PHP119 Is There No Optimal Approach for Orphan Drugs to Patient Access? Rare Disease is Not Rare and Needs to Develop the New Pricing and Reimbursement Solution

This study aimed to analyze four current pathways affecting the listing and post-listing prices of new orphan drugs (ODs) in South Korea. These mechanisms were: (1) essential OD, (2) pharmacoeconomic evaluation (PE) waiver OD, (3) weighted average price OD, and (4) PE OD. We analyzed the ratio of the listing price of 48 new ODs to the average adjusted price (AAP) of seven advanced countries and examined the change in the post-listing price. Descriptive statistics were used to analyze the listing and post-listing price changes. The mean and median ratios of the listing price of total new OD to AAP were calculated to be 69.4% and 65.4%, respectively. Essential OD showed the highest mean (93.8%) and median (80.8%) ratios. The mean cumulative price discount rate of the new OD was 7.2% in the third year and 5.7% in the fifth year. The rarity of diseases impacts the listing price of OD, but the political effects of the benefits of OD on the post-listing price of these drugs could not be verified. Further research should be conducted to develop measures that facilitate the practical sharing of budget risks and increase patient access to new ODs.

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The National Drug Formulary Listing Process for Orphan Drugs in South Korea: narrative review focused on pricing and reimbursement pathways

Expert Opinion on Orphan Drugs ◽

10.1080/21678707.2021.1913121 ◽

2021 ◽

Author(s):

Joon Seok Bang ◽

Jong Hyuk Lee

Keyword(s):

South Korea ◽

Orphan Drugs ◽

Narrative Review ◽

Drug Formulary ◽

Formulary Listing ◽

National Drug ◽

Pricing And Reimbursement

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A global perspective on the issue of access to insulin

Diabetologia ◽

10.1007/s00125-020-05375-2 ◽

2021 ◽

Author(s):

David Beran ◽

Maria Lazo-Porras ◽

Camille M. Mba ◽

Jean Claude Mbanya

Keyword(s):

Type 1 Diabetes ◽

Diabetes Management ◽

Multinational Companies ◽

Global Perspective ◽

Patient Access ◽

Middle Income ◽

Middle Income Countries ◽

Wide Range ◽

Pricing And Reimbursement

AbstractThe discovery of insulin in 1921 changed the prognosis for people with type 1 diabetes. A century later, availability and affordability of insulin remain a challenge in many parts of the globe. Using the WHO’s framework on understanding the life cycle of medicines, this review details the global and national challenges that affect patients’ abilities to access and afford insulin. Current research and development in diabetes has seen some innovations, but none of these have truly been game-changing. Currently, three multinational companies control over 95% of global insulin supply. The inclusion of insulin on the WHO’s Prequalification Programme is an opportunity to facilitate entry of new companies into the market. Many governments lack policies on the selection, procurement, supply, pricing and reimbursement of insulin. Moreover, mark-ups in the supply chain also affect the final price to the consumer. Whilst expenses related to diabetes are mostly covered by insurance in high-income countries, many patients from low- and middle-income countries have to pay out of their own pockets. The organisation of diabetes management within the healthcare system also affects patient access to insulin. The challenges affecting access to insulin are complex and require a wide range of solutions. Given that 2021 marks the centenary of the discovery of insulin, there is need for global advocacy to ensure that the benefits of insulin and innovations in diabetes care reach all individuals living with diabetes. Graphical abstract

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PSY187 - THE QUANTITY AND QUALITY OF EVIDENCE SUPPORTING EUROPEAN MARKETING AUTHORISATION OF ORPHAN DRUGS: COMPARISON OF RARE ONCOLOGY VERSUS RARE DISEASE

Value in Health ◽

10.1016/j.jval.2018.09.2761 ◽

2018 ◽

Vol 21 ◽

pp. S468

Author(s):

S. Kelly

Keyword(s):

Rare Disease ◽

Marketing Authorisation ◽

Orphan Drugs ◽

Quality Of Evidence

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VP74 Orphan Black Box: Explanatory Principles

International Journal of Technology Assessment in Health Care ◽

10.1017/s0266462319003295 ◽

2019 ◽

Vol 35 (S1) ◽

pp. 92-93

Author(s):

George Wang ◽

Richard Macaulay

Keyword(s):

Market Access ◽

Economic Value ◽

Orphan Drugs ◽

Regulatory Approval ◽

Marketing Authorization ◽

European Medicines Agency ◽

Reimbursement Mechanisms ◽

Regulatory Pathways ◽

Small Patient ◽

Pricing And Reimbursement

IntroductionOrphan legislations over the past thirty years have successfully increased the number of drugs receiving marketing authorization for rare diseases. However, for a therapy to be accessible to most patients, it requires not only marketing authorization, but market access via public reimbursement. In many major markets, the pricing and reimbursement of new therapies is based on an assessment by a national Health Technology Assessment (HTA) body, for which economic value is typically a key consideration. This research evaluates the outcome of HTAs of orphan drugs in Europe.MethodsHTA decision data (to 31/08/2017) was extracted from Gemeinsame Bundesausschuss (G-BA), Haute Autorité de Santé (HAS), National Institute for Health and Care Excellence (NICE), Pharmaceutical Benefits Advisory Committee (PBAC), and Scottish Medicines Consortium (SMC) websites. EC-approval data was extracted from the European Medicines Agency (to 31/08/2017).ResultsOnly a small minority of drugs for orphan diseases received full recommendations for their licensed indication(s) by NICE (3/35, 9%), SMC (8/66, 12%) and PBAC (1/44, 2%). 37% (26/70) of drugs assessed received positive HTA outcome by HAS (ASMR I-III). In Germany, all approved orphan drugs (100/100) received automatic additional benefit post regulatory approval by G-BA.ConclusionsThere have been significant challenges for manufacturers in converting regulatory approval of orphan drugs into commercial success and optimised market access. Attaining positive HTA appraisals for these drugs, which have been approved under expedited regulatory pathways on a less than fully mature dataset, whilst also having high prices, due to small patient populations, limiting commercial returns, may necessitate increased utilisation of alternative reimbursement mechanisms.

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Post-approval Studies for Rare Disease Treatments and Orphan Drugs

Advances in Experimental Medicine and Biology - Rare Diseases Epidemiology: Update and Overview ◽

10.1007/978-3-319-67144-4_11 ◽

2017 ◽

pp. 197-205 ◽

Cited By ~ 4

Author(s):

William C. Maier ◽

Ronald A. Christensen ◽

Patricia Anderson

Keyword(s):

Rare Disease ◽

Orphan Drugs

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Patient access to orphan drugs in France

Orphanet Journal of Rare Diseases ◽

10.1186/s13023-019-1026-4 ◽

2019 ◽

Vol 14 (1) ◽

Cited By ~ 3

Author(s):

Marion Bourdoncle ◽

Blandine Juillard-Condat ◽

Florence Taboulet

Keyword(s):

Orphan Drugs ◽

Patient Access

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Patient access to orphan drugs

Expert Opinion on Orphan Drugs ◽

10.1080/21678707.2017.1402676 ◽

2017 ◽

Vol 5 (12) ◽

pp. 923-932 ◽

Cited By ~ 2

Author(s):

Joshua P. Cohen ◽

Josephine G. Awatin

Keyword(s):

Orphan Drugs ◽

Patient Access

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Rare disease awareness and perspectives of physicians in China: a questionnaire-based study

10.21203/rs.3.rs-65651/v1 ◽

2020 ◽

Author(s):

Xuefeng Li ◽

Xiangyu Zhang ◽

Shu Zhang ◽

Zijuan Lu ◽

Jianyong Zhang ◽

...

Keyword(s):

Medical School ◽

Rare Disease ◽

Rare Diseases ◽

Strong Support ◽

Orphan Drugs ◽

School Education ◽

Sufficient Information ◽

Medical School Education ◽

Disease Awareness ◽

Career Length

Abstract Background There are over 16.8 million rare disease patients in China, representing a significant challenge for the healthcare system and society. Rare disease patients often experience delayed diagnosis, misdiagnosis, or improper treatment, which may be due to the lack of rare disease awareness among physicians. Results A total of 224 physicians from different hospitals in China participated in the questionnaire, and 9 rare disease experts were interviewed with open-ended questions. Most physicians (83.5%) were from Tertiary hospitals, which have over 500 beds. Only 5.3% of physicians were moderately or well aware of rare diseases. Most physicians (80.1%) had suspected their patients to have rare diseases less than 3 times. There was a strong support for special legislations for rare diseases and orphan drugs. Further, multinomial logistic regression (MLR) was used to determine whether hospitals, gender, and career length has an impact on perspectives and awareness. It was shown that male physicians were more likely to think newborn screening is important (p<0.05). The longer the career length is, the more likely physicians believe that their previous education has not provided sufficient information about rare diseases and that their hospital has paid enough attention to rare diseases. Physicians from Tertiary A hospitals were more likely to rate the affordability of orphan drugs high. In addition, nine experts believed that rare disease awareness is essential for early diagnosis and timely treatment. These experts also made recommendations on how to improve rare disease awareness through medical school education and continuing training. Conclusions Our study highlighted the importance of improving rare disease awareness among physicians in China. Recommendations about how to improve rare disease awareness in medical school education and establish an online ‘information hub’ are made for considerations of policy-makers.

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