Rare Disease Strategies in the Booming US Market

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Rare disease – defined in the US as a disease with a maximum patient population of 200,000 – is perhaps the most salient issue at the nexus of innovation, access, and affordability in the country.

 

As FDA CDER director Dr Janet Woodcock indicates, “rare diseases have been rising as a target of pharmaceutical development. Last year around 41 percent of approvals were novel approvals for rare diseases. Part of that is because of targeted therapies that make more rare diseases, and part of it is because rare diseases themselves are being targeted for development. That is a big change from two decades ago when most development was in common diseases, antimicrobials, cardiovascular diseases and so forth.”

 

Last year around 41 percent of [US FDA] approvals were novel approvals for rare diseases

Dr Janet Woodcock, CDER, FDA

 

Another supporting factor is legislation like the Orphan Drug Act (ODA), which among other things offer sponsors development incentives like qualified clinical testing and a waiver of the prescription drug user fee for the NDA filing. As the National Organization for Rare Disorders (NORD) president and CEO Dr Peter Saltonstall expresses, “before ODA, there were only 34 orphan drugs approved in the US and today, we have over 700.”

 

At the same time, these innovations have come at significant cost. According to AHIP data, the average annual orphan drug cost has risen from USD 7,136 in 1997 to USD 186,758 in 2017. However, NORD president and CEO Saltonstall cautions that the conversation should not center myopically on price: “in 2019, only 9.2 percent of the overall US drug spend was on orphan drugs so we should not obsess on ‘fixing’ that 10 percent. We believe rare diseases R&D should be encouraged, we want to retain the incentives for doing so under ODA, and we believe that it is important that companies have the ability and incentives to develop therapies for these small patient populations”, since, as he points out, “90 percent of patients with rare diseases still do not have any approved therapies for their conditions, so the unmet medical needs are urgent and critical.”

 

90 percent of patients with rare diseases still do not have any approved therapies for their conditions, so the unmet medical needs are urgent and critical

Peter Saltonstall, NORD

 

Furthermore, he laments, “we are very concerned about access, and it is not about price alone. For instance, a few years ago, we did not require prior authorization from insurers for many rare disease therapies. Today, of the nearly 50 therapies we support through our Patient Assistance Program, 100 percent of them require prior authorization. It means [insurers] are influencing the decision of whether patients receive access to these therapies.”

 

Another aspect of promoting access, Kyowa Kirin North America president Gary Zieziula asserts, is “patient-finding initiatives”, which explains the fact that Kyowa Kirin “have digital patient-finding programs in place, and we are building much more robust digital education and communication programs to reach patients and healthcare practitioners (HCPs). We are also actively engaging with patients and advocacy groups, as well as participating in conferences and congresses.” He also hopes to leverage digital tools more fully in this endeavour, sharing, “we look forward to having a much more comprehensive digital infrastructure by 2021 or 2022, supported by a new Digital Center of Excellence, to grow our role in the community.”

 

[Kyowa Kirin has] digital patient-finding programs in place, and we are building much more robust digital education and communication programs to reach patients and HCPs

Gary Zieziula, Kyowa Kirin

 

Takeda US president and president of global portfolio commercialization Ramona Sequeira agrees on the importance of patient-centricity, pointing out, “with rare disease, a healthcare provider might have just one patient with that type of disease using our medication. So naturally, the provider has not had the chance to build experience around that disease or therapy. The patient might feel alone, and the doctor might feel alone.” Based on Takeda’s value system ‘Patient-Trust-Reputation-Business’, she continues, “we want to figure out how to alleviate this problem. The first step is getting close to patient communities and patient advocacy groups to really understand their needs and challenges. Based on what we learn, the second step is to increase support through medical and patient services.”

 

As an example, she suggests, “a provider might not need as much promotional support but would welcome more medical information to help identify the right patients and learn more about the disease progression. Patients might need help navigating a difficult diagnostic process or complex reimbursement systems.” This is what being “committed to delivering success to our customers and patients” means to Takeda.


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