A 1-Day Training Course Delivered by
David Schwicker

David Schwicker

  • Founder of ORPHA Strategy Consulting, David is a sought-after expert for accelerating time to launch and patient access for OMPs and ATMPs. 
  • David's experience includes rare oncology and haematology diseases (ALL, AML, MM, CD30+ HL/NHL, CTCL, ALK+/EGFR+ NSCLC, breast cancer), metabolic and endocrine diseases (MPS, Graves’ disease, acromegaly), neurology diseases (CIDP, RTT, MG, narcolepsy, Friedrich’s ataxia), inherited retinal dystrophies (retinitis pigmentosa, LCA), USHER syndrome, hepatic conditions (PSC), and rare immunological diseases (AMR, GvHD).
  • Strategic Advisor to Ziphius Therapeutics, a startup biopharmaceutical company, with a focus on the planning of clinical and value evidence generation.
  • Author and co-author of numerous peer-reviewed publications, David is a regular speaker at international meetings (ISPOR, RAPS EU Congress, Orphan Drugs Development and Commercialisation).


  1. Why market access challenges for OMPs and ATMPs, including autologous and allogeneic gene therapies, tissue engineered products and somatic cell therapies, are different from other drugs.
  2. How to collaborate with patients and caregivers in rare conditions to generate patient-relevant outcomes.
  3. Leveraging applications of RWE throughout the OMP and ATMP value chain.
  4. To mitigate payer uncertainty by addressing the challenge of early market access with accelerated approvals and fewer clinical data.
  5. How to develop a next-generation market access strategy with a full-spectrum lifecycle evidence generation plan that maximises product value from launch to loss of exclusivity.
  6. To differentiate and succeed in the increasingly competitive rare disease space with a “fast to patient” approach.

Additional Benefits

  • You will receive an up-to-date collection of demonstrator cases and literature focused on rare patient engagement, PROs, patient-centric RWE applications in rare conditions, value demonstration and market access.
  • You will be able to share your unique OMP- or ATMP-related challenges and to discuss practical solutions with the expert and delegates.


Why You Should Attend

The Content: The fundamental challenge for the successful market access and commercialisation of OMPs and ATMPs is that the evidence generated for regulatory approval cannot satisfy the requirements of HTA bodies, pricing & reimbursement, and contracting negotiations with payers. Approval does not guarantee a premium price nor commercial success. In this course you will learn to address the challenge of early market access based on fewer and less mature data, with the creation of an Early Value Proposition and a Patient-Centric Evidence Generation Plan that both comprehensively support value demonstration, product positioning, and that both meet the requirements of all healthcare stakeholders (regulators, patients & caregivers, physicians & KOLs, HTA bodies and payers).

The Expert: David Schwicker has developed a unique understanding in supporting clients with a “fast to market” strategy by integrating rare disease development, regulatory and market access strategies with the joint view of the earliest feasible and sustainable patient access to innovative orphan drugs and advanced therapies.

The Accelerating Market Access in Rare Diseases course is the only available public training course delivered by David Schwicker on this topic.

Agenda of Accelerating Market Access in Rare Diseases



Welcome & Audience Expectations


Opportunities and Challenges in OMPs and ATMPs

  • Defining rare and ultra-rare conditions - Industry outlook to 2024: Enormous potential, but commercial challenges too
  • Why the OMP & ATMP regulatory approval package is often insufficient for market access
  • Orphan legislation and incentives for developers of OMPs and ATMPs in the US and EU
  • Key to OMPs and ATMPs: Fast track, breakthrough therapy, PRIME and accelerated approvals
  • Price premiums – is rarity valued by payers & the public?
  • De-risking and increasing ROI: The economics of rare disease R&D
  • Pricing and reimbursement challenges for OMPs and ATMPs – The reasons QALYs often don’t work in rare conditions, and why payers dislike uncertainty
  • Million-dollar cures and the affordability debate: Hyper-personalized, hyper-accurate, hyper-expensive - who will have access?


Empowerment & Involvement of Rare Disease Patients

  • Transforming orphan drug development and evidence generation through patient-led research
  • Patient engagement objectives and ROI: Identifying populations, faster & smaller clinical trials, incorporating patient-relevant endpoints, accelerated patient & market access
  • Collaborating with rare disease patients, caregivers and patient advocacy organisations: Principles and guidelines
  • How patients & caregivers influence regulatory and HTA decision-making
  • Patient Reported Outcomes (PROs) in labelling: An underutilised high-risk, high-reward differentiation opportunity
  • PRO strategy: Selection, research, validation and regulatory/HTA qualification of tools


Coffee Break


Leveraging RWE Throughout the Value Chain


  • Key RWE objectives and applications throughout the OMP and ATMP lifecycle

  • How RWE can accelerate regulatory and HTA decision-making (FDA, EMA, HTA bodies)

  • Using the full evidence spectrum: The RCT to RWE continuum

  • Biomarker & surrogate endpoint-based strategies – Increased speed, decreased risk

  • Non-traditional and hybrid Real World Data (RWD) – Advances driven by digital health solutions

  • Regulatory- & HTA-grade RWD quality criteria (FDA and Europe)

  • Strategic partnerships with commercial and academic RWD curators, RWE/RWD initiatives and sources, The European Rare Disease Reference Networks (ERN) and Orpha.net




Learning from High-Impact Demonstrator Cases

  • The current “state of play” of RWE application by biopharma, regulators, HTA bodies and payers
  • Discussion of practical solutions for challenges based on real world case examples, such as:
    • Understanding the rare condition: Natural history, epidemiology, patient identification, subpopulations, burden of disease, unmet medical needs
    • Biomarker and Genotype-Phenotype identification, validation of novel endpoints
    • Primary regulatory approval based on nonrandomised studies with historical controls
    • RWE-based label extensions (lifecycle management and iterative development)
    • Post-authorisation commitments following conditional marketing authorisation
    • Leveraging RWD & RWE from expanded access and compassionate use
    • Pragmatic clinical trials
    • Value demonstration and market access in rare and ultra-rare conditions 


Early Value Proposition and Evidence Generation Planning

  • Creating an evolving Early Value Proposition (EVP) as a complement to the labelling concepts and the Target Product Profile
  • Integrating the EVP into the asset strategy, strategic imperatives and critical success factors
  • Core value drivers: Early value demonstration with limited clinical data based on accelerated regulatory approvals - Addressing few data and uncertainty
  • Developing a full-spectrum lifecycle evidence generation strategy plan to employ the complete toolbox from RCTs over PROs to patient-centric RWE
  • Early dialogues: Seeking scientific advice in parallel EMA/HTA and single HTA consultations


Coffee Break


Next-Generation Market Access Strategies for OMPs and ATMPs

  • Differentiated value communication: Involving stakeholders and improving education to prepare the community and accelerate patient access
  • Value assessment trends - holistic approaches: Mechanism of Coordinated Access (MoCA), ORPH-VAL, the new AIFA Innovation Algorithm
  • Why compassionate use (US: expanded access) is ideally suited for generating an early market presence & revenue and enabling rapid commercial uptake
  • Managed Entry Agreements (MEAs): Risk sharing & pay-for-performance contracts, value- and indication-based pricing, the “Netflix” model - Current challenges and limitations
  • Orphan-specific launch, commercialisation and product differentiation strategies for increasingly crowded rare disease markets


Key Takeaways & Final Q&A



>> Click here if you wish to receive the PDF brochure of this course

Learning Methodology

David Schwicker is very experienced in transferring his expertise in small class executive courses. The key learning tool he employs in his course is interactive discussion of the content, based on numerous highly current real world case examples.

One of the most valuable aspects of attending any C.E.L.forpharma course is not only being able to have your specific questions answered by a leading expert, but also having the opportunity to share experiences and have in-depth discussions with your international peers.

infoThe typical audience size of our courses ranges from 6 to 24 (max) participants.

Who Should Attend?

This is an invaluable course for executives who want to learn about optimising market access and pricing of orphan drugs and advanced therapies for the 6-7.000 rare and ultra-rare conditions.
For example: 

  • Experienced pricing managers who want a review of the pricing strategies and innovative value-based approaches for OMPs and ATMPs
  • Marketing and commercial executives who want to create next-generation patient-centric rare disease marketing plans
  • R&D and regulatory executives who want to integrate an early value proposition and patient-centric RWE generation into their strategies 
  • Finance executives who need to understand the market access and pricing mechanisms of orphan drugs and advanced therapies
  • Venture capitalists, strategists and portfolio executives who are interested in building or expanding a rare disease pipeline
  • Patient advocates who want to learn more about real-world evidence and the biopharma approach to market access and pricing
  • Epidemiologists, quality-of-life experts, outcomes researchers and health economists who want to apply their expertise to rare diseases
  • Digital health, big data and real-world evidence researchers who want to leverage their fields in orphan drugs and advanced therapies

As this course is delivered by David Schwicker from ORPHA Strategy Consulting, a leading pharma consultancy company, executives from other professional service agencies should request approval prior to registering for this course. Please contact Inge Cornelis, Director Commercial Communications, for more information (inge.cornelis@celforpharma.com, tel +32 (0)2 709 01 43).


Past Participants

As this is the first session of the Accelerating Market Access in Rare Diseases course, there is no past participants list available.



As this is the first session of the Accelerating Market Access in Rare Diseases course, there are no testimonials available.

Dates & Locations

All C.E.L.forpharma courses are held in top-class four or five star hotels situated in easily accessible locations, either close to an international airport or near public transportation links in city centre locations.


  • 19 November 2019, Brussels


This session is fully booked. If you want to be on the waiting list, please send an e-mail to Inge Cornelis.

This course takes place at the Courtyard Marriott Brussels Hotel which is located 15 minutes from Brussels National Airport by complimentary hotel shuttle or taxi and 30 minutes by taxi from Brussels South train station (Thalys, TGV, Eurostar). 


Address: Avenue des Olympiades 6, 1140 Brussels, Belgium
Tel: +32 2 337 08 08

  • 2 April 2020, Brussels

This course takes place at the Courtyard Marriott Brussels Hotel which is located 15 minutes from Brussels National Airport by complimentary hotel shuttle or taxi and 30 minutes by taxi from Brussels South train station (Thalys, TGV, Eurostar). 


Address: Avenue des Olympiades 6, 1140 Brussels, Belgium
Tel: +32 2 337 08 08

  • 8 October 2020, London

This course takes place at the Renaissance London Heathrow Hotel which is located on the perimeter of Heathrow Airport, a short taxi or bus journey from the terminal where the Heathrow Express service to Central London is also situated.


Address: Bath Road, Hounslow, TW6 2AQ, United Kingdom
Tel: +44 20 88 97 63 63

  • 1 December 2020, Zurich

This course takes place at the Hilton Zurich Airport Hotel which is located just five minutes from Zurich Kloten Airport with the complimentary shuttle. Zurich’s vibrant city centre is located only a fifteen minutes’ drive away.


Address: Hohenbuehlstrasse 10, Opfikon-Glattbrugg 8152, Switzerland
Tel: +41 44 828 50 50

Hotel Booking Assistance

Having built a strong relationship with each hotel, C.E.L.forpharma has secured preferential room rates for our delegates who book their accommodation three weeks or more prior to the course. Upon your registration, we will help you make your hotel booking. For any assistance in this matter please do not hesitate to contact Sarah Nissen, Senior Programme Coordinator (sarah.nissen@celforpharma.com, tel +32 (0)2 709 01 46).

Included in the Registration Fee​

  • Course Material (Digital & Print Versions)
  • Coffee, Tea & Refreshments During the Course
  • Lunch During the Course
  • Certificate of Attendance Signed by the Expert


Group Discounts

Team discounts can be offered to 3 or more delegates from the same company.
Contact Inge Cornelis, Director Commercial Communications, (inge.cornelis@celforpharma.com) for more details.


How to Register

  1. Click the "Register Here" button on this page.
  2. Choose a course date, then fill out your personal details and company / invoicing details.
  3. Choose to pay via bank transfer / invoice or by credit card.
  4. Click "Confirm Registration". You will be sent an automatic email confirming your registration followed by a personal email containing an invoice and further payment instructions.

For assistance in registering, raising a PO or invoicing, please do not hesitate to contact Kealeigh Steel.
(email: kealeigh.steel@celforpharma.com or call: +32 2 709 01 45)


Transfer & Cancellation Policy

Flexible Transfer

  • If a registrant cannot attend the scheduled course, he/she can avoid any cancellation charge by sending a suitable replacement participant.
  • Alternatively, the registrant can transfer once on a “space available” basis at no extra cost, until 3 weeks prior to the event, to another course held within one year of the original course date.

Participant Cancellation

  • Up to 6 weeks prior to the course: cancellation fee of € 400 per participant.
  • Up to 5 weeks prior to the course: 25% of the invoiced registration fee.
  • Up to 4 weeks prior to the course: 50% of the invoiced registration fee.
  • Up to 3 weeks prior to the course: 75% of the invoiced registration fee.
  • Fewer than 3 weeks or if no notification received: registrant liable to pay invoiced registration fee.
  • If a registrant postpones his/her participation to a future course, and cancels again, no refund can be claimed for registration fees.