2020: What does the future hold for the manufacturing and clinical landscape?

Pharma IQ spoke to Jeremy Schafer and Scott Marshall to find out their predictions.




The manufacturing and clinical landscapes are set to see further change over the coming years. Pharma IQ spoke to Jeremy Schafer and Scott Marshall to find out their predictions.

 

Jeremy Schafer, PharmD, MBA, SVP, Access Experience Team, Precision for Value

Gene therapies and potential cures

Diseases that have been managed for decades through chronic drugs may see dramatic paradigm shifts in care through the development of gene therapies.

2019 could see a monumental change in how diseases like hemophilia, immunodeficiency, and cancer are treated.

We will also begin to gather more data on the durability of gene and CAR-T therapies already on the market, which will help us understand whether these therapies are worthwhile investments and how manufacturers will continue to evolve the science.

 

Federal and State pressure on prices

Democrats, who will assume control of the House in 2019, found a winning message on healthcare during the midterms. Thy will likely keep up the pressure with a focus on drug prices, an issue where the electorate places a high priority.

Combined with a President who has shown a willingness to be aggressive and inventive in implementing ways to reduce costs, manufacturers will be more pressured on price than ever before.

 

Dwindling sacred cows

In 2019, we will see PBMs introducing exclusions in categories like hemophilia and HIV. CMS has proposed rules that would allow stricter management of protected classes, including ending grandfathering and excluded drugs with high price increases. Another PBM will offer a program that allows clients to exclude certain drugs not deemed cost-effective.

These moves indicate a shift in the market that no drug therapy is too sacred, too rare, or too unique to aggressively manage. This will pressure all manufacturers, regardless of therapeutic space, to take threats of limited access more seriously while at the same time requiring a value story that resonates with more customers.

 

Combinations in cancer care

Manufacturers looking for success in the oncology space will increasingly have to put aside their rivalries and instead work together to study combination regimens in oncology. Combination regimens have the potential to strike cancers in multiple ways that can delay progression and even extend survival compared with monotherapies.

Increasingly, the question doctors will have regarding cancer drugs will be not only “what is it used for?” but also “what is it used with?”.

 

Scott Marshall, PhD, Managing Director, Translational Informatics & Diagnostic Sciences, Precision for Medicine 

Machine Learning, AI and Technology Platforms to Address the Challenge of Big Data in Clinical R&D

With the continued evolution of biomarker-guided drug development and advancements in technology platforms to characterize biological variation and its impact on treatment response, considerations to address the big data challenge in clinical drug development will continue to be a hot topic.

Ranging from artificial intelligence and machine learning approaches to interrogate complex biological data for patient stratification to technology platforms to integrate disparate high throughput and high content data sets.

Novel informatics and data science driven approaches will be key to realizing the value of biomarkers as they continue to penetrate clinical development.

 

New Regulatory Developments in Gene Therapy and CDx

On the heels of FDA’s November 2017 release of a comprehensive regenerative medicine policy framework is FDA’s July 2018 release of 6 new draft guidance documents for gene therapy products.

Three of the guidance documents were directed toward disease-specific indications, including hemophilia, rare diseases, and retinal disorders. Most notably, in the rare disease draft guidance, FDA made reference to the need for enrollment of study populations using a companion diagnostic capable of measuring antibodies to the host vector.

This is a significant shift from use of total antibody (TAb) and neutralizing antibody (NAb) assays from informational to potentially required for commercialization. The science has not fully confirmed the association between antibody measurements and safety or efficacy of the gene therapy;however, the regulatory direction suggests the need to initiate studies and assays that meet the requirements for FDA clearance/approval.