Implementing Rules and Regulations of Republic Act No. 10747 "Rare Diseases Act of the Philippines"

December 7, 2017

IMPLEMENTING RULES AND REGULATIONS OF REPUBLIC ACT NO. 10747 — AN ACT PROMULGATING A COMPREHENSIVE POLICY IN ADDRESSING THE NEEDS OF PERSONS WITH RARE DISEASE, OTHERWISE KNOWN AS THE "RARE DISEASES ACT OF THE PHILIPPINES"

The following Rules and Regulations hereby promulgated to implement the Republic Act No. 10747 an act promulgating a comprehensive policy in addressing the needs of persons with rare disease otherwise known as "Rare Diseases Act of the Philippines."

RULE I

Policy and Application

ARTICLE I

SECTION 1. Purpose. — These Implementing Rules and Regulations shall provide the concerned national government agencies, local government units and other public institutions, non-government organizations, people's organizations and private institutions with guidelines for the implementation of a comprehensive national policy institutionalizing a system towards the provision of early and sustainable care of persons with rare diseases.

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 to have full and healthy development as individuals through timely access to health information and adequate medical care. In pursuit of such policy, the State shall institutionalize a system that is comprehensive, integrative and sustainable that facilitates collaboration among government and non-government agencies and organizations at the national and local levels, private sectors, health professional organizations, academic institutions, communities and families towards the provision of care for persons afflicted with rare diseases. The State recognizes the crucial role of research in defining health programs and activities to address the needs of persons with rare disease. 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.

SECTION 3. Objectives. — The objectives of this IRR are as follows:

These Implementing Rules and Regulations shall provide the procedures for the implementation of RA 10747. Specifically, it shall:

a. Guide the integration of comprehensive care (i.e., identification, referral, and management) for persons with rare diseases within the public healthcare delivery system.

b. Clarify the process for establishing a National Rare Disease Registry that will provide evidence for policies, programs, plans, strategies, and interventions.

c. Guide the creation of health promotion, public information and education campaigns on rare diseases.

d. Establish the roles and responsibilities of different stakeholders.

e. Provide regulations and fiscal incentives to support research and development on rare diseases and to facilitate the manufacture and importation of affordable orphan drugs and products. CAIHTE

RULE II

Definition of Terms

ARTICLE II

SECTION 4. Definition of Terms. — For the purpose of this IRR:

a) Commercial use refers to the selling of orphan drugs at a profit.

b) Healthcare Practitioner refers to any doctor of medicine, dentist, nurse, midwife, allied health professional and other healthcare professional duly licensed by the Professional Regulation Commission.

c) Health care institutions refer to hospitals, health infirmaries, health centers, rural health units, lying-in clinics or puericulture centers, whether public or private.

d) Medical care refers to a comprehensive and professional care that encompasses correct diagnosis, treatment and prevention of rare diseases.

e) Medical food refers to special milk formula preparation devoid of offending amino acids, organic acids or fatty acids, amino acid supplements, essential amino acid mixtures, amino acid gels or juices, and low protein food products that are part of the regimen for the medical treatment of infants, children, adolescents and adults with inherited metabolic diseases.

f) Medical specialist refers to a pediatrician for patients zero to eighteen (0-18) years old or an adult physician for above eighteen (18) years old adequately trained in the field of inherited metabolic diseases to diagnose and treat persons with rare diseases

g) National Comprehensive Newborn Screening System refers to the Newborn Screening (NBS) system established in Republic Act No. 9288.

h) Newborn Screening Continuity Clinics refers to an ambulatory clinic based in a secondary or 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.

i) Orphan drug refers to any drug or medicine used to treat or alleviate the symptoms of persons afflicted with a rare disease and declared as such by the DOH upon recommendation of the National Institutes of Health (NIH).

j) Orphan product refers to any healthcare or nutritional product, other than a drug or medicine, including but not limited to, medical food, diagnostic kits, medical devices and biological products, used to prevent, diagnose, or treat rare diseases and declared as such by the DOH upon recommendation by the NIH.

k) PWD refers to Persons with Disability. Persons with rare diseases shall be considered as PWDs and shall have same opportunities and benefits as per Republic Act 7277.

l) Rare disease refers to disorders such as inherited metabolic disorders and other diseases with rare occurrence as recognized by the DOH upon recommendation of the NIH but excluding catastrophic (i.e., life threatening, seriously debilitating, or serious and chronic) forms of more frequently occurring diseases.

m) Rare Disease Management Program refers to a comprehensive management program encompassing the diagnosis, clinical management, genetic counseling and drug research development for people with rare diseases.

n) Rare Disease Registry refers to the secure health information system, including the electronic database system, relating to data on rare diseases, persons with rare disease, and orphan drugs and orphan products.

o) Rare Disease Technical Working Group (RDTWG) refers to the DOH designated pool of experts on rare diseases, which shall include experts from the NIH, tasked with identifying rare diseases, orphan drugs and orphan products.

p) Telegenetics Referral System refers to telehealth using a computer network system that provides remote clinical genetic consultations to physicians in the provinces for their patients.

RULE III

Identification, Referral, Management and Registration of Persons with Rare Disease

ARTICLE III

SECTION 5. Identification and Referral of Persons with Rare Disease. — Persons 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 diagnosis and treatment of rare diseases under the National Comprehensive Newborn Screening System. For patients from remote areas, the Telegenetics Referral System will be utilized. For inclusion in the registry and for appropriate management and services, the diagnosis shall be confirmed by centers or specialists recognized by the DOH.

SECTION 6. Creation and Management of Registry of Persons with Rare Diseases. — The DOH, in coordination with the NIH, shall develop a system for the identification and referral of persons with rare diseases to a specialist for diagnosis and management. All persons diagnosed with rare disease shall be included in a Rare Disease Registry for the main purpose of monitoring prevalence and incidence of rare diseases.

All healthcare practitioners and health care institutions shall be required to report to the Rare Disease Registry based in NIH diagnosed cases of rare disease and provide reports on the status of patients. Such report shall be subjected to the guidelines issued by the NIH to protect the privacy of persons with rare disease.

SECTION 7. Availability of Specialist for the Management of Persons with Rare Diseases. — The DOH, with the assistance of NIH, shall develop a system to develop the capacities of a sufficient number of medical specialists to diagnose and manage persons with rare disease through the Newborn Screening Continuity Clinics.

SECTION 8. Creation of an Oversight Committee for an Integrated Rare Diseases Management Program under DOH. — The DOH shall create an oversight committee for the integration of the various existing programs and in collaboration with different offices to address persons with rare diseases following a life cycle, patient-centered coordinated care and health systems approach. This shall be under the Disease Prevention and Control Bureau, formerly the National Center for Disease Prevention and Control, to be headed by at least a Director III.

The DOH in collaboration with the NIH, shall ensure the provision of improved and sustainable access to the services and health commodities to persons with rare diseases through the Integrated Rare Diseases Management Program.

RULE IV

Persons with Rare Disease as Persons with Disabilities (PWDs)

ARTICLE IV

SECTION 9. Designation of Persons with Rare Disease as Persons with Disabilities (PWDs). — Persons with a rare disease shall be considered as persons with disabilities (PWDs), in accordance with Republic Act No. 7277, as amended, or the Magna Carta for Disabled Persons.

SECTION 10. Rights and Privileges of Persons with Rare Disease. — The appropriate national government agency shall ensure that they are accorded the same rights and privileges as PWDs, to wit:

a) The DSWD shall provide assistance to persons with rare disease for non-medical expenses to ensure that their social welfare and benefits provided under Republic Act No. 7277, as amended, or the Magna Carta for Persons with Disabilities, are granted; and

 These shall be in the form of, but not limited to the following: transportation costs, accommodation, meals, and livelihood assistance programs.

b) The DOLE shall adopt programs that promote the availability of opportunities for work and employment of able-persons with rare disease.

 In partnership with local government units' public employment service office, persons with rare diseases shall be given equal opportunities as described above.

RULE V

Designation of Rare Disease, Orphan Drug, and Orphan Product Status

ARTICLE V

SECTION 11. Designation of Rare Disease. — The DOH, upon recommendation of the RDTWG, shall have the authority to designate any disease that is recognized to rarely afflict the population of the country as a rare disease. Within one hundred twenty (120) days from effectivity of this IRR, the DOH shall publish a list of rare diseases. This shall be regularly reviewed by the RDTWG.

SECTION 12. Designation of Orphan Drug. — The DOH, motu proprio, or upon application by any interested person, and with the recommendation of the RDTWG, may designate any drug or medicine indicated for use by persons afflicted with any of the rare diseases as an orphan drug. Within one hundred twenty (120) days from effectivity of this IRR, the DOH shall publish a list of orphan drugs for these rare diseases. This shall be regularly reviewed by the RDTWG, in partnership with FDA and a revised/updated list shall be published accordingly.

SECTION 13. Designation of Orphan Product. — The DOH, motu proprio, or upon application by any interested person, and with the recommendation of the RDTWG, 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. Within one hundred twenty (120) days from effectivity of this IRR, the DOH shall publish a list of orphan products for these rare diseases. This shall be regularly reviewed by the RDTWG, in partnership with FDA and a revised/updated list shall be published accordingly.

SECTION 14. Access to Orphan Drugs and Products shall be increased through:

a) Research and Development. The government, through DOH and DOST, with the recommendation and assistance of the RDTWG, can give research grants to local researchers that aim to develop supportive, diagnostic, and therapeutic modalities including low cost medical food for rare diseases.

b) Importation of Orphan Drug/Orphan Product.

i. The Food and Drug Administration (FDA) shall be responsible for issuing of Certificate of Product Registration (CPR) for orphan drugs and orphan products; DETACa

ii. The FDA shall prioritize the process of issuance of the CPR;

iii. The FDA shall certify Orphan drug and orphan products that shall be included in the list of conditionally-free importations under Section 105 of the Tariff and Customs Code of the Philippines.

SECTION 15. Permit for Restricted Use of an Orphan Drug/Orphan Product. — Any orphan drug/orphan product for compassionate use may be imported provided that they secure a compassionate special permit from the FDA in accordance with DOH Administrative Order No. 4, series of 1992, and any future guidelines that may be issued on the same.

Within thirty (30) days from receipt of requirements, the FDA shall issue a permit for restrictive use of an orphan drug/orphan product which shall be effective for a period of three (3) years, renewable for a period of three (3) years thereafter. Provided, that the FDA shall expedite the said permit in cases of emergency.

RULE VI

Implementation

ARTICLE VI

SECTION 16. The DOH shall be the lead agency in the implementation of this IRR. For purposes of achieving the objectives of this IRR, the DOH shall:

a) Establish the Rare Disease Technical Working Group (RDTWG);

b) Integrate rare diseases in existing health and nutrition programs across the life stages;

c) Collaborate with NIH for the technical assistance in the implementation of this IRR;

d) Collaborate with all government and non-government agencies, and all other stakeholders (i.e., patient groups, donors, etc.) that are involved in the implementation of this IRR;

e) Designate referral centers in strategic locations in the country for the timely and sustainable medical management of persons with rare disease integrated in the local health service delivery network;

f) Organize a pool of medical specialists who shall be responsible in the diagnosis and management of persons afflicted with rare disease and their families;

g) Conduct culturally-sensitive health promotion and education campaigns on the nature of rare diseases to help the general public understand the special needs of afflicted persons and their right against ridicule and discrimination, and to increase referral and identification of possible persons with rare disease;

h) Incorporate rare diseases in the development of the National Objectives for Health, other medium and long term strategic and investment plans, and monitoring and evaluation system of related health and nutrition programs;

i) Identify mechanisms and institute measures to improve access to medicines and products for persons with rare diseases;

j) Allot budget for the implementation of this IRR.

SECTION 17. The Rare Disease TWG shall have the following roles and responsibilities:

a) Determine the disorders or diseases that shall be recommended to the DOH as rare disorders or diseases;

b) Determine the orphan drugs and orphan products;

c) Identify policy gaps on the regulation of orphan drugs and orphan products and recommend the necessary policies needed to be developed; and

d) Establish a system to ensure the regular updating of information, diagnosis and treatment of rare diseases in order to provide for the comprehensive healthcare of these patients.

SECTION 18. Other Implementing Agencies. —

a) Health Care Institutions

i. Integrate the Rare Diseases in its delivery of health and nutrition services;

ii. Provide adequate and sustained services such as information, education, communication, and management of identified cases;

iii. Conduct orientation and/or training of hospital staff on the Rare Diseases.

b) Food and Drug Administration (FDA)

i. Ensure that medical foods, orphan drugs and orphan products are permitted in the country for purposes of managing conditions of persons with rare diseases including necessary devices or tests needed in the screening and diagnosis of their conditions;

ii. Develop guidelines that shall clearly define systems and identify responsible units that shall facilitate the immediate approval of the CPR of orphan drugs and orphan products as the need may arise; and

c) Department of the Interior and Local Government (DILG) shall:

i. Encourage LGUs to implement RA 10747 and extend total cooperation in the implementation of the said law; and

ii. Assist the DOH in the monitoring and evaluation of the implementation of the IRR;

iii. Provide technical assistance to local government units planning offices in the development of annual operations and investment plans for health.

d) Department of Social Welfare and Development (DSWD) shall:

i. Ensure that persons with rare disease are given the opportunity to be productive members of society; and

ii. In pursuant to RA 7277, ensure that persons with rare diseases are accorded the same rights and privileges as PWDs.

e) Department of Education (DepEd) shall:

i. Include rare diseases in the curricula of elementary and high school students;

ii. In pursuant to RA 7277, ensure that persons with rare disease are provided with access to quality education and ample opportunities to develop their skills; and,

iii. Come up with guidelines on how schools can accommodate the needs of persons with rare disease, ensuring that they are not discriminated and isolated;

iv. Enhance capability of kindergarten, elementary and secondary schools to address the health and nutrition needs of pupils and students with rare diseases.

f) Department of Labor and Employment (DOLE) shall:

i. Ensure that the persons with rare disease are given the opportunity to be productive members of the society; and

ii. In pursuant to RA 7277, ensure that persons with rare diseases are not denied access to opportunities for suitable employment.

g) Department of Budget and Management (DBM) shall:

i. Ensure that National Government Agencies (NGAs) and local government units are able to provide budgetary allocations to implement the provisions of this law; and

ii. Incorporate in the national expenditure framework and medium and long term expenditure program of concerned government agencies.

h) Department of Science and Technology (DOST) shall:

i. Provide mechanisms to further research for a better understanding of rare diseases in the country;

ii. Assist in the development of orphan drugs, orphan products; and low-cost medical foods for the persons with rare diseases; and

iii. Mobilize corresponding agencies under DOST to implement the law.

i) National Institutes of Health (NIH) shall:

i. Provide technical assistance to the DOH in the implementation of this IRR and shall provide advice to other relevant agencies;

ii. Assist DOST in conducting and supporting research on rare diseases, orphan drugs and orphan products;

iii. Through the Institute of Human Genetics, lead in the training of metabolic specialists who shall be responsible in the diagnosis and management of persons afflicted with rare disease and their families;

iv. Through the Institute of Human Genetics, be the central referral agency for the Telegenetics Referral System;

v. Create and manage Rare Disease Registry; and

vi. Engage other Institutes and/or Centers of the NIH in the implementation of the law.

j) Academe, Health Professional Organizations, and other National Organizations of Health Professionals shall:

i. Ensure that all its members are capacitated in the identification and initial management of rare diseases and the care of patients and their families, and the society at large;

ii. Recommend the inclusion of rare diseases as part of the curricula of all medical and allied health professions;

iii. Conduct educational and information campaigns on the nature of rare diseases and help the general public understand the special needs of persons with rare diseases; and

iv. Assist DOH and NIH in the release of instructional materials on rare diseases.

SECTION 19. Obligation of Healthcare Practitioners. — A healthcare practitioner who attends to a person with rare disease has the responsibility of informing the patient and their family of available resources and refer them to the nearest available specialist.

SECTION 20. Continuing Education and Training of Health Personnel. — To ensure awareness of all health personnel, the DOH, LGUs, and the academe, with the assistance of the NIH and other government agencies, professional societies and non-government organization shall:

a) Conduct continuing information, education, re-education and training programs for health personnel on the signs and symptoms, assessment and initial management of rare diseases;

b) Disseminate information materials on rare diseases at least annually to all health personnel involved in pediatric care; and

c) Integrate information in existing education programs for medical and paramedical professionals. aDSIHc

RULE VII

Resource Generation and Fiscal Incentives

ARTICLE VII

SECTION 21. Financial Assistance for Persons with Rare Disease. — A person with rare disease may avail of the following:

a) Basic benefit package from the Philippine Health Insurance Corporation, which shall be provided in accordance with its guidelines;

b) Medical assistance as provided in Section 8 of Republic Act 10351 or the Sin Tax Reform Act of 2012.

SECTION 22. Fiscal Incentives. — The following shall be exempted from all taxes and custom devices, as applicable whether national or local:

a) Donations intended for researches and treatment of persons with rare diseases, maintenance of the Rare Disease Registry, or purchase of orphan drugs or orphan products for use solely by persons with rare diseases; and

b) Orphan drugs and orphan products for use solely by persons with rare diseases, as verified by the FDA.

RULE VIII

Final Provisions

ARTICLE VIII

SECTION 23. Repealing Clause. — All general and special laws, decrees, executive orders, proclamations and administrative regulations, or any part or parts thereof, which are inconsistent with this IRR are hereby repealed or modified accordingly.

SECTION 24. Separability. — If, for any reason or reasons, any part or provision of this IRR shall be declared or held to be unconstitutional or invalid, other parts or provisions hereof which are not affected thereby shall continue to be in full force and effect.

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

(SGD.) HERMINIGILDO V. VALLE, MD, MPAOIC-Secretary of Health