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Most medicines that enter human trials never reach patients, and the diseases with the smallest markets attract the least investment. This page shows how research gets from lab to patient, where government, industry and charity money each goes, what changed recently for sickle cell disease and dementia, how to judge a research charity, and the medical research causes you can support with crypto.

By Global Relief Group · Last updated:

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Medical research is slow, expensive and mostly unsuccessful, which is exactly why where the money goes matters. Governments and drug companies spend far more than charities ever will. Charity money counts in the places they leave thin: diseases too rare to be profitable, cancers in children, infections that mainly affect poor countries, and the unglamorous work of registries, trial networks and getting proven treatments used.

From lab to patient: how few medicines make it

Start human trials (Phase I)about 2.3 years
100
Reach Phase IIabout 3.6 years
52
Reach Phase IIIabout 3.3 years
15
Submitted for approvalabout 1.3 years
9
Approved
8

Out of every 100 drug programmes that start human trials, about this many reach each stage. Years show the average time spent in each stage. Source: BIO, Informa and QLS, 2011–2020 data.

An industry analysis of more than 9,000 drug programmes from 2011 to 2020 found that only 7.9% of those entering human trials were approved, about ten and a half years later on average. Phase II, where a medicine first has to show it actually works, has the lowest success rate of any stage: fewer than three in ten move on. One encouraging exception: medicines for rare diseases succeeded much more often than average, 17.0% against 5.9% for common chronic conditions. Once a rare-disease treatment reaches trials, its chances are relatively good; the hard part is getting it that far.

Can a small donation make a difference?

Not to the total. The US National Cancer Institute alone had a budget of about US$7.2 billion in 2024. But charity money is large in some countries, and it is concentrated where other funders are thin. UK medical research charities that belong to the Association of Medical Research Charities spent about £1.6 billion on research in the UK in 2024, funding more than 1,200 research posts.

Gifts tend to go furthest when they are pooled by a charity that chooses projects through independent expert review, and when they pay for things that unlock more research: pilot studies that win bigger grants, patient registries, biobanks and trial coordinators. Charities often say small grants lead to larger ones; that is plausible, but it has rarely been measured rigorously.

Who pays for what

Area of researchDrug industryGovernmentsCharities and philanthropy
Common adult diseasesHeavyHeavyModerate
Rare genetic diseasesUneven; depends on market sizeModerateOften decisive early on
Childhood cancersLightModerateSignificant
Neglected tropical diseasesVery lightModerate, and fallingSignificant
Getting proven treatments usedVery lightLight to moderateSignificant
An editorial summary of where money tends to concentrate, not a measured breakdown.

Why rare and childhood diseases fall behind

Rare diseases are rare one by one but common together. A 2020 analysis of the Orphanet database counted 6,172 distinct rare diseases affecting an estimated 263 to 446 million people worldwide; about seven in ten are genetic, and about seven in ten begin in childhood. Each one is too small a market to attract much private investment, which is why patient charities often fund the natural-history studies, registries and early trials that make later drug development possible. The Genetic Disease Research Fund supports trial capacity and evidence for Duchenne muscular dystrophy.

Childhood cancer is a similar story with a twist. You may have read that it gets “less than 4%” of the National Cancer Institute’s budget. That figure is out of date: the National Cancer Institute’s spending on childhood cancer was about 8% of its total in 2022, according to NIH data compiled by St. Baldrick’s Foundation. The bigger gap is in drug development, because few medicines are designed for children’s tumours. The Pediatric Cancer Research Fund funds early-stage clinical trials for childhood cancers.

Diseases markets ignore

Neglected tropical diseases are a group of infections, such as sleeping sickness, Chagas disease and leishmaniasis, that mainly affect the poorest people, so there is no paying market for new treatments. Progress is real: WHO reported in 2025 that 1.495 billion people needed interventions against these diseases in 2023, 32% fewer than in 2010. But official aid for them fell by 41% between 2018 and 2023. The non-profit Drugs for Neglected Diseases initiative, which works with companies, universities and governments rather than running its own labs, has delivered 17 treatments for six diseases since 2003. The Tropical Disease Research Coalition supports its work in Latin America.

Sickle cell disease: a hidden burden

A 2023 analysis in The Lancet Haematology estimated that 7.74 million people were living with sickle cell disease in 2021, with numbers rising fastest in western and central sub-Saharan Africa and the Caribbean. Only 34,400 deaths were recorded as caused by sickle cell disease, but the total number of deaths attributable to it was about 376,000, nearly eleven times higher, because children with the disease often die of infections, malaria or anaemia recorded under other causes.

Cheap, proven tools exist. A trial in four African countries, published in the New England Journal of Medicine in 2019, found that the medicine hydroxyurea was safe and roughly halved complications such as pain crises and malaria in children, compared with before treatment. The gap is detection and delivery: screening newborns, starting treatment early and keeping families in care. The Sickle Cell Research Fund works on affordable screening and treatment in Nigeria.

Dementia: what the new drugs changed

For the first time, medicines have been shown to slow decline in early Alzheimer’s disease. In their main trials, lecanemab and donanemab slowed decline on a standard rating scale by roughly a quarter to a third over about 18 months. The effect is modest, the drugs require regular brain scans to watch for swelling and bleeding, and access varies by country: England’s health technology body, NICE, recommended against routine NHS use in 2025 on cost grounds; after the manufacturers won appeals in early 2026 the decision is being reconsidered, and no new decision had been published as of mid-2026. One result is that early diagnosis and groups of people ready to join trials have become much more valuable, which is the work behind the Alzheimer’s Research Fund.

Jargon decoder

  • Phase I, II, III: the three main stages of human trials. Phase I tests safety in small groups, Phase II whether the treatment works, Phase III whether it works, compared with placebo or existing care, in large groups.
  • Orphan drug: a medicine for a rare disease, given incentives such as extended market exclusivity in the US and EU.
  • Natural history study: research that follows how a disease progresses without a new treatment, needed before trials can show a treatment helps.
  • Registry and biobank: organized collections of patient information and samples that let researchers find participants and study a disease.
  • Peer review: independent experts assess a grant application before money is awarded.
  • Health technology assessment: the review, such as NICE in England, that decides whether a health system will pay for an approved treatment.
  • Product development partnership: a non-profit that brings companies, universities and governments together to develop medicines with no commercial market.
  • Implementation research: research on how to get treatments that already work to the people who need them.

How to judge a research charity

Ask how it chooses what to fund. The Association of Medical Research Charities sets out principles for this: proposals reviewed by independent experts, reviewers who are not staff or trustees, a published conflicts-of-interest policy, and a published process. Beyond that, good research charities publish what they fund and what came of it, expect results to be published openly, disclose any industry funding, and name their scientific advisers. Be cautious of appeals that promise a “cure” from a single gift.

Every Medical Research cause we list

Each cause names the organization that receives the grants and when our team last verified it. Open a cause to see what it pays for, or donate directly.

Common questions

Will my gift fund a specific experiment?

Gifts are passed to the partner organization as a grant for that cause. The partner then allocates it within that cause through its own process; for research charities this is usually independent expert review.

Why do some research causes fund screening or registries rather than laboratories?

Because research does not end at a discovery. Patient registries, trial networks and studies on getting treatments used are often what stands between a proven medicine and the people who need it.

Is medical research the same as health care?

No. For causes that pay for treatment and care now, see Health & Medical Aid.

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