Lupus Research Institute Applauds 21st Century Cures Initiative in US House of Representatives


House Committee on Energy and Commerce
2125 Rayburn House Office Building
Washington, D.C. 20515

                  The Honorable Fred Upton                                    The Honorable Henry Waxman
                              Chairman                                                                 Ranking Member

                  The Honorable Joe Pitts                                            The Honorable Frank Pallone
           Chairman, Health Subcommittee                       Ranking Member, Health Subcommittee

                                                    The Honorable Diana DeGette
                                                Committee Member, Co-chair 21st
                                                        Century Cures Initiative                                                 


July 14, 2014

Dear Committee Members,

On behalf of the Lupus Research Institute, its National Patient Coalition, and lupus patients and their families nationwide, I would like to offer our heartfelt appreciation for the attention you are devoting to the importance of clinical trials and patient engagement in the 21st Century Cures initiative.

The hearings held last week on Modernizing Clinical Trials and on Incorporating the Patient Perspective both showcased the urgent need to innovate as we push for new cures and treatments for disease while also involving patients as equal partners in all stages of the research continuum.

Lupus is the prototypical autoimmune disease. It afflicts over 1.5 million persons -- 90 percent of whom are women. At the same time it disproportionately affects African Americans, Hispanics, Asians, and Native Americans and is up to three times more common among those populations. The nature of the disease, its destructive treatments and the wide range of the body's organs affected make it a particularly complex disease for conducting clinical trials. Lupus patients only recently celebrated the first FDA approval of a new drug to treat their disease and many more are needed. Other, more widely used treatments today still involve use of steroids and chemotherapy drugs - all of which are toxic and ravage the body and its organs.

Since its founding in 2000, the driving mission of the Lupus Research Institute has been to invest in pioneering, innovative research searching for better, safer treatments and for the cause and cure by raising funds in the private sector. The LRI program has been highly successful and our substantial investment has provided academic-based investigators with the ability to initiate studies, publish groundbreaking breakthroughs and become successful in obtaining highly competitive NIH funding to continue their research. Our investment of over $45 million has leveraged another $127 million in NIH research which has reinvigorated lupus research, leading to multiple new discoveries and the identification of over 20 new lupus biomarkers for new investigation. Thus far we have awarded over 150 grants to researchers in 70 academic and research institutions.

Patient engagement and participation has been highly integrated into all aspects of our efforts leading to research momentum and progress moving discoveries along the continuum from basic investigations, through clinical and transitional work toward clinical trials.

Currently we are focused on expanding patient-centric research and clinical trials in the belief that the scientific foundation is now in place to transform lupus patients' lives with a new emphasis in these areas. From the very beginning, innovation has been the cornerstone of our Institute and our philosophy. We focus on cutting-edge, novel research with the intention to deliver new treatments, prevention and a cure, and to stop disease progression and damage in the very near term. Advised by a world class Scientific Advisory Board, we work hand-in-hand with the biopharmaceutical industry, academia, other lupus research organizations, NIH, CDC and the FDA in pursuing our common goals.

As you move forward in the 21st Century Cures initiative toward developing legislation, we encourage you to:

  • Provide incentives to foster more clinical research, networks and clinical trials, accelerate drug discovery, and allow innovation to develop safe and effective drugs;
  • Insure that FDA has the flexibility needed to innovate and deal with clinical trials for complex diseases like lupus;
  • Integrate greater -- and meaningful -- patient participation at all stages of the research and drug approval processes, including representative levels of women and minorities; and
  • Support measures to enhance the numbers and conduct of clinical trials by the NIH and strengthen appropriations for the agency's biomedical research programs in general.

We are most grateful for the collaborative approach you have undertaken to consider these important topics. We thank you for devoting precious Congressional time and attention to patient engagement and clinical trials and we stand ready to work with you in any way as you move toward developing legislation to enhance the lives of lupus patients.


Margaret Dowd
President and CEO