Hot topics in pharma and orphan drugs

I attended this year’s pharma pricing and market access conference in London with attendees across the pharmaceutical industry, payer and HTA bodies and patient representatives. While following the talks, panel discussions and roundtable debates, a few overarching key topics crystallised:

–          Germany – AMNOG changes

AMNOG, the German pharmaceutical law is regularly adapted to changes in the environment, the latest update is just discussed by the German government and is expected to be finalised around April or May 2017 (click here for our AMNOG review). These changes will guide future market access activities in Germany. A successful drug assessment from the Joint Federal Committee’s (G-BA) is highly dependent on the study design. During the conference, Thomas Muller of the G-BA highlighted “Pricing begins with the study design” Relevant and objective endpoints are key for a successful G-BA assessment and will continue to be. HTA bodies are offering early advice to discuss study design and HTA related questions. In the future, manufacturers will be able to obtain joint advice from national HTA bodies as well as the EMA.

–          The concept of value

Interesting discussions came up during a panel that focused on the regulatory aspects of market access. Payer representatives and audience discussed value as the buzz word. What is value? Is value defined in the same way across countries? Is value interpreted in the same way by patients and by payers and regulators? Does value have the same meaning across different disease groups? What does this mean for orphan drugs? What does this mean for companies when developing their value story? How will the value story impact the reimbursement and market access decision? How can companies navigate and channel their value story? When companies are preparing for launch they need to understand which value story resonates with their individual audience, there is no one size fits all approach.

–          International reference pricing and innovative pricing models

The ever-growing pressure on healthcare budgets were discussed by several speakers and during discussions on site. International reference pricing and its implications for patient access were a big topic, how can manufacturers make international reference pricing an opportunity? Advances in medical treatment opportunities are leading to new challenges, high priced drugs will continue to challenge current funding systems. How can healthcare systems and payers react? We observed several innovative pricing models starting from pay for performance systems, risk pooling funds or dose cap schemes that are applied to be able to provide patients with the appropriate treatment while keeping the healthcare budget under control.

What does this mean for orphan drugs?

The European Medicines Agency defines orphan diseases as diseases with a prevalence of less than 5 in 10,000. Although it is estimated that about 30 million people in the EU suffer from a rare disease only a fraction of the 5,000 to 8,000 diseases is known and understood[i]. This makes the orphan drugs are a special case within the healthcare environment. Most countries introduced schemes to foster the development of orphan drugs, including designated market access pathways and support for smaller manufacturers. The orphan drug sector is booming and big and small companies are investing.

Reviewing the conference, orphan drugs were discussed from several angles. Under AMNOG, the German system assumes that products with an orphan designation have an added benefit and do not require the extensive HTA review if the yearly sales volume does not exceed €50 million. Although some groups asked for an end of this rule considering studies that have shown that several orphan drugs do not have an additional benefit[ii], the AMNOG proposal keeps this rule.

Value in orphan drugs is a sensitive topic, is the value of an orphan drug higher than of a non-orphan drug that it justifies a higher price? Does the general public support higher prices and thresholds for orphan drugs? Is it fair that some individuals within the healthcare system have access high priced drugs? Research has shown that so far, the higher prices and the need for incentives to develop such products is publicly accepted. However, high prices have sparked criticism among consumer groups and companies have to be prepared to explain these prices.

Many new products and developments are using new technologies and change current treatment pathways, as for example gene-therapy.  With those new products, the patient might be able to move from a chronic to a one-off therapy. This affects how the health system pays for it, even though a product might offer a financial benefit over the long run, healthcare budgets are set on a yearly basis and do not immediately account for benefits that occur in the years ahead. Manufacturers need to work together with HTA bodies and payers to find individual solutions to pay for promising high priced new products.


At Partners4Access, we believe patients with rare diseases deserve the right to access the most appropriate treatment. Our vision is ensuring fair access for all key stakeholders. We partner with our clients to think carefully, plan smartly and implement effectively to successfully secure access for orphan drugs. To receive more information please contact Christina Poschen [email protected] or visit our website and follow our LinkedIn page




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