PARIS – In Europe, patient involvement in drug development and regulatory assessment is evolving from an ad hoc occurrence into a more structured process, driven by a shared interest on the part of regulators and drug developers in having patient input influence the development and assessment process.
Some patients – and their advocates – are pushing for this too, as was evident during a session on the topic at the DIA Euromeeting here Wednesday. One patient representative challenged an EMA official on when the agency's Committee for Human Medicinal Products (CHMP) would recruit one or more patients. The response was not encouraging. "The composition of the CHMP is defined by legislation, so that's not going to happen easily," replied Francesco Pignatti, the EMA's head of oncology, hematology and diagnostics.
Nevertheless, patient involvement in the EMA's activities is a work in progress, he told delegates. It has followed a natural evolution, and the level of interaction is increasing year on year. "We've had a very good experience with scientific advice groups," he said. It's now standard that two or three patients participate in these groups. Patient involvement at more formal levels of the assessment process is less mature.
"The most challenging part has been at the level of CHMP," he said.
Sceptics still raise several concerns, he said. There is no such thing as an average patient, even though regulators frame their questions in that way. Patients' views are anecdotal and biased. Patients themselves are uninformed, or they may need extensive training if sophisticated research methods are going to be employed and ultimately, their input may have little real impact.
None of these objections are necessarily barriers to patient participation, he said, given the potential of patients to contribute to drug regulators' core decision-making process in assessing a drug's risk-benefit profile. "The most important part of this, where patients can be involved, is in [assessing] the trade-off," he said. There are still challenges to this, he added, including questions about the robustness of patient input and its feasibility in the context of a marketing authorization application (MAA). A 6,000-patient-preference study is not a viable option.
So far, patients and their caregivers have provided input into the CHMP's decision-making process just once, in the case of an MAA on the erythropoietic protoporphyria drug Scenesse (afamelanotide), developed by Melbourne, Australia-based Clinuvel Pharmaceuticals Ltd. That occurred under a pilot program, in which patient testimony is considered when the CHMP is itself uncertain of a drug's risk-benefit profile. The drug subsequently received approval, and reimbursement negotiations in key European markets are currently under way. (See BioWorld Today, Sept. 29, 2014.)
The present assessment process does not always capture information about a drug's benefits that may be important to patients, Francois Houyez, health policy advisor at Euroordis, a pan-European alliance of rare disease organizations, told delegates. "I think patients can help select the best outcome and measurement tools."
Speaking from the floor, CHMP chairman Tomas Salmonson evinced an appetite for further patient involvement. "Understanding patient preferences and values is essential," he said. And receiving informal communication is not unusual for the CHMP – experts and patients already engage this way. "We are quite used to getting input in various ways," he said. However, his preference is for a structured engagement process, involving professional facilitators. Public patient testimonies, a feature of FDA advisory committees, are not on the agenda. "Whether that's a good model or not – I'm not convinced. I think we can find a better model."
He welcomed an industry proposal, currently in preparation, for a new Innovative Medicines Initiative (IMI) project in this area, which would focus on developing clear guidance for industry, regulators and health technology assessment agencies on how and when to elicit patient perspectives in benefit-risk assessments. This is in parallel to an existing internal EMA pilot study on patient preferences in multiple myeloma and melanoma. "An IMI initiative can give us so much more," Salmonson said.
In practical terms, how to include patients – and how to fund their involvement – has not been fully worked out.
"Yes, that is an issue, just as it is with experts. We have to deal with it in order to be transparent," he said.
One industry delegate raised the danger of active patient participants becoming "professionalized" by their involvement, although Houyez dismissed the risk. "When patients are appointed to these committees, they are not put in gaol [jail]. They still meet other patients and are accountable to other patients," he said.
The conference ended Wednesday.