Module 0: The Opening
模块 0：开幕

Every resource spent is a choice made.

Every choice made is a life affected.

寻求治愈的人们啊，

你考虑过你的决定的分量吗？

这不是一门关于
这是一门关于拯救生命的选择的课程。

一个国家的转变

Muscat, 1970. The Ministry of Health is born.

在部门之前

2
Hospitals in all of Oman
118
Infant deaths per 1,000 births

By 2022

85+
从穆桑代姆到佐法尔的医院
8.8
Infant deaths per 1,000 births

118 → 16 → 8.8

Infant mortality per 1,000 births

1970 → 2002 → 2022

In one generation, Oman saved 109 children per thousand.
Life expectancy rose from 49 to 77 years.

你没有亲眼目睹这一切吗？奇迹？

Then consider what comes next.

俄勒冈州选择的故事：

1994年，俄勒冈州面临着一场危机：医疗补助无法覆盖一切。他们没有秘密配给，而是创建了一个公开的优先列表。条件治疗对按成本效益和公共价值进行排名。髋关节置换术的排名高于整容手术。针对罕见癌症的挽救生命的治疗方法的排名低于预防数千例病例的疫苗。

明确的权衡存在争议，但很明显。俄勒冈州证明配给无处不在；选择是公开还是隐藏。

您是 1994 年的俄勒冈州卫生官员。医疗补助无法涵盖一切。你会做什么？

Path A: Ration secretly like other states 继续秘密决定谁得到什么护理。
结果：公众的不信任加剧。对不公平和隐藏议程的指控。没有人明白为什么他们的治疗被拒绝。
Path B: Create a transparent priority list 根据成本效益和公共价值观发布排名。让每个人都看到其中的权衡。
结果：争议爆发，但公众理解。信任随着时间的推移而建立。俄勒冈州成为诚实医疗配给的典范。

这是困扰每个卫生系统的问题。

5.3%
Oman's GDP on healthcare (2020)
$2M+
Cost of one gene therapy dose
10+
New therapies approved yearly

Innovation outpaces budgets. Choices must be made.

What is Health Technology Assessment?

在本课程结束时，您将能够：

  • Define HTA and explain its role in healthcare decision-making
  • 计算和解释 QALY 和 ICER 值
  • Apply Oman's cost-effectiveness threshold correctly
  • 对4 年范围
  • Perform mandatory sensitivity analyses
  • Identify appropriate comparators for different scenarios

使用 scientific evidence to determine the value of health technologies.

七项原则的多学科流程：

1. Every resource spent is a choice made.

2. Value must be measured, not assumed.

3。比较器定义问题。

4. A QALY is a year of life, lived well.

5. Uncertainty must be explored, not hidden.

6。可负担性与价值不同。

7. Transparency protects the public trust.

Every resource spent is a choice made.

模块 1：问题

模块 1：问题

比较器定义了问题。

你有没有考虑过

that every comparison reveals truth differently?

将新药与水进行比较，这似乎很神奇。将其与现有最好的治疗方法进行比较，它的真正价值就显现出来了。

两位肿瘤学家的故事

Sultan Qaboos Comprehensive Cancer Care Centre, Muscat

Dr.法蒂玛和艾哈迈德医生都治疗同一种癌症。 Fatima 医生的患者接受了 18,000 OMR 的新免疫疗法。 Ahmed 医生的患者接受 4,000 OMR 的化疗。

两名患者的结果相似。免疫疗法有效，但针对该特定病例的化疗也有效。

谁的选择是明智的？答案取决于您所比较的内容。

"A new technology without a comparator is like a scale with only one weight — it cannot measure value."

Decision Tree: Choosing the Comparator

此适应症有授权的可报销治疗吗？
YES
Use the standard of care as comparator
NO
Use Best Supportive Care (BSC)

What makes a valid comparator?

It must be authorized 适应症。

It must be reimbursed and available.

It must be evidence-based 已证实有效。

It must be what the new technology will actually replace.

Oman's Epidemiological Profile

Ministry of Health, 2022
“当本地数据不可用时，可以接受国际流行病学参考，优先考虑来自 GCC 的数据”
4.5M
Population (2023)
~45%
Under age 30
Rising
NCDs burden

当他们询问未满足的需求时，

智者回答：

“明确阐明尚未完全解决的具体公共卫生需求 - 例如早期发现、低治愈率、治疗耐药、依从性问题或严重副作用等领域。”

— Oman HTA Guidelines, 2024

Module 1 Quiz

当罕见疾病没有有效治疗方法时，应该使用什么作为比较药物？

比较器定义了问题。

模块 2：证据

模块2：证据

Value must be measured, not assumed.

行医者啊，

你没有看到主张需要证据吗？

两项研究的故事

Clinical Trial

500 patients, strict inclusion criteria, controlled conditions

Shows efficacy

Real-World Study

5,000 patients, routine practice, diverse population

Shows effectiveness

Both are needed. Neither alone is sufficient.

What outcomes truly matter?

Mortality: Does it extend life? (Overall Survival)

Morbidity: Does it reduce suffering? (Event-free survival)

Quality of Life: Does it improve daily experience? (PROs)

Function: Can patients return to work, to family, to life?

Hard vs. Surrogate Endpoints

Hard Endpoints (Preferred)

Overall survival, major clinical events, mortality

Direct patient benefit — no extrapolation needed

Surrogate Endpoints

Tumor response, biomarkers, lab values

Require validation that they predict hard outcomes

缩小的肿瘤（替代）可能并不总是转化为更长的寿命（硬终点）。在信任该措施之前验证链接。

决策树：证据综合

Are there multiple RCTs available?
YES
Conduct meta-analysis or network meta-analysis
NO
Is there one pivotal trial?
Justify the choice, assess transferability

指导方针命令：

“如果有的话，利用海湾国家联合临床评估作为所调查卫生技术的主要证据来源。”

— Oman HTA Guidelines, Section 2

Regional collaboration strengthens individual nations.

生存分析：试验之外的推断

当试验时间短但疾病寿命长时，我们必须使用参数模型（Weibull、对数正态、Gompertz）仔细推断生存曲线。
Kaplan-Meier
Observed data
Parametric
Extrapolation
Hazard Ratio
Relative effect

Justify model choice. Test sensitivity to alternative extrapolations.

Value must be measured, not assumed.

模块 3：平衡

模块 3：THE平衡

可负担性与价值不同。

您是否思考过成本与价值之间的差异

？

无法治愈的廉价治疗方法是昂贵的。昂贵的治愈治疗可能是最划算的。

Decision Tree: Type of Economic Evaluation

Does the new technology show statistically significant improvement in primary endpoints?
YES
Cost-Utility Analysis
Measure outcomes in QALYs
NO
Cost-Minimization
Outcomes assumed equal

四种评估类型

Cost-Benefit
All outcomes in OMR
Rarely used (ethical issues)
Cost-Minimization
When outcomes equal
Cost-Effectiveness
Natural units (LY, events)
Cost-Utility
Preferred: QALYs

Perspective: Who Bears the Cost?

Healthcare Perspective (Mandatory)

All direct medical costs: drugs, hospitalization, diagnostics, nursing, palliative care — regardless of who pays.

Societal Perspective (Optional)

Broader costs: transportation, caregiver burden, lost productivity, school impact.

Oman Requirement
“医疗保健观点被强制作为基本情况。”

我们必须展望未来多远？

对于糖尿病或心脏病等慢性疾病，着眼于一生。对于急性疾病，较短的窗口可能就足够了，但要证明您的选择是合理的。

Discount Rate: 3% per year

Applied to both costs and outcomes (Oman requirement)

Examples: Time Horizons by Disease

Diabetes (Type 2)

Horizon: Lifetime

Complications develop over decades (nephropathy, retinopathy, CVD)

Acute Infection

Horizon: Weeks to months

Resolution or death occurs quickly

Cancer

Horizon: 5-10 years or lifetime

Survival curves, recurrence patterns

Cardiovascular

Horizon: Lifetime

Secondary prevention, long-term outcomes

NCDs account for a growing share of Oman's disease burden.

可负担性与价值不同。

模块 4：测量

模块 4：测量 (QALY)

A QALY is a year of life, lived well.

Consider two patients.

Patient A

Lives 5 more years
In constant pain
Cannot work or enjoy family

Patient B

Lives 3 more years
In good health
Returns to full life

Which outcome is truly better?

QALY 公式

QALY = Life Years × Utility

Where utility = 1 (perfect health) to 0 (death)

计算两者患者

Patient A: 5 years × 0.4 utility = 2.0 QALYs

Patient B: 3 years × 0.85 utility = 2.55 QALYs

Patient B has more quality-adjusted life, despite fewer years.

因此智者宣称：

“QALY对所有人一视同仁。生命的一年对于年轻人和老年人、富人和穷人、权贵和卑微来说具有相同的价值。”

这既是它的力量，也是它的价值。

Measuring Utility: EQ-5D

Preferred Instrument
EQ-5D 是首选实用工具，最好在可用时具有 GCC/阿曼特定值集。
Mobility
Walking ability
Self-Care
Washing, dressing
Activities
Work, family, leisure
Pain
Discomfort level
Anxiety
Depression

Patient-Reported Outcomes (PROs) capture what matters most to patients.

Real Case: Cancer Therapy in Muscat

Illustrative Example: Royal Hospital Oncology

Outcome Standard Care New Therapy Difference
Life Years 2.5 4.0 +1.5
Utility (EQ-5D) 0.65 0.70 +0.05
QALYs 1.63 2.80 +1.17

Note: This is an illustrative example based on Oman HTA methodology.

A QALY is a year of life, lived well.

模块 5：比率

模块 5：比率 (ICER)

Every resource spent is a choice made.

现在的问题变为：

How much for each year of quality life?

ICER 公式

ICER = ΔCost / ΔQALY

Incremental Cost per Quality-Adjusted Life Year Gained

Translation: "How many extra rials do we spend for each additional QALY?"

Continuing the Royal Hospital Case

添加成本数据

Measure Standard New Therapy Incremental
Total Cost (OMR) 8,500 18,200 +9,700
QALYs 1.63 2.80 +1.17

ICER = 9,700 / 1.17 = 8,291 OMR/QALY

成本效益平面

Northwest: REJECT

More costly, less effective

Northeast: TRADE-OFF

More costly, more effective

Southwest: TRADE-OFF

Less costly, less effective

Southeast: DOMINANT

Less costly, more effective

When a technology dominates,

决策很明确。

It costs less AND heals more. Accept without hesitation.

Decision Tree: The Four Quadrants of Value

Is the new technology MORE effective than current care?
YES
Is it also LESS costly?
YES
DOMINANT
Always adopt
NO
TRADE-OFF
Calculate ICER
NO
Is it LESS costly?
YES
TRADE-OFF
Calculate ICER
NO
DOMINATED
Never adopt

Every resource spent is a choice made.

模块 6：阈值

模块 6：阈值

Value must be measured, not assumed.

If ICER is 8,291 OMR per QALY...

这是好还是坏？

我们需要一个阈值 - 一条将“值得”与“不值得”分开的线。

Oman's Cost-Effectiveness Threshold

CET = GDP per capita × Multiplier

Baseline: 1× GDP per capita

~8,000
OMR GDP per capita (approx.)
1-3×
Multiplier range
Data Source
来自阿曼中央银行的人均 GDP 数据（每年更新）。提交时使用最新的官方数字。

但并非所有条件都是平等的。

Orphan diseases: Multiplier = 2×

Priority diseases (cancer): Multiplier = 2×

Major relative health gain: Up to 3×

社会愿意为治疗最严重和最罕见的疾病支付更多费用。

Decision Tree: Is It Cost-Effective?

Is ICER below the calculated threshold?
YES
Cost-Effective
考虑报销
NO
Are there special circumstances? (Soft threshold)
Negotiate or decline

返回我们的案例

Royal Hospital Cancer Therapy Decision

ICER: 8,291 OMR/QALY

Disease: Cancer (priority) → Multiplier = 2×

Threshold: 8,000 × 2 = 16,000 OMR/QALY

8,291 < 16,000 → COST-EFFECTIVE

Value must be measured, not assumed.

模块7：预算

模块7：预算

可负担性与价值不同。

你没看到差异吗

between a wise purchase and a breaking flood?

索磷布韦的故事

United States, 2014: 吉利德推出Sovaldi （索磷布韦），第一种治疗丙型肝炎的药物。每位患者为期 12 周的疗程费用为 84,000 美元。临床试验显示治愈率超过 90%。 ICER 非常有利——预防肝衰竭、肝硬化、移植和癌症。

“这很划算！”分析师宣称。付款人不同意。

为什么？考虑一下规模。

How many Americans had Hepatitis C?

3.2M
患有慢性丙型肝炎的美国人
$84,000
Cost per patient
$268B
Total potential cost

Cost-effective per person. Unaffordable at scale. Medicaid programs rationed access.

您是 2014 年的医疗补助主任。Sovaldi 的费用为每位患者 84,000 美元。 320万患者需要它。您做什么？

Path A: Fund treatment for all patients immediately 批准 Sovaldi 用于所有丙型肝炎患者，无论疾病阶段如何。
Outcome: Budget collapses. $268 billion cost forces cuts to cancer care, mental health, and preventive services. Other patients suffer.
Path B: Implement tiered access based on disease severity Prioritize patients with advanced liver disease. Others wait.
Outcome: Controversy and lawsuits follow, but budget survives. Some patients progress while waiting. Explicit rationing is painful but sustainable.
Path C: Negotiate price reductions aggressively 利用集体谈判、仿制药竞争和批量折扣来降低成本。
Outcome: Takes time, but prices eventually drop 90%. By 2020, treatment becomes affordable. Patience and negotiation win.

Two Questions, Two Answers

Cost-Effectiveness Analysis

“物有所值吗？”

Per-patient perspective

✓ YES

预算影响分析

"Can we afford it at scale?"

Population perspective

✗ NOT YET

两个答案都是正确的。双方都必须告知该决定。这就是阿曼需要两项分析的原因。

Oman's BIA Requirements

4 Years
Projection horizon
0%
Discount rate (no discounting)
Gradual
Patient uptake assumption

BIA 仅包括医疗保健付款人 — 卫生部承担的费用。

糖尿病药物：4 年的预算影响

Item Year 1 Year 2 Year 3 Year 4 Total
Patients treated 40,000 80,000 120,000 160,000 —
New therapy cost (OMR) 8M 16M 24M 32M 80M
Avoided complications -1M -3M -5M -8M -17M
Net impact (OMR) 7M 13M 19M 24M 63M

Gradual uptake (10%→40% of eligible patients over 4 years)

因此决策者要求：

"What can we negotiate?"

The Resolution: 卫生部谈判了一项分阶段的计划与制造商的介绍。第一年：仅限高危患者。第 2 年：扩展到中等风险。价格降低 20%，以换取销量保证。

价值和承受能力都得到了满足。

可负担性与价值不同。

模块 8：不确定性

模块 8：不确定性

Uncertainty must be explored, not hidden.

你没有看到信心如何能够改变吗？欺骗？

自信分析师的故事：

一位分析师展示了他的模型：“这种药物每 QALY 的成本为 12,000 OMR。它具有成本效益。”委员会批准了。

一年后，真实世界数据显示该药物的效果只有试验建议的一半。真实的 ICER 为每个 QALY 28,000 OMR。

Had he explored uncertainty, he would have known: "There is only a 40% chance this drug is cost-effective."

决策树：敏感性分析

Deterministic SA (Mandatory)
Change each input by ±10%
Is there significant parameter uncertainty?
YES
Probabilistic SA
Run 1,000+ iterations
LOW
场景分析可能就足够了

The Tornado: Finding What Matters

赫赛汀批准的故事：

2006 年， NICE 评估了赫赛汀治疗早期乳腺癌的效果。每位患者的药物费用为 20,000 英镑。临床试验显示了生存获益，但随访时间较短——生存估计是外推的。 NICE 的敏感性分析显示，一切都取决于一个不确定的数字：长期生存。如果推断乐观，ICER 为 18,000 英镑/QALY（可接受）。如果悲观，£40,000/QALY（边界）。

NICE 有条件批准，需要现实世界的生存监测。龙卷风图揭示了不确定性最重要的地方。

您是 2006 年的一名 NICE 分析师。赫赛汀显示出前景，但生存数据不确定。 ICER 的范围可以从可接受到临界。您有什么建议？

Path A: Approve based on optimistic extrapolation 相信试验数据并以较低的 ICER 估计值批准。
Outcome: If extrapolation proves wrong, resources are wasted on a less effective treatment than assumed. Opportunity cost: other patients lose access to better interventions.
Path B: Reject until long-term survival data is available Wait 5-10 years for definitive survival outcomes before approving.
结果：潜在有效的治疗被推迟了数年。今天可以受益的患者被拒绝进入。如果药物有效，您就造成了可预防的死亡。
Path C: Approve with mandatory real-world monitoring Grant access now, but require ongoing data collection to verify survival assumptions.
结果：患者可以使用。证据不断积累。如果结果令人失望，可以修改决定。不确定性得到管理，而不是被忽视。

Oman requires: Display at least the top 10 most impactful parameters.

What does honest uncertainty look like?

分析师的诚实报告
“我们的基本情况 ICER 为每 QALY 8,291 OMR。但是，如果生存率比试验建议的低 20%（在有限的随访情况下这是一个合理的情况），ICER 会升至每 QALY 14,500 OMR QALY。

在 16,000 OMR/QALY 的阈值下， there is an 85% probability this technology is cost-effective."

这是睁大眼睛做出决策。

Cost-Effectiveness Acceptability Curve

CEAC 显示了一项技术在不同支付意愿阈值下具有成本效益的概率，它回答：“我们有多大信心。是吗？”
50%
at 5,000 OMR/QALY
85%
at 10,000 OMR/QALY
95%
at 16,000 OMR/QALY

Plot probability of cost-effectiveness (y-axis) against WTP threshold (x-axis)

Uncertainty must be explored, not hidden.

模块 9：决策

模块 9：决策

Transparency protects the public trust.

Now all threads come together.

证据、成本、价值、不确定性。

Oman's HTA Journey: The Roadmap

2024

First Edition of HTA Guidelines

Ministry of Health publishes comprehensive methodology

1-2 yrs

Short-term: Capacity Building

Train-the-trainers, innovative pharmaceuticals focus

3-5 yrs

Medium-term: Expansion

Medical devices, Oman-specific threshold

6-10 yrs

Long-term: Full Transparency

Publish all reports, revision of decisions

当 21 位专家齐聚一堂时马斯喀特...

October 27, 2022: 来自卫生部、苏丹卡布斯癌症中心和皇家医院的二十一位专家齐聚一堂，接受高级 HTA 培训。他们被问到：“您希望阿曼的 HTA 系统变成什么样子？”

Their answers would shape the nation's healthcare future.

阿曼专家之声

95%
更喜欢。多标准决策分析
90%
Demand full transparency
81%
想要强制执行本地数据

When asked about cost-effectiveness thresholds, 57% chose explicit soft thresholds — flexible enough for special cases, firm enough to guide decisions.

透明度如何？

PHARMAC 透明度的故事：

当新西兰的 PHARMAC 拒绝资助癌症药物时，他们会以充分的理由公布每一项药物资助决定。 2010 年，患者感到愤怒，但也能明白原因：每个 QALY 的成本超过了阈值，而且预算影响会取消其他治疗的资金。相比之下，PHARMAC 面临批评，但调查显示，即使新西兰人不同意决定，他们也相信这一流程。

Transparency doesn't prevent controversy—it makes controversy productive.

您是一名卫生部官员，决定如何传达药物资助决定。

Path A: Keep decisions confidential Simply announce "approved" or "not approved" without explanation. Avoid public scrutiny.
结果：没有立即受到批评，但阴谋论盛行。当否认影响到有同情心的病人时，反弹是毁灭性的——而你没有防御。
Path B: Publish full reasoning for every decision 显示成本效益数据、预算影响、考虑的权衡。
结果：最初的批评是激烈的。但随着时间的推移，公众学会了利用证据。即使是那些不同意的人也会尊重这一过程。

透明度不是弱点，而是合法性的盔甲。

Decision Tree: The Transparency Ladder

Level 0: Nothing published (current state for 95% of Oman)
Level 1 (Years 1-5): Recommendations published
Level 2 (Years 3-5): Clear timelines established
Level 3 (Years 6-10): Full critical appraisal reports published

Oman Vision 2040

The Promise
“建立一个促进正义和提供高质量服务的透明医疗保健系统......以维持可持续和持续的卫生资助......以培养科学研究和健康方面的领导力。 ”

HTA is how Oman will keep this promise.

Transparency protects the public trust.

Final Assessment

您已经了解了七项原则。

七项原则

1. Every resource spent is a choice made.

2. Value must be measured, not assumed.

3。比较器定义问题。

4. A QALY is a year of life, lived well.

5. Uncertainty must be explored, not hidden.

6。可负担性与价值不同。

7. Transparency protects the public trust.

Final Quiz (1/10)

1. What is Oman's baseline cost-effectiveness threshold?

Final Quiz (2/10)

2. What discount rate does Oman require for economic evaluations?

Final Quiz (3/10)

3. 预算影响分析项目持续了多少年？

Final Quiz (4/10)

4. Which utility instrument is preferred for measuring health-related quality of life?

Final Quiz (5/10)

5. What type of sensitivity analysis is MANDATORY in Oman?

Final Quiz (6/10)

6. For orphan diseases, what threshold multiplier applies?

Final Quiz (7/10)

7. 基本情况分析必须采用哪种观点？

Final Quiz (8/10)

8. When a new therapy is MORE effective AND LESS costly, it is called:

Final Quiz (9/10)

9. 如果有临床证据，应首先使用什么来源？

Final Quiz (10/10)

10. ICER 公式为：

您已经完成了旅程。

用智慧进行评估。

Based on the Oman HTA Guidelines, First Edition 2024
Ministry of Health, Sultanate of Oman

注意：案例研究是基于阿曼 HTA 方法的说明性示例。