Funding Gene Therapy for Rare Diseases

The question

How to approach this two-part question question

A two-part question asks two direct questions. Answer both fully, one in each body paragraph.

1. Paraphrase the topic and say how you will answer both questions.
2. Body 1 answers question one with reasons and an example.
3. Body 2 answers question two with reasons and an example.
4. Conclude with a short summary of both answers.

How to plan this answer

Structure

1. Introduction: paraphrase the high cost of gene therapies for rare inherited conditions and signal that you will answer both parts of the question separately.
2. Body 1: the first part, explaining why these treatments cost so much, such as complex development, small patient groups and individual manufacturing, with an example.
3. Body 2: the second part, first suggestion for governments, such as outcomes-based payments linked to how well patients respond, with its mechanism and one limitation.
4. Body 3: the second part, a second suggestion, such as pooled national purchasing or public investment in manufacturing, with its likely effects and costs.
5. Conclusion: a short summary of your recommendation for governments.

Idea bank

• Small markets: when only a few patients need a treatment, the development cost must be recovered from a small number of buyers.
• Manufacturing: some gene therapies are made for each patient using complex processes, which raises the cost of every dose.
• Price example: one gene therapy for spinal muscular atrophy launched in the United States at a list price of more than two million dollars.
• Outcomes-based payments: a government pays in instalments depending on whether a patient responds, which spreads the cost and shifts risk to the manufacturer.
• Pooled purchasing: several countries or regions negotiating together could strengthen their position and secure lower prices.
• Public investment: funding non-profit manufacturing or public research could reduce dependence on high-priced private suppliers, although it requires taxpayer money.

Useful vocabulary

• gene therapy: Gene therapy aims to correct the faulty gene that causes an inherited condition.
• an inherited condition: Some rare conditions are caused by an inherited mutation.
• to recoup development costs: Companies must recoup development costs from a small number of patients.
• a one-off treatment: A one-off treatment can be far more expensive than years of standard care.
• an outcomes-based agreement: An outcomes-based agreement ties payment to proven results.
• to negotiate a price: Governments can negotiate a price with manufacturers in bulk.
• a bespoke manufacturing process: The therapy requires a bespoke manufacturing process for each patient.
• to be unaffordable for: The treatment is unaffordable for most health systems without negotiation.
• to pool resources: Countries could pool resources to buy treatments collectively.
• to shoulder the cost: Governments must decide who should shoulder the cost.

Common mistakes

• Treating the high price as only the result of greed, without considering development, small markets and manufacturing.
• Answering the second part with a vague suggestion such as 'governments should spend more', without a mechanism.
• Blending the two parts into one paragraph, when the task asks for each part to be explained separately.
• Describing gene therapy inaccurately, for example by suggesting it cures every genetic disease.
• Ignoring the trade-off that spending on rare diseases may limit funding for more common conditions.

Examiner tip: Keep the two parts clearly separate, and for the second part name one mechanism, such as payment linked to results, and explain how it would protect taxpayers.