Supporting the Kidney Disease Community

Kabir NathPresident and CEO
I’m proud and excited to be working for Otsuka.In case you missed the news today, the U.S. Food and Drug Administration approved the first treatment for autosomal dominant polycystic kidney disease (ADPKD).Thanks to the dedication and focus of my Otsuka colleagues, we’re bringing hope to patients and families facing this serious genetic disorder.It’s one of those moments that all of us here at Otsuka live for.  For many of us, the development of a new treatment for an unmet medical need is one of the reasons to work in the life sciences industry.You can read all about the details of this news in the press release, but I wanted to share some of my thoughts about what this new treatment means to the patient community.

The Impact of ADPKD

ADPKD is a genetic disorder diagnosed in about 140,000 people in the US [1] and is the fourth leading cause of end-stage renal disease in the U.S.[2]It is not just debilitating, but also painful.  Over time, fluid-filled cysts develop in the kidneys, enlarging them and limiting their ability to function normally, leading to kidney failure in most patients. [3]To give you some sense of what this can be like, keep in mind that an average kidney is about the size of a fist.  In patients with ADPKD, kidneys can get as big as a football and weigh 30 pounds each.[4]ADPKD can be passed from generation to generation.  Parents with ADPKD have a 50% chance of passing the disease to their kids.[5] [6] The consequences of the disease include end stage renal disease, kidney dialysis, and, in some cases, kidney transplantation.  And, to date, there have been few options. This approval, which provides the first drug treatment for adults with ADPKD, is a testament to the hard work and dedication of many of my colleagues in research and development and throughout Otsuka.However, it isn’t enough to gain approval for new treatments.  At Otsuka, we also want to make sure patients have access to the medications they need. 

Access

Otsuka is committed to supporting patients, and will work hard to make sure that those patients who could benefit can get this our medicines.To that end, we’ve established a number of patient support programs for eligible patients, including a co-pay savings program.The Otsuka Patient Assistance Foundation (OPAF) provides another option for eligible patients who are unable to afford their medication.  A separate and independent entity, OPAF’s mission is to eliminate the financial barriers to Otsuka pharmaceutical therapies for eligible uninsured or underinsured patients.  To learn more about this program, please visit www.otsukapatientassistance.comAt Otsuka, we take great care to price our medicines and products based on the innovation involved in their development, as well as the value they bring to patients and society.  In this case, we took into consideration a variety of pricing factors, including the costs associated with our clinical development program.Pharmaceutical research and development can be extremely time and resource intensive, with numerous hurdles associated with designing and executing clinical trials and obtaining regulatory approval.Our development program spanned more than 10 years, and included more clinical studies in ADPKD than any other clinical development program. This clinical research included two of the largest late stage (Phase 3) trials in ADPKD to date and involved 2,815 participants.  In fact, through our research program, we played an important role in advancing the scientific body of knowledge about ADPKD.When deciding the pricing of the new treatment, we also examined the value this new medication delivers to patients and society.By impacting the progression of the disease, patients may have more time before the advent of end stage renal disease and kidney transplant or dialysis, both of which bring additional health issues for patients and significant costs for the healthcare system.  

Taking a Long-Term View

We also realize that there is still research to be done to better understand ADPKD, and this new treatment.  We are committed to the continued investigation of products to provide healthcare providers and patients with further evidence of long-term safety, efficacy, and cost-effectiveness. The pricing also took into account the anticipated costs of other future research and development in our ongoing efforts to discover and develop new and innovative therapies that may help improve patients’ lives.At Otsuka, we take a long-term view.  When setting pricing, we recognize that our activities will be ongoing, and will involve additional research to attempt to solve some of the world’s most complex health issues.We’re pleased with today’s news which we hope will represent a significant development for members of the ADPKD community. Our commitment is not just for today, but for tomorrow.  By ensuring access to this medication while also ensuring financial resources are available for future research and development, we believe we not only serve today’s patients, but tomorrow’s as well.April 2018          01US18EUC0085[1] Data on file. Otsuka America Pharmaceutical, Inc., 2018[2] Lentine, K. L. (2015). Renal Function and Healthcare Costs in Patients with Polycystic Kidney Disease. Clinical Journal of the American Society of Nephrology, 5, 1471-1479.[3] Lentine, K. L. (2015). Renal Function and Healthcare Costs in Patients with Polycystic Kidney Disease. Clinical Journal of the American Society of Nephrology, 5, 1471-1479.[4] https://www.pkdinfo.com/about-adpkd.html[5] Autosomal Dominant Polycystic Kidney Disease Fact Sheet. NIH Reports. Accessed online on March 30 2018 at https://report.nih.gov/NIHfactsheets/ViewFactSheet.aspx?csid=29&key=A#A\[6] Torres VE, Grantham JJ. Cystic diseases of the kidney. In: Taal MW, Chertow GM, Mardsen PA, Skorecki K, Yu ASL, Brenner BM, eds. Brenner & Rector’s The Kidney.Philadelphia, PA: Elsevier Saunders; 2012:1626-1667  
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