• 
SIXTEENTH CONGRESS OF THE 
REPUBLIC OF THEPHILIPPINES 
First Regular Session 
SENATE 
S.B. NO -IllS a 
INTRODUCED BY SENATOR PIA S. CAYETANO 
S{'I111h' 
,.tt.;. ,,! I~. ~HT,br~ 
'\4 FEB -3 P2 :55 
AN ACT PROMULGATING A COMPREHENSIVE POLICY IN ADDRESSING THE NEEDS OF 
PERSONS WITH RARE DISEASE 
EXPLANATORY NOTE 
A "rare diseas.e". otherwise called an "orphan disorder", is any health condition 
resulting from genetic defects that rarely affect the general population, There are 6,000 to 
8,000 rare diseases, majority are genetic in origin and manifest at birth or early in childhood, 
Rare diseases are often chronic, progressive, degenerative, and life-threatening, The 
quality of life of patients is often compromised by the lack or loss of autonomy, high level of 
pain and suffering for the patient and their family. Although these diseases inflict a small 
number of individuals, treatment is usually life term and costly, making it beyond the reach 
of most Filipino patients. 
The right of the person to be provided proper health care finds anchor in the 1987 
Constitution. In particular, Section 15 of Article 2, states that "The State shall protect and 
promote the right to health of the people and instill health consciousness among them". 
Furthermore, the United Nations Convention on the Rights of the Child, which the 
Philippines ratified on July 26, 1990, requires State Parties to "recognize the right of the 
child to the enjoyment of the highest attainable standard of health" (Art. 24[1]) and to 
"ensure the provision of necessary medical assistance and health care to all children" (Art. 
24[2b]). 
This bill provides for the creation of a comprehl?nsive and sustainable health system 
for rare diseases integrated into existing public health care system. This will ensure the 
provision of early and sustainable care for patients suffering from rare disease, relevant 
researches on rare diseases, and integration of the health care activities for informational 
program on rare diseases for the general public and health care practitioners. This bill will 
help provide patients with rare diseases and their families better access to adequate 
medical care, health information, and healthc'lfe products needed to treat their condition. It 
establishes a system to coordinate sustainable research & development initiatives and 
resource generation efforts among relevant agencies of government and the private sector 
toward improving the quality of life of patients with rare diseases and their families. 
The r~tionale for establishing a national health care system for rare disorders as part 
• of the country's healthcare delivery system finds further justification and expression in 
Section 1'1 'of Article 13 of the Constitution: 
"The State shall adopt an integrated and 
comprehensive approach to health development which shall endeavor to make essential 
goods, health and other services available to an people at affordable cost." 
In recognition of our constitutional and international obligations to improve the health 
of the people, the immediate enactment of this bill is therefore requested. 
P~.!AY~L/ 
senat~~TONO 10 


SIXTEENTH CONGRESS OF THE 
REPUBLIC OF THE PHILIPPINES 
First Regular Session 
) 
) 
) 
SENATE 
s.B.No 2098 
INTRODUCED BY SENATOR PIA S. CAYETANO 
'14 
FEB -3 P2:55 
Rf;C'EIVED BY;--f-
AN ACT PROMULGATING A COMPREHENSIVE POLICY IN ADDRESSING THE 
NEEDS OF PERSONS WITH RARE DISEASE 
1 
ARTICLE 1 
2 
GENERAL PROVISIONS 
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SECTION 1. Short Title.- This Act shall be known as the "Rare Disease Act of 
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2014." 
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SEC. 2. Declaration of Policy. - It is the policy of the State to protect and 
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promote the right to health of the people, including the right of persons suffering from 
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rare disease, to survival and full and healthy development as individuals through access 
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to timely health information and adequate medical care. In pursuit of such policy, the 
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State shall institutionalize a system that is comprehensive, integrative and sustainable 
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and will facilitate collaboration among government and non-government agencies and 
'11 • organizations at the national and local levels, private sector, professional health 
) 2 
organizations, academic institutions, communities, and families towards the provision of 
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early and sustainable care of every person afflicted with rare disease. The State 
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recognizes the crucial role of research in defining health programs and activities 
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addressing the needs of patients with rare disease_ The State also recognizes that an 
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effective public education program is vital in helping ensure the early diagnosis and 
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treatment of rare diseases and in preventing those afflicted with them from being the 
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subject of ridicule and stigmatization. The State further recognizes the leading role of 
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the Department of Health (DOH) in implementing the Rare Disease Program, 
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overseeing the provision of care, and working with the other government agencies, the 
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private sector and non-governmental organizations, in designing and implementing 
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programs, including research & development activities on rare diseases for the benefit 
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of those afflicted with them. 
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SEC. 3. Objectives. - The objectives bf this Act are as follows: 
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1. Endeavor that every patient diagnosed to have a rare disease has access to 
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timely health information and, adequate medical care, including drugs and 
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other healthcare products to treat or otherwise help them cope with their 
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condition: 
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i) 
ii) 
Establish a comprehensive and sustainable health care system 
integrated within the public health care delivery system that will 
endeavor to provide early and sustainable care for patients 
suffering from rare disease; 
Design and maintain the Rare Disease Registry which shall include 
data on rare disease in the Philippines, patients afflicted with rare 
'disease, and orphan drugs and'products_ This data shall be utilized 
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in formulating policies, identifying program interventions, and 
'designing researches that will' eventually address the needs of 
patients with rare disease; 
iii) 
Integrate public educational and informational campaigns in the 
current programs of the DOH to identify persons afflicted with rare 
disease and help the Pllblic understand the special needs of such 
persons; and 
iv) 
Facilitate the regular collaborative activities among stakeholders 
regarding the realization of the objectives of this Act 
2. Provide regulatory and fiscal incentives to support research and development 
activities on rare disease and the import or manufacture of affordable orphan 
drugs or orphan products; and 
3. Institutionalize a financial incentive system for agencies involved in clinical 
researches, patient care, medical information management, and other similar 
activities for the benefit of persons afflicted with rare disease. 
ARTICLE 2 
DEFINITION OF TERMS 
SEC. 4. Definitions. - For the purpose of this Act, the following terms shall be 
defined as follows: 
1) Commercial use means the selling of orphan drugs, imported from sources 
abroad, at a profit; 
2) Healthcare practitioner means any doctor of medicine, dentist, nurse, midwife, 
allied health professionals or other health care professionals duly licensed by 
the Professional Regulatory Commission; 
3) Healthcare institution means hospitals, health infirmaries, health centers, 
lying-in centers or puericulture centers, whether public or private; 
4) Medical care means any method used by a health care practitioner to inform, 
brief, prevent, diagnose, manage, or remove the symptoms and cause of a 
disease; 
5) National Comprehensive Newborn'Screening System, as established by R.A. 
9288, is the existing network of medical specialists, nurses, laboratories and 
hospitals screening and treating t~ese genetic diseases, many of which are 
also rare disease; 
6) Newborn Screening Center (NSC) means a facility equipped with a newbom 
screening laboratory that complies with the standards established by the 
National Institutes of Health (NIH) and provides all required laboratory tests 
and recall/follow-up programs for newboms with metabolic, genetic, or rare 
disease; 
7) Orphan Drug means any drug or medicine used to treat or alleviate the 
symptoms of persons afflicted with a rare disease and designated as such by 
the Food and Drug Administration (FDA) upon recommendation of the 
National Institutes of Health (NIH); 
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8) Orphan Product means any healthcare' or nutritional product, other than a 
drug or medicine, including but not limited to diagnostic kits, medical devices 
and biological products, used to prevent, diagnose, or treat rare diseases and 
designated as such by the FDA upon recommendation of the NIH; 
. 9) Rare Disease means disorders such as Gaucher Disease, Maple Syrup Urine 
Disease, Pompe Disease, Galactosemia, Phenylketonuria, Methylmalonic 
Acide(T1ia, Urea Cycle Defects, Hurler Syndrome, Hunter Syndrome, Prader-
Willi Syndrome, and other diseases with similar rare occurrence as 
recognized by the DOH upon recommendation of the NIH but excluding 
catastrophic (Le. life threatening, seriously debilitating, or serious and chronic) 
forms of more frequently occurring diseases; 
10) Rare Disease Registry means the health information system, including the 
electronic database system, relating to data on rare diseases, persons 
afflicted with rare diseases, and orphan drugs and orphan products; 
11) Rare Disease Technical Working Group (RDTWG) means a DOH-designated 
pool of experts on rare disease tasked with identifying rare disease, orphan 
drugs, and orphan products; 
12) Telegenetics Referral System is an established system utilizing electronic 
communications, including but not limited to video conferencing and emails, 
which aims to make genetics services accessible to all patients with genetic 
conditions; 
ARTICLE 3 
IDENTIFICATION, REFERRAL, AND MANAGEMENT OF PERSONS WITH RARE 
DISEASE 
SEC. 5. Identification of Persons with Rare Disease. -
The DOH, in 
coordination with the NIH, shall create a Rare Disease Registry. It shall endeavor to 
comply with set global standards, if applicable. All patients diagnosed with rare disease 
shall be induded in this registry. 
All healthcare practitioners and health institutions shall be required to report to 
the Rare Disease Registry of the DOH and NIH diagnosed cases of rare disease and 
provide reports on the status of patients; Provided, that such reports shall be subject to 
guidelines issued by the NIH to protect the privacY,of patients afflicted with rare disease. 
SEC. 6. Referral of Patients with 'Rare Disease. -
Patients suspected or 
diagnosed with rare disease shall be referred to Regional Newborn Screening Centers 
(NSC) identified by the DOH as referral centers for medical care of rare disease. This 
ensures that the afflicted person receives the adequate care for his/her condition and is 
referred to a healthcare practitioner specializing on rare disease. In the absence of a 
specialist in the area, the referral center must coordinate with NIH through the DOH 
Centers for Health Development (CHD) for co-management of the patient with a 
specialist. 
There shall also be a system established in collaboration with local units and 
agencies that will endeavor to provide the afflicted person with sustainable medical care 
of the disease and have his/her case reported to NIH for its inclusion to the Rare 
Disease Registry. 
SEC. ,7. Management of Persons with Rare Disease. -The DOH, with the 
assistance of NIH, should ensure the proper management of persons with rare disease 
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through the creation of a Rare Disease Management Program under the National 
Center for Disease Prevention and Control of the DOH. 
ARTICLE 4 
PERSONS WITH RARE DISEASE AS PERSONS WITH DISABILITIES (PWDs) 
SEC. 8.·· Defiignation of Persons with Rare Disease as Persons with 
Disabilities (PWDs). - Individuals with rare disease shall be considered as persons 
with disabilities (PWDs), in accordance with Republic Act No. 7277, as amended, or the 
Magna Carta for Disabled Persons. 
SEC. 9. Rights and Privileges of Persons with Rare Disease. -
The 
appropriate national government agency shall ensure that they accorded the same 
rights and privileges as PWDs, to wit: 
1) The Department of Social and Welfare Development (DSWD) shall provide 
assistance to persons with rare disease to ensure that their social welfare and 
benefits as mandated under Republic Act No. 7277, as amended, or the 
Magna Carta for Disabled Persons, are granted. 
2) The Department of Labor and Employment (DOLE) shall ensure that able-
persons with rare disease are given the opportunity for work and employment 
to become productive members of the society. 
ARTICLE 5 
DESIGNATION OF RARE DISEASE, ORPHAN DRUG, AND ORPHAN PRODUCT 
STATUS 
SEC. 10. The Rare Disease Technical Working Group (RDTWG) . • The DOH 
shall convene the RDTWG which shall have the following roles and responsibilities: 
1) Designate rare disease and update the list periodically; 
2) Designate and periodically update orphan drugs and products corresponding 
to the rare disease; and 
3) Formulate policies that shall regulate the approval and certification of orphan 
drugs and products. 
SEC. 11. Designation of Rare Disease. - The DOH, upon recommendation of 
the NIH and RDTWG, shall have the authority to deSignate any disease that is 
recognized to rarely afflict the population of the country. Gaucher Disease, Maple Syrup 
Urine Disease, Pompe Disease, Galactosemia, Phenylketonuria, Methylmalonic 
Acidemia, Urea Cycle Defects, Hurler Syndrome, Hunter Syndrome and Prader-Willi 
Syndrome are hereby designated as rare disease. Additional diseases should be 
approved by the DOH, as recommended by the NIH'and RDTWG. 
SEC. 12. Designation of Orphan Drug. - The FDA, motu proprio or upon 
application by any interested person, may designate any drug or medicine indicated for 
use by patients afflicted with a rare disease as an orphan drug; Provided, that no 
existing drug or medicine in the Philippines that can yield the same or superior results 
shall be designated as an orphan drug. Within one hundred twenty (120) days from the 
effectivity cif this Act, the FDA shall publish a list of orphan drugs for rare disease and 
shall periodically update the said list. 
SEC. 13. Designation of Orphan Product . • The FDA, motu proprio or upon 
application by any interested person, may designate any healthcare or nutritional 
4 


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product, other'than a drug or medicine, including but not limited to diagnostic kits, 
2 • medical devices and biological products, used primarily to prevent, diagnose, or 
3 
alleviate the symptoms of rare disease as an orphan product; Provided, that no existing 
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product in the Philippines that can yield the same or superior results shall be designated 
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as an orphan product. Within one hundred twenty (120) days from the effectivity of this 
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Act, the FDA shall publish a list of orphan products for rare disease and shall 
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periodically update the said list. 
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SEC. 14. Permit for Restricted Use of an Orphan Drug/Orphan Product. -
Any person may import any orphan drug or orphan product without need of obtaining a 
Certificate of Product Registration; Provided, that the said importation shall not be for 
commercial use; Provided, farther, that a Permit for Use of an Orphan Drug/Orphan 
Product shall be secured from the FDA. The Permit for Restricted Use of an Orphan 
Drug/Orphan Product shall be issued by the, FDA if the applicant meets the following 
requirements: 
1) A sworn application for the issuance of a Permit for Restricted Use of an 
Orphan Drug/Orphan Product, containing the name and address of the 
applicant and the estimated 'annual volume requirement of the drug or 
product; 
2) Certification from the FDA that the drug or product qualifies as an orphan 
drug or orphan product; 
3) In the case of a drug or medicine, medical device, or diagnostic kit: (i) the 
names and addresses of medical specialists qualified and authorized to use 
them; (ii) a written commitment on the part of all the authorized specialists to 
submit to the FDA with copies to the DOH no later than January 15 of each 
year, a Clinical Study Report for every patient administered with the drug or 
product, describing the quantity administered or used, the therapeutic or 
desired effect, and adverse reactions, if any; 
4) Certification that the drug or product is registered in the country of origin; and 
5) An affidavit stating that the applicant shall hold the FDA and its officials and 
employees free and harmless from any death, injury, or damage arising from 
the use of the orphan drug or orphan product. 
Within thirty (30) days from receipt of the following requirements, the FDA shall 
issue a Permit for Restricted Use of an Orphan Drug/Orphan Product which shall be 
effective for a period of three (3) years, renewable for periods of three (3) years 
thereafter. 
ARTICLE 6 
IMPLEMENTATION 
SEC. ,15. Lead Agency. - The DOH shall be the lead agency in the 
implementatioh of this Act, For the purposes of achieving the objectives of this act, the 
DOH shall:," 
1) 
2) 
3) 
Establish the RDTWG; 
Develop the implementing rules and regulation for the implementation of this 
Act within one hundred twenty (120) days from the enactment of the Law; 
Coordinate with the NIH for the technical assistance in the implementation of 
this Act; 
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organizations involved in the implementation of this Act; 
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Designate referral centers in strategic locations in the country for the timely 
and sustainable medical management of persons afflicted with rare disease; 
and 
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Organize a pool of medical specialists who will be responsible In the 
management of persons afflicted with rare disease and their families. 
SEC. 16. Other Implementing Agencies. - The FDA, NIH, DSWD, DOLE, and 
other relevant government agencies shall perform the following tasks: 
1) The FDA shall ensure that orphan drugs and products are permitted in the 
country for the purpose of treating rare disease; 
2) The NIH shall serve provide the technical assistance to the DOH in implementing 
this Act; 
3) The DSWD and DOLE shall ensure that persons with rare disease are given the 
opportunity to be productive members of the society and that they are given the 
same rights and benefits as PWDs; and 
4) All other relevant government agencies shall assist in the full implementation of 
this Act. 
SEC. 17. Obligation of Healthcare Practitioners. - Any health care practitioner 
who attends to a person with rare disease is obligated to the following: 
1) To give the patient and his family SUbstantial information about the 
significance of diagnosis and management or refer them to a healthcare 
practitioner specializing on rare disease; 
2) To ensure -that the afflicted person is referred to a Regional NSC identified by 
the DOH as referral centers for treating rare disease; 
3) . To report the case for entry into the Rare Disease Registry; and 
4) To inform the patient afflicted with rare disease of relevant orphan drugs and 
orphan products. 
SEC. 18. Continuing Education and Training of Health Personnel. -The DOH 
and the NIH, together with health professional societies, and academic health 
institutions, shall: 
1) Conduct continuing education, information, and training programs for health 
personnel on the identification and referral of persons with rare disease for 
medical management; and 
2) Educate health personnel on the importance of reporting cases for the Rare 
Disease Registry. 
ARTICLE 7 
RESOURCE GENERATON AND FISCAL INCENTIVES 
SEC. 19. Source of Funds for Maintaining Medical Management of Persons 
with Rare Disease. - The DOH shall ensure the establishment of a system that will 
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facilitate the qualification of an afflicted person as one of the beneficiaries of the 
services for sustainable compliance to the medical management of the rare disease: 
1, To the extent actuarially possible, the .treatment of rare disease shall be 
included in the benefit package, to be provided in the guidelines set by the 
Philippine Health Insurance Corporation (PHIC), 
2, Incremental Revenues from the Excise Tax on Alcohol and Tobacco Products 
as provided in Republic Act No. 10351 shall include medical assistance to 
patients with rare disease, to be provided in the guidelines set by DOH, 
SEC. 20. Fiscal Incentives. - The following shall be exempted from all taxes 
and customs duties, as applicable, whether national or local: 
1) Donations intended for researches on rare disease, maintenance of the Rare 
Disease Registry, or for purchase of orphan drugs or orphan products for use 
solely, by P?tients with rare disease; and 
2) Orphan Drugs and Orphan Products for use solely by patients with rare 
disease, as certified by the FDA. 
ARTICLE 8 
FINAL PROVISIONS 
SEC. 21. Implementing Rules and Regulations. - Within one hundred twenty 
days (120) from effectivity of this Act, the DOH, in consultation with the NIH, shall issue 
the implementing rules and regulations to this Act. 
SEC. 22. Repealing Clause. - All general and special laws, decrees, executive 
orders, proclamations and administrative regulations, or any parts thereof, which are 
inconsistent with this Act are hereby repealed or modified accordingly. 
SEC. 23. Separability Clause. - If, for any reason or reasons, any part of 
provisions of this Act shall be declared or held to be unconstitutional or invalid, other 
provision or provisions hereof which are not affected thereby shall continue to be in full 
force and effect. 
SEC. 24. Effectivity. - This Act shall take effect fifteen (15) days after its 
publication in at least two (2) newspapers of general circulation. 
Approved, 
7