Jan Wyatt, RN, PhD
Rheumatoid Arthritis Patient
Volunteer – Arthritis Foundation (AF)
Chair, AF Public Policy Committee
Member, AF Board of Directors
before a meeting of the
FDA Arthritis Advisory Committee
(NDA) 203214, tofacitinib tablets, Pfizer Inc.,
May 9, 2012
Good morning, Chairman and members of the Committee. My name is Jan Wyatt. In 2004, at the height of my professional career as a nurse executive and nurse practitioner, I was diagnosed with rheumatoid arthritis (RA). For me and the 1.5 million other Americans with rheumatoid arthritis, the pain and loss of function that we suffer is, and will always be unacceptable. As I have struggled to manage different biologic and other drug therapies to achieve disease improvement, I have been pleased to volunteer with the Arthritis Foundation (AF), the nation’s leading voluntary health agency working for the past 60 years on behalf of the more than 50 million Americans with arthritis, including more than 1.5 million Americans and 300,000 children with rheumatoid and juvenile arthritis respectively. As a member of the Arthritis Foundation’s Board of Directors and as the volunteer Chair of the Arthritis Foundation’s Public Policy Committee, I am pleased to have the opportunity to appear before you this morning.
For over six decades, the Arthritis Foundation has been committed to programs and activities to prevent, control and cure arthritis. While we have made progress in our efforts, much more needs to be done. RA is a complex autoimmune systemic disease. It is not fully understand why women and young girls are disproportionally affected. It is not fully understand why patients with RA have an increased risk of cardiovascular disease and an increased risk of malignancy. There is no cure and as the progression of this disease is managed, as patients we are fully aware of the expectation of a shortened life span. Though we are anxious for a cure, patients with RA want and need better treatments now.
A little over a decade ago the care and treatment of RA was transformed with the introduction of biologic disease modifying agents. To date, the FDA has approved nine biologic formulations for the treatment of rheumatoid arthritis. These treatments have indeed been transformational for many, though not all. Instead of the expectation of progressive disability that could lead to the need for a wheelchair, biologic therapies have been a significant reason for the improvement of function, the limitation of pain and the improvement of quality of life for both adults and children.
While the Arthritis Foundation is not in a position to comment specifically on the applicant's supporting data or on Tofacitinib itself, we support all efforts to help ensure that the widest range of safe and effective therapies are available to people who have rheumatoid arthritis and related diseases. As scientific development continues, the AF supports the promise of a safe, effective oral pill formulation for treatment of RA. An oral form of RA treatment is especially needed for patients, who for a wide variety of reasons, are unable to manage biologic infusion or injection administration.
With the opportunities offered by new therapies focusing on inflammatory pathways, the Arthritis Foundation recommends that the FDA emphasize patient education and strengthen patient related “pharmacovigilance” for this and any RA therapy. As the FDA moves forward with its review, we ask that the potential launch of tofacitinib, serves as an opportunity for the FDA to renew policies to strengthen trust and transparency among and between patient’s, providers, and policy makers. Today, selection of the best treatment strategies for continues to be limited because of our lack of knowledge of the influence of gender, race, age, co-morbidities, and other drug to drug reactions. For the past decade we have little or no comparative effectiveness research that would assist in identifying the right medication for the right patient at the right time. Many RA patients try as many as three biologic therapies in an 8-12 year period as they work with their providers to secure the best responses. Unfortunately, many of the current therapies do not work for some patients so the need for novel therapies such as this one is especially acute for this patient population of non-responders.
As new treatments are launched, policies must be directed at engaging patients and providers in active monitoring of the safety and effectiveness of these new and older therapies. As both the disease and treatment response patterns for patients are often unpredictable, we urge a renewed focus on the involvement of patients in pharmacovigilance - the important on-going monitoring of both positive and negative effects of drug treatment.
The key will be increased patient education and engagement to ensure monitoring for safety in the “real world” vs. clinical trial conditions. While pre-production safety occurs within strict boundaries of clinical trials, long-term safety and monitoring for the safety and efficiency of new drugs remains a challenge. We continue to experience reports of serious health threats created by “approved” drugs. These threats heighten the need for long term patient-focused monitoring. If approved, tofacitinib will be the first in its class of small molecule disease modifying pills for the treatment of rheumatoid arthritis – communication about the risks, benefits, and potential side effects associated with this new therapy will be critical.
The Arthritis Foundation urges development of a systematic way to engage patients meaningfully in post marketing activities including providing them with appropriate information and communication tools. We recommend that drug related patient education, beginning with manufacturer’s packaging inserts, be re-tailored. Lengthy, fine-printed, highly technical package insert materials are often ignored by patients and providers and more is needed to provide relevant clinical information so treatment monitoring can be improved. Engagement of patients in monitoring for the effects of drug therapy could be facilitated through secure patient registries and engagement of the Arthritis Foundation and other volunteer health related organizations.
The Arthritis Foundation also urges the development of policies to support full access to new RA treatments. Tofacitinib is a new oral small molecule therapy - we understand that economic efficiencies may have been achieved in the production and manufacturing compared to biologics. If this is the case, these savings should be passed onto patients. Lower cost therapies will indeed significantly increase access to care for thousands of patients with RA.
In summary, my remarks today center on patient access and the patient-driven perspectives that are relevant to the Committee's work. The AF supports FDA’s efforts to provide the widest possible range of safe and effective therapeutic options to help patients appropriately manage the pain and disability of rheumatoid arthritis. In addition the potential launch of this new treatment provides a unique opportunity to broaden patient engagement in the long term monitoring of the efficacy and safety of this new drug. The Arthritis Foundation urges the Committee to strengthen patient education and engagement of RA patients in the long term monitoring of drugs. We thank you for the opportunity to share the RA patient perspective and look forward to additional opportunities to work with the FDA to improve the lives of all patients with arthritis. I would be pleased to address any questions or concerns.