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Orphan drugs and where to launch them: The keys to Europe’s forgotten territories

Pharmaceutical Technology

The commercial investment required to research and develop an innovative drug, prove its safety and efficacy, and bring it to market is staggering. Go-to-market strategies. The challenge of providing adequate access to the peripheral EU markets is quite evident. Efforts have been made to change this.

Drugs 147
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Risk-sharing agreements are growing at a rate of 24%

Pharmaceutical Technology

This is not the first treatment to come with a high price tag. Access to oncology medicines varies across countries, and their cost against safety/efficacy is generally perceived as being low, such as in the US. Undoubtedly, this has played a factor in the success of market-leading PD-1 inhibitors, Merck & Co.’s

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Cannabinoids receptors: popular preclinical target but banned in 137 countries

Pharmaceutical Technology

Collectively, cannabinoid receptors (CB1 and CB2) are currently the most popular targets in preclinical stage of development, with 391 drugs tagged in total. In the US, the world’s largest pharmaceutical market, most states have legalized cannabinoids for medical use. This is closely followed by CB2 receptors in second place.

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Smart pharmaceutical and healthcare labels: Lets trace medicines from its origin

Roots Analysis

It is worth noting that smart labels contain a transponder code which can be read by sophisticated devices, including radio frequency identification device (RFID) tags and near-field communication (NFC) chips. While most smartphones can read NFC chips, RFID tags can only be read by specialized receivers.

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A broad range of unmet needs remains in the immuno-oncology space

Pharmaceutical Technology

There are more than 20 marketed ICIs with approvals across a very wide spectrum of solid tumour indications. Marketed bispecific antibodies are largely bispecific T-cell engagers (BiTEs), which work by directing T-cells to antigen-expressing tumour cells. Cost-related unmet needs also scored highly.

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Free access to Zolgensma curbed, says Novartis

pharmaphorum

Novartis’ programme providing free access to its spinal muscular atrophy (SMA) gene therapy Zolgensma is being scaled back to a dozen countries worldwide, according to the company. ” Zolgensma is one of the most expensive therapies available, with a price tag of around $2.1 Pause in UK access.

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Rare Disease Spotlight – tracing the rise of orphan drug designations over almost 40 years

Pharmaceutical Technology

Additionally, pricing and access for rare disease therapies continue to be scrutinized closely. For example, while orphan drug designations have more than doubled in the last decade compared to the previous one, only 16% of therapies with orphan tags have managed to gain FDA approval in some indications.

Drugs 246