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Patient-Focused Drug Development

Amplifying the patient voice at every stage of the drug development process

At the Lupus Foundation of America, our mission is to improve the lives of people living with lupus. We know that to be successful, we need to understand them – their needs, their experiences, and their priorities. Since 1977, our work has been guided by those perspectives.

With only one drug approved specifically to treat lupus in the last 50 years, people with lupus need safer, more effective treatments. That’s why we’re funding research to identify new targets for therapies, partnering with leading researchers to design better clinical trials, and producing new, more precise tools to evaluate the effectiveness of potential medicines.

Patient-focused drug development is an initiative of the Food and Drug Administration (FDA), mandated by Congress, to incorporate the patient perspective into the drug development and approval process.

We believe that patient-focused drug development will lead to safer, more effective treatments for people with lupus in the near future. 

Patient-focused drug development (PFDD) will develop systems to collect meaningful information from patients and provide that information to researchers to guide the creation of new medicines and the FDA’s evaluation of them. That information includes:

Burden of Disease

How does lupus affect the people living with it? What are the biggest challenges of coping with lupus? Which symptoms are the most difficult to manage?

Burden of Treatment

People with lupus take an average of 8 drugs to manage their disease. Which side effects are the worst? How difficult is it to manage the medications?

Medicine Benefits and Risks

Every medicine comes with benefits and risks. For people with lupus, what is the right balance? How much risk is acceptable for a moderate benefit?

Clinical Trials

What prevents people with lupus from enrolling in clinical trials? How can clinical trials be designed to make lupus patients want to participate?

Through the patient-focused drug development process, pharmaceutical companies and the FDA can better understand how diseases like lupus affect the people living with them, and use that information to more effectively tailor their decision-making to patients’ needs. 

For drug companies, this could mean focusing on designing medicines that treat the most debilitating symptoms of a condition first, and constructing clinical trials that meet the needs of patient groups and encourages their participation. 

For the FDA, PFDD will mean evaluating and approving drugs based on the full range of a medicine’s impact. In 2015, for example, FDA heard from people with melanoma (a form of skin cancer) that there was a psychological benefit to seeing their skin lesions shrink, and the FDA approved a therapy in part based on that information. Prior to PFDD, FDA likely would not have had that information and would not have been equipped to incorporate it into their decision-making. 

We have been involved with patient-focused drug development since the beginning. And we continue to engage in its implementation to ensure the systems and guidelines being established will accelerate the development of new lupus drugs.

On September 25, 2017, the Lupus Foundation of America partnered with the Lupus and Allied Diseases Association and the Lupus Research Alliance to host an externally-led PFDD meeting. At the meeting, members of the lupus community shared with the FDA their perspectives on living with the disease.

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