companies should engage with patient groups and how patient groups should engage with drug companies.

Where she had problem with drug companies it involved their wanting patient input on what she called “small things” rather than allowing them a seat at the table across the board. She’s grown suspicious of companies that tell the foundation that it can help them without these companies first listening to patients and learning from them about the burden of the disease. And, she has also found a catchall excuse some companies use for excluding patients from certain discussions is “it is a compliance problem.”

For Stratton, companies that are patient-centric are ones that see that the patient community has a seat at every table so the patient voice is represented in discussions with medical, compliance, marketing, other parts of the company and that voice is valued from the top down.

In one case, she said her group broke off relations with one company because it didn’t like the way the company communicated with the patient community, bypassing the group and going directly to patients, who it felt weren’t in as good a position to evaluate what the company was telling them. The company has since been acquired and the new company has repaired its relationship with the foundation.

When Stratton co-founded United Lipodystrophy, she said that the group had internal discussions as to whether they would accept funding from drug companies. While they decided they would accept funding, they also decided that they need to look at everything they do through the lens of whether what they were doing helped patients.

“We wanted to be a resource for the patient. Everything for us comes back to that,” she said. “The question to ask in working with a company is, ‘Does this serve the purpose of the patient, or the purpose of the company.’ If it serves a purpose for the patient alone, or the patient and the company, we’re in. If it doesn’t serve a purpose for the patient, we’re not.”

United Lipodystrophy is trying to build on the lead of the International Fibrodysplasia Ossificans Progressiva Association, which last year published a set of guidelines that spells out how the organization interacts with industry.

“The advice I’d give to the patient community is that if it doesn’t feel comfortable, then you absolutely say, ‘No,’ she said. “It doesn’t matter if they are a funding source. Our job to protect the patient. That’s our sole responsibility no matter what we do as a foundation, our first role is to make sure we understand compliance issues and that we protect the patient.”
 
The lesson here is that drug companies may not always be patient-centric. They have many interest to balance. On the other hand, there’s no excuse for patient groups being anything but patient-centric.

October 15, 2017

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