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Access to Therapy Around the World

As you know, the Histiocytosis Association has a multifaceted mission and under that umbrella, oversees many different activities all aimed at providing support, resources, and advocacy along every step of the histiocytosis journey. One of the areas of our work involves providing information about the latest treatment options for each histiocytic disorder. Patients, families, and physicians turn to the Histiocytosis Association for information about new therapy clinical trial opportunities, clinical guidelines and consensus guidelines, protocols, and details on various options for treatment based on the location and severity of the histiocytic disorder and age of the individual impacted.  

While this information is very beneficial for guiding next steps and possible courses of treatment, accessing these treatments is not always straightforward or easy. Histiocytic disorders are rare orphan diseases, meaning 5-6% of the population, or 1:200,000 in the US have this diagnosis. Treatment for rare orphan diseases can be challenging.

While many traditional or conventional treatments (such as chemotherapy) can be used for histiocytosis, other treatments have been developed and/or tested for use with histiocytosis and may have a better outcome and therefore may be considered.

Some treatments, while effective, are still part of a clinical, which means that a patient may not be able to use that therapy without joining the trial or may need to request special access. Some treatments are very expensive and insurance companies/government/payors hesitate to approve coverage of the treatment. Treatments approved by the U.S. Food and Drug Administration (FDA) are not always approved in other countries due to regulatory differences, and vice versa.

Challenges in Access to Treatment

Some of the challenges to access of treatment and/or development of new treatments include:

  • Small patient population: with so few people impacted by a particular diagnosis, pharmaceutical companies may be hesitant to invest in research, development, and production of drugs for rare diseases. This is why the Histiocytosis Association continues its Annual Research Program, which funds new research into histiocytic disorders through the generosity of the community.
    • Additionally, the patient population may be spread out due to the rarity of the diagnosis, which can make distribution difficult for pharmaceutical companies.
  • High development costs: developing a drug for any disease or condition is an expensive and time-consuming process. Pharmaceutical companies need to recover their investments to sustain operations, which can lead to higher prices for these treatments.
  • Regulatory challenges: the process of having a treatment approved can be challenging for any treatment. However with smaller or limited resources, the patient population size for example, this process can be even more challenging for rare disease treatments. (See additional reading for ways the FDA is trying to make this less challenging for rare orphan diseases!)
  • Pricing and reimbursement: the higher costs associated with developing these treatments often result in higher prices for the medications, which can create challenges with reimbursement by healthcare systems and insurance providers. This can limit patient access to these drugs.

Addressing these challenges requires collaboration across various stakeholders, including the Histiocytosis Association, healthcare providers, other patient advocacy groups around the world (including the OR Asociacion, the ECDGA, the HA of Canada, and many others), pharmaceutical companies, regulatory authorities, policymakers, and more.

What We’re Doing to Help

In August, the Histiocytosis Association will be participating in EveryLife Foundation’s Rare Across America, which is an organized opportunity to meet with Members of Congress at their in-district offices and educate them on the issues that are most important to the rare community by sharing our story. Many of you signed up to participate in this alongside us!  This is one way to continue to advocate for change.

In October, we will meet with our long-standing partners, the Histiocyte Society, at their Annual Meeting. During this meeting, we will sit down with international advocacy organizations and the Histiocyte Society Board to discuss other ways to work together to positively impact treatment access, information sharing, and other important issues surrounding histiocytosis.

We look forward to sharing more about these efforts, including some exciting new projects in the coming months, as they continue to take shape!

Additional Reading:

More information on the approval process and regulatory terms within the United States can be found at the link below; while this site is specific to COVID-19, the terminology and processes are the same: Understanding the Regulatory Terminology of Potential Preventative and Therapeutic Drugs for COVID-19 | FDA

CDER’s ARC Program | FDA –  ARC’s Vision: Speeding and increasing the development of effective and safe treatment options addressing the unmet needs of patients with rare diseases.

ARC’s Mission: To drive scientific and regulatory innovation and engagement to accelerate the availability of treatments for patients with rare diseases.