Regulatory Focus™ > News Articles > PhRMA Launches Patient-Focused Drug Development Initiative in Hopes of Influencing FDA

PhRMA Launches Patient-Focused Drug Development Initiative in Hopes of Influencing FDA

Posted 05 November 2013 | By Alexander Gaffney, RAC

Patient-focused drug development has caught the attention of both US legislators and regulators in recent years, with the former instructing the latter to make it a part of the US Food and Drug Administration's (FDA) assessment process. And while FDA is still in the midst of setting up meetings, asking questions of patients and-crucially-figuring out how to incorporate their views into regulators' benefit-risk paradigms, a prominent member of the pharmaceutical industry has just announced its own foray into patient-focused drug development as well.


Patient-focused drug development is based on the idea that some patients may be willing to accept greater risks, lesser benefits or less certainty of a benefit in some cases. In particular, for patients who have a serious disease and few-or sometimes no-treatment options, FDA's current benefit-risk assessment process is sometimes seen as being too risk-averse.

In 2012, in response to advocacy efforts by several patient groups, legislators inserted a provision into the Food and Drug Administration Safety and Innovation Act (FDASIA) that required FDA to set up 20 patient-focused drug development meetings over the next five years, with more groups to be added at later dates.

The pharmaceutical industry has-not surprisingly given the potential for laxer drug approvals-been supportive of the move.

PhRMA Initiative

But on 4 November 2013, the US pharmaceutical industry's more prominent trade group, the Pharmaceutical Research and Manufacturers of America (PhRMA), announced it will partner with several disease groups to crowd-source input from additional patients not able to attend FDA's patient-focused drug development meetings.

The groups chosen represent three disease areas already selected by FDA for its disease meetings: sickle cell disease, idiopathic pulmonary fibrosis (IPF) and irritable bowel syndrome (IBS). The sickle cell disease community will be represented by the North Alabama Sickle Cell Foundation, Sickle Cell Disease Association of America, Sickle Cell Warriors, and The William E. Proudford Sickle Cell Fund. Meanwhile, the Coalition for Pulmonary Fibrosis will engage with the IPF community, while the Gastroparesis Patient Association for Cures and Treatments and the Share and Care Cockayne Syndrome Network will work with the IBS community.

All will be working with a system known as the Platform for Engaging Everyone Responsibly (PEER), a customized technology platform that will allow patients to share information based on their own personal privacy preferences. "Using the Platform for Engaging Everyone Responsibly (PEER), there is an opportunity to demonstrate the power of a secure, crowd-sourced approach to provide additional insight into patients' experience with a disease or condition," stated Sharon Terry, president and CEO of Genetic Alliance, one of PhRMA's partners on the project.

So what's the goal of collecting all this information? Simple, explained Genetic Alliance: "Our goal is to collect a robust dataset of information, and to offer a diverse range of perspectives to the FDA and to the participating [disease advocacy organizations]."

PhRMA Statement

Genetic Alliance Statement

Tags: PhRMA

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