oF RESE, oy

nk
ERA,

Republic of the Philippines
HOUSE OF REPRESENTATIVES
Quezon City

SIXTEENTH CONGRESS
Second Regular Session

5082

HOUSE BILL NO.

Introduced by Hon. GABRIEL LUIS R. QUISUMBING

Explanatory Note

The Amyotrophic Lateral Sclerosis (ALS) ice bucket challenge recently
flooded news articles and social media. This phenomenon raised awareness for
ALS, and to a certain extent, other rare diseases. 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,
Although these diseases afflict a small number of individuals, treatment is
usually life long and costly, making it beyond the reach of most Filipino patients.

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 aims to
provide patients with rare diseases and their families a better access to adequate
medical care, health information, and healthcare products needed to treat their
condition. It also establishes a unified system to coordinate the sustainable
research & development initiatives and resource generation efforts among
relevant agencies of government and the private sector towards improving the
quality of life of patients with rare diseases and their families.

This legislative measure is in consonance with the right of the person to be
provided proper health care. Section 15 Article 2 of the 1987 Constitution
provides, “The State shall protect and promote the right to health of the people
and instill health consciousness among them”. Section 11 Article 13 of the 1987
Constitution provides that “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

*EURORDIS.RARE DISEASES EUROPE: bep//wsse gureditong/sbout rae disease

2 National Onganbation for Rase Disondues (US National nsinates of Fea:



cost”, The Bill also finds harmony with the State policy enshrined in Section 2,
Article 13 of the 1987 Constitution of the Republic of the Philippines declaring
“the State shall adopt an integrated and comprehensive approach to health
development which shall endeavor to make essential goods, health and other
social services available to all the people at affordable cost. Priority of the needs
of the underprivileged, sick, elderly, disabled, women, and children shall be
recognized. Likewise, it shall be the policy of the State to provide free medical
care to paupers.”

Hence, the early approval of this bill is earnestly sought.

GABRIEL . QUISUMBING


Republic of the Philippines
HOUSE OF REPRESENTATIVES
Quezon City

SIXTEENTH CONGRESS
Second Regular Session

HOUSE BILLNo.__5082

Introduced by Hon. GABRIEL LUIS R. QUISUMBING

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

Be it enacted by the Senate and the House of Representatives of the Philippines in
Congress assembled:

ARTICLE 1
GENERAL PROVISIONS

SECTION 1. 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
recognizes the crucial role of research in defining health programs and activities
to address 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 or patients highly
suspected of having a rare disease has access to comprehensive medical care,
including drugs and other healthcare products to treat or otherwise, as well as
timely health information, to help them cope with their condition;

2) 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;

3) 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;

4) 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;

5) Facilitate the regular collaborative activities among stakeholders regarding
the realization of the objectives of this Bill;

6) Provide regulatory and fiscal incentives to support research and development
activities on rare diseases and the manufacturing of affordable orphan drugs or
orphan products or the importation of these; and

7) 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) 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.

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

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

4) Medical Food are food products specifically formulated for the dietary
management of specific inherited metabolic disorders. These are prescribed by


physicians with special training in the care of patients with rare disorders to
provide the necessary nutrition and treatment that is essential for the survival of
these patients.

5)National Comprehensive Newborn Screening System, as established by RA.
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.

6)Newborn Screening Continuity Clinic refers to an ambulatory clinic based in a
tertiary hospital identified by the DOH to be part of the National Comprehensive
Newborn Screening System Treatment Network. It is equipped to facilitate
continuity of care of patients confirmed with conditions included in the
expanded newborn screening in its area of coverage.

7) NIH means the National Institutes of Health.

8) 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.

9) 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.

10) 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 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 (ie, life threatening, seriously
debilitating, or serious and chronic) forms of more frequently occurring diseases.

11) Rare Disease Registry means the secure health information system, including
the electronic database system, relating to data on rare diseases, persons
afflicted with rare diseases, and orphan drugs and orphan product.

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

13) Telegenetics Referral System refers to a computer network system that
provides remote genetic clinical cénsultations to physicians in the provinces for
their patients


ARTICLE 3
RARE DISORDERS

Section5. Obligation of healthcare practitioners - 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 and the rights of the affected

individual to benefits of persons with disability;

2) To ensure that the afflicted person is referred to Newborn Screening
Continuity

Clinics identified by the Department of Health (DOH) as referral centers
for treating rare diseases; and

Section 6.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 Welfare and Development shall provide
assistance to person with rare disease to ensure that their social welfare
and benefits are provided as mandated in the Magna Carta for disabled

pers
ARTICLE 4

IDENTIFICATION, REFERRAL AND MANAGEMENT OF PERSON WITH RARE
DISORDERS

Section 7. Continuing Education and Training of Health personnel -
‘The Department of Health and the National Institutes of Health in
collaboration 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; and

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

Section 8. Public Information about rare disorders -The Department
of Health with the assistance of the NIH 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 9. Referral of patients with rare disease- Patients highly
suspected of or diagnosed with rare disease shall be referred to a Newborn
Screening Continuity Clinic identified by the DOH as referral centers for
treatment of rare diseases under the National Comprehensive Newborn
Screening System.

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 metabolic specialist, geneticist or genetic counselor for
genetic counseling.

2) In the absence of a medical specialist in the area, the Newborn
Screening Continuity Clinic shall coordinate with the National Institutes of
Health (NIH) for co-management of the patient with a specialist.

3) Establish a system in collaboration with local government 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 10. Availability of specialist for the management of afflicted
person with rare disorders - The DOH with the assistance of NIH shall ensure
the availability of a medical specialist for the management of persons afflicted
with rare diseases

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

Section 11. 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 _

4) Establish a system to ensure the regular updating of information,
diagnosis and treatment of rare disorders in order to provide for the
comprehensive health care of these patients.


Section 12. Designation of Rare Disease - The Department of Health,
upon recommendation of the NIH and RDTWG, shall have the authority to
designate any disease that is recognized to rarely afflict the population of the
country,

Section 13, 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.
Within one hundred twenty days from the effectivity of this Act, the DOH shall
publish a list of orphan drugs for these rare diseases.

Section14. 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 rare diseases are hereby considered as orphan products. Within
one hundred twenty days from the effectivity of this Act, the Department of
Health shall publish a list of orphan products for these rare diseases

Section 15. 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 (FDA) within thirty days from receipt of requirements
issued by the FDA.

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 16. 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 17. 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
diagnosis and management of persons afflicted with rare disorders and
their families; and

7) Allot budget for the implementation of the law.

Section 18. Other implementing agencies - The Food and Drug
Administration, NIH, Department of Interior and Local Government (DILG),
Department of Education (DepEd), Department of Social and Welfare
Development (DSWD), Department of Labor and Employment (DOLE) and the
Department of Science and Technology (DOST) shall each perform the mandated
task in this Bill.

1) Food and Drug Administration shall ensure that medical foods, orphan
drugs and products are permitted in the country for the purposes of
treating rare diseases;

2) NIH shall provide the technical assistance to the DOH in the
implementation of this Act.


3) DILG, DepEd, 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,

4) DOST shall provide mechanisms to further research for a better
understanding of rare diseases in the country. DOST shall develop low
cost medical foods and products for the patients.

ARTICLE 7
RESOURCE GENERATON AND INCENTIVES FOR RARE DISEASES FUNDING

Section 19. 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 in the benefit package.

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

Section 20. 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 21, 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 22. Repealing Clause - All general and special laws, decrees,
executive orders, proclamations and administrative regulations, or any parts
thereof, which are inconsistent with this Act are hereby repealed or modified
accordingly.

Section 23. 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 24, Effectivity - This Act shall take effect fifteen (15) days after
its publication in at least two (2) newspapers of general circulation.

Approved,