A fair share for patients living with rare diseases

The Stratbase ADR Institute, in partnership with UHC (Universal Health Care) Watch and the Philippine Society of Orphan Disorders (PSOD), held a forum last week titled “Sustaining Gains and Balancing Priorities – Implementation of Rare Disease Act for 2023.” This was the third discussion, since 2022, organized by the same groups. Previous forums provided venues for different stakeholders to tackle the milestones and as a continuing support for the implementation of the Rare Disease Law of 2016 (Republic Act No. 10747).

This year, the discussions were held in line with the recent celebration of Rare Disease Week, marked every 4th week of February, and the 7th anniversary of the signing of the Act. We also needed to tackle a much more pressing concern involving the funding allocation for 2023, which was significantly reduced to only P28.089 million from an initial funding of P104.9 million in 2022. This drastic reduction seriously compromises how the existing programs for rare disease patients will be sustained.

Prof. Victor Andres “Dindo” Manhit, President of the Stratbase ADR Institute, in his welcome address said it is vital to provide and expand our healthcare system with adequate resources to constantly respond to the needs of the public. We must all work together to make universal healthcare a reality for everyone and make it so that “no one will be left behind.”

Dr. Carmencita D. Padilla, current Chancellor of University of the Philippines Manila and founding Chairman of PSOD, said in her opening that “we’ve been able to get some funding but it’s not really enough. We really need long term funding because of the patients who need them.”

The Department of Health’s (DoH) representative, Dr. Manuel V. Vallesteros, the Division Chief of Disease Prevention and Control Bureau’s Child, Adolescent and Maternal Health, where the program for rare diseases is currently included, said that “there’s still a lot of work that must be done. Our list of diseases has become longer because it will include rare diseases other than those from inborn errors of metabolism. The DoH continuously encourages our medical societies to help us establish referral pathways for these other rare diseases.”

Dr. Celeste Mae Campomanes of the Philippine College of Chest Physicians and, at the same time, chair for the Council on Occupational and Interstitial Lung Diseases (ILD) said that their group looks forward to collaborating with the DoH. She has been involved in the management of patients suspected with ILD, a type of rare disease that is mostly detected in adult patients.

Lourdes Desiree Cembrano, the Director for Healthcare Policy at the Pharmaceutical and Healthcare Association of the Philippines (PHAP) mentioned that “the establishment of the private sector advisory council with health as a major component is a tangible cross-channel [for us] to put together [our] shared goals and aspirations to provide an environment that facilitates health for our fellow Filipinos, especially children with rare diseases.” She also said that “this administration recognizes the important role of the private sector, and the health representative on that private sector Advisory Council is a member company of PHAP.”

On the other hand, Dr. Marita Tolentino-Reyes, Chairperson of the Health Technology Assessment (HTA) Council, an independent advisory body created under Republic Act 11223 or the Universal Health Care Act, with the overall role of providing guidance on the coverage of health interventions and technologies to be funded by the government, identified challenges to the prioritization of rare diseases — lack of local data on burden of illness, healthcare costs, unharmonized list of international and local rare diseases, lack of local studies on clinical efficacy, effectiveness and safety, high costs of innovative drugs, and the lack of data on prevalence and access to care in subpopulations.

The overwhelming consensus in the forum is the need to build a “rare disease coalition” that will ensure the progressive implementation of the Rare Disease Law, and that includes the provision of appropriate and sustainable funding.

Funding should be based on the plans, activities, and programs indicated on a national action plan, which in this case, is the recently released Integrated Rare Disease Management Program Plan 2022-2026. It is essential that this strategic plan consider the wide spectrum of rare disease types, and the roles and responsibilities of different sectors in support of the implementation and achievement of the objectives stipulated in the Rare Diseases Act and its Implementing Rules and Regulations vis-a-vis the UHC Act.

As advocates of universal healthcare, we want everyone, most of all patients living with rare diseases, to receive a fair share of the pie — the support and attention being given by the government and all stakeholders. As we continue this advocacy, we will constantly be on the watch so that more patients will have access to needed health services.

Indeed, the journey has been long and we have much to do, but we have been progressing and gradually realizing our goals. Working together, we will see the objectives of the Rare Disease Law being realized — hopefully soon.

Alvin Manalansan is the health and nutrition fellow of Stratbase ADR Institute and co-convenor of the UHC Watch.