REPUBLIC OF THE PHILIPPINES
HOUSE OF REPRESENTATIVES
Quezon City, Metro Manila

SIXTEENTH CONGRESS
First Regular Session

3896

House BiLLNo.

INTRODUCED BY HONORABLE RANDOLPH S. TIN

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 goverment 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.

RANDOI S. TING
Re itative:
3" Distric} of Cagayan


REPUBLIC OF THE PHILIPPINES
HOUSE OF REPRESENTATIVES
Quezon City, Metro Manila

SIXTEENTH CONGRESS
First Regular Session

3896

Aan
HOUSE BIEL NO.

INTRODUCED BY HONORABLE RANDOLPH S. TING

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 afilicted 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) FDAmeans 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,i8

the existing network of medical specialists, nurses, laboratories and hospitals

screening and treating these genetic diseases, many of which are also rare disorders

Ly

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 Registrymeans 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.

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 afilicted person receives sustainable medical management of the disease.

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

Zz

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 ot
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 swom 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;

Certification from the Department of Health that the drug or product qualifies as an

orphan drug or orphan product;

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.

2

3

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 drags 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) Alot 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 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 - 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.

RANDOIPH S. TING
Reprsentative
3™ District of Cagayan