On ALS and rare diseases

On ‘ALS Ice Bucket Challenge’ and Rare Diseases
Privilege Speech
By Senator Pia S. Cayetano
03 September 2014

Mr. President, esteemed colleagues,

Social media has become a remarkable tool for spreading information and raising awareness. In the past few weeks, the ALS Ice Bucket Challenge has gone viral over social media to promote awareness and encourage donations to research on ALS, or amyotrophic lateral sclerosis. Many prominent personalities have participated in the challenge, including Senate President Drilon, Senator Bongbong Marcos, Senator Bam Aquino, and Senator Sonny Angara, who nominated me last week. I would like to congratulate those who participated in the campaign by taking the challenge, and I respond to Senator Sonny’s nomination through this speech.

Why? Why am I responding on the Senate floor? Allow me to take you back 10 years ago, even before I became a Senator.

In 2004, at the end of the 12th Congress, Congress passed the Newborn Screening Act. This law is able to identify and reverse the complications of a number of these rare diseases. And because of this law, Filipinos who would otherwise be disabled for life or die an early death will now be able to live a normal lives.

Fast forward to today….

Mr. President, prior to the Ice Bucket Challenge, few people knew of ALS and its devastating effects. I take this opportunity to point out that ALS forms part of the 48 rare diseases affecting patients in the Philippines, of which we know very little about. Many more remain undiagnosed.

ALS is a motor neuron disease that affects the nerve cells in the brain and the spinal cord. Motor neurons send signals from the brain to the different muscles of the body [1], in order to control voluntary muscle activity, such as speaking, walking, or swallowing. [2] ALS causes the progressive degeneration of motor neurons, which means that the brain loses its ability to control muscle movement. When the muscles stop receiving signals from the brain that they need in order to function, the muscles begin to atrophy, or become smaller.

During the early stages of ALS, symptoms include increasing muscle weakness, especially affecting the arms and legs, speech, and swallowing or breathing. In later stages, patients with ALS may become completely paralyzed. Although 10% of cases are linked to genetics, there is no known cause for ALS. Further, despite the development of Riluzole, a drug that slows the progress of ALS, there is still no cure or treatment that halts or reverses the disease. [3]

Early this year, I filed Senate Bill No. 2098 or the Rare Disease Bill, which seeks to provide access to medical treatment and drugs for persons with rare diseases. Rare diseases do not discriminate who they affect.

In order to adequately address a health issue, research and data gathering are very important. To this end, SB No. 2098 mandates the following:

Identification of Persons with Rare Disease and Creation of a Rare Disease Registry
Section 5 of B No. 2098 provides for the identification of persons with rare disease and the creation of a Rare Disease Registry. Healthcare practitioners and health institutions shall be required to report diagnosed cases of rare diseases to the Rare Disease Registry of the Department of Health, and provide reports on the status of patients.

Referral of Patients with Rare Disease
Section 6 further provides for the referral of patients suspected or diagnosed with rare disease to the Regional Newborn Screening Centers to ensure that the afflicted person receives adequate medical care for his or her condition, and is referred to a healthcare practitioner who specializes on rare diseases.

Benefits of Persons with Rare Disease
Under Sections 8 and 9 of this bill, persons with rare disease shall enjoy the same benefits as persons with disabilities, as mandated by Republic Act No. 7277, or the Magna Carta for Disabled Persons.

Resource Generation
Under Section 19, the DOH shall ensure that a system is in place to facilitate the qualification of an afflicted person as a beneficiary of medical management services of a rare disease. Thus, treatment of rare disease shall be included in the benefit package, to be provided in the guidelines set by the Philippine Health Insurance Corporation. Further, revenues generated by RA 10351 or the Sin Tax Reform Act shall include medical assistance to patients with rare disease.

Mr. President, heightened awareness about rare diseases, as well as diagnosis and accessible treatment are crucial if we are serious about helping persons with rare diseases. Poor families caring for a family member with a rare disease have no fighting chance if they do not have access to quality and affordable medical care. It is my hope that the attention given to ALS can somehow carry over to other rare ailments, and that more work and research may be undertaken to diagnose and manage these rare diseases.

Mr. President, my child had trisomy 13, a condition that can be likened to a rare disease. I know treatment for such conditions is expensive. I know sometimes there is no cure. But like any mother, like any parent who has gone through this experience, we live by hope: everyday, the hope for a cure, the hope for a breakthrough, even the hope for temporary relief – let us be part of that hope.

And this is my response to the ice bucket challenge, not just to broaden awareness for ALS, and the other rare diseases that affect the Filipinos and people all over the world. But for the allocation of funding for health care. We passed the sin tax law that provides for budget for universal health care, attainment of the millennium development goals and health awareness programs as well as for medical assistance and health enhancement facilities programs. But, until today, I am still awaiting the response from the Department of Health as to how these funds have been allocated, which I believe should include funds to deal with these rare diseases. Thus, as Vice-Chair of the Committee on Health and with the permission of our chair, I plan to hear Senate Bill No. 2098 in the hopes of truly improving the quality of life of patients with rare diseases and their families, and help alleviate the burden of finding appropriate medical care to manage the illness.

To Senator Angara who nominated me, if we go to Geneva together for the Inter-Parliamentary Union, I will jump in Lake Geneva for this cause …if you will join me. That is my counter-challenge.

Thank you.

Sources:
[1] http://www.alsa.org/about-als/what-is-als.html
[2] http://en.wikipedia.org/wiki/Motor_neuron_disease
[3] http://www.alsa.org/about-als/what-is-als.html

Notes:
For more information on rare diseases in the Philippines, visit the Philippine Society of Orphan Disorders website: www.psod.org.ph and www.fightpompe.com

READ/DOWNLOAD: PDF copy of Sen. Pia S. Cayetano’s SBN 2098, the proposed Rare Disease Act of 2014

Photo: Senator Pia S. Cayetano’s guests at the Senate gallery on Wednesday (Sept.3) when she delivered her privilege speech on ALS and rare diseases included members of the Philippine Society of Orphan Disorders.

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