Asia-Pacific Roundup: Japan’s PMDA shares thinking on showing clinical efficacy in chronic skin condition

Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) has shared points to consider for evaluating the clinical efficacy of drugs to treat the chronic skin condition palmoplantar pustulosis (PPP).

PMDA exchanged views with academia, industry, and other groups at the annual meeting of the Japan Society of Clinical Trials and Research in March 2024. The discussion informed a paper in The Japanese Pharmacology & Therapeutics journal and led to the publication of early regulatory considerations for PPP drug development on PMDA’s website.

The early considerations provide information to promote “the practical application of new technologies and the development of innovative pharmaceuticals, although scientific knowledge and information have not yet been fully accumulated,” PMDA said.

PMDA’s early consideration for PPP drug development describes the condition, which is characterized by recurrent and multiple aseptic pustules on the palms and soles, and notes geographic differences in how the term is used. In the US and Europe, PPP “generally refers to the palmoplantar-localized form of pustular psoriasis, which is rare,” PMDA said. In Japan, PPP “is not necessarily a rare disease, which is primarily due to focal infections and smoking habit,” the agency said.

Drug developers have used the change in the PPP Area and Severity Index (PPPASI) total score after 16 weeks as the assessment scale in regulatory submissions. PMDA “has generally accepted the applicant's explanation of the primary endpoint,” the agency said, but “there is no consensus on the least clinically meaningful changes in the PPPASI total score.”

The agency said the percentage of patients who achieve a certain improvement in PPPASI, such as a 50% change from baseline, and report quality of life improvements “are important in interpreting the efficacy of a drug for PPP. That conclusion led PMDA to state “the efficacy of a drug for PPP should be evaluated comprehensively by including the results of these endpoints as secondary endpoints.”

Quality of life is important in assessing efficacy in PPP, the agency said, but “it is currently considered appropriate to primarily evaluate the PPPASI Total Score to assess the severity of skin findings and lesion area.” In the future, “endpoints that are closer to clinical remission such as PPPASI 75 achievement rate may be desirable,” PMDA added.

PMDA said the primary endpoint may be evaluated after 24 weeks and long-term efficacy, for example after 52 weeks, “may also be considered as an important endpoint.” The agency’s position reflects “the fluctuations in symptoms in PPP and generally slow onset of the effect of biologics on PPP.”

PMDA Notice

Malaysia’s MDA seeks feedback on guidance for importing devices for personal use

Malaysia’s Medical Device Authority (MDA) has released draft guidance on importing medical devices for personal use for consultation.

MDA published the first version of the guidance in January 2024. The agency is proposing to retain most of the existing text but make changes to some sections to clarify its advice. MDA is proposing to add lines that explain the guidance applies to unregistered devices and that applicants can request a letter about personal use status via email either before a product is imported or, in some cases, at the point of entry.

The draft also includes two new examples. In one example, a clinic is importing blood pressure monitors. The clinic submits “a purchase order and invoice issued by an establishment that acts as an importer, rather than using a medical device registration certificate by MDA.” In that case, the devices would be deemed to be for commercial or clinical use and may be seized at the border.

The second new example describes a man who buys a pulse oximeter online but makes the purchase via his company. Because the company name was used, MDA would deem the transition to be commercial, not personal, and may seize the device at the border.

MDA is accepting feedback on the draft until 3 February.

Draft Guidance

Philippine FDA suspends some fees over issues raised by implementation of new rules

The Philippine Food and Drug Administration (FDA) has suspended the pre-assessment fee for certain licensing and product notification and registration applications.

FDA published guidelines on its new fee schedule for fees and charges late last year and brought the new rules into effect on 12 January. The agency published a notice on 22 January to explain that the new rules were in effect and would apply to all applications submitted from 12 January.

However, FDA published another notice on 24 January to disclose the temporary suspension of certain pre-assessment fees. The suspension will stay in place until further notice. FDA will process applications without collecting the fee during the temporary suspension. The agency will collect pre-assessment fees “from licensing applications lodged in the e-Services following its pilot implementation.”

FDA said it adopted the temporary suspension “in the interest of effective and efficient public service delivery and to address concerns encountered in the implementation” of the new fees and charges.

FDA Notice

Malaysia’s NPRA advises physicians after assessing safety issue linked to GLP-1 drugs

Malaysia’s National Pharmaceutical Regulatory Agency (NPRA) has advised physicians to be aware of the potential risk of aspiration and pneumonia aspiration when using GLP-1 receptor agonists during general anesthesia or deep sedation.

GLP-1 drugs such as Eli Lilly’s tirzepatide and Novo Nordisk’s semaglutide are blockbuster treatments for diabetes and obesity. European regulators discussed the risk of using the class of drugs during general anesthesia or deep sedation last year, concluding that, while a causal link could not be confirmed, the effect of the products on gastric emptying may increase the risk of aspiration and aspiration pneumonia.

NPRA assessed the local adverse drug reaction reports for four GLP-1 drugs — dulaglutide, liraglutide, semaglutide and lixisenatide — and found no cases of aspiration or aspiration pneumonia. However, the European analysis led the agency to provide advice to physicians.

“Be aware of the potential risk of aspiration and pneumonia aspiration associated with the use of GLP-1 RAs during general anesthesia or deep sedation,” NPRA said. “When prescribing GLP-1 RAs, educate patients to inform their doctors if they are scheduled for procedures under anesthesia while taking any GLP-1 RA medication.”

NPRA Notice

Singapore’s Health Sciences Authority formalizes partnership with Vietnamese agency

Singapore’s Health Sciences Authority (HSA) has signed a memorandum of understanding (MOU) with the Vietnam Metrology Institute (VMI).

HSA is Singapore’s designated institute for chemical metrology, a branch of science related to the measurement of chemical substances. As the designated institute, HSA provides certified reference materials and manages external quality assessment programs to help clinical laboratories maintain standards of accuracy.

VMI is central to Vietnam’s measurement standards system. HSA has collaborated with VMI since 2015, providing training and engaging Vietnam’s clinical laboratories. The MOU advances and formalizes the relationship, setting the stage for joint research, knowledge dissemination, sharing of best practices and capacity building.

HSA Notice

Other News:

PMDA has updated the precautions for the oral COVID-19 antivirals Xocova (ensitrelvir) and Lagevrio (molnupiravir). The update advises physicians to carefully consider the necessity of administering the drug to women of childbearing potential and to confirm a patient is not pregnant “through sufficient patient interviews prior to administering the drug.” PMDA made the update after learning of multiple cases of pregnancy being detected after Xocova or Lagevrio have been administered. PMDA Notice

Asia-Pacific Roundup: Japan’s PMDA shares thinking on showing clinical efficacy in chronic skin condition

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