04 AUG 2014
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Republic of the Philippines
HOUSE OF REPRESENTATIVES
Quezon City, Metro Manila

SIXTEENTH CONGRESS
‘Second Regular Session

House ano. 4781

Introduced by Honorable MARK A. VILLAR

EXPLANATORY NOTE

The 1987 Philippine Constitution states that: "The State shall protect and
promote the right to health of the people and instil health consciousness among
them." (Section 15, Article I!)

Moreover, our country, being a party to the Convention on the Rights of the
Child (CRC), committed itself to "recognize the right of the child to the enjoyment of
the highest attainable standard of health” (Article 24 [1], CRC) and to take
appropriate measures to "ensure the provision of necessary medical assistance and
health care to all children” (Article 24 [2b], CRG).

In recognition thereto, this Bill seeks to provide for the creation of a
comprehensive and sustainable health system for rare diseases integrated into
existing public health care system

"Rare disease", otherwise called an "orphan disorder’, is any health condition
resulting from genetic defects that rarely affect the general population, which are often
chronic, progressive, degenerative, and life-threatening. Rare diseases are disabling
the quality of life of patients is often compromised by the lack or loss of autonomy.
There is high level of pain and suffering for the patient and his/her family and there is no
existing effective cure for most rare disease. '

"hte: prodorg phar diseases!



According to the Philippine Society for Orphan Disorders, there are 6,000-
8,000 rare diseases, 75% of which affect children. Also, 30% of rare disease patients
die before the age of five (5) years, and 80% of rare diseases have identified genetic
origins.

In the Philippines, persons born with and afflicted with rare or orphan
disorders are a vulnerable and special population. A disease or disorder is
considered rare if it affects 1 in 20,000 individuals or less, as defined by the Institute
of Human Genetics of the National Institutes of Health, University of the Philippines
Manila.

As of March 2011, the Institute of Human Genetics (IHG) has a record of 244
patients with rare inborn errors of metabolism while the Philippine Society for Orphan
Disorders, Inc. (PSOD) has a record of 145 patients with rare disorders (including
rare inborn errors of metabolism). Majority of the patients in PSOD's registry is part
of the IHG registry.

Inborn errors of metabolism individually are rare but collectively they are
common. In the United States, the collective frequency or incidence of these
disorders is estimated at 1 in every 4,000 live births (of term infants who develop
symptoms of sepsis without known risk factors, as many as 20% may have an inborn
error of metabolism).

With the foregoing facts, the enactment of this proposed measure will help the
patients with rare diseases, and their families, cope with the financial and non-
financial challenges of living and dealing with rare diseases. Specifically, this
proposed measure will provide them better access to adequate medical care, health
information, and healthcare products needed to treat their condition. 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.

Hence, the early passage of this Bill is eamestly sought.
‘

MARK A. VILLAR

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Republic of the Philippines
HOUSE OF REPRESENTATIVES
Quezon City, Metro Manila

SIXTEENTH CONGRESS
Second Regular Session

HOUSE BILL NO. _ 4? 81

Introduced by Honorable MARK A. VILLAR

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

Be it enacted by the Senate and 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."

SEC. 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 and development
activities on rare diseases for the benefit of those afflicted with them.

SEC. 3. Objectives. - the objectives of this Act are as follows

4. 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 Act.

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

SEC. 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 (NSFQ) 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) N/H 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

SEC. 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

SEC. 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.

SEC. 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 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

SEC. 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

SEC. 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.

SEC, 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 newbom 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


SEC. 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

SEC. 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;
and

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

SEC. 13. Designation of Rare Disease. - The Department of Health, upon
recommendation of the National Institutes of Health and RDTWG, shall have the
authority to designate any disease that is recognized to rarely afflict the population of
the country,

Gaucher Disease, Maple Syrup Urine Disease, Pompe Disease,
Galactosemia, Phenylketonuria, Methylmalonic Acidemia, Urea Cycle Defects,
Hurler Syndrome, Hunter Syndrome and Prader-Willi Syndrome are hereby
designated as rare diseases. Additional diseases should be approved by the DOH as.
recommended by the NIH and RDTWG.

SEC, 14. Designation of Orphan Drug. - The Department of Health, motu
proprio or upon application by any interested person, may designate any drug or
medicine indicated for use by patients afflicted with any of the rare diseases as an
orphan drug; provided, that there is no existing drug or medicine in the Philippines
that can provide the same or superior alternative therapy.

The drugs or medicines for the treatment or for the alleviation of symptoms of
Gaucher Disease, Maple Syrup Urine Disease, Pompe Disease, Galactosemia,
Phenylketonuria, Methyimalonic Acidemia, Urea Cycle Defects, Hurler Syndrome,
Hunter Syndrome and Prader-Willi Syndrome are hereby deemed to be orphan
drugs.

Within one hundred twenty (120) days from the effectivity of this Act, the
Department of Health shall publish a list of orphan drugs for these rare diseases.


SEC.15. Designation of Orphan Product. - The Department of Health, motu
proprio or upon application by any interested person, may designate any healthcare
or nutritional product, other than a drug or medicine, including but not limited to
diagnostic kits, medical devices and biological products, used primarily to prevent,
diagnose, or alleviate the symptoms of rare diseases as an orphan product;
provided, that there is no existing product in the Philippines that can provide the
same or superior results, as certified by the Food and Drug Administration

Any healthcare or nutritional product, other than a drug or medicine, including
but not limited to diagnostic kits, medical 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 (120) days from the effectivity of this Act, the
Department of Health shall publish a list of orphan products for these rare diseases

SEC. 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.

SEC.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
SEC.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
Act 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 famil

7) Alot budget for the implementation of the law.

SEC. 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.


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 provide the technical assistance to the DOH in implementing
this Act.

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

SEC. 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

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

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

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

1) Donations 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

SEC. 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


SEC. 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.

SEC. 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.

SEC. 28. Effectivity - This Act shall take effect fifteen (15) days after its
publication in at least two (2) newspapers of general circulation.

Approved,