Why Patient Advocacy Organizations are Essential to Families Coping with Rare Diseases

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In the US, a rare disease is defined as a debilitating condition affecting only one in 1,630 people. According to the National Press Foundation, around 350 million people worldwide have a rare disease. Almost 85% of diseases have a genetic origin, stemming from unique gene defects. Considering there are around 7,000-10,000 rare diseases, it’s unsurprising that only 5% of rare diseases have approved therapy. This has often led to individuals being misdiagnosed and pursuing the wrong treatment, which has burdened not only sick members but also their families who have suffered financially and socially.To help patients and families cope with these issues, more patient advocates and social programs are helping them through the complex social and medical processes of living with a rare disease. With an increasing demand for patient support, it has led to over 400 universities implementing updated Global Standards for Social Work Education and Training, with many of the new programs aligning with healthcare. This wider social focus allows those in patient advocacy organizations to help a wider pool of people navigate life’s intricacies and challenges, especially those suffering from rare diseases. And with more professionals on the ground, more patient advocacy organizations will become essential support to families affected by rare conditions.

Importance of patient advocacy organizations

Patient advocacy organizations (PAO), also known as patient support groups, are volunteer-led charities or large-scale, professional organizations. Regardless of size, they all provide patients, families, and caregivers with resources and guidance related to a serious disease, such as a rare disease. These include educational materials, emotional support, and clinical referrals, but sometimes other groups go above and beyond in their assistance. Listed below are some specific ways they can help and why they are essential to patients with rare diseases:

Ease patient burden

Aside from the financial strain, it can be difficult for patients already experiencing uncomfortable or painful symptoms to go out of their way for on-site visits. PAOs can provide patients access to health technology that makes data more efficient to collect and leverage in researching treatment options. Furthermore, PAOs can also pave the way for developing orphan devices, tech designated for patients with rare diseases, to improve the quality of life of patients in some ways. For example, children with thoracic insufficiency syndrome, a rare disease that prevents normal breathing, can use a vertical expandable prosthetic titanium rib to improve their condition.

Advocate for more inclusive and cost-effective drugs

PAOs play a crucial role in advancing drug development. Aside from helping share meaningful input from patients during the drug approval pathway, they also can demand that clinical trial populations should be more representative of patients with the disease. In the case of the controversial drug Aduhelm, a treatment for Alzheimer’s, many people protested against its country-wide usage. The drug’s trial only included 11 Black and 67 Hispanic participants, despite Alzheimer’s affecting more Black and Hispanic people than white individuals — which became a point of stand for many PAOs.

Work towards greater inclusivity

Inclusivity is something PAOs and medical groups are seeking to develop, ensuring that people are able to get help whatever their racial background, or economic circumstances. A great example of this is the link between WeightWatchers and Amerigroup Georgia, which is targeting heart disease in the area. Heart disease is not just as common as you think – there are many rare types of heart disease which can be staved off by lifestyle changes, such as takotsubo cardiomyopathy and prinzmetal angina. By leveraging a third-party such as Amerigroup, WeightWatchers are able to get vouchers to those at risk, helping them towards natural weight loss which will reduce the risk of both rare heart diseases and those that are all too common. Disease, rare or otherwise, does not discriminate, and neither should prevention advice or help. Thanks to this example of patient advocacy, steps are being taken to ensure that this is the case.

The role of IndoUSrare

Although medical studies have been shifting towards diversity, it largely doesn’t reflect the racial and ethnic makeup of the global population affected by the targeted disease. This lack of diversity is concerning, as these underrepresented minorities and people in low-and-middle-income countries (LMICs), such as India, are denied access to life-saving therapies and potential treatment for common and rare diseases.

In the US alone, there are over 4.2 million Indian diasporas, yet less than 2% of global clinical trials have involved India. For rare diseases that already suffer from poor research, having minimal clinical trials for afflicted populations can keep patients out of recovery. Instead of facing a long wait with questionable safety and efficacy of treatments, patient support groups, including the Indo-US Organization for Rare Diseases (IndoUSrare), are advocating for the inclusion of people of Indian origin in global clinical trials.

Our team at IndoUSrare aims to address the needs of patients with rare diseases by accelerating research and the development of effective treatments. This group harnesses partnerships with not only companies involved in drug and treatment development but also collaborations with other stakeholders to leverage shared resources. In addition to research advancement, IndoUSrare offers different resources and helps with following up on the progress in finding patient diagnoses.

Challenges patient advocacy organizations face

Although PAOs are a reckoning force, they still face multiple challenges in mobilizing patient advocacy. Smaller organizations may struggle with business negotiations with potential partners, as their members may not necessarily be professional, privately paid advocates. On the other hand, there is also a struggle to meet the increasing demand, as groups find it difficult to balance their advocacy efforts and marketing.

With rising healthcare costs and an increasing number of patients, PAOs have become a critical part of the industry— helping patients overcome medical barriers from representation to treatment access. While there are several hurdles, their impact is profound within underrepresented populations, ultimately leading them to a better quality of life and improved outcomes for patients and their families.

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