“A child afflicted with a rare disease is painful and burdened by expensive medications, check-ups, and therapies to alleviate the symptoms of their sickness”
Interstitial Lung Disease, Agammaglobulinemia, Dystrophic Epidermolysis Bullosa, Homocystinuria, Phenylketonuria, Tyrosinemia … these are just five of the estimated 5,000 to 8,000 rare diseases that have been discovered globally. Does not sound so rare, does it?
According to the University of the Philippines National Institute of Health, there are at least 6,500 Filipinos suffering from rare diseases.
Most patients belong to the most financially challenged segment of the population who can only rely on government to help them.
Statistics show that approximately 80 percent are of genetic origin striking children with 30 percent dying before reaching the age of five. A tragedy for any parent and family.
A child afflicted with a rare disease is painful and burdened by expensive medications, check-ups, and therapies to alleviate the symptoms of their sickness.
As they are dependents, their parents and family must attend to the treatment and basic daily needs. You can imagine the difficulty and how economically catastrophic this is.
There was hope for institutionalized support with the enactment of the 2016 Rare Diseases Law or Republic Act 10747. However, stakeholders have since been lamenting on the apparent neglect because of inadequate funding.
In the recently held hybrid forum hosted by the Stratbase ADR Institute in partnership with the Philippine Society of Orphan Disorders and UHC Watch, the institute’s president, Prof. Victor Andres “Dindo” Manhit said “Due to the low prevalence and the widely dispersed distribution of patients with rare diseases, there is an evident disproportion in the availability of treatments and resources.”
He pointed out it took six years after the enactment of the Rare Disease Law for specific funding amounting to P104.9 million that it was successfully allocated in the 2022 General Appropriations Act.
“While inequality is a deeply complex issue with no clear-cut solution, good governance, political reforms, social investments, and multi-sector efforts are consistently considered essential steps to be taken,” Prof. Manhit said.
UHC Watch co-convenor and Healthcare and Education convenor of CitizenWatch Philippines Mr. Alvin Manalansan emphasized that delivery of timely, responsive and sustainable health services should be provided to all and called on government and all of society to work together so that “everyone, every Filipino has the right to health” and that “no one should be left behind.”
The Department of Health Director for Disease Prevention and Control Bureau, Dr. Razel Nikka M. Hao, shared a Five-Year Strategic Plan that outlined six main objectives:
First is to ensure that we have integrated and comprehensive care for rare disease patients.
Second is to provide access to the entitlements and benefits in the form of reimbursements and government assurances for necessary services.
Third is the creation of health promotion, public information, and education campaign materials to increase the level of knowledge of health personnel, the academe, students and the general public.
Fourth is to provide evidence for policy and program planning through Research and Development.
Fifth is to increase the availability and access of the orphan drugs and products through fiscal incentives and streamlined processes to fast-track both importation and access of these commodities.
And sixth and most important is to provide financial assistance through different mechanisms that are to be put in place.
Director for Healthcare Policy of the Pharmaceutical and Healthcare Association of the Philippines Ms. Daisy Cembrano suggested to build an agile registration process to establish a health information system national registry that will provide data on the burden of disease, and in enabling patient support programs.
She pointed out the need for faster Food and Drug administration donation processes including importation, customs, clearance among others.
Most welcome was the remarks of the Senate Committee on Finance Chair, Senator Sonny Angara, who assured continued support in the legislature and recognized that current appropriations are hardly enough and more should be allocated in the next years to provide treatment and funding research.
Durhane Wong-Rieger, Ph.D, President and CEO of the Canadian Organization for Rare Disorder, perhaps gave the most apt illustration of the tragedy of rare disease.
She said that for a child with a rare disease, discrimination strikes very early in life with access to education and adulthood difficult to find, maintain or return to work.
“Women are disproportionately discriminated and excluded from society, either with a person with rare disease or as mothers. Families with a member living with a rare disease are at greater risk of impoverishment and they have more expenses and less income,” Reiger said.
The capacity of the Philippine healthcare system to address the needs of Filipino rare disease patients will greatly depend on the accessibility of universal health care coverage.
The pandemic has exposed the disjointed state of our healthcare system that will need deep focus in investments and policy reforms from government and the participation of all health stakeholders, which is all of us.