, 
, ' 
SIXTEENTH CONGRESS OF THE ) 
REPUBLIC OF THE PHILIPPINES ) 
First Regular Session 
) 
SENATE 
'11l 
JUN 10 P 3 :46 
S.B. No. 22 'i' 9 
Y.' 
------------------------------------------------------4't~,~.~;~+I'~lr:+:~'~;H\~'·~O 
Introduced by SENATOR CYNTHIA A. VILLAR 
AN ACT 
PROMULGATING A COMPREHENSIVE POLICY IN ADDRESSING THE NEEDS OF 
PERSONS AFFLICTED BY RARE DISORDERS 
EXPLANATORY NOTE 
Section 15, Article II of the 1987 Philippine Constitution states that: 
"The State shall protect and promote the right to health of the people and 
instill health consciousness among them." 
Additionally, the Republic of the Philippines, being a party to the Convention on the 
Rights of the Child (CRC), committed itself to "recognize the right of the child to the enjoyment 
of the highest attainable standard of health" (Article 24 [1], CRC) and to take appropriate 
measures to "ensure the provision of necessary medical assistance and health care to all 
children" (Article 24 [2b], CRG). 
In furtherance of the foregoing, this Bill seeks to provide for the creation of a 
comprehensive and sustainable health .system for rare diseases integrated into existing public 
health care system. The rationale for establishing a national health care system for rare 
disorders as part of the country's healthcare delivery system is further expressed in Section 11, 
Article XIII of the 1987 Philippines Constitution, to wit: 
"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 all people at affordable cost." 
Briefly, a "rare disease", otherwise called an "orphan disorder", is any health condition 
resulting from genetic defects that rarely affect the general population, which are often chronic, 
progressive, degenerative, and life-threatening. Currently, there are about 6,000-8,000 identified 
rare diseases, 75% of which affect children. Of this percentage, 30% of them die before they 
reach the age offive (5) years. 
In the Philippines alone, these rare diseases affect 1 in every 20,000 Filipino children. 
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. Furthermore, the quality of life of 
patients is often compromised by the :Iack or loss of autonomy, high level of pain and suffering 
for the patient and their family.' 
, 
The passage of this Bill will help provide patients with rare diseases, and their families, 
better access to adequate medical care, health information, and healthcare products needed to 
treat their condition. This will ensure the provision of early and sustainable care for patients 
suffering from rare diseases, 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. 
Hence, in recognition of our constitutional and international commitment to improve the 
health of the people, the early passage of this Bill is recommended . 
. ~.~~ 
1 "Rare Diseases", Philippine Society for Orphan Disorders, http://www.psod.org.ph/rare-diseasesi. 
• 
, 


, ' 
SIXTEENTH CONGRESS OF THE 
REPUBLIC OF THE PHILIPPINES 
First Regular Session 
) 
) 
) 
'i4 
,JUN 10 P3 :46 
SENATE 
S.B. No. 2279 
Introduced by SENATOR CYNTHIA A. VILLAR 
AN ACT 
PROMULGATING A COMPREHENSIVE I;lOLICYIN ADDRESSING THE NEEDS OF 
PERSONS AFFLICTED BY RARE DISORDERS 
1 
ARTICLE 1 
2 
GENERAL PROVISIONS 
3 
Section 1. Short Title - This Act shall be known as the "Rare Diseases Act of 
4 
the Philippines." 
5 
Section 2. Declaration of Policy - It is the policy of the State to protect and 
6 
promote the right to health of the people, including the right of persons suffering from 
7 
rare diseases to survival and full and healthy development as individuals through 
8 
access to timely health information and adequate medical care. In pursuit of such policy, 
9 
the State shall institutionalize a system that is comprehensive, integrative and 
10 
sustainable and will facilitate collaboration among government and non-government 
11 
agencies and organizations at the national and local levels, private sector, professional 
12 
health organizations, academic institutions, cornmunities and families towards the 
13 
provision of early and sustainable care of every person afflicted with rare disorders. The 
14 
state recognizes the crucial role of research in defining health programs and activities 
15 
addressing the needs of patients with rare disorders. The State also recognizes that an 
16 
effective public education program is vital in helping ensure the early diagnosis and 
17 
treatment of rare diseases and in preventing those afflicted with them from being the 
18 
subject of ridicule and stigmatization. The State further recognizes the leading role of 
19 
the Department of Health in implementing the Rare Disease Program, overseeing the 
20 
provision of care, and working with the other government agencies, the private sector 
21 
and non-governmental organizations, in designing and implementing programs, 
22 
including research and development activities on rare diseases for the benefit of those 
23 
afflicted with them. 
24 
Section 3. Objectives - the objectives of this Act are as follows: 
25 
26 
27 
28 
29 
30 
31 
32 
33 
34 
35 
1. 
Ensure that every patient diagnosed to have a rare disease 
has access to timely health information and adequate medical care, 
including drugs and other healthcare products to treat or otherwise help 
them cope with their condition: 
i) 
Establish a comprehensive and sustainable 
health care system integrated within, the public health care 
delivery system that will ensure' the provision of early and 
sustainable care for patients suffering from rare diseases. 
ii) 
Design 
and 
maintain 
the 
Rare 
Disease 
Registry which shall include data on rare diseases in the 
2 
• 
• 
• 


1 
2 
3 
4 
5 
6 
7 
8 
9 
10 
11 
12 
Philippines, patients afflicted with rare diseases, and orphan 
drugs and products. This data shall be utilized 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 public 
understand the special needs of such persons. 
iv) 
Facilitate the regular collaborative activities 
among stakeholders regarding 
the realization of the 
objectives of this Bill. 
13 
2. 
Provide regulatory and fiscal incentives to support research 
14 
and development activities on rare diseases and the import or 
15 
manufacture affordable orphan drugs or orphan products; 
16 
3. 
Institutionalize a financial incentive system for agencies 
17 
involved 
in 
clinical researches, 
patient care, 
medical information 
18 
management, and other similar activities for the benefit of persons afflicted 
19 
with a rare disease. 
20 
ARTICLE 2 
21 
DEFINITION OF TERMS 
22 
Section 4. Definitions - Under this Act, the following terms shall have the 
23 
meanings respectively given to them below: 
24 
25 
1) 
2) 
DOH means the Department of Health. 
DOLE means the Department of Labor and Employment. 
26 
3) 
DSWD means the Department of Social and Welfare 
27 
Development. 
28 
4) 
FDA means the Food and Drug Administration. 
29 
5) 
Healthcare practitioners means any doctor of medicine, 
30 
dentist, nurse, midwife, allied health professionals and other health care 
31 
professionals duly licensed by the Professional Regulatory Commission. 
32 
6) 
Healthcare institutions means hospitals, health infirmaries, 
33 
health centers, lying-in centers or puericulture centers, whether public or 
34 
private. 
35 
7) 
Medical care means any method used by a health care 
36 
practitioner to prevent, diagnose, and remove the symptoms and cause of 
37 
a disease. 
38 
8) 
National Comprehensive· Newborn Screening System, as 
39 
established by R.A. 9288, is the existing network of medical specialists, 
40 
nurses, laboratories and hospitals screening and treating these genetic 
41 
diseases, many of which are also rare disorders. 
42 
9) 
Newborn Screening Follow-up Clinics (NSFQ) are regional 
43 
medical centers recognized by the DOH for having the expertise and 
44 
capability for follow-up care of newborn's screened with metabolic, genetic 
45 
and rare disorders. 
3 
• 
, 


1 
2 
3 
4 
5 
10) 
NIH means the National Institutes of Health. 
11) 
Orphan Drug means any drug or medicine used to treat or 
alleviate the symptoms of persons afflicted with a rare disease and 
declared as such by the Department of Health upon recommendation of 
the National Institutes of Health. 
6 
12) 
Orphan Product means any healthcare or nutritional product, 
7 
other than a drug or medicine, including but not limited to diagnostic kits, 
8 
medical devices and biological products, u~ed to prevent, diagnose, or 
9 
treat rare diseases and declared as such by the Department of Health 
10 
upon recommendation of the National Institutes of Health. 
11 
13) 
Rare Disease Registry means the health information system, 
12 
including the electronic database system, relating to data on rare 
13 
diseases, persons afflicted with rare diseases, and orphan drugs and 
14 
orphan products. 
15 
14) 
Rare Disease means disorders such as Gaucher Disease, 
16 
Maple 
Syrup 
Urine 
Disease, 
Pompe 
Disease, 
Galactosemia, 
17 
Phenylketonuria, Methylmalonic Acidemia, Urea Cycle Defects, Hurler 
18 
Syndrome, Hunter Syndrome, Prader-Willi Syndrome, and other diseases 
19 
with similar rare occurrence as recognized by the Department of Health 
20 
upon recommendation of the National Institutes of Health. For the 
21 
avoidance of doubt, it does not include catastrophic (I.e., life threatening, 
22 
seriously debilitating, or serious and chronic) forms of more frequently 
23 
occurring diseases. 
24 
15) 
RDTWG means Rare Diseases Technical Working Group, a 
25 
DOH designated pool of experts on rare diseases tasked with identifying 
26 
rare diseases, drugs and products. 
27 
16) 
Tefegenetics Referral System is an established system 
28 
utilizing electronic communications (I.e. video conferencing, emails, 
29 
among others) which aims to make genetics services accessible to all 
30 
patients with genetic conditions. 
31 
ARTICLE 3 
32 
RARE DISORDERS 
33 
Section 5. 
Obligation of healthcare practitioners • Any health care 
34 
practitioners who attend to a person with rare disorders are obligated to the following: 
1) 
To give the patient and their family substantial information 
35 
36 
about the significance of diagnosis and management. 
37 
2) 
To ensure that afflicted person is referred to a Regional 
38 
Newborn Screening Follow-Up Clinic Centers identified by the DOH as 
39 
referral centers for treating rare diseases. 
40 
3) 
To report the case for entry into the Rare Disease Registry. 
41 
Section 6. Referral of patients with rare disease- Patients· suspected or 
42 
diagnosed with rare disease shall be referred to a Regional Newborn Screening Follow-
43 
Up Clinic Centers identified by the DOH as referral centers for treatment of rare 
44 
diseases. 
45 
1) 
Timely referral ensures that the afflicted person receives the 
46 
adequate care of his/her condition and referral of the person afflicted and 
47 
her/his families to a geneticist or genetic counselor for genetic counseling. 
4 
, 
• 
, 


1 
2) 
In the absence of a specialist in the area, the referral center 
2 
must coordinate with NIH through the DOH Centers for Health 
3 
Development for co-management of the patient with a specialist. 
4 
3) 
Establish a system in collaboration with local unit and 
5 
agencies that will ensure that the afflicted person receives sustainable 
6 
medical management of the disease. 
. 
7 
8 
4) 
Registry. 
Reports the case to NIH for its inclusion to the Rare Disease 
9 
Section 7. Designation of persons with rare disease$ as persons with 
10 
disabilities -
Individuals with rare disease shall be included among those with 
11 
disabilities and enjoy the same rights under the Magna Carta for disabled persons as 
12 
mandated in Republic Act 9442. 
13 
1) 
The Department of Labor and Employment shall ensure that 
14 
abled persons with rare disease are given opportunity for work and 
15 
employment to become productive members of the society. 
16 
17 
18 
19 
2) 
The Department of Social and Welfare Development shall 
provide assistance to person with rare disease to ensure that their social 
welfare and benefits as mandated in the Magna Carta for disabled 
persons. 
20 
ARTICLE 4 
21 
IDENTIFICATION, REFERRAL AND MANAGEMENT OF PERSON WITH RARE 
22 
DISORDERS 
23 
Section 8. Continuing Education, and Training of Health personnel -The 
24 
Department of Health and the National Institutes of Health together with health 
25 
professional societies and academic health institutions shall: 
26 
1) 
Conduct continuing education, information and training 
27 
programs for health personnel on the identification a"nd referral of persons 
28 
with rare disorders for medical management. 
29 
2) 
Educate health personnel on the importance of reporting of 
30 
any cases of orphan disorders for the Rare Disease Registry. 
31 
Section 9. Public Information about rare disorders -
The Department of 
32 
Health with the assistance of the National Institutes of Health and other govemment 
33 
agencies, professional societies and non-government organizations shall conduct 
34 
culturally sensitive public educational and information campaigns on the nature of rare 
35 
diseases, identify persons afflicted with· rare disease and help the general public 
36 
understand the special needs of the afflicted persons and be spared for being the 
37 
subject of ridicule and discrimination. 
38 
Section 10. Referral of person afflicted with rare disease - All patients 
39 
suspected or diagnosed to have a rare disease should be referred to the DOH 
40 
designated newborn screening follow-up clinics for follow up and management. AJI 
41 
persons afflicted with a rare disorder must be seen and managed by a specialist 
42 
regularly. In the absence of a specialist in the designated newborn screening follow-up 
43 
clinics, designated persons should coordinate with NIH through the CHD for co-
44 
management with a specialist. 
45 
Section 11. Availability of specialist for the management of afflicted person 
46 
with rare disorders - The Department of Health with the assistance of NIH should 
47 
ensure the availability of a specialist for the management of persons afflicted with rare 
48 
diseases 
5 
• 


• 
1 
ARTICLE 5 
2 
DESIGNATION OF RARE DISEASE, ORPHAN DRUG, AND ORPHAN PRODUCT 
3 
STATUS 
4 
Section 12. The Rare Disease Tec/:mica/ Working Group - The Department of 
5 
Health shall convene the Rare Diseases Technical Working Group (RDTWG) which 
6 
shall have the following roles and responsibilities: 
7 
1) 
Designate diseases that are 'rare diseases'; 
8 
2) 
Designate orphan drugs and products corresponding to the 
9 
rare diseases; and 
10 
3) 
Formulate policies that shall regulate the approval and 
11 
certification of orphan drugs and products. 
12 
Section 13. Designation of Rare Disease. - The Department of Health, upon 
13 
recommendation of the National Institutes of Health and RDTWG, shall have the 
14 
authority to designate any disease that is recognized to rarely afflict the population of 
15 
the country. 
16 
Gaucher Disease, Maple Syrup Urine Disease, Pompe Disease, Galactosemia, 
17 
Phenylketonuria, Methylmalonic Acidemia, Urea Cycle Defects, Hurler Syndrome, 
18 
Hunter Syndrome andPrader-Willi Syndrome are hereby designated as rare diseases. 
19 
Additional diseases should be approved by the DOH as recommended by the 
20 
NIH and RDTWG. 
21 
Section 14. Designation of Orphan Drug - The Departl']1ent of Health, motu 
22 
proprio or upon application by any interested person, may designate any drug or 
23 
medicine indicated for use by patients afflicted with any of the rare diseases as an 
24 
orphan drug; provided, that there is no existing drug or medicine in the Philippines that 
25 
can provide the same or superior alternative therapy. 
26 
The drugs or medicines for the treatment or for the alleviation of symptoms of 
27 
Gaucher Disease, Maple Syrup Urine Disease, Pompe Disease, Galactosemia, 
28 
Phenylketonuria, Methylmalonic Acidemia, Urea Cycle Defects, Hurler Syndrome, 
29 
Hunter Syndrome and Prader-Willi Syndromeare hereby deemed to be orphan drugs. 
• 
30 
Within one hundred twenty (120) days from the effectivity of this Act, the 
' 
31 
Department of Health shall publish a list of orphan drugs for these rare diseases. 
32 
Section15. Designation of Orphan Product - The Department of Health, motu 
33 
proprio or upon application by any interested person, may designate any healthcare or 
34 
nutritional product, other than a drug or medicine, including but not limited to diagnostic 
35 
kits, medical devices and biological products, used primarily to prevent, diagnose, or 
36 
alleviate the symptoms of rare diseases as an orphan product; provided, that there is no 
37 
existing product in the Philippines that can provide the same or superior results, as 
38 
certified by the Food and Drug Administration. 
39 
Any healthcare or nutritional.product, other than a drug or medicine, including but 
40 
not limited to diagnostic kits, medical devices and biologic~1 products, used primarily to 
41 
prevent, diagnose, or alleviate the symptoms of Gaucher Disease, Maple Syrup Urine 
42 
Disease, Pompe Disease, Galactosemia, Phenylketonuria, Methylmalonic Acidemia, 
43 
Urea Cycle Defects, Hurler Syndrome, Hunter Syndrome, Prader-Willi Syndrome are 
44 
hereby considered as orphan products. 
45 
Within one hundred twenty (120) days from the effectivity of this Act, the 
46 
Department of Health shall publish a list of orphan products for these rare diseases. 
6 


1 
Section 16. Permit for Restricted Use of an Orphan Drug/Orphan Product -
2 
Any person may import any orphan drug or orphan product without need of obtaining a 
3 
Certificate of Product Registration; provided, that he first secures a Permit for Use of an 
4 
Orphan Drug/Orphan Product from the Food and Drug Administration within thirty days 
5 
from receipt of the following requirements, shall issue a Permit for Use of an Orphan 
6 
Drug/Orphan Product: 
7 
1) 
A sworn application for the issuance of a Permit for 
8 
Restricted Use of an Orphan Drug/Orphan Product, containing the narne 
9 
and address of the applicant and the estimated annual volume 
10 
requirement of the drug or product; 
11 
2) 
Certification from the Department of Health that the drug or 
12 
product qualifies as an orphan drug or orphan product; 
13 
14 
15 
16 
17 
18 
19 
20 
3) 
In the case of a drug or medicine, medical device and 
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 Food and Drug Administration with 
copies to the Department of Health no later than January 15 of each year, 
a Clinical Study Report for every patient administered the drug or product 
describing the quantity administereQ or used, the therapeutic or desired 
effect, and adverse reactions, if any; 
21 
4) 
Certification that the drug or product is registered in the 
22 
country of origin; and 
23 
5) 
An Affidavit sta~ing that the applicant shall be responsible for 
24 
any death, injury or damage arising from the use of the orphan drug or 
25 
orphan product and holding the Food and Drug Administration and its 
26 
officials and employees free and harmless therefrom. 
27 
The Permit for Use of an Orphan Drug/Orphan Product shall be effective for a 
28 
period of three years, renewable for periods of three years thereafter. 
29 
Section 17. The Rare Disease Registry - All patients diagnosed with a rare 
30 
disease shall be included in this national database for rare disease case registries. 
31 
32 
1) 
All healthcare practitioners and health institutions shall be 
33 
required to report to the Rare Disease Registry of the National Institutes of 
34 
Health diagnosed cases of rare diseases and provide reports on status of 
35 
patients; provided, that such reports shall be subject to guidelines issued 
36 
by the National Institutes of Health to protect the privacy of patients 
37 
afflicted with rare diseases. 
38 
2) 
Health practitioners and health institutions shall inform 
39 
patients afflicted with rare diseases of relevant orphan drugs and orphan 
40 
products in the Rare Disease Registry. 
41 
. 
ARTICLE 6 
42 
IMPLEMENTATION 
43 
44 
45 
46 
47 
Section 18. Lead Agency - The Department of Health shall be the lead agency 
in the implementation of this Act. For the purposes of achieving the objectives of this 
act, the DOH shall: 
• 
1) 
Diseases 
Establish the Technical Working group for the Rare 
.7 
, 
• 


1 
2) 
Develop the implementing rules and regulation for the 
2 
implementation of this Bill within one hundred eighty (180) days from the 
3 
enactment of the Law 
4 
3) 
Coordinate with the National Institutes of Health for the 
5 
technical assistance in the implementation of the Act. 
6 
4) 
Coordinate with 
all 
government and 
non-government 
7 
agencies that will be involved in the implementation of the Act. 
8 
5) 
Designate referral centers in strategic location in the country 
9 
for the timely and sustainable medical management of persons afflicted 
10 
with rare disorders; 
11 
6) 
Organize a pool of medical specialists who will be 
12 
responsible in the management of persons afflicted. with rare disorders 
13 
and their families; 
14 
7) 
Allot budget for the implementation of the law. 
15 
Section 19. Other implementing agencies· The Food and Drug administration, 
16 
NIH, Department of Social and Welfare Development and Department of Labor and 
17 
Employment shall each perform the mandated task in this Bill. 
18 
1) 
The Food and Drug Administration shall ensure that orphan 
19 
drugs and products are permitted in the country for the purposes of 
20 
treating rare diseases. 
21 
2) 
The NIH shall sEirve provide the technical assistance to the 
22 
DOH in implementing this Bill. 
23 
24 
25 
26 
3) 
The DSWD and DOLE shall ensure that persons with rare 
diseases are given the opportunity to be productive members of the 
society and that they are given the same rights and benefits as persons 
with disability. 
27 
ARTICLE 7 
28 
RESOURCE GENERATON AND INCENTIVES FOR RARE DISEASES FUNDING 
29 
30 
Section 20. Source of funds for maintaining medical management of 
31 
persons afflicted with rare diseases • The Department of Health shall ensure the 
32 
establishment of a system that will facilitate the qualification of afflicted person as one of 
33 
the beneficiaries of the services for sustainable compliance to the medical management 
34 
of the rare disease: 
. 
35 
1. 
The Philippine Health Insurance Corporation shall include 
36 
the cost of treatment of rare disease as part of its Catastrophic Illness 
37 
Resource Fund. 
38 
2. 
Provisions from the Sin Taxes collection shall be directed to 
39 
cover the cost of care for patients with rare diseases 
40 
Section 21. Fiscal Incentives - The following shall be exempted from all taxes, 
41 
whether national or local:. 
42 
1) 
Donations to the intended for researches on rare diseases, 
43 
maintenance of the Rare Disease Registry, or for purchase of orphan 
44 
drugs or orphan products for use solely by patients with rare diseases; 
45 
and 
8 
• 
, 


1 
2) 
Orphan Drugs and Orphan Products for lIse solely by 
2 
patients with rare diseases, as certified by the Food and Drug 
3 
Administration. 
4 
In addition, Orphan Drugs and Orphan Products for donation solely to patients afflicted 
5 
with rare diseases or institutions, as .certified by the National Institutes of Health, shall 
6 
be exempt from payments of all tariffs and duties. 
7 
8 
ARTICLE 8 
FINAL PROVISIONS 
9 
Section 22. Implementing Rules and Regulations -
Within one hundred 
10 
twenty days from effectivity of this Act, the Department of Health, in consultation with 
11 
the National Institutes of Health, shall issue the implementing rules and regulations to 
12 
this Act. 
13 
Section 23. Repealing Clause - All general and special laws, decrees, 
14 
executive orders, proclamations and administrative regulations, or any parts thereof, 
15 
which are inconsistent with this Act are hereby repealed or modified accordingly. 
16 
Section 24. Separability - If, for any reason or reasons, any part of provisions of 
17 
this Act shall be declared or held to be unconstitutional or invalid, other provision or 
18 
provisions hereof which are not affected thereby shall continue to be in full force and 
19 
effect. 
20 
Section 25. Effectivity - This Act shall take effect fifteen (15) days after its 
21 
publication in at least two (2) newspapers of general circulation. 
22 
Approved, 
9 
• 
, 
•