The RWE era has come, how can pharmaceutical companies win by surprise?

Real World Evidence (RWE) has become indispensab.

There are more and more application scenarios of RWE, and a brand new situation is gradually opening .

These areas deserve attenti.

RWE used to revise drug "effects"

In 2016, the FDA promulgated the 21st Century Cures Act, which clearly stated that a dedicated program should be established to evaluate Real World Evidence (RWE) to support the approval of new indications and post-marketing studi.

In 2018, the FDA issued the RWE Program Framework to support the goals of the 21st Century Cures Act to assess the possibility of RWE being used to revise the "effects" of drugs, including the following three aspects:

(1) Addition or modification of indications , such as changes in dose, duration of treatment and administration (2) Addition of new populations for adaptation (3) Addition of efficacy comparison and safety information

▲Figure 1 FDA clarifies the application scope of RWE Source: FDA

In 2021, the FDA approved an expanded indicationof Astellas' Pluronic® (tacrolimus) , which is combined with other immunosuppressive drugs , based on an RWE reflecting efficacy provided by a non-interventional stu.

Proxafil was initially approved to prevent organ rejection in patients undergoing liver transplants, and was later approved to prevent organ rejection in kidney and heart transplan.

Astellas used real-world data on all lung transplants from the.

Easier listing approval

In 2013, the European Medicines Agency (EMA) participated in the GetReal Initiative to develop new methods for collecting and synthesizing RWE for earlier use in drug discovery and healthcare decision-maki.

(1) Provide evidence of efficacy and safety for the registration and marketing of new drugs (2) Provide evidence for the change of the package insert of the marketed drug: The change of the package insert includes six different situatio.

RWE and randomized controlled trials complement each other and can provide comprehensive information on product safety and efficacy, and meet real-world evidence needs for different product life cycl.

From 2013 to 2017, CSPC's flagship product, amlodipine tablets, carried out RWE of amlodipine and original research drugs in the treatment of hypertension, including more than 10,000 patients and covering more than 100 hospita.

Roche's entrectinib is in patients with ROS1-mutated non-small cell lung cancer, and a placebo-controlled trial is not feasible due to the small number of patien.

Roche accelerated the submission of entrectinib to the EMA and PMDA by using the "single arm + RWE synthetic control arm" strate.

▲Figure 2 Roche uses "single arm + RWE synthetic control arm" to speed up the listing application of entrectinib 

Source: Digital technology and advanced analytics in Roche, 2020

Globally, East Asia, dominated by China, is the most active region for RWE declaratio.

In early September 2020, pratinib was approved by the FDA for the treatment of adult patients with locally advanced or metastatic non-small cell lung cancer who had previously received platinum-containing chemotherapy with a transfection-rearranged (RET) gene fusion positi.

At the end of September of the same year, it was introduced into Boao Leche.

In October of the same year, it entered the Boao Lecheng Drug Clinical Real-World Data Application Pil.

Boao Super Hospital comprehensively collected patient clinical use feedback data to provide auxiliary evidence for the efficacy evaluation and safety evaluation of the drug in the Chinese advanced non-small cell patient populati.

In March 2021, the State Food and Drug Administration approved the listing of pratinib with conditions through the priority review and approval proce.

It took only half a year for Pratinib to be approved, and RWE has contributed a l.

China is taking Boao Lecheng as an experimental field, and RWE makes it possible for international new drugs to be launched in China "rapidly" and affects the drug market structu.

Market access and (re)pricing

RWE is increasingly playing a role in listing pricing, Medicare/market access bargaining, and early communication with Health Technology Assessment (HTA) agencies and paye.

In the UK, the share of RWE in initial submissions to the National Institute for Health and Clinical Excellence has steadily increased, from 9% in 2015 and 22% in 2016 to 37% in 201

About 25% of initial filings submitted to the French National Health Service included R.

Even Germany, which is skeptical of RWE, had an initial G-BA (Federal Joint Committee (G-BA)) filing in 2017 that included RWE and received positive revie.

In 2019, Roche Pertuzumab was approved by the German HTA and successfully entered the German medical insuran.

RWE played an important role in its successful access to the medical insuran.

Roche used real-world data from Flatrion's Pertuzumab patients to demonstrate that Pertuzumab can shorten the duration of first-line treatment for early-stage breast cancer patients compared to standard therapy, bringing an additional CHF 25 million net to the German government present val.

▲Source: Digital technology and advanced analytics in Roche, 2020

Guideline revisions and prescribing confidence building

In addition to the above application scenarios, RWE is also being used to develop clinical practice guidelines with healthcare stakeholde.

The value of RWE is that it derives from real-world clinical practice, thereby overcoming the limitations of highly controlled randomized controlled trials in representing real-world patient populations and treatment optio.

This provides an opportunity for pharmaceutical companies to jointly develop clinical guidelines with healthcare stakeholders through R.

Guidelines such as the European Respiratory Society Guidelines for the Treatment of Idiopathic Pulmonary Fibrosis and the European Organization for Crohn's Disease and Colitis Treatment Guidelines refer to the RWE eviden.

In addition to clinical societies, HTA agencies also incorporate real-world evidence when issuing clinical practice guidelin.

In the UK, real-world data from clinical practice research in the UK public healthcare system has been used to confirm the safe use of the combined measles, mumps and rubella vaccine, to inform the UK National Institute for Health and Clinical Excellence Cancer Guidelines, and to Improve hypertension management in people with diabet.

The application of RWE has gradually expanded from meeting post-marketing regulatory needs to more convenient marketing review, market access and (re)pricing, guideline revision and prescribing confidence buildi.

The value of RWE cannot be ignor.

How to apply it skillfully and invest wisely in RWE may be a question that every pharmaceutical company needs to think about in the RWE e.

【References】

FDA, State Food and Drug Administration, CStone's official website

Digital technology and advanced analytics in Roche, 2020

"Real World Evidence (RWE): From "Dispensable" to "Indispensable", IQVIA, 2018

"FDA approves expanded scope of immunosuppressants based on real-world data", WuXi AppTec, July 2021

"Real-World Research Drives New Acceleration", Medical Economic Journal, June 2022

"Guiding Principles for Real-World Evidence Supporting Drug Development and Review (Trial)", State Food and Drug Administration, 2020