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Republic of the Philippines lL? RECE van
HOUSE OF REPRESENTATIVES is ECEIVED cA
Quezon City

SEVENTEENTH CONGRESS
First Regular Session

HOUSE BILL NO. 5154

Introduced by HON. GUS S. TAMBUNTING

EXPLANATORY NOTE

A “rare disease" otherwise called an "orphan disorder" is any health condition
resulting from genetic defects that rarely affect the general population. There are
6,000-8,000 rare disease, 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 patient 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 States 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 comprehensive 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 the 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. This bill will help provide patients with rare diseases and their
families better access to adequate medical care, health information, and healthcare
products needed for their condition. It establishes a system to coordinate a sustainable
research & development initiatives and resource generation efforts among relevant
agencies of government and private sector toward improving the quality of life of
patients with rare diseases and their families.

The rationale 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 11 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 all péople at affordable
cost."

In recognition of our constitutional and international obligations to improve
health of the people, the immediate enactment of this bill is therefo1 juested.

Hence, we seek the early passage of this Bill.

GUS S. TAMBUNTING


Republic of the Philippines
HOUSE OF REPRESENTATIVES,
Quezon City

SEVENTEENTH CONGRESS
First Regular Session

5154

HOUSE BILL NO.

Introduced by HON. GUS S. TAMBUNTING

AN ACT PROMULGATING A COMPREHENSIVE POLICY IN ADDRESSING THE
NEEDS OF PERSONS AFFLICTED BY RARE DISORDERS

ARTICLE 1
GENERAL PROVISIONS

SECTION 1. Short Title. - This Act shall be known as the "Rare Diseases Act of
the Philippines.”

SECTION 2. Declaration of Policy. — It is the Policy of the state to protect and
promote the right to health of the people, including the right of persons suffering from
rare diseases to survival and full and healthy development as individuals through
access to timely health information and adequate medical care. In pursuit of such
policy, the State shall institutionalize a system that is comprehensive, integrative and
sustainable and will facilitate collaboration among government and non-government
agencies and organizations at the national and local levels, private sector, professional
health organizations, academic institutions, communities and families towards the
provision of early and sustainable care of every person afflicted with rare disorders.
The State recognized the crucial role of research in defining health programs and
activities addressing the needs of patients with rare disorders. The State also
recognizes that an effective public education program is vital in helping ensure the
early diagnosis and treatment of rare diseases and in preventing those afflicted with
them from being the subject of ridicule and stigmatization. The State further
recognizes the leading role of the Department of Health in implementing the Rare
Disease Program, overseeing the provision of care, and working with the other
government agencies, the private sector and non-government organizations, in
designing and implementing programs, including research & development activities on
rare diseases for the benefit of those afflicted with them.

SECTION 3. Objectives. — The objectives of this Act are as follows:

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 of 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.

Design and maintain the Rare Disease Registry which shall include
data on rare diseases in the 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.

2. Provide regulatory and fiscal incentives to support research and
development activities on rare diseases and the import or manufacture
affordable orphan drugs or orphan products;

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 a rare disease.

ARTICLE 2
DEFINITION OF TERMS

SECTION 4. Definitions. — Under this Act, the following terms shall have been
defined as follows:

1) DOH means the Department of Health.

2) DOLE means the Department of Labor and Employment.

3) DSWD means the Department of Social Welfare and Development.

4) FDAmeans the Food and Drugs Administration.

5) Healthcare practitioners means any doctor of medicine, dentist, nurse,

midwife, allied health professionals and other health care professionals duly
licensed by the Professional Regulatory Commission.


6) Healthcare institutions means hospitals, health infirmaries, health centers,
lying-in centers or puericulture centers, whether public or private.

7) Medical care means any method used by a health care practitioner to
prevent, diagnose, and remove the symptoms and cause of a disease.

8) 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 these genetic diseases, many of which are
also rare disorders.

9) Newborn Screening Follow-up Clinics (NSFC) are regional medical centers
recognized by the DOH for having the expertise and capability for follow-up
care of newborn's screened with metabolic, genetic and rare disorders.

10) MH 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.

12) 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
diseased and declared as such by the Department of Health upon
recommendation of the National Institutes of Health.

13) 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.

14) Rare Disease means disorders such as Gaucher Disease, Maple Syrup Urine
Disease, Pompe Disease, Galactosemia, Phenylketonuria, Methylmalonic
Acidemia, Urea Cycle Defects, Hurler Syndrome, Hunter Syndrome, Prader-
Willi Syndrome, and other diseases with similar rare occurrence as
recognized by the Department of Health upon recommendation of the
National Institutes of Health. For the avoidance of doubt, it does not include
catastrophic (i.e., life threatening, seriously debilitating, or serious and
chronic) forms of more frequently occurring diseases.

15) RDTWD means Rare Diseases Technical Working Group, a DOH designated
pool of experts on rare diseases tasked with identifying rare diseases, drugs
and products.

16) Telegenetics Referral System is an established system utilizing electronic
communications (i.e., video conferencing, emails, among others) which


aims to make genetics services accessible to all patients with genetic
conditions.

ARTICLE 3
RARE DISORDERS

SECTION 5. Obligations of healthcare practitioners. — Any health care
practitioners who attend to a person with rare disorders are obligated to the following:

1) To give the patient and their family substantial information about the
significance of diagnosis and management.

2) To ensure that afflicted person is referred to a Regional Newborn
Screening Follow-up Clinic Centers identified by the DOH as referral
centers for treating rare diseases.

3) To report the case of entry into the Rare Disease Registry.

SECTION 6. Referral of patients with rare disease. — Patients suspected or
diagnosed with rare disease shall be referred to a Regional Newborn Screening Follow-
up Clinic Centers identified by the DOH as referral centers for treatment of rare
diseases.

1) Timely referral ensures that the afflicted person receives the adequate care
of his/her condition and referral of the person afflicted and her/his families
to a geneticist or genetic counselor for genetic counseling.

2) Inthe absence of a specialist in the area, the referral center must coordinate
with NIH through the DOH Centers for Health Development for co-
management of the patient with a specialist.

3) Establish a system in collaboration with local unit and agencies that will
ensure that the afflicted person receives sustainable medical management
of the disease.

4) Reports the case to NIH for its inclusion to the Rare Disease Registry.

SECTION 7. Designation of persons with rare diseases as persons with
disabilities. — Individuals with rare disease shall be included among those with
disabilities and enjoy the same rights under the Magna Carta for disabled persons as
mandated in Republic Act 9442.

1) The Department of Labor and Employment shall ensure that abled person
with rare disease are given opportunity for work and employment to
become productive members of the society.

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.


ARTICLE 4
IDENTIFICATION, REFERRAL AND MANAGEMENT OF PERSON WITH RARE
DISORDERS

SECTION 8. Continuing Education, and Training of Health Personnel. — The
Department of Health and the National Institutes of Health 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
disorders for medical management.

2) Educate health personnel on the importance of reporting of any cases of
orphan disorders for the Rare Disease Registry.

SECTION 9. Public Information about rare disorders. ~ The Department of
Health with the assistance of the National Institutes of Health and other government
agencies, professional societies and non-government organizations shall conduct
culturally sensitive public educational and information campaigns on the nature of rare
diseases, identify persons afflicted with rare disease and help the general public
understand the special needs of the afflicted persons and be spared for being the
subject of ridicule and discrimination.

SECTION 10. Referral of person afflicted with rare disease. — All patients
suspected or diagnosed to have a rare disease should be referred to the DOH
designated newborn screening follow-up clinics for follow up and management. All
persons afflicted with a rare disorder must be seen and managed by a specialist
regularly. In the absence of a specialist in the designated newborn screening follow-
up Clinics, designated person should coordinate with NIH through the CHD for co-
management with a specialist.

SECTION 11. Availability of specialist for the management of afflicted person
with rare disorders. - The Department of Health with the assistance of NIH should
ensure the availability of a specialist for the management of persons afflicted with rare
diseases.

ARTICLE 5
DESIGNATION OF RARE DISEASE, ORPHAN DRUG, AND ORPHAN
PRODUCT STATUS

SECTION 12. The Rare Disease Technical Working Group. - The Department
of Health shall convene the Rare Diseases Technical Working Group (RDTWG) which
shall have the following roles and responsibilities:

1) designate diseases that are ‘rare diseases’

2) designate orphan drugs and products corresponding to the rare diseases


3) formulate policies that shall regulate the approval and certification of
orphan drugs and products

SECTION 13. Designation of Rare Disease. - The Department of Health,
upon recommendation of the National Institutes of Health 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 diseases. Additional diseases should be approved by the DOH as recommended
by the NIH and RDTWG.

SECTION 14. Designation of Orphan Drug. — The Department of Health,
motu proprio or upon application by any interested person, may designate any drug
or medicine indicated for use by patients afflicted with any of the rare diseases as an
orphan drug; provided, that there is no existing drug or medicine in the Philippines
that can provide the same or superior alternative therapy. The drugs or medicines for
the treatment or for the alleviation of symptoms of 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 deemed to be orphan drugs. Within one hundred twenty days
from the effectivity of this Act, the Department of Health shall publish a list of orphan
drugs for these rare diseases.

SECTION 15. Designation of Orphan Product. ~The Department of Health,
motu proprio, or upon application by any interested person, may designate any
healthcare or nutritional product, other than a drug or medicine, including but not
limited to diagnostic kits, medical devices and biological products, used primarily to
prevent, diagnose, or alleviate the symptoms of rare diseases as an orphan product;
provided, that there is no existing product in the Philippines that can provide the same
or superior results, as certified by the Food and Drug Administration. Any healthcare
or nutritional product, other than a drug or medicine, including but not limited to
diagnostic kits, medical services and biological products, used primarily to prevent,
diagnose, or alleviate the symptoms of Gaucher Disease, Maple Syrup Urine Disease,
Pompe Disease, Galactosemia, Phenylketonuria, Methylmalonic Acidemia, Urea Cycle
Defects, Hurler Syndrome, Hunter Syndrome, Prader-Willi Syndrome are hereby
considered as orphan products. Within one hundred twenty days from the effectivity
of this Act, the Department Health shall publish a list of orphan products for these rare
diseases.

SECTION 16. 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 he first secures a Permit
for Use of an Orphan Drug/Orphan Product from the Food and Drug Administration
within thirty days from receipt of the following requirements, shall issue a Permit for
an Orphan Drug/Orphan Product:


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 Department of Health that the drug or product
qualifies as an orphan drug or orphan product;

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 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 be responsible for any death,
injury or damage arising from the use of the orphan drug or orphan
product and holding the Food and Drug Administration and its officials and
employees free and harmless therefrom.

The Permit for Use of an Orphan Drug/Orphan Product shall be effective for
a period of three years, renewable for periods of three years thereafter.

SECTION 17. The Rare Disease Registry. — All patients diagnose with a rare
disease shall be included in this national database for rare disease case registries.

1) All healthcare practitioners and health institutions shall be required to
report to the Rare Disease Registry of the National Institutes of Health
diagnosed cases of rare diseases and provide reports on status of
patients; provided, that such reports shall be subject to guidelines issued
by the National Institutes of Health to protect the privacy of patients
afflicted with rare diseases.

2) Health practitioners and health institutions shall inform patients afflicted
with rare diseases of relevant orphan drugs and orphan products in the
Rare Disease Registry.

ARTICLE 6
IMPLEMENTATION

SECTION 18. Lead Agency. — The Department of Health shall be the lead
agency in the implementation of this Act. For the purpose of achieving the objectives
of this act, the DOH shall:


1) Establish the Technical Working group for the Rare Diseases.

2) Develop the implementing rules and regulations for the implementation of
this Bill within one hundred eighty (180) days from the enactment of the
Law.

3) Coordinate with the National Institutes of Health for the technical
assistance in the implementation of the Act.

4) Coordinate with all government and non-government agencies that will be
involved in the implementation of the Act.

5) Designate referral centers in strategic location in the country for the timely
and sustainable medical management of persons afflicted with rare
disorders;

6) Organize a pool of medical specialist who will be responsible in the
management of persons afflicted with rare disorders and their families;

7) Allot budget for the implementation of the law.

SECTION 19. Other implementing agencies. — The Food and Drug
Administration, NIH, Department of Social and Welfare Development and Department
of Labor and Employment shall each perform the mandated task in this Bill.

1) The Food and Drug Administration shall ensure that orphan drugs and
products are permitted in the country for the purpose of treating rare
diseases.

2) The NIH shall serve provide the technical assistance to the DOH in
implementing this Bill.

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.

ARTICLE 7
RESOURCE GENERATION AND INCENTIVES FOR RARE DISEASES FUNDING

SECTION 20. Source of funds for maintaining medical management of persons
afflicted with rare diseases. — The Department of Health shall ensure the
establishment of a system that will facilitate the qualification of afflicted person as one
of the beneficiaries of the services for sustainable compliance to the medical
management of the rare disease:

1) The Philippine Health Insurance Corporation shall include the cost of
treatment of rare disease as part of its Catastrophic Illness Resource Fund.

2) Provisions from the Sin Taxes collection shall be directed to cover the cost
of rare for patients with rare diseases.

SECTION 21. Fiscal Incentives. — The following shall be exempted from all
taxes, whether national or local:


1) Donations to the intended for researches on rare diseases, maintenance of
the rare Disease Registry, or for purchase of orphan drugs or orphan
products for use solely by patients with rare diseases; and

2) Orphan Drugs and Orphan Products for use solely by patients with rare
diseases, as certified by the Food and Drug Administration.

In addition, Orphan Drugs and Orphan Products for donation solely to patients afflicted
with rare diseases or institutions, as certified by the National Institutes of Health, shall
be exempt from payments of all tariffs and duties.

ARTICLE 8
FINAL PROVISIONS

SECTION 22. Implementing Rules and Regulations. — Within one hundred
twenty days from effectivity of this Act, the Department of Health, in consultation with
the National Institutes of Health, shall issue the implementing rules and regulations to
this Act.

SECTION 23. 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.

SECTION 24. Separability Clause. — If, for any 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.

SECTION 25. Effectivity. - This Act shall take effect fifteen (15) days after its
publication in at least two (2) newspapers of general circulation.

Approved,