RE Vi
Republic of the Philippines CEN

HOUSE OF REPRESENTATIVES
Quezon City

SIXTEENTH CONGRESS
First Regular Session

3634

HOUSE BILL NO.

Introduced by Representative Alfredo D. Vargas Ill
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 family2. 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, the Constitution declares that it 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 State Parties to “recognize the right of the
child to the enjoyment of the highest attainable standard of health*” and to “ensure the
provision of necessary medical assistance and health care to all children."

This bill provides for the creation of a comprehensive and sustainable health
care 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.

‘What is a Rare Disease?, Available:
<chttp./Avww.eurordis, ora/sites/defaultfiles/publications/Fact Sheet_RD.pdf> . (12 December 2013]
? About Rare Diseases, 2009. Available: http://www.eurordis.ora/about-rare-diseases. [12 December 2013]
® Section 15 of Article Il of the 1987 Constitution
{Atticle 24(1] of the United Nations Convention on the Rights of the Child (UNCRD)
* Article 24[2b] of the UNCRD

_ 1 0EC 73)


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 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 the
health of the people, the immediate enactment of this bill is therefore requested.

ALEREDO’D, VARGAS Il

® Section 11 of Article 13 of the 1987 Constitution


Republic of the Philippines
HOUSE OF REPRESENTATIVES
Quezon City

SIXTEENTH CONGRESS
First Regular Session

3634
HOUSE BILL NO.

Introduced by Representative Alfredo D. Vargas Ill

AN ACT
PROVIDING A COMPREHENSIVE POLICY IN ADDRESSING THE NEEDS OF PERSONS
AFFLICTED BY RARE DISEASES

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

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
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:

(a) 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;

(b) 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;

(c) 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;

(a) 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;

(e) Facilitate the regular collaborative activities among stakeholders
regarding the realization of the objectives of this Act;

(6) 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; and

(g) 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:

(a) DOH refers to the Department of Health.
(b) DOLE refers to the Department of Labor and Employment.
() DSWD refers to the the Department of Social and Welfare Development.

(a) FDA refers to the Food and Drug Administration.


(e.) 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.

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

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

(h) 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.

(a) 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.

6) NIH refers to the the National Institutes of Health.

(k) 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.

(0) 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,

(m) 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

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

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


(p) 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:

(a) To give the patient and their family substantial information about the
significance of diagnosis and management.
(b) 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.
(c) 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.

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.

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

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.

‘The Department of Social and Welfare Development shalll 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:

(a) Conduct continuing education, information and training programs for
health personnel on the identification and referral of persons with rare disorders
for medical management; and

(b) 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:

(a) designate diseases that are ‘rare diseases’;

(b) designate orphan drugs and products corresponding to the rare
diseases; and

() 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, 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 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:

(a) 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;

(>) Certification from the Department of Health that the drug or product
qualifies as an orphan drug or orphan product;
(c) 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, ifany;

@ Certification that the drug or product is registered in the country of
origin; and
(e) ‘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.

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.

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:

(a) Establish the Technical Working group for the Rare Diseases;

i Develop the implementing rules and regulation for the implementation of
this Bill within one hundred eighty (180) days from the enactment of the Law;
(c) Coordinate with the National Institutes of Health for the technical

assistance in the implementation of the Act;

(a) Coordinate with all government and non-government agencies that will
be involved in the implementation of the Act;

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

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

(gs) ‘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 Act.


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

The NIH shall serve provide the technical assistance to the DOH in implementing
this Act.

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:

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

(a) 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

(>) 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 the approval of this Act, the Department of Health, in consultation with the
National Institute 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.