Republic of the Philippines
HOUSE OF REPRESENTATIVES
Quezon City, Manila

SIXTEENTH CONGRESS
First Regular Session

3343

House Bill No.

Introduced by HONORABLE ROSE MARIE ‘BABY’ J. ARENAS

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 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
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 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 to treat 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 people at affordable cost.”

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

Hence, we seek the early passage of this Bill.

OSE MARIE ‘B; an ay


Republic of the Philippines
HOUSE OF REPRESENTATIVES
Quezon City, Manila

SIXTEENTH CONGRESS
First Regular Session

3343

House Bill No.

Introduced by HONORABLE ROSE MARIE ‘BABY’ J. ARENAS

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 - \t 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
recognizes 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-governmental 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 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 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 the
meanings respectively given to them below:

1) DOH means the Department of Health.

2) DOLE means the Department of Labor and Employment.

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

4,) FDA means the Food and Drug 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.) 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.

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 diseases 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) __ RDTWG 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. Obligation 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 for 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.) In the 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 persons
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 persons 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 afflictthe population of
the country. Gaucher Disease, Maple Syrup Urine Disease, Pompe Disease, Galactosemia,
Phenylketonuria, Methylmalonic Acidemia, Urea Cycle Defects, Hurler Syndrome,
Hunter Syndrome andPrader-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,
motuproprio 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 Syndromeare
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.

Section15. Designation of Orphan Product - The Department of Health,
motuproprio 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 devices 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 of Healthshall 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 Use of 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 diagnosed 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 purposes 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 regulation 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 specialists 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 purposes 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 GENERATON 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
care 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 6
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 - 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.

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

Approved,