The much awaited European Federation of Pharmaceutical Industries and Association (EFPIA) Waiting to Access Innovative Therapies (W.A.I.T.) Indicator Survey has been released. First released in 2004, this annual survey has gathered greater momentum over the years and now includes data from 39 European countries (27 EU, 12 non-EU) that examine access to 160 innovative medicines over a 5-year period (2017-2021).

However, this year, and in the wake of the European Commission’s (EC) Revision of the Pharmaceutical Legislation, EFPIA’s National Association and Manufacturers membership is linking these data to some bold commitments and proposals to address the worsening inequity of access to medicines in Europe.

What does the W.A.I.T indicator survey show?

  • There are two key indicators to dissect
    • Rate of availability: Whether the EU-approved medicine makes an individual country’s reimbursement list and is made available to patients
    • Average time to availability: Time from marketing authorisation to date the medicine becomes available to patients.
  • Both indicators are going in the wrong direction: there are fewer medicines available to patients and on average it was taking longer for patients to access them in 2021 compared to 2020.
  • As with prior years, there is a significant geographical disparity between Northern & Western compared to Eastern & Southern European countries. Out of the 160 medicines examined:
    • Less than 30% are available in smaller and Eastern European Member States compared with 92% in Germany and 46% in average in the EU.
    • For the medicines that were available, patients in Germany wait around 133 days to access new medicines compared to patients in Romania that wait more than 899 days.   
  • There were also differences observed across specific therapeutic areas.
    • In good news, on average more oncology products were available across the region (compared to the average of all products) and their average time to availability improved by almost one month compared to 2020.
    • The story has worsened for orphan medicines: the rate of availability of orphan drugs fell across the region. In fact, many countries did not make any orphan drugs available in 2020 or 2019.

What are the root causes for the access problems?

With the EC looking at ways to ensure greater equity across EU members states, alongside these data EFPIA released an analysis from Charles River Associates that places 10 root causes of delays and unavailability front and centre:

Potential root causes:

  1. The speed of the regulatory process
  2. Accessibility of medicines 
  3. Initiation of the process 
  4. The speed of the national timelines and adherence 
  5. Misalignment on value and price
  6. The value assigned to product differentiation and choice 
  7. Insufficient budget to implement decisions 
  8. Diagnosis, supporting infrastructure and relevance to patients 
  9. Multiple layers of decision-making processes

What is the industry doing and why is that important?

To cement greater equity of access to medicines in place without damaging the EU innovation eco-system, EFPIA is making meaningful promises. These will help increase transparency around the process and reduce delays. This will also up the ante on regulators, member states and EU institutions to play their part, as the EC eyes overhauling how medicines are accessed across Europe.

The key actions/commitments made are:

  • Commitment to File: For pricing and reimbursement in all EU countries as soon as possible and no later than 2 years from the central EU market authorisation.
    • Modeling shows that this commitment can increase the availability of medicines from 18% to 64% and reduce the time patients wait for these medicines by 4-5 months in several under-served countries.
  • Launch an Online Portal: Where manufactures will provide timely information regarding the timing and processing of pricing and reimbursement applications in the EU-27 countries. The data will be confidential and to be used only in aggregate and/or therapeutic level.
  • Equity-Based Tiered Pricing (EBTP): Opening discussions on a conceptual EBTP framework to ensure that ability to pay across countries is considered in the prices of innovative medicines. Anchored in a principle of EU solidarity, the countries will be divided into Tier 1 and 2 (based on Gross National Income) with the idea that Tier 1 pay more for innovative medicines than Tier 2.

Here is what manufacturers need to consider as EFPIA moves forward with these commitments

  • Emerging biotechs need to take a position: Emerging biotechs on average account for 33% of all EMA drug approvals every year. Most of them are not EFPIA members and should consider voluntary sign-up to adhere to the new pledge. Otherwise, they risk becoming an outlier and being targeted to explain their efforts on ensuring availability and access to their medicines in Europe.
  • Equity-Based Tiered Pricing (EBTP) needs a global and coordinated communication strategy: It is a bold proposal, but the devil will be in the details.
    • EBTP sets a framework that will define scope for negotiation and therefore revenue. This can cause tensions on both sides of the table. Some possible contention areas can be:
      • Will all Member States accept Tier 1 and 2 designations?
      • Will there be agreement on amending the External Reference Pricing (ERP) systems? How much lower should the Tier 2 price be compared to Tier 1?
    • Companies also need to be prepared to answer whether the EBTP should be utilized globally. Often the industry messaging stresses the need for local/country-specific pricing strategies. With EBTP, tough questions can be raised around:
      • Should other regions such as LATAM and APAC also adopt this structure?

What about the United States? Using the same theory, should innovative medicines cost more in richer states such as California? Why is this model workable in the EU but not in the US?   

About the Author:

Aneeb leads the health policy and patient engagement team, advising clients on strategic communication and corporate reputation strategies from a global public affairs, value & access and advocacy standpoint. His background is in politics and healthcare policy with vast experience working in the US and Europe. He is based in London.