Trying to harness the Power of 11

This week, Rare Diseases South Africa officially turned 11 years old. Over the past decade, our journey in advocating for rare diseases has been nothing short of extraordinary, and we owe many of our success to your unwavering support and dedication, as well as our team who pull together every day for the benefit of our community.

Together, we have achieved remarkable milestones and touched countless lives. As we celebrate yet another year in existence, we want to share some of the highlights and successes we've accomplished over the years.

The Good

Some of our key successes over the years have included:

• Raising Awareness: From our humble beginnings, we have tirelessly worked to bring rare diseases to the forefront of public and medical community attention. Through social media campaigns, partnerships with media outlets, and active communication campaigs, we have successfully increased awareness and understanding of rare diseases, ensuring that they receive the recognition they deserve.
• ?Support for Patients and Families: Understanding the unique challenges faced by those affected by rare diseases, we have established comprehensive support programs. Our patient advocacy network now includes over 6,000 families, offering them access to resources, support groups, and navigation services. Our annual patient family days have become an exciting event of the calander, bringing hope, community and an opportunity providing to share experiences.
• Policy and Advocacy: Our organisation has played a pivotal role in influencing policy changes to benefit the rare disease community. Through relentless advocacy, we have contributed to the passage of key legislation that ensures better healthcare access, increased funding for rare disease research, and enhanced patient rights. Our voice in the policy arena has never been stronger, thanks to your support.
• Educational Initiatives: Education remains at the core of our mission. Over the past 11 years, we have developed and distributed educational materials to healthcare providers, patients, and the general public. Our online webinars and capacity building events have reached thousands of participants, equipping them with the knowledge to better understand and manage our rare conditions.
• Global Outreach: Our commitment to rare disease advocacy extends beyond borders. We have established partnerships with international organizations to share knowledge, resources, and support, and continue to actively assist with leading the African Rare Disease Alliance. Our global initiatives have helped improve diagnostic accuracy and treatment options in underserved regions, bringing hope to many who previously had none.?

As we celebrate our 11th anniversary, we reflect on these achievements with immense pride and gratitude. However, our work is far from over. With your continued support, we aim to expand our reach, and advocate even more vigorously for the rare disease community.

The Bad

As is with any organisation, there have been some things that haven't gone our way. Over the past decade, healthcare delivery in South Africa, particularly for rare disease patients, has faced numerous obstacles. These challenges underscore the critical need for our continued advocacy and support.

• Limited Access to Specialized Care: One of the most pressing issues has been the limited access to specialised care for rare disease patients. South Africa’s healthcare system often struggles to provide the necessary expertise and facilities required for the diagnosis and treatment of rare diseases. Many patients face long waiting periods and must travel great distances to receive appropriate care, leading to delays in diagnosis and treatment.
• Shortage of Healthcare Professionals: The shortage of trained healthcare professionals, and suitable posts to provide further training to doctors for improved knowledge about rare diseases has been a persistent problem. This gap in expertise means that many rare diseases go undiagnosed or are misdiagnosed, causing significant distress and health deterioration for patients. The lack of continuous medical education on rare diseases further exacerbates this issue.
• Financial Barriers: The high cost of healthcare poses a substantial barrier for rare disease patients and their families. The expense of diagnostic tests, treatments, and necessary medications is often prohibitive, especially since many rare disease treatments are not accessible in either the private or government sector. This financial strain forces many families to make difficult decisions, sometimes foregoing essential treatments.
• Inadequate Policy and Legislation: Despite some progress, there remains a lack of comprehensive policy and legislation specifically addressing rare diseases in South Africa. This absence of a supportive legal framework means that rare disease patients often do not receive the protection and consideration they need within the healthcare system. Advocacy for improved policies has been a slow and arduous process. Whilst we have worked hard on developing our Rare Disease Policy Framework, the implementation of National Health Insurance, and the lack of government prioritisation continues to hinder our efforts.
• Supply Chain Disruptions: The past decade has seen significant disruptions in the medical supply chain, exacerbated by economic challenges and global events. These disruptions have led to shortages of essential medications and medical supplies, critically affecting the continuity of care for rare disease patients. With many global pharmaceutical companies making the decision to withdraw from the local market, due to the lack of certainty regarding reimbursement, and the tough economic climate, the issue of supply chain shortages and lack of availability will likely continue.
• Stigma and Lack of Awareness: Stigma and lack of awareness about rare diseases persist, contributing to social isolation and psychological stress for patients and their families. Misunderstanding and prejudice within communities, and sometimes even within the healthcare system itself, hinder effective care and support.?
• Research and Data Gaps: A lack of robust data and research on rare diseases in South Africa impedes the development of effective treatments and healthcare strategies. The scarcity of epidemiological data means that many rare diseases remain poorly understood, limiting the ability to advocate for and implement necessary healthcare services.

And the Ugly...

• #TheFightForZach legal saga: The case of Rare Diseases SA against in Medical Aid, for young Zachary de Wet has starkly highlighted the pervasive delays in the South African legal process and the resulting inability to obtain timely justice. The De Wet family has endured years of procedural setbacks and administrative bottlenecks, which have severely hindered our quest for a fair resolution. Despite clear evidence and a pressing need for adjudication, the legal system’s inefficiencies have prolonged the ordeal, causing emotional and financial strain, to both the De Wet family and RDSA. This case underscores the critical need for judicial reform to ensure that justice is both accessible and prompt, particularly in matters involving essential healthcare rights.

With our next High Court date set for November 14th, and an outstanding date still be determined at the Council For Medical Schemes, the emotional and financial fatigue has set in. To date, we still continue to fundraise for not only the outstanding legal fees for this matter (which are still in excess of 150k), but also those still to come, as we aim to ensure Prescribed Minimum Benefit legislation is appropriately applied.

Donations towards our efforts in this regard are welcomed.

You can make a direct donation to our legal fund: https://www.givengain.com/campaign/legal (Section 18A available) or you can make a contribution to our legal fees directly to our attorney trust account.

Banking Details: Account Name: HJW Attorneys Trust Account: 627 475 134 49 Bank: First National Bank Branch Number: 204809 Ref: M02024

• Impact of the COVID-19 Pandemic: The COVID-19 pandemic further strained the healthcare system, diverting resources and attention away from non-communicable diseases, including rare diseases. Many patients experienced interruptions in their treatment plans, reduced access to healthcare services, and increased vulnerability to health complications.

Furthermore, the pandemic has had a profound negative impact on charities worldwide, and RDSA was no exception. The pandemic-induced lockdowns and restrictions led to the cancellation of numerous fundraising events, severely diminishing financial support crucial to cover our operational costs. Additionally, the economic downturn resulted in reduced donations from both individuals and corporate sponsors, with a 116% reduction in donations received during the period of March 2020 - Feb 2023.

RDSA also faced significant logistical challenges, as the healthcare system's focus shifted to addressing the immediate crisis, causing disruptions in ongoing projects and support services. This diversion of resources and attention has hindered our ability to provide critical assistance and advocacy for rare disease patients, exacerbating the vulnerabilities of an already marginalised group.

?This year we have reduced our staff headcount significantly, leaving us running on proverbial fumes. We have closed our brick and mortar offices, and have cut back on projects due to capacity limitations. April 2024 marked the first time since our inception, where RDSA could not assist parents and patients with transport or socio-economic assistance due to the depletion of our Patient transport and services fund. A last minute call to support this fund resulted in R25 000 being raised, however, this only covers 6 weeks of transport for our indigent patients being seen in state facilities.

The Present

While these challenges are daunting, they also fuel our determination to continue fighting for better healthcare delivery for rare disease patients in South Africa. We are committed to advocating for policy changes, increasing awareness, supporting research, and providing the necessary assistance to those affected.

?We continue to seek your support in our efforts to address these issues. Your continued financial support is crucial to sustain our programs, fund vital research, and advocate for better healthcare policies.

We want you to know that we deeply appreciate your generosity and commitment to our cause. Together, we can overcome these obstacles and ensure that every rare disease patient receives the care and attention they deserve.

Thank you for standing with us and making a difference in the lives of those who need it most.

Get Involved

Should you wish to donate, please make use of the following options listed below. Please note that all donations over R100 are entitled to a Section 18A certificate. Kindly send your proof of donation to [email protected] to request your certificate be issued.

• DIRECT EFT

Account Holder:

Rare Diseases South Africa Bank:?First National Bank Branch Code:?251655

Acc No:?624 11658 034 Ref:?Your email address?

• PAYPAL: [email protected]
• SNAPSCAN
• ZAPPER
• YOCO