Proposed measure to address rare diseases of Filipino children pushed

MANILA  — A young lawmaker has filed a bill establishing a comprehensive and sustainable health system for rare diseases, which affect 75 percent of Filipino children.

Las Pinas Rep. Mark A. Villar said House Bill 4781 establishes a system that will help patients and their families cope with the financial and non-financial challenges of living and dealing with rare diseases.

“The creation of a comprehensive and sustainable health system for rare diseases will provide better access to adequate medical care, health information, and health care products needed to treat their condition,” Villar said.

The bill identified some of the rare diseases as Gaucher Disease, Maple Syrup Urine Disease, Pompe Disease, Galactosemia, Phenylketonuria, Methymalonic Acidemia, Urea Cycle Defects, Hurler Syndrome, Hunter Syndrome and Prader-Willi Syndrome.

Citing a report of the Philippine Society for Orphan Disorders’ (PSOD), Villar said there are 6,000 to 8,000 rare diseases in the country affecting 75 percent of children population in the country. He said 30 percent of rare disease patients die before they reach five years old.

The bill defines rare disease as any health condition resulting from genetic defects that rarely affect the general population, which are often chronic, progressive, degenerative, and life-threatening.

“Rare diseases are disabling, with the patient’s quality of life often compromised by the lack or loss of autonomy, coupled with high level of pain and suffering of the patient and family,” Villar explained.

Under the bill, individuals with rare disease shall be included among those with disabilities and must enjoy the rights under the Magna Carta for Disabled Persons as mandated in Republic Act 9442.

All healthcare practitioners and health institutions shall be required to submit status report to the Rare Disease Registry of the National Institutes of Health (NIH) diagnosed cases of rare diseases, subject to the guidelines provided by the NIH to protect the privacy of patients afflicted with rare diseases.

As the lead agency, the DOH shall establish the Technical Working group for the Rare Diseases, coordinate with the NIH for technical assistance, coordinate with all related government and non-government agencies, such as the Food and Drug Administration (FDA), Department of Social Welfare and Development (DSWD) and Department of Labor and Employment (DOLE).

The DOH shall also designate referral centers in strategic location in the country for the timely and sustainable medical management of persons afflicted with rare disorders, and organize a pool of medical specialists who will be responsible in the management of persons with rare disorders and their families.

For the sustainable compliance to the medical management of the rare diseases, the DOH shall ensure the system will facilitate qualified afflicted person to benefit from its intended services, with its cost of treatment as part of Philippine Health Insurance Corporations’ Catastrophic Illness Resource Fund, and its cost of care be charged to the Sin Taxes collection.

Donations intended for researches on rare disease, maintenance of the Rare Disease Registry, for purchase of orphan drugs or products for use solely by patients with rare diseases, as certified by the FDA and NIH shall be exempted from payments of all tariff, duties and taxes.

The DOH and the NIH, together with health professional societies and academic health institutions, shall conduct continuing education, information and training programs for health personnel on the identification and referral of afflicted persons for medical management.

The measure mandates the DOLE to ensure that abled persons with rare disease be given opportunity for work and employment to become productive members of the society.

Likewise, the DSWD shall provide assistance to the afflicted person, ensuring their social welfare and benefits as mandated in the Magna Carta for Disabled Persons.

The DOH shall convene the Rare Diseases Technical Working Group (RDTWG) which will designate diseases that are considered rare disease, designate orphan drugs and products, and formulate policies that will regulate the approval and certification of orphan drugs and products.