Include rare disease in public health policy

Rare Disease Act
Senate Bill No. 2990
Under Committee Report No. 286

Sponsorship Speech
By Senator Pia S. Cayetano

04 November 2015

Mr. President, esteemed colleagues,

The right of every person to proper health care is espoused in the 1987 Constitution, which declares that: “The State shall protect and promote the right to health of the people and instill health consciousness among them.” It further mandates that: “The State shall adopt an integrated and comprehensive approach to health development, which shall endeavor to make essential goods, health and other social services available to all the people at affordable cost. There shall be priority for the needs of the underprivileged, sick, elderly, disabled, women, and children.”

Today, I rise to sponsor Senate Bill No. 2990 under Committee Report No. 286 or the Rare Disease Act. Rare disease, otherwise known as ‘orphan disorder,’ is any health condition resulting from genetic defects that rarely affect the general population. There are between 7,000 and 8,000 rare diseases that are known, and less than four hundred (400) approved treatments for them.[1] Seventy-five percent (75%) of rare diseases affect children.[2] The Philippine Registry of Rare Diseases at the Institute of Human Genetics revealed 41 diseases affecting 300 diagnosed patients in the country, while many more remain undiagnosed.

The thing about rare diseases is that they are often chronic, debilitating, and life-threatening. The quality of life of patients is compromised by loss of autonomy. Parents and their families experience a high level of pain and suffering, and the cost of treatment is often beyond the means of most families.[3]

Rare disease is particularly close to my heart. I lost my son at nine months to complications from a rare congenital disease called trisomy 13. Trisomy 13 is just one of the many rare diseases.

In the Senate here today are some brave persons living with rare diseases. Princess Paula Javate is nine years old and living with Moebius syndrome, a rare neurological disorder that leaves patients unable to move certain muscles in their faces. Although there is no definitive treatment for Moebius Syndrome, physical, occupational, and speech therapy may be beneficial for patients.

Eighteen-year-old Roseann Ibay has Osteogenesis Imperfecta, or brittle bone disease, in which bones break easily. Management of this disease may include rehab medicine, orthopedic therapy, dental care, and hearing loss treatment.

Joaidda Ziv Morejon is seventeen years old. She has Blue Rubber Bleb Nevus Syndrome, which is a rare disorder frequently associated with serious and potentially fatal bleeding and anemia. Treatment may include blood transfusions, and sirolimus for severe bleeding.

Twenty-six-year-old Michael Capoquian also joins us today. He is living with MPS Type II Hunter Syndrome, which symptoms may include enlarged liver and spleen, stiff joints, cardiac disease, and prolonged neurological involvement leading to developmental delays and regression. To treat this rare disease, patients may need to undergo bone marrow transplant or enzyme replacement therapy.[4]

And we have Juan K. Magdaraog, who is thirty-eight years old and living with Pompe disease, which is a disorder that disables the heart and skeletal muscles. Pompe disease eventually renders a person weak, needing the help of people and equipment to go on with their daily activities. For Juan, this disease started when he was just ten years old, when he started having trouble getting up from the floor and climbing stairs. Many did not expect him to live past the age of 30. But he is now 38 years old, and in a wheelchair and reliant on a breathing machine. He finished school, obtained a degree in Industrial Design, and is now gainfully employed.

Sadly, Mr. President, many others who are afflicted with rare disease do not have ready access to the treatment and support they need. These diseases are medically and economically catastrophic – medically, because they progress to multiple disability or multiple organ failure which, without help, lead to early death; and economically, because the cost of care is too high and can push a patient and his family to poverty.[5]

Under the Sustainable Development Goals or SDGs of the United Nations General Assembly, one of our global goals is to “ensure healthy lives and promote well-being for all at all ages.” As we currently stand, the Philippines has yet to include the rare disease sector in mainstream public health programs, despite studies suggesting that 11.2% of child mortality in the Philippines are caused by birth defects and congenital anomalies attributed to rare diseases.[6]

Mr. President, it is time we include the Rare Disease Sector in our public health policy because health is a human right, and universal health care means that nobody is left behind. We need a policy that will address the plight of persons affected by rare disease and their families. The proposed bill which was ably crafted and prepared by Dra. Carmencita Padilla, now chancellor of UP Manila, will establish a Health Delivery System that is comprehensive, permanent, and sustainable for the benefit of the Rare Disease Sector. It also seeks the inclusion of rare disease treatment in the health benefit package of PhilHealth, and the inclusion of medical assistance to persons with rare diseases as provided by Republic Act No. 10351 or the Sin Tax Law. Finally, it lays down regulatory and fiscal incentives to support research and development, import, and manufacture of orphan drugs and products.

Mr. President, on a policy level, this is a health bill which aims to reduce child mortality and reduce poverty. However, for the Filipinos who are directly affected by this condition, this measure will relieve some of the economic burden of managing rare diseases. More importantly, they can hope for better quality of life for the ones they love. With that, I earnestly seek the swift passage of this bill. Thank you.

END NOTES:

[1] Padilla, C. “Orientation on Rare Disorders and the Proposed Rare Disease Act of the Philippines” as presented in the Senate Committee Hearing on 26 August 2014.
[2] Ibid.
[3] Ibid.
[4] Rare disease information care of Philippine Society for Orphan Disorders, Inc.
[5] Padilla, C. “Orientation on Rare Disorders and the Proposed Rare Disease Act of the Philippines” as presented in the Senate Committee Hearing on 26 August 2014.
[6] Child mortality in 2010. DOH website as cited in Padilla, C. “Orientation on Rare Disorders and the Proposed Rare Disease Act of the Philippines” as presented in the Senate Committee Hearing on 26 August 2014.

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