Diagnosis
38What is AL amyloidosis?
Plain-language primer on AL amyloidosis, why early diagnosis matters so much, and how modern therapy works.
What is hyperemesis gravidarum?
Plain-language primer on hyperemesis gravidarum, why it is more than morning sickness, and what comprehensive care can offer.
What is systemic sclerosis?
Plain-language primer on systemic sclerosis (scleroderma), why it affects multiple organ systems, and what modern therapy can offer.
What is allergic bronchopulmonary aspergillosis?
Plain-language primer on ABPA, why it is different from typical asthma, and how modern therapy works.
What is achondroplasia?
Plain-language primer on achondroplasia, why it is the most common skeletal dysplasia, and what the new therapy options can offer.
What is thyroid cancer?
Plain-language primer on thyroid cancer, why molecular profile now matters, and how modern therapy works.
What is dementia with Lewy bodies?
Plain-language primer on dementia with Lewy bodies, why it is different from Alzheimer's, and how modern care works.
What is retinal vein occlusion?
Plain-language primer on retinal vein occlusion, why it is a vascular event, and what modern therapy can offer.
Autism spectrum disorder care explained
Plain-language primer on ASD care, why it is multidisciplinary, and what is changing in pharmacotherapy.
What is ANCA-associated vasculitis?
Plain-language primer on ANCA-associated vasculitis, why it can affect multiple organs, and how modern therapy works.
What is cholangiocarcinoma?
Plain-language primer on cholangiocarcinoma, why molecular profiling now matters so much, and how modern therapy works.
What is overactive bladder?
Plain-language primer on overactive bladder, why it is common, and what the modern therapy options can offer.
What is chronic inflammatory demyelinating polyneuropathy?
Plain-language primer on CIDP, why immune therapy is the foundation, and what the modern options can offer.
What is phenylketonuria?
Plain-language primer on phenylketonuria, why early diagnosis matters so much, and what the modern therapy options can offer.
What is allergic conjunctivitis?
Plain-language primer on allergic conjunctivitis, the different types, and what the modern range of therapy can offer.
Severe COPD and biologic therapy explained
Plain-language primer on severe COPD, why biologic therapy is now an option for some patients, and what the modern combined approach can offer.
What is lupus nephritis?
Plain-language primer on lupus nephritis, why kidney involvement is a turning point in lupus, and how modern therapy works.
What is spinal muscular atrophy?
Plain-language primer on SMA, why genetics drives the disease, and how the modern therapy options work.
What is glioblastoma?
Plain-language primer on glioblastoma, why it has been so hard to treat, and what is changing in glioma therapy.
What is obstructive sleep apnea?
Plain-language primer on obstructive sleep apnea, why CPAP has been the standard, and what is changing in OSA therapy.
What is spasticity?
Plain-language primer on spasticity, why it happens after neurological injury, and what the therapy options can offer.
What is heart failure?
Plain-language primer on heart failure, why the modern therapy is built around four foundational classes, and what is changing.
Acute ischemic stroke explained
Plain-language primer on acute ischemic stroke, why time matters, and how modern therapy works.
What is hepatocellular carcinoma?
Plain-language primer on hepatocellular carcinoma, why it usually develops on a background of liver disease, and how the modern therapy options compare.
What is type 1 diabetes?
Plain-language primer on type 1 diabetes, what is changing, and how modern therapy works.
What is obsessive-compulsive disorder?
Plain-language primer on OCD, why it has multiple subtypes, and what therapy can offer.
What is thyroid eye disease?
Plain-language primer on thyroid eye disease, why it is linked to thyroid problems, and what modern therapy can offer.
What is Sjogren disease?
Plain-language primer on Sjogren disease, why it has been hard to treat, and what is changing.
What is hereditary angioedema?
Plain-language primer on hereditary angioedema, why it is different from allergy, and how modern therapy works.
Postmenopausal osteoporosis explained
Plain-language primer on postmenopausal osteoporosis, why bone changes after menopause, and how modern therapy works.
What is refractory chronic cough?
Plain-language primer on refractory chronic cough, why it is a distinct condition, and what is changing in therapy.
Preeclampsia screening: where biomarker-based pathways are converting
Preeclampsia screening has moved from clinical-risk-factor-driven to biomarker-augmented pathways in several markets. The methodology, the access frame, and the implications for both maternal outcomes and commercial planning around adjacent assets are worth understanding.
What are CSF biomarkers, and how do they fit alongside plasma and PET?
CSF biomarkers - measured in cerebrospinal fluid obtained by lumbar puncture - are well-validated tests for Alzheimer's pathology that pre-date both plasma biomarkers and broad amyloid PET access. They remain in routine clinical use, particularly where PET is not available and where the diagnostic question is complex enough to warrant the procedural friction.
What is tau PET, and how is it different from amyloid PET?
Tau PET is a brain scan that shows the build-up of tau, the second protein associated with Alzheimer's disease. The image is closer to the clinical picture than amyloid is - tau accumulation tracks more directly with where and how a person is currently impaired. The trade-off is that tau PET is harder to access than amyloid PET, and the access gap is now a rate-limiter on multiple fronts.
What is mild cognitive impairment, and how is it different from "early Alzheimer's"?
Mild cognitive impairment (MCI) is a diagnostic category, not a disease. It describes cognitive decline that is more than expected for age but not severe enough to count as dementia. When that decline is caused by underlying Alzheimer's pathology, the term you will increasingly hear in clinical-trial and treatment settings is "early Alzheimer's disease" - which means MCI due to Alzheimer's plus mild dementia due to Alzheimer's, taken together.
What is co-pathology in dementia, and why does it matter?
Co-pathology means more than one disease process is contributing to someone's symptoms at the same time. In older patients with cognitive symptoms, mixed pathology is the rule, not the exception.
What is amyloid PET, and when is it used?
Amyloid PET is a brain scan that shows whether the amyloid protein associated with Alzheimer's disease has built up in the brain. It is used to confirm or rule out Alzheimer's pathology when the diagnosis matters.
What are plasma biomarkers in Alzheimer's disease?
Plasma biomarkers are blood tests that look for proteins associated with Alzheimer's disease pathology. The most clinically useful one in 2026 is plasma p-tau217.
Biomarkers
3ctDNA-guided treatment decisions are entering routine oncology care
Circulating tumour DNA testing has moved from research-grade tool to a routine decision aid in several oncology indications. The pathways from blood draw to clinical decision are now described well enough to plan around, even though reimbursement remains uneven.
Why 'tumor-agnostic' approvals are reshaping reimbursement reviews
When a drug is approved for a molecular target across tumor types rather than for one cancer, the HTA review framework has to evaluate efficacy across heterogeneous indications with different unmet needs, comparators, and standard-of-care benchmarks. That has practical consequences for access timelines.
COPD's biomarker problem and how the field is solving it
COPD has, until recently, been managed as a single condition with a stepwise pharmacological ladder. The arrival of biologics in COPD - and the subgroup-driven trial designs that supported their approval - has forced the field to confront a biomarker question that asthma resolved a decade ago: which patient, which mechanism, which therapy, and how do we know.
Treatment
17What is treatment-resistant schizophrenia?
Plain-language primer on treatment-resistant schizophrenia, why clozapine matters, and what comprehensive care looks like.
PCSK9 inhibitors and modern lipid therapy explained
Plain-language primer on PCSK9 and the modern range of lipid-lowering options.
What is tobacco use disorder?
Plain-language primer on tobacco use disorder, why quitting is difficult, and what the modern combined approach can offer.
What are vasomotor symptoms?
Plain-language primer on hot flashes and night sweats, why they happen, and what the modern therapy options can offer.
What is presbyopia?
Plain-language primer on presbyopia, why it affects nearly everyone with age, and what the modern range of options can offer.
How obesity coverage frames are diverging between cardiovascular-prevention and obesity-only indications
GLP-1 obesity therapy coverage is bifurcating across markets between cardiovascular-prevention indication framing and obesity-only indication framing. The differences in coverage breadth, prior-authorisation criteria, and prescriber pathway are material and are reshaping commercial planning across the class.
How contraception innovation is being reshaped by the long-acting reversible class
Contraceptive innovation has shifted from oral and short-acting options toward long-acting reversible contraception (LARC) including hormonal IUDs, copper IUDs, and contraceptive implants. The commercial logic, the access frame, and the implications for the broader reproductive health pipeline are worth understanding.
How pediatric mental health prescribing differs from adult and why it matters
Pediatric mental health prescribing operates under different evidence frameworks, regulatory expectations, and access structures than adult prescribing. Understanding the differences is essential for any sponsor with mental health assets that are eligible for paediatric expansion or that interact with paediatric-eligible populations.
How combination regimens are restructuring late-line oncology trial design
Late-line oncology trials are increasingly testing combination regimens rather than single-agent comparisons. The trial-design conventions, the regulatory framework, and the commercial implications of combination-as-standard are different enough from single-agent design that cross-functional teams need to understand the shift.
Why HFpEF is the chapter cardiology has been waiting to write
Heart failure with preserved ejection fraction was a diagnostic and therapeutic puzzle for thirty years. The arrival of SGLT2 inhibitors with HFpEF outcome benefit, combined with a more nuanced phenotype framework and the GLP-1 obesity-HFpEF subgroup data, has turned a disease without a treatment into a disease with a small but growing set of evidence-based options.
What are GLP-1 receptor agonists, and why are they being tested in Alzheimer's?
GLP-1 receptor agonists are a class of drugs originally developed for type 2 diabetes and now widely used for obesity. The class is now in late-stage Alzheimer's trials. The mechanistic case spans metabolic, vascular, inflammatory, and direct neuronal pathways - and the access shape would be very different from anti-amyloid therapy.
What is a subcutaneous anti-amyloid antibody, and why does it matter for access?
Subcutaneous formulations of the anti-amyloid antibodies are reformulations that allow the same active drug to be given as a small under-the-skin injection rather than an intravenous infusion. The biology is the same. The delivery is dramatically simpler. The access implications are real but partial - subcutaneous administration removes the infusion-chair constraint, but does not change the MRI surveillance requirement.
What is iADRS, and how is it different from CDR-SB?
iADRS - the Integrated Alzheimer's Disease Rating Scale - is a composite endpoint that combines a cognitive score (ADAS-Cog) with a functional score (ADCS-iADL) into a single number. It is the primary endpoint donanemab used in its pivotal trial and is reported alongside CDR-SB in many late-stage Alzheimer's programs. It answers a slightly different question than CDR-SB does.
What is CDR-SB, and what does a small change on it actually mean?
CDR-SB is the Clinical Dementia Rating - Sum of Boxes, the cognitive and functional scale used as the primary endpoint in most late-stage Alzheimer's trials. It is a six-box, 0–18 scale, scored by a clinician from a structured interview with the patient and a caregiver.
What is Medicare coverage with evidence development?
Coverage with evidence development (CED) is a Medicare coverage mechanism that pays for a treatment on the condition that clinical data about its use is collected and reported back to CMS. It is how Medicare currently covers anti-amyloid antibodies.
What is ALZ-NET, and what does it do with patient data?
ALZ-NET is the Alzheimer's Network for Treatment and Diagnostics - the main patient registry collecting real-world data on people receiving anti-amyloid therapy in the US. It is the registry that Medicare's coverage-with-evidence-development framework routes patients through.
What are anti-amyloid antibodies, and how do they work?
A plain-language explanation of the disease-modifying drug class that defines the current Alzheimer's treatment landscape.
Safety
2What ARIA is, and why it gates how anti-amyloid antibodies can be used
ARIA - amyloid-related imaging abnormalities - is the side effect that defines the operational and clinical experience of being on lecanemab or donanemab. Understanding what it is, who it affects more, and why it requires MRI surveillance is essential context for any conversation about anti-amyloid treatment.
What is ARIA, and why does it matter for treatment?
Amyloid-related imaging abnormalities are the defining safety consideration of the anti-amyloid antibody class - and the reason MRI surveillance is built into treatment.
Genetics
1Pipeline
4How chronic cough has emerged as a discrete indication with its own pipeline
Chronic cough was historically managed within the broader respiratory and ENT framework as a symptom rather than as an indication. The emergence of P2X3 receptor antagonists and adjacent novel mechanisms has established chronic cough as a discrete therapeutic indication with a distinct pipeline, regulatory pathway and commercial logic.
How inherited retinal dystrophy gene therapy is moving beyond RPE65
The voretigene neparvovec approval for RPE65-mediated inherited retinal dystrophy validated AAV-based gene therapy in ophthalmology. The pipeline has moved substantially beyond RPE65 to address adjacent inherited retinal dystrophy genotypes, with implications for diagnostic infrastructure, surgical delivery, and commercial planning.
Why 'women's health' is being rebuilt from a neglected indication frame to a commercial category
For most of the modern pharmaceutical era, 'women's health' meant contraception and hormone-replacement therapy. The conditions outside that frame - endometriosis, polycystic ovary syndrome, menopause symptoms, female-specific cardiovascular and metabolic conditions - were under-funded relative to disease burden. That is changing structurally, and the commercial implications are larger than any single therapy.
What is the FDA accelerated approval pathway, and why does it matter for Alzheimer's?
Accelerated approval is a 1992 FDA regulatory pathway that lets a drug come to market based on a surrogate endpoint reasonably likely to predict clinical benefit, with a confirmatory trial obligated to follow. In Alzheimer's, the pathway is closely associated with the aducanumab episode and a recalibrated bar for what surrogate evidence the agency now considers persuasive.
Patient journey
1Drug development
2How natural history studies are being co-designed with sponsors
Natural history studies in rare disease are increasingly being designed jointly between patient organisations, academic networks, and sponsors with assets in development. The co-design model has implications for evidence quality, regulatory acceptability, and the commercial use of the resulting data.
How to read a modern phase 3 oncology readout: hazard ratios, crossover and what regulators care about
Phase 3 oncology trial readouts have a vocabulary, a set of conventions, and a regulatory frame that the headline numbers obscure. This is a plain-language guide to the parts of the readout that decide approval, label and reimbursement.
Market access
1Delivery
1Regulatory
2How orphan drug designation reshapes commercial planning across markets
Orphan drug designation is more than a marketing-exclusivity tag. It carries fee waivers, accelerated review pathways, market-exclusivity protection, and HTA-layer implications that shape commercial planning materially across markets.
How natural-history studies became regulatory currency for rare-disease approvals
In conditions where a randomized controlled trial is impractical or unethical, well-conducted natural-history studies have become the comparator of record for FDA submissions. The methodology, the patient-advocate organizations that often run these studies, and the regulator-sponsor dialogue have all matured into a recognizable framework.