Developing new medicines with the patient in mind
“The silent hero of the entire medical story is the patient,” said Dr. Salim Mujais, Senior Vice President at Astellas, while introducing a panel discussing patient-focused drug development. “Their tenacity is what really motivates us, their suffering is what inspires us and, ultimately, most of us will become patients at some point in our lives.”
This sentiment was echoed throughout the session that followed, which was moderated by Susan Winckler, Chief Risk Management Officer at Leavitt Partners and featured Theresa Mullin, Director, Office of Strategic Programs at the Center for Drug Evaluation and Research at the FDA; Kenneth Hobby, President of Cure SMA; Ubavka DeNoble, Corporate Vice President, Patient Technology Solutions at PAREXEL; and Bernhardt Zeiher, President of Development at Astellas.
Engaging with the FDA
The first question of the session concerned patient advocacy groups’ interaction with the FDA: what are the current opportunities for them to do so?
“The most effective place for advocates to get involved with the FDA is at the very beginning,” said Mullin. “We need patient evidence of benefit and burden that we can bring in and use as a basis for an approval.”
Those same data insights should be distilled into endpoints for trials, explained Mullin, and that’s why the communication with the FDA needs to be as soon as possible.
Backing up Mullin’s opening statement was Hobby. His own association worked directly with the FDA en route to its first ever approval of a medicine for spinal muscular atrophy.
His experience did come with a warning though – ensure there is a balance between emotional anecdotes and data. “An FDA decision based on emotions could lead to potentially dangerous drugs getting approved,” said Hobby.
An evolving patient-provider relationship
“Despite all our attempts and knowledge, drug development has become more and more expensive,” said DeNoble. “We are moving to the next level. Previously, incorporating the patient voice was mostly to do with the launch of a product, but now there is a shift to more engagement with the patient at every stage of development.”
DeNoble’s answer was in response to a question about how the process of drug development had changed during her time in the pharma industry – a period that, according to DeNoble, has seen average drug development spending rise from $890 million to around $2.5 billion.
“For us, as a drug company, a lot has changed in how we develop medicines,” added Zeiher. “We’re starting to think more about what really matters to the patient.”
Patients are more informed than they ever have been, he said, with more patients and their families wanting to know about their diseases.
“It’s really important that families understand clinical trials, what the challenges are, what’s really needed in the regulatory process,” added Hobby, in agreement with Zeiher. “It’s our responsibility to make sure they’re as well informed as possible.”
The proposed Patient Affairs Office
When turned over to the audience, the first question posed to the panel asked for their opinions on the proposed FDA Patient Affairs Office – a department intended to be a single point of contact between patient advocacy groups and the agency.
“The creation of a patient affairs FDA office is a great way to streamline communications with patients and hopefully will make the process easier,” said Hobby, an opinion the remainder of the panel agreed with. “If it can do exactly that, then it will be a great addition.”
The question was naturally passed on to Mullin, herself an FDA representative, for the closing remarks of the session: “The patient-focused approach is really a culture change for the entire ecosystem and everyone is going to have to undergo it.”