Rare diseases patients should not be left behind
February 2, 2022
By Alvin Manalansan, originally published in BusinessWorld
The continuing global pandemic posed several challenges and showed the gaps all throughout the health ecosystem. Undeniably, addressing the country’s COVID-19 situation has been the priority and concern of the government for two years now, and still counting. Patients who have other health conditions, such as those who were diagnosed with rare diseases, are sadly kept on the sideline.
According to the Department of Health, as of Dec. 31, 2021, there are 887 cases registered with rare diseases. For sure, there are more undetected and most of them likely belong in the lower socio-economic group who really require external support either from the government or other stakeholders. Adhering to the principle of universal healthcare, every Filipino should have equal access to health services and “no one should be left behind.” Access to quality, safe, and effective healthcare and treatment should be for all, regardless of age, sex, status, and/or condition.
The Rare Disease Act of the Philippines or Republic Act No. 10747, signed into law on March 2016, is a comprehensive policy that addresses the needs of persons with rare diseases. The law primarily envisioned that those patients afflicted with the diseases are diagnosed early and have access to available interventions so that their rate of survival is increased, and they potentially can live a normal way of life.
Like all law-mandated programs, adequate funding is crucial for implementation. For the first time, stakeholders have successfully advocated for its long-delayed funding in the 2022 General Appropriations Act (GAA). The allocation, worth P104.9 million, was lodged in the University of the Philippines-National Institutes of Health (UP-NIH) which is specifically intended for the implementation of the Rare Disease Act.
On Jan. 27, the Stratbase ADR Institute, in partnership with UHCWatch and the Philippine Society for Orphan Disorders, held a virtual roundtable discussion entitled “No One Should be Left Behind Amid the Continuing Pandemic — Facilitating the Implementation of the Rare Disease Law for 2022.” The roundtable gathered relevant stakeholders from the academe, government agencies, civil society/patient organizations, and private sector to discuss ongoing programs and needed support for the effective utilization of the allocated budget. Avenues of collaboration on how different stakeholders can collaborate were tackled in line with the implementation of the Rare Diseases Act.
Prof. Dindo Manhit, President of Stratbase ADR Institute, said in his welcome remarks that 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. Various stakeholders can proactively collaborate in the decision-making process so that through this whole-of-society approach the country’s health systems could achieve better health outcomes, he added.
According to Dr. Carmencita Padilla, Chancellor of the University of the Philippines-Manila, “we [Philippines] are very fortunate because we are the model for the rest of developing world, wherein we were able to take care of patients with rare metabolic disorders. However, no single agency can make this happen.”
Dr. Eva Maria C. Cutiongco-dela Paz, Executive Director of the UP-NIH, mentioned that there was an on-going project for the development of the Integrated Rare Disease Management Plan (IRDMP) for the period of 2022-2026. Part of this five-year plan is the development of the guidelines for medical societies in preparation of the rare diseases list.
Daisy Cembrano, Director for Healthcare Policy of the Pharmaceutical and Healthcare Association of the Philippines, emphasized that the importance of ensuring sustainable patient access to diagnosis, treatment, and care is foremost. She explained that over the past two years, most resources were reallocated to the pandemic response and it is worth looking at some innovative financing models implemented by other countries for the treatment of rare diseases.
Dr. Lizette Kristine Lopez, Chair of the Subcommittee on Drugs of the Health Technology Assessment Council, in her reaction to the presenters mentioned the importance of inclusiveness and her preferential regard for the underserved and unserved. According to her, “rare disease patients need to be included because despite the rarity of their conditions, they are definitely underserved and some of them are probably unserved considering the financial impact and also the lack of specialists.”
The start of the year provided a brighter tomorrow for patients with rare diseases. The current challenge is how to facilitate the development of the IRDMP for the optimal utilization of the allocated funding for this year. This plan will also serve as the basis for funding the succeeding years and will determine the additional types of rare diseases to be covered.
To be true to the mandate of the Universal Health Care Act and the Rare Disease Act, all rare disease patients must receive the assistance and treatments they need to enable them to be productive citizens of society.