21st Century Cures Initiative

The Honorable Fred Upton
Chairman, House Committee on Energy and Commerce
2125 Rayburn House Office Building
Washington DC 20515

The Honorable Frank Pallone
Ranking Member, House Committee on Energy and Commerce
2125 Rayburn House Office Building
Washington DC 20515

Re: 21st Century Cures Initiative

Dear Chairman Upton:

On behalf of 7.5 million Americans living with psoriasis and psoriatic arthritis – our nation's most common autoimmune disease – National Psoriasis Foundation (NPF) is writing to commend you for undertaking the 21st Century Cures Initiative. We applaud your effort to improve the discovery, development and delivery of medical treatments and cures. It is clear that a significant amount of work has gone into this effort over the past year. We applaud the Committee's recently release draft legislation that both includes patients in the biomedical research process and addresses unmet medical needs.

As the Committee seeks to build upon the foundation in the discussion draft, the National Psoriasis Foundation (NPF) urges you to strongly consider the following specific recommendations:

  • Increase funding for NIH;
  • Promoting Pediatric Research;
  • Further strengthen and enhance the Patient-Focused Drug Development and related provisions to maximize the level of meaningful patient engagement throughout the Food and Drug Administration (FDA) review process and recognize that the personalized nature of many diseases and conditions often require multiple points of view; and
  • Establishing the Bipartisan Telemedicine Working Group

Furthermore, while we do not have specific recommendations for the following sections, we applaud the following sections of the draft:

  • National Institutes of Health Planning and Administration
  • Supporting Young Emerging Scientists
  • Advancement of National Institute of Health Research and Access
  • Modern Trial Design and Evidence Development
  • Streamlining Clinical Trials

Title I: Discovery

The NIH Health Reauthorization aligns with the NPF's strategic plan of accelerating the discovery to cure psoriatic disease. We plan to double the number of researchers studying psoriasis and psoriatic arthritis while also cutting in half the number of people who say these chronic conditions are a problem in their daily lives as part of our strategic plan and mission to cure psoriatic disease and improve the lives of those affected. Accelerate discovery to cure psoriatic disease by involves:

  • Increasing the number of dollars invested annually by the National Institutes of Health (NIH) to more than $18 million—a 50 percent increase;
  • Increasing the number of scientists studying psoriatic disease by 50 percent;
  • Creating a community of 1,000 people with psoriasis and psoriatic arthritis who collaborate in research;
  • Initiating annual progress reports in key areas of psoriatic disease research such as, causes, diagnosis, prevention and cures;
  • Invest 30 percent or more of annual NPF expenses in research.

We commend the committee leaders for drafting language that would authorize and provide increased funding for the NIH, including $10 billion over the next five years in mandatory funding through an NIH Innovation Fund. We are encouraged the committee leadership recognizes the critical importance of maintaining NIH as a national priority. NIH funded-research is essential if we are to continue to improve our nation's health, sustain our leadership in medical research, and remain competitive in today's global information and innovation-based economy. Research means hope for patients with psoriatic disease and other serious illnesses such as cancer, depression, and several comorbid conditions associated with psoriasis. We urge Congress to find a way to support NIH with mandatory funds so as to protect the agency from the uncertainty and ups and downs of annual appropriations.

We applaud this section, as the primary funder of scientific research in this country, it is important that the NIH is transparent in how it decides what to fund, effective in funding the most important research, and flexible enough to adapt to the rapidly changing research environment. We are aware that NIH's 27 institutes and centers already develop their own strategic plan, but it helps the overall impact of discovery if each of the institutes where aligned with their respective strategic plans. Further, we believe that high-risk, high-reward science can yield major breakthroughs, yet the current federal research structure often does not incentivize this type of research. This sections addresses some of these needs.

Fostering the next generation of researchers is critical to maintaining America's status as a leader in biomedical research. This provision fits in line with NPF's strategic plan to cultivate young and future scientists in pursuing careers in research, though we do not view this provision as preferential treatment that will hurt midcareer and senior investigators. We urge you to provide additional funding, to NIH to fund more emerging scientists, as it's critical to the long-term health of our nation's scientific efforts.

The needs of children with chronic disease are sorely unmet in this country, as data on the efficacy and safety of adult therapies are lacking. Building a dataset across multiple institutions sharing the common goal of helping kids will help meet this need. We ask that the committee boarders the aspects of both sections, particularly the Global Pediatric Clinical Trial Network to include all pediatric care.

There are no psoriasis treatments appropriately studied and labeled for pediatric use. Treatments currently on the market are used off-label for children, are not consistently effective, and are difficult to use with pediatric patients. Physicians are reluctant to use many treatments approved for adults with children due to possible long term or delayed side effects. Other times, certain long-standing treatments for psoriasis are used off-label for children and health professionals are placed in the untenable position of either prescribing a medication without sufficient information about the impact of the treatment on the pediatric population or withholding the treatment from children. When doctors do prescribe off-label for children, there are significant problems having these treatments approved for insurance coverage.

The NPF believes that data-sharing can help everyone in the research community to better understand and by extension support discovery and development of treatments for disease. We support Sec. 498E- Sharing Data through NIH funded research. Having access to NIH data would help non-profit foundation like NPF and others that support research best direct their resources to find a cure. Further, section 1102 (C) information required to be submitted to the registry and results data bank, including recruitment information helps to make to make data clear and accessible to anyone interested in exploring it. This has tremendous value to our country's research efforts as you never know who will make the next big discovery; thus we applaud this effort and ask that

Title II – Development

Patient perspective during the drug development process are vital to developing drugs that will best suit the needs of patients. Patients often bring a unique, valuable, and arguably the most important perspective to the disease they live with. Many people living with psoriasis and psoriatic arthritis are on multiple therapies; all FDA approved psoriasis treatment options have side effects and most have inadequate efficacy as the majority of patients and dermatologists note that they are unsatisfied with the results obtained from past and current treatment options. Although specific therapeutic recommendations exist for psoriasis, it is important that future treatment options are tailored to meet individual patient needs. Therefore it is vital the FDA understand the patient experience, including what types of reactions and side effects they have to medications, and the level of difficulty in accessing and adhering to medications. We support this Cures effort to strengthen the patient perspective in FDA's regulatory processes, and agree that a more structured framework for incorporating patients will help ensure their needs are addressed at every step of the process. However, we also urge you to identify ways patients can be involved early in the drug development process, and we seek clarification on how drug sponsors may be able to communicate with patients on drugs they are working on without legal ramifications. The drug development and approval process is not a one-size-fits-all proposition, as various treatment options work very differently for each psoriasis patient.

Biomedical research is critical to achieve scientific breakthroughs to develop improved treatments with fewer side effects and, ultimately, cures or means of prevention. At present, strict data use agreements largely prohibit researchers who have access to these limited data sets from building stronger and more meaningful data sets, which greatly limit the usefulness of the data.

We support both provisions of building a 21st Century streamlined data sharing as well as accessing sharing and using health data for research purposes. Researchers must be given more flexibility to work with the data within specified parameters in order to fully realize the potential value of the data as it pertains to clinical research and discovery. Presently, patient data is not operationally used for researchers while patients have shown to be generally willing to share their own medical records to support the uses of data to research.

Facilitating multicenter studies is critical to success of many scientific endeavors, particularly in the case of rare diseases. The Institutional Review Board (IRB) approval process is a necessary safeguard to ensuring appropriate patient protections; however, aspects of the process have proven to be cumbersome and redundant. We commend the Committee for supporting ways to create efficiencies and reduce duplications, which will help expedite reviews and ultimately allow research to progress faster. We support the regulatory changes proposed and encourage Congress to continue to identify other areas where other redundancies may be eliminated.


We appreciate your efforts to address telemedicine. Reducing or containing the cost of healthcare is one of the most important reasons for funding and adopting tele-health technologies. Telemedicine has been shown to reduce the cost of healthcare and increase efficiency through better management of chronic diseases, shared health professional staffing, reduced travel times, and fewer or shorter hospital stays. There is a dramatic shortage in pediatric rheumatologists, as many states have only 1 pediatric rheumatologist, and several states have none. Additionally, it bridges the big care gap for those who don't have easy or convenient access to a dermatologist.

We encourage you to continue to find pathways to navigate state licensure issues, so that physicians in one state can provide tele-health services to patients in other states. We also urge that any provisions include payment parity so that providers delivering services via telehealth are not unfairly punished with a lower reimbursement when the identical service is delivered in a face-to-face encountered.


The NPF thanks the Committee, again, for this opportunity to comment on the draft legislation. We believe that our recommendations and comments can make a tremendous difference in the lives of 7.5 million Americans living with psoriatic disease. We are optimistic about the legislation and working with the Committee and members further as the legislation is finalized. If you have any questions about these comments, please contact Mr. Quardricos Driskell, NPF's Health Policy Manager at [email protected] or at 404-455-5650. Thank you in advance for your consideration.

Leah McCormick Howard, J.D.
Vice President, Government Relations and Advocacy