Rare diseases are a group of disorders which individually affect a few patients. These diseases are often considered chronic, progressive, degenerative, and life-threatening. Often, they are termed as “orphan diseases” due to the neglect from the medical community.
Patients living with rare diseases (PLWRDs) are unceasingly confronted with several challenges, the most predominant of which is the requirement of lifetime medical management.
Imagine experiencing a similar condition. Perhaps one cannot fathom the quality of life of these patients. Consider the lives of their families, beset with significant social and economic burdens as they struggle to make ends meet for their loved ones.
The start of this year provided some relief and hope for PLWRDs, their carers and families when funding worth P104.9 million was included in the General Appropriation Act of 2022, specifically for the implementation of the Rare Disease Law or Republic Act 10747.
Passed in 2016, the said Law aims to improve access of PLWRD or patients suspected of having a rare disease, to comprehensive medical care. This includes access to available medicines and other health technologies that will treat or otherwise help them cope with their condition.
On Aug. 15, the Stratbase ADR Institute, in partnership with the Philippine Society for Orphan Disorders (PSOD) and UHCWatch, held a hybrid town hall discussion (THD) entitled “The State of Rare Disease Law: Continuing Implementation and the Delivery of Responsive Health Services to the Affected Population.”
The THD, which is actually a continuation of an initial discussion held last January, brought together different stakeholders from the academe, government agencies, civil society/patient organizations, and private sectors. It showcased milestones and updates, continuing challenges and a possible way forward that will support the progressive implementation of the Law.
Prof. Dindo Manhit, President of Stratbase ADR Institute, mentioned in his opening remarks: “continuous support and advocacy is needed to sustain a responsive healthcare system and that adequate resources is vital to constantly respond with the public needs, including those patients living with rare diseases.”
Dr. Carmencita Padilla, Chancellor of the University of the Philippines-Manila and Founding Chairman of PSOD, said that “it’s not a societal commitment. It is a commitment of the world to make sure that it is inclusive, that all patients will be able to benefit from the policy.”
According to Dr. Razel Nikka Hao, Director IV of the Disease Prevention and Control Bureau of the Department of Health, “a recently approved five-year strategic plan for the integrated management of [rare disease] condition… is rooted in the five guiding principles of access, integrated comprehensive care, evidence-based policies, inclusive communication and enhanced collaboration.”
“A total 159 rare diseases made it into the initial list that was submitted to the Department of Health,” noted Dr. Eva Maria C. Cutiongco-dela Paz, Executive Director of the UP-National Institutes of Health. “Additionally, a checklist provided assistance in deciding which rare diseases are the most important to be included in the list that will be covered by the Law.”
Dr. Durhane Wong-Reiger of the Asia Pacific Alliance of Rare Disease Organizations, who joined the discussion virtually from Canada, said that “there is a need to address the health of the most vulnerable for an inclusive society.” PLWRD are counted as among the most vulnerable and marginalized populations.
“Only 5% of rare diseases have an approved orphan drug,” Daisy Cembrano of the Pharmaceutical and Healthcare Association of the Philippines pointed out. She reiterated the pharma industry’s commitment to advocate for policy reforms based on four principles, namely, 1.) rare diseases as a public health priority, 2.) empowering patients and their communities, 3.) promoting continued research and development, and, 4.) ensuring sustainable patient access to diagnosis and care.
Senator Sonny Angara, in his message, asserted that “funding is a challenge, but hopefully the Legislature can provide more, as well as the National Expenditure Program provided by the Executive.” He expressed gratitude to the private sector for their help and encouraged people to partner with the government in helping make progress against rare diseases.
The senator also assured the public of continued support and motivated them to continue the dialogue to keep the attention on rare diseases.
Among those who also shared their insights in the event were Chris Muñoz of the Philippine Alliance of Patient Organizations, Cynthia Magdaraog of the PSOD, and Dr. Lizette Kristine Lopez of the Health Technology Assessment Council.
As the one who was tasked to moderate the said event, I would like to send my sincere appreciation to the PLWRDs who personally attended the event. Indeed, you are an inspiration to all patients. I hope that someday we will no longer use the term “orphan disorders” to describe your diseases, as all will have corresponding treatment.
Alvin Manalansan is the health and nutrition fellow at Stratbase ADR Institute and a co-convenor of UHC Watch.