Es mostren les entrades ordenades per rellevància per a la consulta cancer. Ordena per data Mostra totes les entrades
Es mostren les entrades ordenades per rellevància per a la consulta cancer. Ordena per data Mostra totes les entrades

15 de juny 2017

Is there any justification for interventions that aren't cost-effective?

Ethics, priorities and cancer

This is one of the most challenging questions nowadays. Anthony Culyer sheds light n this difficult issue in a recent article applied for cancer care. These are his nine  arguments:
Argument 1: the whole health maximisation assumption underlying the approach is misconceived. health care is about more than just promoting health. Other objectives commonly include financial protection (e.g. from the out-of-pocket expenseof costly interventions), innovation, and all those listed earlier
Argument 2: innovation is stifled by the strict application acost-effectiveness threshold that is too low
Argument 3: the use of standard outcome measures, like theEQ-5D QALY or averted DALYs, underestimates the health benefits of cancer treatments
Argument 4: the assessment of benefit excludes the beneficial effects that treatment and its consequences have on those who care for cancer patients
Argument 5: the opportunity cost argument is weak. There are always efficiency savings that can be found in any systemwhich mean that the alleged sacrifice of health represented by the threshold is spurious. the actual sacrifice is much smaller
Argument 6: cancer is a scary disease and people who suffer from it deserve to have access to treatments that would fail aconventional cost-effectiveness test
Argument 7: for some cancer patients a costly and not very effective treatment may offer a “last chance” to someone in despair. such a situation might exist if no intervention of any kind existed for these patients or if the patient suffered from a rare form of cancer
Argument 8: cancer is a “severe” disease and should accordingly be given a higher priority than less severe diseases
Argument 9: many cancer patients have a short life expectancy even with treatment. a quasi-utilitarian argument might cite the law of diminishing marginal value: even small gains for such people are to be valued more highly than the same gains of equivalent quality of life for people with an already long expectation of life. alternatively, there is the more direct emotional appeal “Our moral response to the imminence of death demands that we rescue the doomed proof"
These arguments fall into two broad groups. Some are questionsof social value: how should we value health gains of particular kindsand should we value them differently according as they accrueto different people? Others are questions of fact: would informa-tion about the quantitative size of the effects in question lead us to conclude that cancer is indeed a special case? The burden of proof in both cases lies with those making the assertion that cancer is, indeed, special. That burden of proof is not impossible to bear.
Is cancer a special case? The question may apply to many diseases and will provide more difficulties than answers. In the end any analysis relies on distributive justice principles and according to different views you'l apply different prioritisation criteria.

PS. The article was published in a cancer journal. I was surprised by the new perspective by Tony Culyer.

PS. What do you think about a new cancer inmunotherapy service that may cost $750.000???




Le Corbusier Guitariste (1960)

02 de maig 2024

El cost d'oportunitat dels preus desmesurats dels medicaments pel càncer (2)

 Cancer medicines: a private vice for public benefit?

El que costa un medicament pel càncer:

Over 23 years of cancer medicine R&D the mean cost in USD of developing a cancer medicine to pharmaceutical companies has been around $4.4 billion. But this hides a huge range of costs from ‘just’ 276 million USD for Dinutuximab for post consolidation therapy for childhood high risk neuroblastoma to 13.4 and 15.8 billion USD, respectively, for Durvalumab (used to treat certain types of bladder, lung, and biliary tract cancer) and Isatuximab (used in treatment of blood cancer multiple myeloma).

I el retorn de la inversió (ROI):

 Overall, ROI for cancer medicines with sufficient maturity i.e., launched between 1997-2015 is between 435 to 551%. Again, this hides huge variation. A substantial number of cancer medicines to date have negative or flat ROI’s as low as minus 78-87% in some cases. However, some cancer medicines launched in late 1990’s to mid 2000 have generated astronomical ROIs; for example, Erlotinib (2794%) (pancreatic and lung cancer), Trastuzumab (3421%) (breast cancer), Rituximab (2523%) (Non Hodgkin’s Lymphoma) and Bevacizumab (3200%) (colon, lung, glioblastoma and renal cell cancers). This reflects the fact the the oncology business model is still driven by blockbusters.

I el missatge:

 The problem thus is that the entire structure and incentive framework governing the biotechnology and pharmaceutical industry are geared towards a specific type of behaviour. Short of completely reforming the entire capital-industrial market, starting in the USA, expecting industry to behave any differently from what it is doing right now is a dead end. It is an ideological and technical cul-de-sac. Arguing about the rights and wrongs of industry profits in oncology misses the point. The system is geared towards profit maximisation that is completely independent of R&D costs, it is insensitive to whether the drugs deliver meaningful therapeutic benefit, or whether the cancer medicines are priced ‘fairly’ for any given country.

Les opcions:

One is faced with two choices. Accept that progress is a private vice with public benefit. Essentially align with Bernard Mandeville’s position in Fable of the Bees (1705) that vicious greed, properly channelled by skilful politics, will lead to invisible co-operation and public benefit. Espousing any higher virtue is mere hypocrisy. In this world the only challenge is external. If, for example, China was to exercise its considerable biopharmaceutical muscle in oncology to massively undercut global prices. The other choice falls more in line with the Rawlsian idea of social justice. In this world a new social contract is constructed that truly reflects equitable value. Respective institutions all align along this common public good backbone. Prices truly reflect clinical benefit and are set to a fair level that maximises patient access. R&D is incentivised on societal worth and not profit.

Article per guardar i per reflexionar. El cost d'oportunitat dels medicaments pel càncer és precisament tot aquest valor social que som incapaços de capturar i que deixem de dedicar a altres usos més valuosos.


World Press Photo 2024


 

 

15 de gener 2021

Precision medicine

 Precision Medicine for Investigators, Practitioners and Providers

Many topics under the same umbrella:

Table of Contents

Introduction

2. Role of genomics in precision medicine

3. High throughput omics in the precision medicine ecosystem

4. Infant gut microbiome

5. Paraprebiotics

6. Fecal transplantation in autoimmune disease

7. Drug pharmacomicrobiomics

8. CRISPR technology for genome editing

9. Engineering microbial living therapeutics

10. Organ on a chip

11. Multicellular in-vitro organ systems

12. The role of biobanks in biomarker development

13. Translational interest of immune profiling

14. Organoid pharmacotyping

15. Large datasets for genomic investigation

16. Modern applications of neurogenetics

17. Genomic profiling in cancer

18. Genomics in pediatrics

19. Genomics of gastric cancer

20.  Genomics of prostate cancer

21. MicroRNAs and inflammation markers in obesity

22. MiRNA sequencing for myocardial infarction screening

23. Cell free DNA in hepatocellular carcinoma

24. Non coding RNA in cancer

25. Germline variants and childhood cancer

26. Pharmacogenomics in cancer

27. Proteomic biomarkers in vireoretinal disease

28. Proteomics in respiratory diseases

29. Cardiovascular proteomics

30. Host genetics, microbiome, and inflammatory bowel disease

31. Sampling, Analyzing, and Integrating Microbiome ‘omics Data in a Translational Clinical Setting

32. Omics and microbiome in sepsis

33. Molecular and omics methods for invasive candidiasis

34. Lipid metabolism in colorectal cancer

35. Salivary volatolome in breast cancer

36. immunodiagnosis in leprosy

37. decision support systems in breast cancer

38. Electronic medical records and diabetes phenotyping

39. Clinical signature of suicide risk

40. Machine learning and cluster analysis in critical care

41. Artificial intelligence in gastroenterology

42. Algorithms for epileptic seizure prediction

43. Precision medicine in ophthalmology

44. Phenotyping COPD

45. Lifestyle medicine

46. Precision medicine for a healthier world

47. Aging and clustering of functional brain networks

48. Nutrigenetics

49. Genome editing in reproductive medicine

50. MRI guided prostate biopsy

51. Precision Nutrition

52. Theranostics in precision oncology

53. Precision medicine in daily practice

54. Imaging in precision medicine

55. Organoid for drug screening

56. Printing of personalized medication using binder jetting 3D printer

57. 3 D printing in orthopedic trauma

58. Consumer genetic testing tools in depression

59. The future of wearables

60. Tumor heterogeneity and drug development

61. Smartphone based clinical diagnosis

62. Smartphone biosensing for point of care use

63. Data security and patient protection

64. Blockchain solutions for healthcare

65. Ethical questions in gene therapy

66. Pitfalls of organ on a chip technologies

67. Regulatory issues of artificial intelligence in radiology

68. Academic industrial alliance

69. The future of precision medicine

70. Precision Medicine Glossary

71. Useful internet sites



07 de setembre 2023

El cost d'oportunitat dels preus desmesurats dels medicaments pel càncer

 The cancer premium – explaining differences in prices for cancer vs non-cancer drugs with efficacy and epidemiological endpoints in the US, Germany, and Switzerland: a cross sectional study

Què està passant amb els preus dels medicaments pel càncer? Per què són tant cars? Per què són molt més cars que els altres medicaments?

Doncs si ho voleu saber cal que consulteu aquest article al Lancet (bon treball d'en Miquel Serra i col.laboradors). La resposta és clara, ajustant per eficàcia i característiques epidemiològiques els medicaments per càncer costen tres vegades més que els que els altres. La conclusió és aquesta: 

Our regression estimates indicated that after adjusting for efficacy and epidemiological characteristics, cancer drugs were on average approximately three times more expensive compared to non-cancer drugs in all three countries, indicating a cancer premium; i.e., treatment prices of cancer drugs were on average USD 74,412 (95% CI [62,810; 86,015]) more expensive in the US compared to non-cancer drugs, USD 37,770 (95% CI [26,175; 49,367]) more expensive in Germany, and USD 32,801 (95% CI [27,048; 38,555]) more expensive in Switzerland.

I la pregunta que resta a l'aire és per què són més cars? Què fa que la societat estigui disposada a pagar aquesta subvenció diferencial? Fins quan haurem d'acceptar-ho? La magnitud del cost d'oportunitat és massa alt com per no respondre ben aviat a la qüestió. 

PS. Ja en vaig parlar del mateix fa dies.


L'habitació d'en Van Gogh a Arles

05 de febrer 2021

Clinical utility of genetic testing for breast cancer

 Breast Cancer Risk Genes — Association Analysis in More than 113,000 Women

Genetic testing for breast cancer susceptibility is widely used, but for many genes, evidence of an association with breast cancer is weak, underlying risk estimates are imprecise, and reliable subtype-specific risk estimates are lacking.

However,

 We found strong evidence of an association with breast cancer risk (Bayesian false-discovery probability, <0.05) for protein-truncating variants in 9 genes, with a P value of less than 0.0001 for 5 genes (ATM, BRCA1, BRCA2, CHEK2, and PALB2) and a P value of less than 0.05 for the other 4 genes (BARD1, RAD51C, RAD51D, and TP53).

  None of the other 25 genes in the panel had a Bayesian false-discovery probability of less than 0.10. Of note, 19 genes had an upper limit of the 95% confidence interval of the odds ratio of less than 2.0, with 2.0 representing a proposed threshold for “pathogenic, moderate risk alleles”9; we therefore conclude that these genes are not informative for the prediction of breast cancer risk. We confirmed that missense variants in BRCA1, BRCA2, and TP53 that would be classified as pathogenic according to clinical guidelines are indeed associated with clinically significant risks. We also found that rare missense variants in CHEK2 overall, as well as variants in specific domains in ATM, are associated with moderate risk.

The summary:

 Variants in 8 genes — BRCA1, BRCA2, PALB2, BARD1, RAD51C, RAD51D, ATM, and CHEK2 — had a significant association with breast cancer risk.

 

25 de novembre 2015

MABS in history of medicine

The Lock and Key of Medicine Monoclonal Antibodies and the Transformation of Healthcare

While reading FT this summer I came across an article quoting a unique book on history of monoclonal antibodies (MABS). Right now there are more than 30 drugs in the market based on hybridoma technology that was created in 1975.
The birth of MABS is explained with full details, how the creators finally didn't patented it and why, the difficulties for research in an unconnected world, etc... An exciting story that is worth reading. Right now, it would be completely different, commercialization of research and medicine has raised considerably.

That a British company spearheaded the first marketing of Mabs, a technology devised in a British laboratory by an émigré Argentinian scientist with his German colleague, highlights the international nature of biotechnology commercialization. Sera- Lab’s venture to sell Mabs took place in the midst of the excitement generated by the founding of Genentech in 1976. The emergence of Genentech, which had been set up
to market recombinant DNA products, galvanized numerous alliances among academics, entrepreneurs, and venture capitalists to launch new companies to commercialize biotechnology. Most of the early enterprises set up in the wake of Genentech’s birth were dedicated to exploiting recombinant DNA for the mass production of natural products such as interferon and insulin for drugs. But the early germination of the modern biotechnology industry did not rest solely on recombinant DNA. By the 1970s a number of pioneering companies were developing Mab products, including Sera- Lab and two startups: Hybritech in San Diego and Centocor in Philadelphia. Entrepreneurs who risked entry into the field had no guarantee of success and were entering totally uncharted
territory. Such individuals faced major fi nancial, personal, professional, and regulatory challenges as well as a great deal of hostility, pessimism, and litigation.

Milstein with Köhler at the time of their receiving the Nobel Prize in 1984 together with Nils Jerne.

Mabs have had their strongest therapeutic impact in the field of cancer. The first Mab to reach the market for cancer was edrecolomab (Panorex), which was granted German regulatory approval in 1995 for the treatment of postoperative colorectal cancer. Developed by Centocor in partnership with the Wistar Institute, it was withdrawn in 2001 because of its poor effi cacy in comparison with other drugs. Since 1997, however, the U.S. Food and Drug Administration (FDA) has approved twelve Mab drugs for cancer treatment, including rituximab (Rituxan), approved in 1998 for the treatment of non- Hodgkin’s lymphoma. By 2012 there were over 160 candidates in clinical trials for cancer, with seventy of them in phase III trials, the stage before a drug is submitted for regulatory approval.

Mabs have enabled the identification and characterization of cancerous tumors previously difficult to detect and diff erentiate from other tumors, thereby providing a better understanding of cancer. They have also opened a path to more personalized medical treatment. Trastuzumab (Herceptin), for example, was specifically developed to target HER2/neu, a protein overexpressed by tumors found in 25 percent of newly diagnosed breast- cancer patients

20 de març 2011

Defenseu-nos dels geneto-entusiastes

Aquesta qüestió està agafant un camí perillós que ens porta de cap al penya-segat. Em refereixo a la medicina predictiva. El diari LV, -sempre el mateix i no troba per ara tema més suggerent- mostra cofoi que neix el primer nen sense un gen que el predisposava al càncer de mama BRCA1. No discutiré la ciència que hi ha al darrera i tampoc el negoci. Evitaré tot comentari ja fet anteriorment sobre genètica i medicina, i l'oblit de l'epigenètica i demés. Evitaré tota reflexió bioètica. Em centraré només en una reflexió tant senzilla com: els pares saben segur que si la prova dona positiva, pot ser que no tingui capacitat predictiva?.
Miro l'acreditada referència labtestonline i diu això:
The degree of risk conferred with a positive result is difficult to quantify for a specific person. Results must be interpreted in conjunction with the tested person's personal and family history. A genetic counselor/trained health care professional should explain the meaning of the results, explain treatment options for the individual that are intended to decrease risk, and testing options for other family members.
A negative result does not mean that a woman will not develop breast or ovarian cancer. It simply indicates that the person tested is not at increased risk for developing hereditary breast cancer or ovarian cancer related to the BRCA mutations for which he/she was tested. It is important to remember that 90-95% of breast cancers are not associated with a BRCA mutation. Furthermore, in the general population, the lifetime risk of developing breast cancer is approximately 12% and the lifetime risk of developing ovarian cancer is about 1.4%. The risks increase with age.
The presence of a BRCA-1 or BRCA-2 mutation means that the person tested is at an increased risk for breast and/or ovarian cancer, but it does not mean that she will ever have them. Even within a family with the same BRCA mutation, not everyone will develop cancer and those that do may develop it at different times during their life.
M'estalvio d'explicar la sensibilitat i especificitat perquè el que diu al prospecte no hi ha referència a cap estudi independent. Els criteris GRIPS sobre predicció de risc genètic tampoc no els trobareu enlloc. També m'estalvio d'explicar quin laboratori està qüestionat als tribunals per la patent sobre aquesta prova perquè ja ho vaig explicar fa dies. Tampoc vull insistir que hi ha competència ferotge en la qüestió. No vull saber el que ens ha costat als catalans. I m'estalvio d'indicar que la publicitat que els estan fent els surt gratuita, perquè és evident i és a la llum de tots nosaltres.
Voldria finalment recordar una vegada més la necessitat d'avaluar l'efectivitat de la prova i el que se'n derivi, no fos cas que s'hagi introduit a la cartera pública sense que algú se n'hagi oblidat de fer-ho. I malauradament sabem que ha estat així i s'ha pagat amb diners de tots, perquè ja s'han encarregat d'explicar-ho al diari i així l'oferta pot crear demanda. En resum, el regulador ha mirat cap a una altra banda i s'ha oblidat de defensar-nos. Una volta i una altra, i fins quan?

PD. I encara més aquí LV i aquí  EP. I aquí m'aturo.


29 d’abril 2012

Sobrediagnòstic

Overdiagnosis of Invasive Breast Cancer Due to Mammography Screening: Results From the Norwegian Screening Program

En tota decisió clínica sabem que hi ha una probabilitat de falsos positius. També sabem que pot haver-hi sobrediagnòstic. El sobrediagnòstic és sovint confós amb el terme fals positiu d'una prova, però són dos conceptes diferents. Un resultat fals positiu d'una prova es refereix a una exploració complementària que suggereix la presència de la malaltia, però es tracta d'un error que es pot corregir amb una repetició de la prova. Als pacients sobrediagnosticats se'ls diu que tenen la malaltia i en general reben tractament. Els cas dels pacients sobrediagnosticats esdevé especialment preocupant. La seva magnitud és desconeguda però hi ha àrees on se sap més que altres.
Així per exemple llegint Annals podem saber que a Noruega i fruit dels programes de cribratge de càncer de mama han sobrediagnosticat entre un 15 i un 25% dels casos. Aquest és el resum:
A total of 39 888 patients with invasive breast cancer were included, 7793 of whom were diagnosed after the screening program started. The estimated rate of overdiagnosis attributable to the program was 18% to 25% (P < 0.001) for approach 1 and 15% to 20% (P < 0.001) for approach 2. Thus, 15% to 25% of cases of cancer are overdiagnosed, translating to 6 to 10 women overdiagnosed for every 2500 women invited.
Al llibre Overdiagnosed, quan es parla dels programes de cribratge de cancer de mama, s'assenyala:
If there were no overdiagnosis, then the total number of individuals diagnosed with cancer would be unaffected by screening. A rise in the number of breast cancers following the initiation of screening is expected (some people do have cancers destined to appear later that can be detected by screening),20 but if there is truly no overdiagnosis, that rise will be offset by reductions in the numbers of cancers detected later. In other words, if all cancers detected early through screening were ultimately going to be clinically evident (typically when a woman notices a new breast lump and then seeks medical care to evaluate it), one would expect a subsequent decline in the number of cancers detected clinically later in time. Since the cancers would have been detected and treated in women of screening age, the reduction should become evident as the women age and stop screening (in Europe, generally around ages sixty-five to seventy). Throughout Europe this reduction has largely failed to appear.
I assenyala que el problema del sobrediagnòstic és el sobretractament i cita la revisió Cochrane:
“The evidence review suggests that for every 2000 women invited to screening for 10 years one death from breast cancer will be avoided but that 10 healthy women will be overdiagnosed with cancer. This overdiagnosis is estimated to result in six extra tumorectomies and four extra mastectomies and in 200 women risking significant psychological harm relating to the anxiety triggered by the further investigation of mammographic abnormalities.”
Tema preocupant al que prestem una atenció limitada. A més de l'impacte en salut i benestar, hi ha l'impacte econòmic del qual desconeixem el seu abast a dia d'avui.

PS. Per a Catalunya cal consultar aquest article. L'estimació del sobrediagnòstic pot assolir fins i tot el 46%. Aquests són els resultats:
Incidence of invasive BC increased, especially in cohorts born from 1940 to 1955. The biggest increase was observed in these cohorts between the ages of 50 to 65 years, where the final BC incidence rates more than doubled the initial ones. Dissemination of mammography was significantly associated with BC incidence and overdiagnosis. Our estimates of overdiagnosis ranged from 0.4% to 46.6%, for women born around 1935 and 1950, respectively.

A l'exposició de Chagall el podreu veure fins el 20 de maig, després se l'endurà la duquessa.

21 de maig 2023

L'assaig clínic mai vist per al cribratge del càncer

Li pregunto a Chat GPT sobre l'assaig clínic més gran en nombre de participants i em diu que és ASPREE, sobre l'aspirina i prevenció cardiovascular. I veig que Galleri, l'assaig clínic de GRAIL sobre cribratge de càncer al NHS hi ha 140.000 participants. I jo penso, això és molta gent i el Chat GPT4 no ho ha copsat. 

This is a trial in a large population in England to assess the performance and clinical utility of GRAIL’s multi-cancer early detection test when added to standard of care. Participants are randomized to either a test or control arm. The study teams remain blinded throughout the study. Participants who test positive will be referred for standard of care investigations and treatment in the National Health Service (NHS).

The study has enrolled approximately 140,000 people aged 50 to 77. Unless diagnosed with cancer, participants in both arms are asked to return for annual visits at approximately 12 and 24 months. All participants whether test positive, test negative or not tested will be followed for cancer and associated outcomes via linkage to NHS routine datasets.

Els detalls de l'assaig els trobareu aquí. I si voleu veure la validació, aquí. I mentrestant el regulador segueix pensant si autoritza la prova, resulta que ja es troba al mercat.



Health information, such as whether someone developed cancer and how it was treated, is collected from centrally held NHS records for up to 10 years after people’s first appointment. This allows the researchers to easily track people for whether they get cancer, even for people who may have moved home.

People who are diagnosed with cancer while they are taking part in the trial may not need to attend further trial appointments to give blood samples.

A quina data s'acaba l'assaig clínic no se sap del cert. Després de llegir tot això espero que se sàpiga quina és la sensibilitat i especificitat de la prova segons càncer detectat. Dic això perquè no he sabut veure com s'avaluen els falsos negatius, ni els falsos positius. I en un moment concret diuen que haurem d'esperar 10 anys. No ho sé, potser m'ha passat per alt.

Més detalls, aquí.

Mentrestant la Unió Europea ha prohibit la fusió Illumina-Grail, i ja veurem com acaba. Per ara els mesos passen i res de res...

Galleri, el test detecta (diuen) 50 tipus de càncer amb una gota de sang que cerca ADN circulant per 1.000€ (diuen, diuen).

Més d'un i més de dos volen saber si estem davant d'un nou cas Theranos, (jo vull pensar que no) i per això han començat aquest assaig clínic. A FT del dijous hi ha més detalls. I ho fan a UK perquè a Europa aquesta empresa hauria d'estar prohibida fins que no compleixi les resolucions de les autoritats.



17 de juny 2018

Cost-effectiveness of genome sequencing (3)

Application of next-generation sequencing to improve cancer management: A review of the clinical effectiveness and cost-effectiveness

Once again, there is no need for cost-effectiveness if there is not a clear message on the analytical validity, clinical validity and utility of a diagnostic test.
A new article want to shed light on cancer and NGS, and says:
Our search for cost‐effectiveness studies on NGS in cancer care yielded 2037 articles. Only 6 articles included cost‐effectiveness studies of the application of NGS (targeted gene panel) in cancer

The 6 selected reports could be separated into 2 types. Three of the articles assessed the cost‐effectiveness of recommending patients receiving targeted therapy matching their genetic mutation identified via NGS; and the remaining 3 articles assessed the cost‐effectiveness of using NGS as part of the screening program to direct patients or high risk family members into prophylactic treatment

Two out of 3 articles in the “targeted therapy” group reported that NGS and targeted therapy was not cost-effective (Table 3A), using an ICER threshold of US$100 000 per Quality Adjusted Life
Year (QALY) gained. An ICER of less than US$100 000/QALYs gained is generally considered favourable for funding in the United States

Two out of the 3 articles in the “screening” group reported that the use of NGS was cost‐effective (Table 3B), that is, under US$100 000 per QALY gained.loser surveillance.
 In our evaluation of the effectiveness of NGS, we found that NGS is effective at identifying mutations in cancer patients, and we reported that 37% of the diagnosed patients proceeded to receive therapy matching their genetic profile. However, with only 6 articles available that assess the cost-effectiveness of NGS in various settings, it remains an area for future research to determine whether the technology is cost-effective in routine cancer management
Summary: the message is that there is no message with such a few observations!


Something is being missed...




19 de gener 2012

Reflexionem-hi

High-Value Testing Begins With a Few Simple Questions

Sabem que el valor de les proves diagnòstiques sorgeix de la informació marginal que aporten i la capacitat per modificar les probabilitats de patir una malaltia o el canvi en el seu curs. Per tant, el valor en si mateix és informació, un bé intangible crucial per a decidir el tractament. Tota prova diagnòstica té un cost, ara bé per tal que aquest cost acabi convertint-se en valor, de veritat, podem fer algunes preguntes clau que proposa l'Annals:
Did the patient have this test previously?
If so, what is the indication for repeating it?
Is the result of a repeated test likely to be substantively different from the last result?
If it was done recently elsewhere, can I get the result instead ofrepeating the test?
Will the test result change my care of the patient?
What are the probability and potential adverse consequences of a false positive result?
Is the patient in potential danger over the short term if I do not perform this test?
Am I ordering the test primarily because the patient wants it or to reassure the patient?
If so, have I discussed the above issues with the patient?
Are there other strategies to reassure the patient? 
i el darrer paràgraf clau:
Data suggest that unnecessary testing abounds. The Congressional Budget Office has estimated that up to 5% of the nation’s gross national product is spent on tests and procedures that do not improve patient outcomes (6). The sixth edition of the ACP Ethics Manual specifically calls out responsible stewardship of resources as an ethical responsibility of physicians (7) and has been lauded for doing so (8). Such stewardship requires substantial and persistent effort with some hard decisions along the way. Addressing a few simple questions before ordering a test seems to be a reasonably easy way to start the journey toward high-value care.
A tots aquells que vulguin una retallada selectiva i no pas lineal, tal com alguns hem vingut demanant des de l'inici, el mateix Annals ofereix la llista de proves diagnòstiques en situacions clíniques que aportaran poc valor. Només fa falta llegir-ho i dur-ho a la pràctica. Qui s'hi apunta?

PS. La llista de proves diagnòstiques inacurades
1. Repeating screening ultrasonography for abdominal aortic aneurysm following a negative study
2. Performing coronary angiography in patients with chronic stable angina with well-controlled symptoms on medical therapy or who lack specific high-risk criteria on exercise testing
3. Performing echocardiography in asymptomatic patients with innocent-sounding heart murmurs, most typically grade I–II/VI short systolic, midpeaking murmurs that are audible along the left sternal border
4. Performing routine periodic echocardiography in asymptomatic patients with mild aortic stenosis more frequently than every 3–5 y
5. Routinely repeating echocardiography in asymptomatic patients with mild mitral regurgitation and normal left ventricular size and function
6. Obtaining electrocardiograms to screen for cardiac disease in patients at low to average risk for coronary artery disease
7. Obtaining exercise electrocardiogram for screening in low-risk asymptomatic adults
8. Performing an imaging stress test (echocardiographic or nuclear) as the initial diagnostic test in patients with known or suspected coronary artery disease who are able to exercise and have no resting electrocardiographic abnormalities that may interfere with interpretation of test results
9. Measuring brain natriuretic peptide in the initial evaluation of patients with typical findings of heart failure
10. Annual lipid screening for patients not receiving lipid-lowering drug or diet therapy in the absence of reasons for changing lipid profiles
11. Using MRI rather than mammography as the breast cancer screening test of choice for average-risk women
12. In asymptomatic women with previously treated breast cancer, performing follow-up complete blood counts, blood chemistry studies, tumor marker studies, chest radiography, or imaging studies other than appropriate breast imaging
13. Performing dual-energy x-ray absorptiometry screening for osteoporosis in women younger than 65 y in the absence of risk factors
14. Screening low-risk individuals for hepatitis B virus infection
15. Screening for cervical cancer in low-risk women aged 65 y or older and in women who have had a total hysterectomy (uterus and cervix) for benign disease
16. Screening for colorectal cancer in adults older than 75 y or in adults with a life expectancy of less than 10 y
17. Repeating colonoscopy within 5 y of an index colonoscopy in asymptomatic patients found to have low-risk adenomas
18. Screening for prostate cancer in men older than 75 y or with a life expectancy of less than 10 y
19. Using CA-125 antigen levels to screen women for ovarian cancer in the absence of increased risk
20. Performing imaging studies in patients with nonspecific low back pain
21. Performing preoperative chest radiography in the absence of a clinical suspicion for intrathoracic pathology
22. Ordering routine preoperative laboratory tests, including complete blood count, liver chemistry tests, and metabolic profiles, in otherwise healthy patients undergoing elective surgery
23. Performing preoperative coagulation studies in patients without risk factors or predisposing conditions for bleeding and with a negative history of abnormal bleeding
24. Performing serologic testing for suspected early Lyme disease
25. Performing serologic testing for Lyme disease in patients with chronic nonspecific symptoms and no clinical evidence of disseminated Lyme disease
26. Performing sinus imaging studies for patients with acute rhinosinusitis in the absence of predisposing factors for atypical microbial causes
27. Performing imaging studies in patients with recurrent, classic migraine headache and normal findings on neurologic examination
28. Performing brain imaging studies (CT or MRI) to evaluate simple syncope in patients with normal findings on neurologic examination
29. Routinely performing echocardiography in the evaluation of syncope, unless the history, physical examination, and electrocardiogram do not provide a diagnosis or underlying heart disease is suspected
30. Performing predischarge chest radiography for hospitalized patients with community-acquired pneumonia who are making a satisfactory clinical recovery
31. Obtaining CT scans in a patient with pneumonia that is confirmed by chest radiography in the absence of complicating clinical or radiographic features
32. Performing imaging studies, rather than a high-sensitivity D-dimer measurement, as the initial diagnostic test in patients with low pretest probability of venous thromboembolism
33. Measuring D-dimer rather than performing appropriate diagnostic imaging (extremity ultrasonography, CT angiography, or ventilation–perfusion scintigraphy), in patients with intermediate or high probability of venous thromboembolism
34. Performing follow-up imaging studies for incidentally discovered pulmonary nodules 4 mm in low-risk individuals
35. Monitoring patients with asthma or chronic obstructive pulmonary disease by using full pulmonary function testing that includes lung volumes and diffusing capacity, rather than spirometry alone (or peak expiratory flow rate monitoring in asthma)
36. Performing an antinuclear antibody test in patients with nonspecific symptoms, such as fatigue and myalgia, or in patients with fibromyalgia
37. Screening for chronic obstructive pulmonary disease with spirometry in individuals without respiratory symptoms

PS. Salut i crisi a Grècia. Explicat al blog de Liaropoulos. Algú creu que Grècia pot pagar un interès del 34% del bo a 10 anys? Tothom sap que això és qüestió de dies però mira cap un altre costat.

PS. Sobre el tema de falsificació de medicaments, documents de la OMS

21 de juliol 2017

Beyond health gain:the value of knowing in precision medicine

Value assessment in precision cancer medicine

Towse and Garrison provide a clear picture of the economics of using drugs with companion diagnostics (precision medicine) in the Journal of Cancer Policy. Three main issues arise:
A. Reducing or avoiding the adverse effects associated with treatment (including the medical and nonmedical costs of man-aging them).
B. Reducing or avoiding time delays in selecting the most appropriate intervention
C. Enabling a treatment effective only in a small fraction of the population to be made available or more widely available.
But there are also psychological gains (difficult to estimate) related to the value of knowing:
1. Reduction in uncertainty reflecting the idea that a companion diagnostic, by increasing the certainty of a patient’s response to a medicine—would be more valuable to individual patients and hence they (or their payer) would be willing to pay more for the combination. Furthermore, as noted above, at the population level, greater certainty could lead to greater uptake and improved compliance.
2. The value of hope is the notion that in some circumstancesindividuals become risk-seekers in the sense that they would be willing to pay more for access to a technology with a long tail indicating that some patients have a much longer survival time than current therapy, even though the average life expectancy may be no greater, or even less, than standard therapy.
3. Real option value for which the best example is that if  a treatment can extend life, this opens up possibilities for individ-uals to benefit from future advances in medicine. Hence, they(or their payer) should be willing to pay more than simply theamount they would pay for a gain in life expectancy alone, ascalculated under conventional methods, because it provides the option of benefiting from further treatments.
4. Insurance value is related to the idea that insurance tocover innovations provides peace of mind, not just by protectingagainst catastrophic financial loss but also by protecting fromcatastrophic health loss. The focus is usually only on financial protection, in the form on an Extended Cost-effectiveness Analysis. Lakdawalla et al. point out that greater value comesfrom the reassurance value of knowing of the existence of a treatment, or even of incentives to develop such a treatment.
5. Scientific spillovers arise because the benefit of scientificadvances cannot be entirely appropriated by those making them. Improving knowledge creates opportunities for additional innovation by others. For example, proving that a particular agentworks on a hypothesized pathway in a particular cancer means that the general understanding of that cancer is enhanced and thus further research can explore other pathways in the same cancer. This creates a commons problem with potential underinvestment, implying that patients may wish to reward developerswith higher prices to encourage knowledge generation.
The authors back value based pricing for drugs and companion diagnostics, just at the same time that The New York Times casts some shadows over this option.



Cantut - El Pomeró

28 de gener 2019

In search for a fair price-setting in cancer drugs

Pricing of cancer medicines and its impacts

We all know that new cancer drugs represent a challenge for the whole society. Expectations from drug firms are high and public and private budgets do not increase according to such expectations. A technical report released by WHO sheds light on the issue.
Just one statement:
Overall, the analysis suggests that the costs of R&D and production may bear little or no relationship to how pharmaceutical companies set prices of cancer medicines. Pharmaceutical companies set prices according to their commercial goals, with a focus on extracting the maximum amount that a buyer is willing to pay for a medicine. This pricing approach often makes cancer medicines unaffordable, preventing the full benefit of the medicines from being realized.
You may find here former posts on the same topic.

PS. My comment on genetics in clinical practice in GCS 69, p.96


05 de novembre 2024

La història de la innovació en la lluita contra el càncer

An Ungovernable Foe: Science and Policy Innovation in the U.S. National Cancer Institute

El llibre "An Ungovernable Foe: Science and Policy Innovation in the U.S. National Cancer Institute" de Natalie B. Aviles narra la història del National Cancer Institute (NCI) i la seva lluita contra el càncer des de 1948 fins a 2018, centrant-se en la investigació sobre virus i vacunes contra el càncer.  

La tesi central del llibre és que l'estreta interconnexió entre les pràctiques científiques i burocràtiques al NCI ha permès als seus agents desenvolupar una expertesa política única que ha donat forma a la innovació en la investigació biomèdica i a la seva governança als Estats Units.

Metodologia i estructura

L'autora basa el seu argument en l'anàlisi de fonts històriques que documenten les activitats científiques i burocràtiques dels investigadors del programa intramural del NCI, com ara informes anuals, actes de reunions i entrevistes orals. El llibre segueix un ordre cronològic, dividit en capítols que cobreixen aproximadament una dècada cadascun, narrant les innovacions científiques i burocràtiques que van sorgir dels esforços dels investigadors per complir la doble missió del NCI:

*Donar suport a la ciència biomèdica.

*Millorar la salut pública.

Temes clau

Al llarg del llibre, es destaquen diversos temes clau:

La importància de l'aprenentatge social entornat en la innovació científica. Aviles argumenta que les innovacions al NCI no només són el resultat de descobriments científics, sinó també de la manera com els actors del NCI interpreten els problemes i les oportunitats que s'obren al seu voltant i adapten les seves pràctiques en conseqüència.

La influència dels factors organitzatius i culturals en la innovació. El llibre mostra com l'estructura organitzativa i la cultura del NCI han influït en la trajectòria de la investigació sobre el càncer, tant en termes de descobriments científics com en la formulació de polítiques.

L'evolució de les pràctiques burocràtiques al NCI.  Aviles analitza com el NCI ha anat adaptant les seves pràctiques burocràtiques per respondre als reptes canviants de la investigació sobre el càncer, des de la creació de noves estructures de programa fins a la implementació de noves formes de gestió de contractes.

Alguns exemples destacats

Durant la dècada de 1960, la creació del Virology Research Resources Branch (VRRB) va permetre al NCI centralitzar i coordinar els esforços de recerca sobre virus del càncer, proporcionant recursos i materials a investigadors de tot el país.

A la dècada de 1970, la implementació del National Cancer Program va donar lloc a una important expansió dels fons per a la investigació sobre el càncer, però també va generar controvèrsia sobre les pràctiques de contractació del NCI.

Durant la dècada de 1990, el concepte de "recerca translacional" va guanyar importància, i el NCI va dedicar esforços considerables a fomentar la transferència dels descobriments científics a la pràctica clínica.

Conclusió

"An Ungovernable Foe" ofereix una visió detallada i perspicaç de la història del NCI i el seu paper en la lluita contra el càncer. El llibre és una lectura essencial per a qualsevol persona interessada en la història de la investigació biomèdica, la sociologia de la ciència o la política científica als Estats Units. 




22 de març 2022

Cancer Economics

 Regulatory and Economic Aspects in Oncology

Topics:

Cost of Cancer: Healthcare Expenditures and Economic Impact

Oncology from an HTA and Health Economic Perspective

Heterogeneous Recommendations for Oncology Products Among Different HTA Systems: A Comparative Assessment

Patient-Reported Outcomes in Oncology, Beyond Randomized Controlled Trials

Patient-Reported Outcomes in Health Economic Decision-Making: A Changing Landscape in Oncology

Approaches to Capturing Value in Oncology

Orphan Drugs in Oncology

Recent Developments in Health Economic Modelling of Cancer Therapies

Drug Pricing and Value in Oncology

Regulatory and Evidence Requirements and the Changing Landscape in Regulation for Marketing Authorisation

Prioritization not Rationing in Cancer Care



08 de maig 2018

Cost-effectiveness of genome sequencing (2)

Application of next-generation sequencing to improve cancer management: A review of the clinical effectiveness and cost-effectiveness

If you want to go deeper on the issue, have a look at this article. It is focused on one disease, cancer and tries to combine clinical effectiveness and cost effectiveness. Sounds good. At the end you'll see that the number of available studies is limited (6), but that's the situation and these are the conclusions:

We report the rate of successfully detecting mutations from the clinical studies. The incremental cost-effectiveness ratio and sensitivity analysis outcomes are reported for the cost-effectiveness articles. Fifty-six articles reported that sequencing patient samples using targeted gene panels, and 83% of the successfully sequenced patients harboured at least 1 mutation.
 In our evaluation of the effectiveness of NGS, we found that NGS is effective at identifying mutations in cancer patients, and we reported that 37% of the diagnosed patients proceeded to receive therapy matching their genetic profile. However, with only 6 articles available that assess the cost-effectiveness of NGS in various settings, it remains an area for future research to determine whether the technology is cost-effective in routine cancer management.
PS. Today this blog has surpassed its 200.000 visits. That's great! Thank you for your loyalty.

Sally Mann, On the Maury, 1992, gelatin silver print, Private collection.
Washington National Gallery, current exhibition


22 de novembre 2020

The time to stop recreational testing has come

 Direct-to-Consumer Genetic Testing: Value and Risk

Piecing together information from a variety of sources, one reporter concluded that by early 2019, more than 26 million people worldwide had been tested by the four leading companies, 23andMe, Ancestry, Gene By Gene, and MyHeritage (1). That volume was fueled by aggressive marketing, including discounts in the lead-up to major holidays to promote gifting of test kits. As of May 2020, the  undiscounted price of the basic test offered by the leading companies was $59–$99.

This is an example of what should not had happened. Recreative genomics doesn't add value and increases uncertainty and anxiety. 

Although many consumers of DTCgenetic testing express an intention to modify their lifestyle to address risk factors, studies typically show no changes at follow-up (15, 30). In the PGen Study, 59% of participants said that test results would influence their management of their health (31). However, an analysis of the 762 participants who had complete cancer-related data found that those who received elevated risk estimates were not significantly more likely to change lifestyle or engage in cancer screening than those who received average or below-average risk estimates (44). It may be relevant that no participants tested positive for pathogenic variants in highly penetrant cancer susceptibility genes. As for population health, the Centers for Disease Control and Prevention identify three conditions—hereditary breast and ovarian cancer syndrome,Lynch syndrome, and familial hypercholesterolemia—that are poorly ascertained despite the potential for early detection and intervention to significantly reduce morbidity and mortality (45). The hope is that DTC genetic testing could improve the situation (15). However,DTC genetic testing as currently carried out is likely to fill gaps in haphazard fashion, given the characteristics of purchasers, the scope of available products, and integration issues.

One message. Right now and until we don't know the implications of recreational genetic testing, direct to consumers testing should stop.


Banksy

 

18 de setembre 2016

The anxiety of inaccuracy

Conflicting Interpretation of Genetic Variants and Cancer Risk by Commercial Laboratories as Assessed by the Prospective Registry of Multiplex Testing

What happens if "one quarter of the clinical genetic results from commercially available multiplex cancer panels and reported at the PROMPT registry had conflicting interpretations" and if "36% of conflicting genetic tests results appeared to be clinically relevant, because they were either reported as pathogenic/likely pathogenic"? Does anybody care about it?.
I would suggest today you have a look at this article and your level of anxiety will increase suddenly.
Clinical data and genetic testing results were gathered from1,191 individuals tested for inherited cancer susceptibility and self-enrolled in PROMPT between September 2014 and October 2015. Overall,participants (603 genetic variants) had a result interpreted by more than one laboratory, including at least one submitted to ClinVar, and these were used as the final cohort for the current analysis.

Of the 603 variants, 221 (37%) were classified as a variant of uncertain significance (VUS), 191 (32%) as pathogenic, and 34 (6%) as benign. The interpretation differed among reporting laboratories for 155 (26%). Conflicting interpretations were most frequently reported for CHEK2 and ATM, followed by RAD51C, PALB2, BARD1, NBN, and BRIP1. Among all participants, 56 of 518 (11%) had a variant with conflicting interpretations ranging from pathogenic/likely pathogenic to VUS, a discrepancy that may alter medical management.
Therefore, 
Clinical interpretation of genetic testing for increased cancer susceptibility as assessed by multiplex panels hinges on accurate curation and interpretation of variants. Discrepant interpretation of some genetic variants appears to be common.
Take care. The regulator remains on vacation, a never ending vacation.

PS. On genetic testing 

08 de gener 2024

Els medicaments que venen i els que ja s'han aprovat el 2023

És bo fer una ullada a quins són els medicaments que previsiblement s'aprovaran l'any 2024, i els de Nature diuen que són aquests:

Medicaments per aprovar el 2024

Biologic name

Sponsor

Properties

Indication

Timing

Zolbetuximab

Astellas

Claudin 18.2-targeted mAb

Gastric cancer

January

Lifileucel

Iovance

Tumour-infiltrating lymphocyte therapy

Melanoma

February

Resmetiroma

Madrigal/Synta

Thyroid hormone receptor β agonist

NASH

March

Sotatercepta

Merck & Co./Acceleron

Fusion protein ligand trap for TGF-β superfamily

PAH

March

mRNA-1345a

Moderna

mRNA-based vaccine

RSV prevention

April

Donanemaba

Eli Lilly

Amyloid-β-targeted mAb

Alzheimer disease

Q1

EB-101a

Abeona

Gene therapy with COL7A2 transgene

RDEB

May

Patritumab deruxtecana

Merck & Co.

HER3-targeted ADC

NSCLC

June

Imetelstat

Geron

Telomerase inhibitor

Transfusion-dependent anaemia with MDS

June

Tarlatamaba

Amgen

DLL3 × CD3 T-cell engager antibody

SCLC

June

Fidanacogene elaparvoveca

Pfizer/Spark

AAV-based gene therapy with factor IX transgene

Hemophilia B

Q2

Bentracimaba

Laboratoires SERB

Ticagrelor-neutralizing antibody

Drug toxicity

1H

Crovalimaba

Roche

C5-targeted mAb

PNH

July

Danicopana

AstraZeneca/Alexion

Factor D inhibitor

PNH

July

Midomafetaminea

MAPS

MDMA

PTSD

August

Xanomeline plus trospium

Karuna/BMS

Muscarinic receptor modulators

Schizophrenia

September

Acoramidis

BridgeBio

TTR stabilizer

TTR amyloidosis

December

Marstacimab

Pfizer

TFPI-targeted mAb

Haemophilia A and B

Q4

Afamitresgene autoleucela

Adaptimmune

MAGE-A4-targeted autologous, engineered T cell therapy

Synovial sarcoma

2024


Fig. 1 | 30 years of novel FDA approvals. Annual numbers of new molecular entities (NMEs) and biologics license applications (BLAs) approved by the FDA’s Center for Drug Evaluation and Research (CDER). See Table 1 for new approvals in 2023. Products approved by the Center for Biologics Evaluation and Research (CBER), including vaccines and gene therapies, are not included in this drug count (Table 2). Source: FDA.

Fig. 2 | CDER approvals by therapeutic area. Indications that span multiple therapeutic areas are classified under only one, based on which FDA office and division reviewed the approval application. Sources: Nature Reviews Drug Discovery, FDA.


Fig. 3 | CDER approvals by modality. Small molecules, including peptides of up to 40 amino acids in length, and oligonucleotides are approved as new molecular entities (NMEs). Protein-based candidates are approved through biologics license applications (BLAs). mAb, monoclonal antibody; siRNA, small interfering RNA. Source: Nature Reviews Drug Discovery.

I la notícia de l'any ha estat CRISPR:
Vertex and CRISPR Therapeutics’ exagamglogene autotemcel (exa-cel; Casgevy) especially is the first CRISPR–Cas9-based gene editor to secure a green light from the FDA, winning an approval for sickle cell disease (SCD). Exa-cel is an ex vivo gene-edited cell therapy: blood cells are harvested from patients, genetically modified at the BCL11a transcription factor to re-enable fetal haemoglobin production, and then re-infused into patients. The therapeutically upregulated fetal haemoglobin compensates for the defects in β-haemoglobin that cause the diseases. Clinical data shows that the gene therapy has curative potential, although longer-term data are needed to assess the durability of the effect.

When Harvard Medical School and HHMI’s Stuart Orkin and colleagues discovered the role of BCL11a in fetal haemoglobin production in 2008, it was unclear how to drug the transcription factor. The arrival of CRISPR–Cas9 gene-editing system in 2012 provided a path forward for haemoglobinopathies. The development of the programme was “remarkably fast”, said Orkin. “It is a perfect example of how the ecosystem can work.”

Vertex and CRISPR have priced the one-off treatment at $2.2 million. It also requires a harsh preconditioning chemotherapy regimen, to make room for the edited cells. The therapy will consequently remain out of reach for many patients. “This is not the end game,” says Orkin, who has his eye on next-generation gene editors and small molecules that might be more accessible.
PS. Un breu missatge per aquells que mitjançant la seva recerca "obren la porta" a tractaments i ho expliquen al Telenotícies. No n'hi ha cap d'aquesta llista del 2024 ni del 2023 d'aquí sota que sigui un d'ells, la porta segueix oberta, o potser no hi havia porta per obrir. Millor no haver d'estar sentint això sempre, sense explicar-ne el resultat.
PS. The economist sobre el tema




PS. El llistat de medicaments:

Table 1 | CDER approvals in 2023

Drug (brand name)

Sponsor

Properties

Indication

Lecanemab (Leqembi)a

Eisai/Biogen

Amyloid-β-targeted mAb

Alzheimer disease

Bexagliflozin (Brenzavvy)

Theracosbio

SGLT2 inhibitor

Glycaemic control in type 2 diabetes mellitus

Pirtobrutinib (Jaypirca)

Loxo/Eli Lilly

BTK inhibitor

Mantle cell lymphoma

Elacestrant (Orserdu)

Stemline

ER antagonist

ER-positive, HER2-negative, ESR1-mutant breast cancer

Daprodustat (Jesduvroq)

GSK

HIF-PH inhibitor

Anaemia caused by CKD for adults on dialysis

Velmanase alfa (Lamzede)a

Chiesi

Recombinant α-mannosidase

Non-CNS manifestations of α-mannosidosis

Sparsentan (Filspari)

Travere

Endothelin and angiotensin II receptor antagonist

Proteinuria in primary IgA nephropathy

Omaveloxolone (Skyclarys)

Reata/Biogen

Mechanism unknown, NRF2 activator

Friedrich’s ataxia

Zavegepant (Zavzpret)

Pfizer

CGRP receptor antagonist

Migraine

Trofinetide (Daybue)

Acadia

Mechanism unknown

Rett syndrome

Retifanlimab (Zynyz)a

Incyte

PD1-targeted mAb

Merkel cell carcinoma

Rezafungin (Rezzayo)

Cidara

Echinocandin antifungal

Candidemia and invasive candidiasis

Leniolisib (Joenja)

Pharming

PI3Kδ inhibitor

Activated PI3Kδ syndrome

Tofersen (Qalsody)

Biogen

SOD1-targeted ASO

SOD1 amyotrophic lateral sclerosis

Pegunigalsidase alfa (Elfabrio)a

Chiesi

PEGylated recombinant α-galactosidase Α

Fabry disease

Fezolinetant (Veozah)

Astellas

Neurokinin 3 receptor antagonist

Hot flashes caused by menopause

Perfluorohexyloctane (Miebo)

Bausch + Lomb

Semifluorinated alkane

Dry eye disease

Epcoritamab (Epkinly)a

Genmab/AbbVie

CD20 × CD3 T-cell engager

DLBCL and high-grade B-cell lymphoma

Sulbactam, durlobactam (Xacduro)

Entasis

β-lactam antibacterial plus a β-lactamase inhibitor

Hospital-acquired and ventilator-associated bacterial pneumonia caused by susceptible ABC

Nirmatrelvir, ritonavir (Paxlovid)

Pfizer

SARS-CoV-2 main protease inhibitor plus a CYP3A inhibitor

Mild-to-moderate COVID-19

Flotufolastat F18 (Posluma)

Blue Earth

Radioactive diagnostic agent

PET imaging in prostate cancer

Sotagliflozin (Inpefa)

Lexicon

SGLT1/2 inhibitor

Heart failure

Glofitamab (Columvi)a

Genentech

CD20 × CD3 T-cell engager

DLBLC or large B-cell lymphoma

Ritlecitinib (Litfulo)

Pfizer

JAK3 inhibitor

Alopecia areata

Rozanolixizumab (Rystiggo)a

UCB

FcRn-targeted mAb

AChR- or MuSK-antibody-positive gMG

Somatrogon (Ngenla)a

Pfizer

Human growth hormone analogue

Growth hormone deficiency

Nirsevimab (Beyfortus)a

AstraZeneca

RSV F protein-targeted mAb

RSV lower respiratory tract disease

Quizartinib (Vanflyta)

Daiichi Sankyo

FLT3 kinase inhibitor

AML

Lotilaner (Xdemvy)

Tarsus

Ectoparasiticide

Demodex blepharitis

Zuranolone (Zurzuvae)

Sage

GABAA receptor PAM

Postpartum depression

Avacincaptad pegol (Izervay)

Iveric/Astellas

C5-targeted aptamer

Geographic atrophy secondary to AMD

Talquetamab (Talvey)a

Janssen

GPRC5D × CD3 T-cell engager

Multiple myeloma

Elranatamab (Elrexfio)a

Pfizer

BCMA × CD3 T-cell engager

Multiple myeloma

Palovarotene (Sohonos)

Ipsen

Retinoic acid receptor agonist

Fibrodysplasia ossificans progressiva

Pozelimab (Veopoz)a

Regeneron

C5-targeted mAb

CHAPLE disease

Motixafortide (Aphexda)

Biolinerx

CXCR4 inhibitor

Hematopoietic stem cell mobilization for autologous transplantation in multiple myeloma

Momelotinib (Ojjaara)

GSK

JAK1/2, ALK2 inhibitor

Myelofibrosis in adults with anaemia

Gepirone (Exxua)

Fabre-Kramer

5HT1A receptor agonist

Major depressive disorder

Cipaglucosidase alfa (Pombiliti)a

Amicus

Recombinant α-glucosidase

Pompe disease

Nedosiran (Rivfloza)

Novo Nordisk

LDHA-targeted siRNA

Primary hyperoxaluria type 1

Etrasimod (Velsipity)

Pfizer

S1P receptor modulator

Ulcerative colitis

Zilucoplan (Zilbrysq)

UCB

Complement C5 inhibitor

AChR-antibody positive gMG

Bimekizumab (Bimzelx)a

UCB

IL-17A/F-targeted mAb

Plaque psoriasis

Vamorolone (Agamree)

Santhera

Corticosteroid

Duchenne muscular dystrophy

Mirikizumab (Omvoh)a

Eli Lilly

IL-23-targeted mAb

Ulcerative colitis

Toripalimab (Loqtorzi)a

Coherus

PD1-targeted mAb

Nasopharyngeal carcinoma

Fruquintinib (Fruzaqla)

Takeda

VEGFR1/2/3 kinase inhibitor

Colorectal cancer

Taurolidine, heparin (Defencath)

Cormedix

Thiadiazinane antimicrobial plus an anticoagulant

Incidence of catheter-related bloodstream infections

Repotrectinib (Augtyro)

Bristol Myers Squibb

ROS1 and TRK kinase inhibitor

ROS1-positive NSCLC

Efbemalenograstim alfa (Ryzneuta)a

Evive

Recombinant leukocyte growth factor

Neutropenia

Capivasertib (Truqap)

AstraZeneca

AKT kinase inhibitor

Breast cancer

Nirogacestat (Ogsiveo)

Springworks

γ-secretase inhibitor

Desmoid tumours

Iptacopan (Fabhalta)

Novartis

Complement factor B inhibitor

Paroxysmal nocturnal haemoglobinuria

Birch triterpenes (Filsuvez)

Chiesi

Mechanism unknown

Epidermolysis bullosa

Eplontersen (Wainua)

Ionis/AstraZeneca

TTR-targeted ASO

hATTR with polyneuropathy