【Intro】

Since the current-term Government took office, new measures on rare diseases have been proposed in every policy address, including increasing the Safety Net support for orphan drugs, optimizing the Safety Net means test mechanism, establishing patient database, conducting public education, increasing manpower, supporting scientific research and clinical research, etc., which have been or are being implemented in phases. RDHK highly appreciates all such initiatives which respond positively to the aspirations of the patient community.

The last policy address of the current-term Government will be delivered in October this year. It would therefore be a suitable time to review the Government’s achievements and shortcomings over the past four years so that governance of the new term Government can be more effective.

The new measures on rare diseases proposed by the current-term Government in the previous years can certainly help to address and solve the specific problems of individual diseases. However, these measures are mostly sporadic, fragmented, unsystematic and largely reliant on clinical means only, which is far from satisfactory in terms of effectiveness of tackling rare diseases comprehensively. Expecting that the new policy address can continue with past successes while creating new achievements in the future, RDHK hopes the Government will adopt a macro perspective and foreword-thinking approach to policy development so that more systematic, comprehensive and integrated measures can be launched in phases. RDHK would like to propose the basic framework and short-term measures as follows:

【Submission Highlights】

2 Basic frameworks:

I. To set up the “Steering Committee on Strategy for Rare Diseases” with tripartite collaboration among the government, the business sector and the community.

II. To define “rare diseases” and compile the rare disease list

5 Short-term measures:

I. To speed up the process of introduction of new drugs

II. To adopt “risk sharing” arrangements, and to speed up the inclusion of orphan drugs in the Safety Net by providing clear guidelines on the review mechanism

III. To enrich the rare disease database

IV. To expand the scope of newborn screening for rare diseases

V. To promote and develop one-stop consultation service with a view to gaining experience in establishing the “Centre of Excellence for Rare Diseases”

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